Cell Therapy Blog

2011 EMA Committee for Advanced Therapies (CAT) classification record. What can be learned?

noreply@blogger.com (Lee Buckler) — Tue, 27 Dec 2011 22:04:00 +0000

What follows is the record of "classifications" done by the ATMP CAT in 2011 related to anything I would call "cell therapies".

In my opinion there are a couple surprises. I'm surprised at the non-cardiac cells (MNCs, CD133s, and MSCs) for cardiac disease/repair being designated TEPs. I'm also surprised at the islets not being classified as an ATMP.

I've tapped into my European and/or regulatory colleagues to help explain those two as well as help us draw any other conclusions or observations we can make in terms of how the CAT is thinking based on the compendium of classifications we have to-date. I'll post an update here when I have something useful.
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In January, the following product was classified as a tissue engineered product - not combined:

Layer of autologous corneal epithelium containing stem cells intended for the treatment of extended corneal lesions

In April, the following product was classified as a tissue engineered product, combined:

Allogeneic human fibroblasts cultured onto a biodegradable matrix, intended for use of conditions in the therapeutic area of dermatology

In May, the following product was classified as a somatic cell therapy medicinal product:

Heterologous human adult liver-derived progenitor cells, intended for the treatment of inborn errors of liver metabolis

In July, the following product was classified as a Tissue Engineered Product, non-combined:

Suspension of allogeneic bone-marrow derived osteoblastic cells, intended for the treatment of non-union, delayed union or other fractures.

In September, the following product was classified as a Tissue Engineered Product, non-combined:

Autologous mesenchymal stem cells (MSC), intended for the treatment of chronic heart failure symptoms by improvement in exercise capacity of NYHA class II and III chronic heart failure patients receiving standard therapy

and the following product was not classified as an ATMP:

Human islets of Langerhans, intended for: Post pancreatectomy for benign pancreatic pathologies (autologous); Treatment of severe forms of type 1 diabetes (Allogeneic)

In October, the following product was classified as a somatic cell therapy medicinal product:

Autologous dendritic cell (DCs) immunotherapy consisting of autologous mature DCs coelectroporated with autologous RCC IVT RNA and synthetic CD40L IVT RNA, intended for the treatment of patients with advanced renal cell carcinoma

In November, the following products were classified as tissue-engineered products:

Concentrate of autologous bone marrow mononuclear cells (MNC), intended for improvement of heart function and quality of life in patients with chronic ischaemic heart disease and after MI.
CD 133+ Autologous bone marrow derived stem cells, intended for Improvement of heart function (LVEF) and quality of life in patients with chronic ischemic heart disease and after MI

In December, the following product was classified as somatic cell therapy medicinal product:

Autologous CD4+ T cells targeted to cells presenting class II restricted epitopes, intended forthe treatment of autoimmune diseases with MHC restricted specific immunity e.g. multiple sclerosis, type I diabetes or graft rejection.

Washing cryopreserved cells. An emerging need or disappearing process?

noreply@blogger.com (Lee Buckler) — Tue, 20 Dec 2011 23:56:00 +0000

I’m not a fan of the allogeneic vs autologous business model debate because I don’t believe it’s a debate that rages other than at conference panel sessions. Most investors, researchers, and executives recognize that there will almost certainly be room for both to succeed and that the winner in any particular indication will be largely determined by proven clinical efficacy over the standard of care and other available treatment alternatives.

Cryopreserved vs Fresh

The oft-touted primary commercial advantage of allogeneic cell therapy products over their autologous counterparts is the ability to inventory standardized products for later on-demand distribution and use. This contributes to the ‘economies of scale’ advantage allogeneic products enjoy. Certainly this is true.

Critics of the autologous business model cite the high cost of single-batch lot sizes and short shelf-life of autologous products- often shipped fresh - as the primary drivers of the high cost of these types of products and the variation in cell composition of therapeutic products derived from patient to patient. Certainly also true.

Nonetheless, the two issues most involved in a debate comparing the business models are cost and price implications of the relative bioprocessing scalability and distribution costs of each model. For sake of convenience it is most often assumed that allogeneic cell therapies are cryopreserved and autologous products are delivered fresh from the manufacturing site to the clinic for delivery to the donor-patient. This is, of course, an over-simplification because it is not always true.

Take, for instance, Opexa Therapeutics’ Toxavin which involves the cryopreservation of multiple doses (potentially representing several years) of treatment from a single patient apheresis. This is in stark contrast with Dendreon’s Provenge which requires a new apheresis for each of three monthly treatments and a limited shelf-life of a fresh product of approximately 72 hours. One cannot avoid concluding that the likely cost implications of such a difference are bound to be significant considering the differences in upstream collection, processing, and distribution costs.

I want, however, in the last few paragraphs of this post to focus attention on a couple of aspects related to cryopreservation of cell therapeutics. Firstly, as a digression, I am often left with the impression that executives and analysts alike often over-estimate the cost of shipping fresh products (with their temperature and time sensitivities) compared to the costs associated with shipping cryopreserved products which most often require heavy and bulky LN2 shippers as well as facilities and personnel experienced with receiving and handling cryopreserved products at regional repositories and local pharmacies.

Does the existence of cryoprotectant excipients dictate point-of-care cell washing?

Currently the short answer is “not necessarily”. The primary point I want to address here, however, is related to the costs currently associated with balancing the excipient and processing requirements involved in cryopreserving, storing, and thawing cells on the one side with the need to have a product that is clinically safe, effective and well tolerated by the patient on the other side. Companies developing cryopreserved cell therapies have three choices in this regard:

Infuse the patient with a product that includes cryopreservant excipients (almost always including some levels of DMSO being the overwhelmingly dominant reagent) recognizing the impact on patient experience and infusion volumes (a more significant concern in bodily regions where capacity is small (e.g., heart) or potentially sensitive (e.g., brain).
Invest in developing an infusion-ready formulation that significantly minimizes the amount of excipients. This may or may not involve a thaw or post-thaw dilution.
Commit to a process that involves point-of-care, post-thaw washing and re-concentration of the product to remove excipients and minimize the volume size of the product to be infused.

In terms of examples, we believe Mesoblast is currently in the first camp, Celgene has pursued the second strategy, and Athersys is an example of a company using the 3rd approach. Each have a similar cell type and all three are pursuing some of the same indications.

Some companies have worked hard to bring to market cryoprservant formulations that reduce the amount of DMSO (e.g., BioLife Solutions). Some predict that in the near future DMSO-free cryoprservants will be a real fourth option (e.g, Essential Pharma's Cryo-Ess currently for Research Use Only).

These products will have to be pioneered by some early-adopters before they are readily considered by the majority of players in a field which is oft-defined both by its dogged pursuit of precedent and reticence to be mold-breaking and innovative.

Stem cell transplanters are also faced with deciding between first and third course. While different considerations and drivers apply to them vs companies developing s.351/ATMP products, they are still faced with the decision to wash or not.

What factors to consider in the “to wash or not wash” debate?

Of the many and somewhat differing factors to take into account, cell therapy developers and stem cell transplanters share a number of common considerations when deciding how to treat cryopreservants in the clinical setting:

Regulator’s general tolerance of DMSO in the final product formulation to-date. Despite this record of tolerance, it is expected that for certain indications and/or for certain types of routes of administration, there may be significantly more regulatory scrutiny concerning injecting DMSO. Indeed, DMSO is classified as a Class 3 (relatively low risk) solvent in ICH Q3C with a recommendation of 50 mg/day as upper threshold below which one does not need to ‘justify’ its presence. The typical DMSO solutions used in cell therapy labs contain about 1 gram (not mg) per mL before dilution-- so if used at 10% in final product, this translates to 100 mg per mL. Therefore a 10 mL cell therapy product (at 10% DMSO) would contain 1000 mg (1 gram) of DMSO (20 times the ICH threshold).

It may be worth noting that apparent regulatory tolerance of the infusion of DMSO may be somewhat tied to the fact that most previous applications have involved the IV injection - allowing for excipients to e rapidly diluted in systemic circulation. For cell therapies delivered some other way, potential toxicity may be a more significant concern.

Concentrations of 10-20% DMSO has been traditionally used since the dawn of stem cell transplantation with minor reports of allergic reactions (e.g., hives, itching or facial or glottal edema) and only rare reports of more serious anaphylactic/oid reactions. Side effects of DMSO include hypernatremia, fluid overload, dysgeusia (distorted taste), nausea, vomiting, elevated liver enzymes, hemolysis, renal failure, and allergic reaction. DMSO toxicity is the most common complication of stem cell transplantation with symptoms including flushing, rash, chest tightness, nausea and vomiting, an cardiovascular instability (as outlined in the Circular of Information for the Use of Cellular Therapy Products). Ruiz-DelGado recently reported dimethyl sulfoxide-induced toxicity in cord blood stem cell transplantation and reviewed the literature (Acta Haematol. 2009;122(1):1-5). The authors reported the incidence of any cord blood infusion reaction ranging from 4% to 65%, with life-threatening infusion reactions occurring in up to 4.6% of patients.

It is worth noting that the FDA’s Pharmacovigilance Review Memo, related to the FDA’s recently approval of the New York Blood Centers BLA for its cord blood progenitor product named “Hemacord”, includes the following statement:

“Exposure to DMSO and Dextran-40, though not completely avoidable, can be limited by proper preparation before infusion of cord blood. Warnings and instructions for preparation (e.g. thawing, washing, dilution) should be included in the label.”

Even for those patients who do not experience any toxicity, allergic, or anaphylactic/oid reaction, there is an undisputed and significant ‘garlic’ odor and taste experience by the patient as well as the issues related to having to consent around the infusion of such excipients.

One of the outstanding regulatory questions is to what extent regulators will consider point-of-care washing steps to be a final manufacturing step. Closely related, but not necessarily intrinsically tied to this issue, is the question of whether a final release assay will be required to test a final product which was washed and re-concentrated post-thaw.

Finally, one is tempted to wonder whether to what extent regulatory opinion, commercial strategies, development pathways are influenced by what has been to-date a lack of commercially acceptable and viable technical solutions to post-thaw washing and re-concentration.

On this last note, Cell Therapy Group is working with Stem Cell Partners on bringing to market what we believe is potentially a simple, quick, cost-effective, easy-to-use density phase washing centrifugation device utilizing commercially available reagents and centrifuges – the EnsuraSep Cell Washer.

Have a quick spin through the brief technology outline at www.cellwasher.com and I would be happy to discuss it further with anyone interested.

We are working on different configurations and sizes of that device for different applications. Stem Cell Partners is working with our clients to design custom canisters and reagent-formulations to meet their specific requirements. Stem Cell Partners is also working on alternative centrifugation-based device that has a wider-capacity range.

While we believe the Ensura-Sep Cell Washer may enable a simple and rapid washing and concentration of a cell suspension in a single centrifugation step, CTG is also working with other companies who are pursuing other solutions using different technologies such as filtration.

There is currently much expert divergence on the question of what role point-of-care cell washing/concentration may play in the future of cell therapies. I invite any and all comments or feedback on this post either using the comment function here or in the discussion thread mentioned below in the LinkedIn Cell Therapy Industry Group.

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Much thanks to a great discussion thread in the LinkedIn Cell Therapy Industry Group called “Clinical preparation of frozen cell therapy products” which inspired must of this content with a special nod to Jon Rowley, Reinout Hesselink, EJ Read, Christopher Bravery, and Ali Mohamed.

Inactive and recently failed or terminated phase III or II/III cell therapy trials

noreply@blogger.com (Lee Buckler) — Sat, 17 Dec 2011 00:25:00 +0000

Updated 20 December 2011

In the two previous posts I have outlined what I believe to be the active phase III and II/III cell therapy trials, as well as the cell therapy products to have 'recently' obtained formal regulatory market approval in some jurisdiction.

In the course of doing that work, I came across the following industry-sponsored phase III cell therapy trials which appear to be inactive and those which failed or were terminated.

INACTIVE INDUSTRY PHASE III or II/III

Aastrom BRCs
Aldagen ALD-101
Arblast AMT-301
Avax Mvax
Vioheart Myocell
HepaLife Tech HepaMate
KeraCure KeraPac
Northwest Bio DCVax-Prostate
t2cure BMCs
TVAX TV-Brain
TVAX TV-Kidney-1

RECENTLY FAILED /TERMINATED INDUSTRY PHASE III

ABH (now Shire) Dermgraft
Cellerix Cx401

I don't imagine this is an exhaustive list but as I have encouraged in previous posts, I welcome feedback as to errors, corrections, or omissions. I'm using the 2009-11 time-frame here. I'll update the post accordingly.

Recently approved cell therapy products

noreply@blogger.com (Lee Buckler) — Sat, 10 Dec 2011 00:26:00 +0000

Following is a list of cell therapy product approved in 2012:

Medipost Cartistem S. Korea
Anterogen Cupistem S. Korea

Following is a list of cell therapy products approved recently (2010-11):

Dendreon Provenge US
FCB-Pharmicell Hearticellgram-AMI S. Korea
Fibrocell Sciences Laviv US
Living Cell Technologies DIABECELL Russia

Honorable mention goes to TiGenix' ChondroCelect approved in late 2009 representing the first EMA approval of an ATMP:

TiGenix ChondroCelect EU

Active phase III or II/III cell therapy trials

noreply@blogger.com (Lee Buckler) — Sat, 10 Dec 2011 00:20:00 +0000

Updated 13 February 2012

What follows is what we are currently tacking as and believe to be a relatively comprehensive list of phase III or II/III cell therapy trials around the world. I am more confident of the industry list than the academic one. More confident of its completeness and accuracy for North American and Europe than of Asia (particularly China).

There is a spreadsheet behind this that includes more data fields like therapeutic category, cell/tissue source, cell type, expansion, indication, expected completion date, clinical trial site locations, etc.

INDUSTRY PHASE III or II/III (active or expected to be active in 1H 2012)

Aastrom Ixmyelocel-T

Baxter ACT34-CMI

Cardio3 Biosciences C-CURE

Cell Medica adoptive cellular therapy

CellSeed France CAOMECS (Cultured Autologous Oral Mucosal Epithelial Cell-Sheet)
co.don co.don chondrosphere
Cook Myosite AMDC

Cytori ADRCs

GamidaCell - Teva StemEx

Genzyme MACI

Harvest Technologies SmartPReP 2 BMAC

Healthpoint HP802-247
Innovaell IES13 (Urocell?)

ISTO Technologies DeNovo ET

Kiaidis Pharma ATIR
MacroCure CureXcell
Miltenyi CliniMACS CD34 Selection System

MolMed TK

Newlink Genetics HyperAcute Pancreas

NovaRx Lucanix

NuVasive Osteocel Plus
Osiris Prochymal

Osiris Prochymal

Pervasis Vasugel
Prima Biomed Cvac
SOTIO DVAC/Pca

Some of the products listed above are in more than one clinical trial - different regions and/or different indications.

In total we are tracking 36 industry sponsored, active (or expected to be active 1H 2010) cell therapy trials in ph III or II/III expected to treat over 8,000 patients.

The 36 trials are sponsored by 26 companies. The trials roughly break down as follows:

36% (13) are autologous
53% (19) are allogeneic
Two are gene-modified allogeneic
One involves autologous and allogeneic cells (we counted it for both)

24% (8) for oncology or related indications
11% (4) for cardiac-related indications
15% (5) for critical limb ischemia, venous leg ulcers chronic wounds
15% (5) for cartilage repair
12% (4) for orthopedic repair

* ACADEMIC PHASE III or II/III

Assistance Publique - Hôpitaux de Paris (France)
Association of Dutch Burn Centres (Netherlands)
Barts and The London NHS Trust (UK)
Erasmus Medical Center (Netherlands)
European Group for Blood and Marrow Transplantation (Europe)
General Hospital of Chinese Armed Police Forces (China)
Leiden University Medical Center (Netherlands)
Meshalkin Research Institute of Pathology of Circulation (Russia)
Meshalkin Research Institute of Pathology of Circulation (Russia)
Ministry of Health (Malaysia)
Royan Institute (Iran)
Rush University Medical Center, University of Sao Paulo, Uppsala University (US, Brazil, Sweden)
Third Military Medical University (China)
University of Minnesota, Masonic Cancer Center (US)
University of Minnesota, Masonic Cancer Center (US)
University Hospital of North Norway (Norway)
University of Utah (US)

* Active trials only - excludes trials which appear inactive, abandoned, and/or only involve a stem cell transplant. Primary source is CinicalTrials.gov.

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I will try to keep this list updated at least once-per-quarter and indicate the date of the last update at the top of the post.

I eagerly encourage all readers to comment below or email direct with any errors and/or omissions.

Sabrina Cohen Foundation Thanks Stem Cell Researchers

noreply@blogger.com (Lee Buckler) — Fri, 25 Nov 2011 20:59:00 +0000

I'm proud to use every available resource at our disposal, including this blog, to highlight the efforts of the charity we support - especially during this holiday season.

I would be so delighted to have you join me in supporting Sabrina Cohen and her efforts. You can start by buying next year's calendar!

The Sabrina Cohen Foundation
The Sabrina Cohen Foundation for Stem Cell Research (SCF) is an internationally recognized nonprofit organization dedicated to building a global network of top scientists and clinicians in the field of Regenerative Medicine, while simultaneously funding cutting edge research and innovative therapies that will reverse spinal cord injury and effectively treat other impairments of the Central Nervous System.

The ‘CELLebrity’ Doctors Calendar
The 2012 CELLebrity Doctors calendar is now available for purchase from www.CELLebrityDocsCalendar.com. All proceeds from calendar sales benefit the Sabrina Cohen Foundation for Stem Cell Research, a 501c3 non-profit organization directly funding stem cell clinical research.

--Lee

_________________________________________________________________

On the night of November 12, David Porosoff’s Artrageous Gallery hotspot was converted into something likely never imagined -- a hotbed of stem cell research. Sabrina Cohen fused the vividly artistic backdrop and venue with gambling, cuban music, great food, and beautiful people all to further her mission of raising money and awareness for stem cell research.

Dr. Sally Temple with Sabrina Cohen

Dubbed the Havana Casino Night, the event had several highlights including the granting of the 2011 Sabrina Cohen Foundation award to stem cell researcher Dr. Sally Temple.

Representing the 3rd recipient of the annual SCF award, Dr. Sally Temple is studying how neural progenitor cells may be employed to create cell-based therapies for neurodegenerative disorders. Dr. Temple is the co-Founder and Scientific Director of the Neural Stem Cell Institute located in Rensselaer, NY. NSCI is the first independent, non-profit stem cell research institute in the USA.

The night, sponsored in part by DMR, Evensky & Katz and Harke Clasby & Bushman, raised $10,000 which will be dedicated toward next year’s SCF Award for Stem Cell Research.

The event also marked the lauch of the the Foundation’s 2012 CELLebrity Doctors Calendar, this year featuring women in the field of stem cell research. The calendar features academics, industry executives, physicians, and advocates primarily from the United States but also representing Sweden, Australia and Canadian covergirl, Dr. Fiona Costello.

“In science you don't have to accept anything anyone tells you, you can come up with a hypothesis and test it yourself. And you can be the first one to do it,” says Dr. Costello, whose research focus is on multiple sclerolsis and other impairments of the central nervous system.

“Stem cell science is often accused of being ‘hyped,” says Cohen, “but that doesn’t necessarily translate into monetary support for or society recognition of the enormous contributions made by stem cell resarchers. They often toil in anonymity making significant discoveries at great personal sacrifice. I consider it my job to find a way to financially support their work and bring profile to them as people.”

The ‘CELLebrity’ Doctors Calendar
The 2012 CELLebrity Doctors calendar is now available for purchase from www.CELLebrityDocsCalendar.com. All proceeds from calendar sales benefit the Sabrina Cohen Foundation for Stem Cell Research, a 501c3 non-profit organization directly funding stem cell clinical research.

The Sabrina Cohen Foundation
The Sabrina Cohen Foundation for Stem Cell Research (SCF) is an internationally recognized nonprofit organization dedicated to building a global network of top scientists and clinicians in the field of Regenerative Medicine, while simultaneously funding cutting edge research and innovative therapies that will reverse spinal cord injury and effectively treat other impairments of the Central Nervous System.

Sabrina Cohen is the Executive Director and President of the foundation. She graduated from the University of Miami with a degree in Communications, double majoring in Advertising and Psychology, and holds a post-graduate degree in Copywriting from the Miami Ad School. She is a C5 Quadriplegic, as the result of a spinal cord injury from a car accident in 1992. In 2006, she established SCF to raise funds for research because she believes the field of Regenerative Medicine will lead to the greatest advances of our time. Sabrina is a Motivational Speaker & Spokesperson continuously speaking in schools, universities and community centers. She has spoken at scientific conferences around the country, including the "World Stem Cell Summit" at the University of Wisconsin, Harvard University, Stanford University, Baylor College of Medicine at the University of Texas, and at the United Nations. Sabrina believes her wheelchair is a vehicle to promote change.

Sabrina Cohen was recognized by WebMD Magazine as a 2009 "American Health Hero”. Sabrina is currently available for interviews highlighting the 2012 “CELLebrity” Doctors Calendar.

Commercializing Cell-based Regenerative Medicines

noreply@blogger.com (Lee Buckler) — Tue, 08 Nov 2011 18:48:00 +0000

When introducing a regenerative medicine cell based product to a commercial setting, there are a host of things to take into consideration to ensure a commercially viable and safe product for patient use.

In this QandA interview by Pharma IQ, William Fodor, Director of Translational Sciences, Cell Therapy Group, gives some teasers into a few of issues to keep in mind relative to commercial manufacturing scale‐up of cell therapies.

Listen to the podcast here (registration required) or read the transcript below:

Pharma IQ: Can you give some advice on the best way for a company to develop standards for
commercialization to improve safety?

William Fodor: Well, with any biological product, you have to do all the appropriate testing and there’s really no standards necessarily to be developed by the company because the regulatory process is pretty well outlined by the FDA and the CBER Division and cell therapy products are regulated by the office of Cell Tissue and Gene Therapy Division. So, it’s not that you need to develop standards for commercialization to improve safety. You need to follow the regulations involved by demonstrating to the FDA that your product is safe, and maintains the identity, in other words, your product doesn’t change during your regular manufacturing process. Purity and then potency are all assays that need to be developed within the manufacturing process for your particular cellular product.

Pharma IQ: And what are some approval processes and pitfalls to be aware of within the
scale‐up process?

William Fodor: So as you are scaling up, you absolutely need to maintain current good
manufacturing practices ‐ it’s known as cGMPs. Typically, during a phase one, you can get
away with certain reagents that may not be fully GMPs. Or in other words, if you use a growth
factor or a certain media that doesn’t have or isn’t manufactured under full GMPs, as long as you test that particular reagent or media that you are using to ensure safety and sterility, you can typically get away with that in the phase one clinical trial process. But when you move to a phase two, you need to make sure that all your reagents and medias and any compounds that come in contact with your product are all manufactured under good cGMP.

Pharma IQ: What are some technology transfer and patent protection concerns to be
cognizant of?

William Fodor: Well, with any cellular‐based product, if there’s a technology that is out there
that a company wishes to pursue, to improve yield, or the manufacturing process, you need to
demonstrate that that technology fits within your manufacturing process. So typically, what is
done is you’ll do validation runs to ensure that that new technology satisfies the regulatory
process for your manufactured product. With respect to patent protection, again, that company needs to maintain their IP portfolio and needs to make sure that they’re not infringing other intellectual property and that’s just standard for the industry.

Pharma IQ: And do you have any tips for ensuring quality and consistency no matter how little
or how much one is producing?

William Fodor: Yes, when you manufacture a cell‐based product, it’s not that much different than any other biologic product. And so, whenever you do manufacture, whatever scale it is, you have to ensure safety, and that’s sterility, tests for microplasma, or other adventitious agents; things like bioburden and endotoxins, so all those tests need to be performed. You need to have an identity test to make sure that your cell product ,whatever scale your manufacturing is, that at the end of that manufacturing run, the product hasn’t changed. Again, no matter what scale you’re at, you need to make sure the identity of the product is
consistent from batch to batch.

For identity, you can do a number of things, and again, for a cell‐based product, if you want to look at cell surface antigens to ensure that the cell surface proteins on your cellular product don’t change over time or through your manufacturing process. And typically, what you like to do is keep it relatively simple. You don’t want to test for a hundred things because you’re just asking for the potential for something within those hundred things to change. So typically, what you do is maybe three to four cell surface antigens to ensure your product identity is consistent and you can also do PCR to determine that an intracellular protein of interest doesn’t change during your manufacturing process.

You also need to ensure for purity, so you want to quantitate your active cell or your tissue type. And then potency; you need to demonstrate the product has a consistent potency and the biological activity of that final product doesn’t change during the manufacturing process. And then typically, what you do is you archive. You archive samples from during your manufacturing process. You cryopreserve those so you can always go back to ensure that that a particular batch was consistent with other batches that were manufactured.
...

Join Dr. Fodor and other industry leaders in Philadelphia, December 12th and 13th 2011 for the IQPC Commercialization of Regenerative Medicine Summit. For more information or to register, visit
www.regenerativemedicinesummit.com.

Cell Therapy & Regenerative Medicine Domains Available

noreply@blogger.com (Lee Buckler) — Fri, 23 Sep 2011 21:10:00 +0000

Please pardon the crass commercial nature of this post. I try to keep self-promotion to a minimum here but I'm looking to unload a number of domains I have the related to cell therapy and regenerative medicine and thought this might be the easiest way to get the word out. Let me know if you are interested in any of the following:

anticyte.com*
biocellutions.com***
.
cardiacregenerativemedicine.com*
cardiacregenmed.com*
cardiactissuerepair.com*
.
cellexpertsolutions.com**
.
celltherapyalliance.com*
celltherapyassays.com***
celltherapycellutions.com****
celltherapycompany.com****
celltherapyconsortium.com*
celltherapydevelopment.com***
celltherapymanufacturing.com****
celltherapymarketing.com*
celltherapypartner.com*
celltherapypatents.com****
celltherapysolution.com***
celltherapystrategies.com***
celltherapysupport.com*
celltheraytech.com**
celltherapytechnologies.com***
celltherapyventures.com***
celltherapyworks.com***
.
cellutionstrategies.com***
.
commerciaizingcelltherapy.com**
commercialisingcelltherapy.com*
commercializingcelltherapies.com**
commercialisingcelltherapies.com*
.
cordbloodshipper.com***
cordbloodshipping.com***
.
developingcelltherapies.com*
.
diagnostacell.com*
diagnostacells.com*
diagnosticstemcells.com**
.
discoverystemcells.com**
.
expertcellutions.com***
.
integratedcelltherapycellutions.com**
integratedcelltherapysolutions.com**
.
ipsbiologics.com**
ipsbiomedical.com**
ipsbioscience.com**
ipsbiosystems.com**
ipsbiotechnologies.com**
ipslifesciences.com**
.
longtailbiomedical.com***
longtailtherapeutics.com***
longtailtherapies.com***
.
personalizedcelltherapy.com***
.
regenerativecellutions.com***
regenerativemedicinepatents.com****
.
stemostics.com**
stemomics.com**
stemonics.com***
.
stemcellcollect.com***
.
strategiccellutions.com**
strategicell.com***
.
thecelltherapycompany.com****
.
totalcelltherapysolution.com***
totalcelltherapysolutions.com***

Funky ones:
cellenterprise.com*
cellsforce.com*
cellsinnovations.com*
cellsmarket.com*
cellspitch.com*
cellsrep.com*
cellstech.com*
cellsventure.com*
iregener8.com**

I have others - some of which are variations of ones listed above (such as with "the" in front) and would be available - some of which I'm still wanting to hold.
_____________________________

* ~$1,500
** ~$5,000
*** ~$10,000
**** ~$20,000

Commercial-stage Cell Therapy Companies and Products

noreply@blogger.com (Lee Buckler) — Fri, 16 Sep 2011 22:05:00 +0000

2012-01-23 update

Below is sample list of companies with cell therapy products* on the market in Europe, USA, or Japan.

Company Product
Advanced BioHealing (now part of Shire) Dermagraft
Altrika MySkin
Altrika CryoSkin
Alphatec Spine PureGen
Amedica BioDfactor
Arthro Kinetics                                           CaReS
Avita Medical ReCell® Spray-On Skin
Bio-Tissue Prokera
Bio-Tissue AmioGraft
BioTissue Technologies BioSeed-C
BioTissue Technologies chondrotissue
Cytori Celution System
Dendreon Provenge
euroderm Epidex
euroderm EpiGraft
Fidia Farmaceuitici Hyalograft 3D
Fidia Farmaceuitici Laserskin
Fidia Farmaceuitici Hyalograft C
Fibrocell laViv
Genzyme (a Sanofi company) Epicel
Genzyme (a Sanofi company)    Carticel
Genzyme (a Sanofi company)    MACI Implant
Japan Tissue Engineering Co. J-TEC Epidermis
Japan Tissue Engineering Co. J-TEC Cartilage
Japan Tissue Engineering Co. J-TEC Corneal Epithelium
Living Cell Technologies DIABECELL
NuTech NuCel
NuVasive Osteocel Plus
Organogenesis Appligraf
Orthofix Trinity Evolution
TiGenix ChrondroCelect
Therakos Therakos Photopheresis

Most of these are only available in one or a few jurisdictions. Sanofi's Genzyme products (Epicel & Carticel) are two of the few which are multi-nationally available.

South Korea is an interesting example of a country with a mature regulatory framework with more than its share of approval. There have been 14 cell therapy products approved for sale in South Korea. They are as follows:

Company Product
Anterogen                                                  Adipocel
Anterogen                                                  Cupistem
Antrogen Queencell
Cellontech RMS ossron
Cellontech Chondron
Chabiotech Hyalgraft-3D+
Chabiotech Autostem
Creagene Creavax-RCC+
FCB-Pharmicell Heartcellgram-AMI
Innocell Immuncell-LC+
Innomedisys Innolak+
MCTT Keraheal+
Medipost                                                     Cartistem
NKBio NKM+
S-Bio Medics Cureskin
Tegoscience Holoderm
Tegoscience Kaloderm

+ These products were brought to market in 2006-7 under a conditional approval unique to South Korea's regulation that allows commercial sale in certain instances while a pivotal trial is underway.

At present, the Korea-FDA is assessing a Biologics License Application (BLA) for CARTISTEM® being developed by Medipost. Once approved, CARTISTEM® will become available as the World's first hUCB-MSC derived adult stem cell drug for treating osteoarthritis and will be marketed in Korea in association with Dong-A pharmaceuticals

* This list does not purport to be exhaustive of all cell therapy products legally sold in these regions. This list does not include approved products in other highly-regulated jurisdictions, such as Australia, New Zealand, or Singapore, for example. This list also excludes those cell-based treatments provided as a hospital or clinic-based service such as stem cell transplantation (hospital) or Regenexx (Regeneration Sciences, Inc.).

For the purposes of this list, “cell therapy” is defined loosely as any product which has in it live cells when administered to the patient including tissue transplants and devices.

Note that some of these products may be subject to emerging regulatory restrictions under the EMA ATMP regulations which may result in them having to be pulled from the market by the end 2012 at the latest.

If you would like to suggest any revisions or additions to this list, please do so in the comment section below.

Cell Therapy Industry Video Channel

noreply@blogger.com (Lee Buckler) — Fri, 16 Sep 2011 21:46:00 +0000

Cell Therapy Group and BioBusiness.TV are pleased to announce they are collaborating to build a "cell therapy" video channel focused on the cell therapy industry. The initiative is part of BioBusiness.TVs "Best of the Web" initiative intended to bring together publicly available video content that complements their original content.

The Cell Therapy channel will focus on cell-based therapeutic products which are in clinical development by or are commercially available from companies around the world. We will update this channel frequently.

To view other video content, see Cell Therapy Group’s YouTube channel.

To recommend video content, submit your comments below.

Potential far-reaching implications of the ongoing fight over point-of-care autologous cell therapy

noreply@blogger.com (Lee Buckler) — Thu, 15 Sep 2011 21:51:00 +0000

The FDA recently issued an untitled letter to Parcell Laboratories and its contract manufacturing organization, New England Cryogenic Center (NECC), pertaining to the product PureGen™ Osteoprogenitor Cell Allograft intended for the "repair, replacement, reconstruction of musculoskeletal defects". The letter stated:

"The PureGen™ Osteoprogenitor Cell Allograft is not the subject of an approved biologics license application (BLA) nor is there an IND in effect. Based on this information, we have determined that your actions have violated the Act and the PHS Act."

This would appear to be an indicator that the FDA does not intend to relax enforcement of its view of how autologous cell therapies are to be regulated despite its ongoing litigation on this very subject with RSI.

As followers of this blog know, since the battle's inception in 2008 I have followed the case of Regenerative Sciences, Inc and their war with the FDA over their right to provide certain autologous cell therapy treatments to patients in certain circumstances without FDA approval. My first blog entry on the topic was in September 2008 in which I pointed out that the FDA had written a letter to RSI that July taking issue with some of their practices.

My next blog on the subject was February 2009 in which I concluded "I think the FDA is building its case and a showdown is on its way to Denver-town."

At the advice of legal counsel, I pointed out in a March 2010 blog entry that my reference to FDA's July 2008 letter to RSI was not officially a "warning letter" as that is defined and as I had referred to it but rather an "untitled letter". I also commented that FDA's lack of enforcement action against RSI to-date was emboldening medical practitioners into thinking FDA was reconsidering their position on the legality of providing autologous, expanded cells to patients outside of an FDA-cleared IND or BLA.

I took some satisfaction in announcing on my blog in August 2010 that the FDA had finally taken action against RSI. My satisfaction was not rooted in a belief that the FDA is right (I've always been agnostic as to which side is right) but in the sense that things had occurred as I had predicted they would.

The action the FDA chose to take against RSI was to seek an injunction against RSI from continuing to provide the "offending" treatment - a version of the Regenexx™ procedure using mesenchymal stem cells (“MSCs”) grown outside the body after harvest for the later infusion back into the donor-patient for the treatment of various orthopedic conditions - which the FDA alleges is a "product" falling under its regulatory authority but for which RSI has never received any FDA clearance to provide to patients.

RSI counterclaimed against the United States, challenging the FDA’s authority to regulate the Regenexx™ Procedure in question and challenging certain FDA regulations. The United States moved to dismiss Defendants’counterclaims and for summary judgment.

I have continued to follow the case relatively closely through a number of source (see this sample media coverage in OthosSpineNews) as it continued to progress. Last month, for example, a blog I follow posted an eloquent case in support of RSI's position with the help of Mary Ann Chirba, J.D., D.Sc., M.P.H. of Boston College Law School.

So it was with much interest that I was recently notified of an order issued by the court which appears to have the potential to take the case in a very unexpected direction with enormous potential ramifications.

The context is that the judge was reviewing the FDA's motion for summary judgment, RSI's response, and FDA's reply when the judge issued this order to show cause.

At its essence the Judge has ordered the FDA to file a brief no later than 26 September showing the court why the term “chemical action” applies to stem cells. It is is a short Order the core of which reads as follows:

The Government finds its definition for a “drug” in the FDCA: “The term ‘drug’means . . . articles intended for use in the diagnosis, cure, mitigation, treatment, or prevention of disease in man or other animals” and “articles (other than food) intended to affect the structure or any function of the body of man or other animals.” 21 U.S.C. § 321(g)(1)(B)&(C).

This definition, at least as to subsection (C), would be broad enough to encompass a boot on a patient’s ankle to hold it secure after ankle surgery. The Court doubts that was Congress’s intent.

Neither party references the definition for “device,” found in the statute at 21 U.S.C. § 321(h). A “device,” is a certain kind of “article” used in diagnosis, cure, mitigation, treatment or prevention of disease, 21 U.S.C. § 321(h)(2), but which, presumably unlike a drug, “does not achieve its primary intended purposes through chemical action within or on the body of man or other animals and which is not dependent upon being metabolized for the achievement of its primary intended purposes.” 21 U.S.C. § 321(h).

These contrasting definitions immediately raise the question of why the Court should not interpret the meaning of the word “drug” to include not only an article for use in diagnosis, etc., and intended to affect the structure or function of a patient, but also an article that “achieve[s] its primary intended purposes through chemical action” and which is “dependent upon being metabolized for the achievement of its primary intended purposes.” Id.

The United States is ORDERED TO SHOW CAUSE why the Court should not read the definition of “device” at 21 U.S.C. § 321(h) as informing and restricting the definition of “drug” at 21 U.S.C. § 321(g)(1)(B)&(C).

It will be most curious to see how the FDA argues out of the corner that many believe the Judge has painted the agency. The FDA recently defined “chemical action” in its draft "Guidance for Industry and FDA Staff: Interpretation of the Term “Chemical Action” in the Definition of Device under Section 201(h)of the Federal Food, Drug, and Cosmetic Act". What is curiously absent from the document is any mention of cells or HCT/P’s despite CBER’s approval stamp on the document.

Another line of argument centers around whether cells - notably 'stem' cells - are “metabolized" as that term is defind.

If one extrapolates the ramifications of where the court appears to be currently leaning, the implications of this judgment may have far-reaching implications for biologics in general well beyond cell therapy and certainly well beyond autologous cell therapy.

As some quite logically argue, one potential scenario is that this judge rules all biologics fail to fall within the legislative "drug" definition. The argument goes like this. The drug regulations live under title 21, which has narrow definitions for what constitutes a drug. The FDA’s authority over biologics comes from title 42, which is merely to control communicable disease transmission in transplants, with no authority to take the drug provisions from title 21 and apply them to title 42. So the agency is risking a loss of control over all biologics.

What’s curious here - and perhaps somewhat ironic for RSI at this stage - is that they only ever set out to challenge their ability to regulate autologous cells used by a physician as part of his or her medical practice yet now the FDA's authority to govern all biologics is currently under question.

The fact that I cannot fathom the courts striking FDA's jurisdiction over all biologics when the dust settles on this case does not make the arguments any less compelling and it does leave open the possibility that a lower court Judge such as the one presiding over this case may be inclined to make a ruling which essentially ensures the issues are punted to the appellate courts for a more considered ruling. In such circumstances, even if the FDA were to prevail at the end of the day (perhaps a decade down road) the uncertainty such a ruling would rain down on the sector would be commercially stifling - even if if were just limited to autologous cell therapy let alone if were any broader.

My dated and unpolished law degree can only take this analysis so far and anyone interested in some further but delightfully light and practical reading on the potential ramifications of the case could do no better than read a paper published recently by the relevant practice groups at the law firm K&L Gates entitled "Cultured Stem Cells for Autologous Use:Practice of Medicine or FDA Regulated Drug and Biological Product in which they review the case and its potential implications - the latter of which the authors are not guilty of underestimating in the following concluding sentence of their analysis:

The court’s decision will, to a large degree, dictate the types of legal strategies and business models that will be necessary to successfully perform stem cell procedures in the future.

_____________

post-script: The potential stink of commercial uncertainty wafting from this case is even more egregious when combined with the uncertainty around what to expect from the FDA in its much-anticipated and typically overdue guidance on adipose-derived cell therapies.

Rumor has it that the FDA is leaning toward considering most (if not all) means of deriving cell populations from adipose tissue (typically lipoaspirate) to be governed as what we colloquially refer to as a '351' thus taking it out of the purview of the practicing physician and into the hands of companies prepared to follow the traditional "drug development' model for new medicines. The rationale here is that the mechanical and/or enzymatic digestion required to separate the desired cell populations from the stroma take the process beyond "minimal manipulation'.

Watch for this guidance from CBER OCTGT in the weeks to come and/or any relevant rulings by the Tissue Reference Group. This would be a serious blow to those building business models around point-of-care, autologous adipose-derived cell therapy treatments.

What makes this even more interesting is the pace of which US-based medical practitioners (and/or companies supporting them) are adopting and selling autologous cell-based products, services and/or treatments for sundry indications in ways which many would argue are apparently in obvious and flagrant disregard for the FDA's regulatory authority over such treatments. Included for consideration on such a list would be the following:

IntelliCell Biosceinces

eternaMD

Arizona Stem Cell Center

.

Good Data? $100. Good Product Development? $100. Good Commercialization Strategy? Priceless.

noreply@blogger.com (Lee Buckler) — Wed, 10 Aug 2011 23:54:00 +0000

I'm not going to fool anyone into believing I'm a therapeutic product development expert but that's not going to stop me from making a few humble observations in light of the Dendreon "fiasco" of last week which I have no doubt will one day be considered an unfortunate pothole on their road to eventual success.

(though perhaps not before certain current management finds themselves polishing their CVs or retiring to spend their time alternating between their yachts and the courtroom defending their questionable stock trading antics)

I received the following message this morning by email. I don't include it here to promote or endorse or even comment on NWBT, DCVax, Linda Powers, or Toucan Capital in any way -- or in a way that is blind to all the things right or wrong about any of them -- but simply to illustrate the 3 points I want to make below the email.

NWBT HIGHLIGHTS COST EFFECTIVENESS OF DCVAX® IN VIEW OF RECENT IMMUNOTHERAPY PRICING CONCERNS

Northwest Biotherapeutics' (OTC.BB: NWBO)... DCVax® immune therapies for a broad range of cancers (including prostate, brain, ovarian and others) hold the promise, based on available data to date, of being cost effective and priced below other immune therapies while still providing substantial profit margins for the Company and longer survival for patients.

The investor concerns in the news relate to the pricing and reimbursement of Provenge for late stage, metastatic prostate cancer. Provenge is priced at $93,000 for one month of treatment and was approved by the FDA based upon having added 4.5 months of patient survival (to reach overall survival of 25.9 months).

NWBT’s DCVax® will be priced in the range of $37,000 per year for up to 3 years of treatments. In NWBT’s Phase I/II multi-center clinical trial in late stage, metastatic prostate cancer, DCVax® added 18 months of patient survival (to reach overall survival of 38.7 months). DCVax® has previously been cleared by the FDA for a 612-patient, randomized, controlled Phase III trial, although the trial has not yet begun. As is typical before a Phase III trial, the manufacturing processes and product costs have already been determined.
...
...

The key to the substantial pricing advantage of DCVax® is NWBT’s proprietary batch manufacturing process together with its cryopreservation technology for frozen storage of the finished vaccine. NWBT has spent a decade developing and improving its manufacturing and cryopreservation processes. The manufacturing of personalized, living cell products is expensive. But the frozen storage of living cells is quite low-cost – once the specialized freezing technology is worked out for a particular type of cells (the culture conditions, rate of freezing, density of cells and many other factors).

NWBT’s manufacturing methods produce – in a single manufacturing run – a large batch of personalized DCVax® product for 3 years of treatments are much less costly than separate manufacturing runs for each treatment. The technology for freezing the master immune cells (dendritic cells) which comprise DCVax® enables these
cells to remain frozen for years and, when needed, to be thawed and “come back to life” with full potency.

This approach makes DCVax® an "off the shelf” product [for that patient] for several years of treatments after just one manufacturing run. In contrast, Dendreon must do a separate manufacturing run for each one month of treatments. In addition, Dendreon's Provenge product is fresh and not cryopreserved, which limits its shelf life to at most a few weeks.

Another important factor in the cost effectiveness of DCVax® is its simplicity and ease of administration. DCVax® is delivered as a small intra-dermal injection under the skin, similar to a flu shot. As such, it can be administered in any physician’s office or clinic. There is no lengthy intravenous infusion, with the attendant patient discomfort, cost and need for a specialty infusion center. In contrast, Dendreon’s Provenge is delivered by intravenous infusion.

The cost effectiveness of NWBT’s DCVax® is enhanced by the fact that DCVax® is targeting a portion of the prostate cancer market that is 4 times the size of the market segment that Dendreon’s Provenge is currently targeting....

Now this is NWBT clearly blowing their horn - nothing wrong with that - in an attempt to woo back frightened investors. I'm agnostic as to whether any of it is true but it does serve to draw out several points of distinction between what some companies might do to optimize their products for commercial success versus what others might do in an overriding belief that clinical benefit is the only precursor to the happiness of investors, the physician community, patients, and partners.

What I say below is not a commentary, in criticism or praise, on any particular company including NWBT or Dendreon. Many others - Luke Timmerman among the best of them - have provided outstanding and in-depth analysis of the Dendreon story along the way.

I'm only interested in what we as an industry might learn from recent experiences or trends and to perhaps facilitate a useful discussion based on 3 simple observations from someone who has swum in this cell therapy pond for now just over a decade:

1. There is a tendency among some to believe that what we are making (cell-based therapies) are so revolutionary and compelling that the products should almost sell themselves - to investors, partners, regulators, insurers, physicians, and patients.

Of course we know it's not true but sometime we act like it is.

Appligraf and Dermagraft didn't sell themselves to physicians even after regulatory approval. Neither is Provenge apparently-- though they did a good job of selling it to very vocal patient groups. Organogenesis and Advanced Biohealing had to work very long and hard to get profitable reimbursement and market penetration. Dendreon will too.

Investing early in understanding potential clinical adoption hurdles, reimbursement issues, and how the product is and will be perceived not by its champions but by its critics and most importantly, the average agnostic practitioner, is not easy to do because it means spending precious resources long before there is a product to sell but it may mean the difference between a product which eventually sells and one which doesn't.

2. There is a tendency to de-emphasize what I call the "ancillary sciences" around a product -- like lowering the cost of goods, optimizing fresh or frozen storage, cell delivery (e.g., injection/application science) mechanisms, in vivo cell tracking, onsite clinical handling, etc.

Product development science is a science. The science that turns good data into something commercially viable. Not everyone is good at it and certainly this is where a lot of companies fail. PD is not the 'second cousin' in the room of esteemed basic and clinical science.

Take these examples when considering the cellular immunotherapy sector:

Several prominent immunotherapy investigators I have spoken with strongly believe cell-based immunotherapies will require long-term administration to be meaningfully effective -- certainly longer than 3 doses in 3 months. Did Dendreon lock into their clinical protocol too early?

Other immunotherapy companies - like Opexa Therapeutics for instance - create multiple doses for a patient from a single patient collection thus saving considerable expense and creating a better patient experience. Did Dendreon lock into their manufacturing protocol too early?

Other companies are investing heavily in finding ways to extend viable shelf-life of their fresh products or to create cryopreserved versions of their product to optimize its commercial viability.

Imagine a Dendreon that only had to build one manufacturing facility (with a backup CMO) to serve the US market rather than 3 facilities. With a cryopreserved version of the product or a version that had longer than its current limited shelf-life (~72 hours I believe?) that might have been possible.

At every point in clinical development there must be concurrent R&D towards the product's:

science (e.g., MOA, characterization, etc),
clinical effect, and
how to optimize its commercial viability - a big part of which is what we think of as 'product development'.

This is the 3-legged footstool of a commercialization strategy geared for success (credit to Bob Preti of Progenitor Cell Therapy for this analogy).

But 'product development' is also not always about the product strictly speaking and even an expansive definition of product development is only part of a good commercialization strategy.

There is significant component of it that is about studying ways to lower the cost of goods, improving manufacturability and scalability, how hard/easy it is to handle, the patient treatment experience, the physician experience, how it impacts patient's QOL (quality of life), not just what side effects it generates but what side effects it prevents (resulting from other treatments or no treatment) and the cost-savings that generates, etc.

Other cell therapy companies have been more proactive in terms of engaging not only KOLs but average practitioners in a meaningful way that might impact their product and clinical design as well as reimbursement and clinical delivery issues.

3. There is an understandable, largely VC-driven, desire to race forward to the next trial phase when a phase-repeat to optimize or better understand different aspects of the product might be the better way to go.

What's worse? Facing the prospect of not being able to get funding or a partner on the terms one wants for a 2nd phase II or burning through a bunch more money in phase III in an attempt to bring a product to market that isn't market ready? I understand its a tough choice -I'm not saying it's an easy one - but one is certainly more strategic and, as pharma says, is certainly a "de-risking" pathway.

Consider the ratio of products we've seen thus far in the cell therapy industry's short life-span that have been raced to phase III or (worse yet) market only to seriously stumble if not fail when they get there. I can think of 8-10 off the top of my head and there are less than 20 cell or tissue based therapies on the market in US/EU that have received any kind of formal regulatory approval.

Some would argue that this is a prime example of why spinning companies out of academia too early is not beneficial because companies want to minimize exploratory science and lock into a "product" too early. I would argue that this may be true if the problem is understanding the product characterization or mechanism of action but not if your problem is related to how best to develop/optimize the product for commercial viability. Few academics are geared to think this way.

Summary

I certainly don't believe Dendreon failed to identify or consider each and every one of the things they might have done better along the way. I'm sure they did and sure they made calculated judgement-calls about how to approach each one.

Since I'm not a shareholder I don't have to worry myself about being critical of their decisions but rather simply to do what I can to ensure that we as an industry do our best to learn from what - with the benefit of retrospect - may be apparent they did right and wrong.

What are your thoughts? To what extent are the problems that Dendreon has experienced along the way with PROVENGE a predicable result of it being a first-generation product or the result of insufficient focus on critical investigation into the less sexy "ancillary sciences" of product and commercial optimization?

(comment below and/or in the LinkedIn Cell Therapy Industry Group)

Clinical trial costs

noreply@blogger.com (Lee Buckler) — Wed, 27 Jul 2011 21:53:00 +0000

Based on a survey* of 21 drug makers, 12 biotechs, nine device makers and 23 contract research organizations, PharmaLot has recently published the following metrics for current clinical trial costs (see full article here)

Average per-patient trial costs across all therapeutic areas:

Phase I: $21,883

Phase II: $36,070

Phase IIIa: $47,523

Phase IIIb: $47,095

Phase IV: $17,042

Average cost per patient for a cardiovascular trial:

Phase II: $33,700

Phase IIIa: $21,750

Phase IIIb: $6,830

In oncology, the average per patient trial cost:

Phase II: $73,303

Phase IIIa: $57,207

Phase IIIb: $65,900

For central nervous system disorders:

Phase II: $28,197

Phase IIIa: $33,768

Phase IIIb: $41,824

For diabetes:

Phase II: $ 8,854

Phase IIIa: $12,667

Phase IIIb: $10,700

Anyone have any thoughts or data as to how this compares to cell therapy trials?

--Lee

*survey conducted by Cutting Edge Information.

Cell Therapy's Got Talent Technology Showcase - A Call for Cell Therapy Manufacturing Technology Presentations

noreply@blogger.com (Lee Buckler) — Thu, 07 Jul 2011 02:24:00 +0000

in collaboration with:

In an effort to showcase the latest technologies driving the production of cell therapies, the Cell Therapy Group and Informa Life Sciences are proud to announce the introduction of the "Technology Showcase" session and award to be held in conjunction with Informa's Cell Therapy Manufacturing conference to be held 30 November to 1 December 2011 in Brussels Belgium.

Having held the same conference last year in London, Informa is committed to building on the success of last year's event by continuing to create a meaningful European forum for the issues related to the clinical and particularly commercial-scale production of cell-based therapies.

The Technology Showcase session, taking place on the main agenda, will feature 6 x 10 minute presentations from innovative companies developing cutting-edge technologies in the field of cell therapy manufacturing, and is particularly relevant to SME and academic groups with limited marketing resources.

All presentations will be reviewed by the Scientific Advisory Board with the winner announced at the end of the session. Exposure on BioProcess International's website is also included.

Technologies we'd like to promote include:

Manufacturing systems including bioreactor technologies
Cell harvest/collection technologies
Cell storage/logistics technologies
Clinical cell delivery and/or other point-of-care technologies
Automation technologies
Cell separation system
Cell process devices
Innovative reagents, scaffolds, matrices, and other “ancillary” tools
Technologies to close currently open systems
Suspension-based production systems
Disposable technologies

How to apply:

To apply to present companies must submit an abstract (<300 words) to daniel.barry@informa.com and lbuckler@celltherapygroup.com outlining the product or service to be presented and why it is a critical technology related to cell therapy manufacturing.

The deadline for applications is SEPTEMBER 15 2011 - Priority given to early submissions

The cost of taking part in the Technology Showcase is £2,700 which includes the following benefits:

1 x 2-day conference pass (normal price £1,599)
10-minute podium presentation within main conference room
1 poster display in the Exhibition Hall
Marketing - company logo displayed on website and event guide
Exposure in BPI Magazine

Terms and conditions:

To be eligible the product or service to be presented must be:

On the market for no less than 2 years or expected to be on the market no later than Q4 2012
Appropriate for, applicable to, and compliant with clinical-grade manufacturing requirements (technologies only available for research use will not be considered)

Plus...

The company must have no more than 15 employees
The company has been running for no more than 5 years, and
The company generates annual revenue of no more than $5m

For further information please contact: daniel.barry@informa.com or lbuckler@celltherapygroup.com

In vivo cell trafficking just took a leap forward

noreply@blogger.com (Lee Buckler) — Mon, 27 Jun 2011 15:49:00 +0000

Today Celsense, Inc. and the University of Pittsburgh Cancer Center announced that the FDA has authorized the use of the Cell Sense imaging reagent for use in a phase I clinical trial of a dendritic cell caccine to treat colorectal cancer patients.

This is the first FDA authorization of the use of Cell Sense in patients. Cell Sense is a novel perfluorocarbon tracer agent used to safely and efficiently label cells ex vivo without the use of transfection agents. Labeled cells are then transplanted into the patient enabling researchers and clinicians to non-invasively track the administration and migration of therapeutic cells using MRI. Applications include tracking cells in immunotherapy or regenerative medicine as well as the diagnosis of inflammatory sites by tracking selected populations of immune cells.

Cell Sense has been studied extensively in preclinical testing with many different human cell types including human cells in animals. For instance, in 2009 a paper was published in Informa's Cytotherapy, in which Celsense’s novel perfluorocarbon tracer agent (product “Cell Sense”) was used to label human DCs ex vivo for the purpose of tracking the cells in vivo post-transplant by 19F MRI. The paper provided an assessment of the technology and demonstrated that human DCs were effectively labeled without significant impact on cell viability, phenotype or function. Furthermore, the labeled dendritic cells were clearly detected in vivo by 19F MRI in a model system, with the labeled cells being shown to migrate selectively towards draining lymph node regions within 18 hours after transplant.

Many investigators looking at various ways to label cells to enable in vivo imaging have expressed concern that the FDA would delay the regulatory progress of their therapeutic candidates if an imaging modality was introduced.

This concern is based on numerous reports of MRI contrast reagents, such as the commonly investigated USPIO (ultrasmall superparamagnet iron oxide), deleteriously affecting the cells (see recent paper in Cell Transplantation).

"We believe that the authorization of this IND will alleviate such concerns and lower the barriers for adoption. The agency’s tangible support for bringing new technologies to bear in the translation of cell-based therapeutics is very encouraging,” s Charlie O'Hanlon, President and CEO of Celsense.

While there have been approved uses of imaging reagents (e.g., Feridex, etc) with cell therapies in other countries (e.g. Isreal), I believe this may be the first FDA-sanctioned use of a particle-based imaging label with a cell-based therapy. Other approaches to cellular imaging include nuclear imaging reagents and genetically modifying cells with reporter genes such as those provided by CellSight Technologies.

Imaging labels are capable of providing investigators with data demonstrating where the cells go, at what volumes, and for how long they stay at the target location.

The industry has been keen to see these kinds of technologies clinically employed but different cell-based labels have created their own technical, clinical, and/or regulatory hurdles. I'm hopeful that Celsense and others like them are now ushering us into a new era where we will eventually be able to use various technologies to monitor and collect valuable data concerning cells after they have been administered as a therapy to a patients.

Additional resources on the topic of imaging for cell therapies:

CIRM recently hosted a webinar - "CIRM/RMC Webinar: Imaging Technology for Cellular Therapies. One of the speakers, Dr. Shahriar Yaghoubi from CellSight Technologies, provides an overview of cell therapy imaging with emphasis on PET. Click hear for the archived playback.

A very interesting article posted today on Harvard's StemBook website. "In-vivo Stem Cell Imaging - Regulatory Challenges and Advances". Nice overview intel from J. Bulte and a snapshot into E. Wirth's (of Geron) perspective re: stem cell imaging.

A new book from CRC Press edited by Dara Kraitchman and Joe Wu will be out soon. It gathers together different methods for comparison. The issue will remain the sensitivity of the methods to track few cells. "Stem Cell Labeling for Delivery and Tracking Using Non-Invasive Imaging".

MRI contrast agents can change stem cell proliferation

There s also a very informative discussion thread on the topic in the Cell Therapy Industry group on LinkedIn.

Cell Therapy Conferences

noreply@blogger.com (Lee Buckler) — Sun, 19 Jun 2011 18:22:00 +0000

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For those of you have been asking, we've now created what we hope is the most comprehensive and current listing of conferences in cell therapy and regenerative medicine. Check it out here and let us know if you believe any should be added.

Cell Therapies: Commercializing a New Class of Biopharmaceuticals

noreply@blogger.com (Lee Buckler) — Thu, 03 Feb 2011 21:05:00 +0000

Over the past six months I have been honored and pleased to have seen and been part of an increasing focus and attention being paid to the unique manufacturing and bioproduction issues related to cell therapy.

Certainly it is the Cell Therapy Group's view, that this is both timely and much-needed as more cell therapies reach later-stage. Issues related to larger scale production and lowering the costs associated with it will be critical to successful commercialization of these products. It is encouraging to see both content-providers and and companies involved in potentially bringing solutions to these issues now bringing their focused energies to this sector.

This focus has come from a number of different sources including conferences focused solely on the topic, companies engaging stakeholders in identifying potential bottlenecks they might be positioned to solve, more conference sessions dedicated to these issues, and now a commitment by one of the leading publications in bioprocessing to engage both the cell therapy industry and the traditional bioprocessing community in stepping up the level of two-way education, dialog, and problem-solving that will be critical to commercializing these products.

In March/April 2011, watch for a special issue of BioProcess International entitled "Cell Therapies: Commercializing a New Class of Biopharmaceuticals".

BioProcess International is a publishing leader of cutting edge technologies, improved processes and breakthrough sciences. With this cell therapy focused issue, in partnership with ISCT and others, BPI is launching what we hope will be a regular supplement and increased focus on the unique bioproduction issues related to cell-based therapies.

BioProcess International aims to accomplish three main objectives with this supplement:

Educate the bioprocess and cell therapy market (suppliers and end-users) on the similarities and differences between the two processes;
Educate and encourage the investor community to keep increasing their interest and investment;
Expedite the commercialization process.

Distribution will include:

BPI's 30,010 qualified readers;
Delegates attending ISCT's 2011 Annual Meeting (included in all delegate bags)
Delegates attending ESACT 2011 (Chair drop at the Cell Therapy Plenary Session)
INTERPHEX 2011 Cell Therapy Roundtable (VIP Invitations, 200 attendees, produced by BPI)
BIO 2011 International Convention (BioProcess Theatre - Cell Therapy track)

If you are interested in advertising, click here for more info.

While the content for this issue is now being finalized, it you are interested in contributing something to BPI, we are looking for more cell therapy related content. As the cell therapy representative on BPI's advisory board I would be happy to champion it through submission.

Cheers.

--Lee

The LinkedIn Cell Therapy Industry Group - 1,000 members strong

noreply@blogger.com (Lee Buckler) — Mon, 10 Jan 2011 19:47:00 +0000

As some of you may know, much of my recent social media energy has been spent on LinkedIn rather than blogging. This was not a conscious decision but I will admit to finding the immediacy and interconnectivity of the LinkedIn/Twitter combo to be more seductive of my limited time than the more laborious and seemingly more unidirectional facets of blogging. I'm still working on a return to more diligent and regular blogging - we'll see how that goes.

In any event, today's blog entry is ironically about the very thing which has replaced my blogging in many ways for the interim: the LinkedIn Cell Therapy Industry Group which I founded in July 2008 (about the same time as I launched this blog).

Primarily due to the outstanding participation of great members, the group has turned out to be what I had hoped would be and I believe has become a fairly valuable resource for those in or interested in the cell therapy industry.

The group grew exponentially throughout 2010 and we are proud today to announce our 1,000th member. Without his knowledge, Luc Gervais today became the 1,000th member of the LinkedIn Cell Therapy Industry Group.

Luc Gervais lists himself on LinkedIn as a "Technologist Entrepreneur" but is also a Researcher at IBM Research, Zurich Research Laboratory in addition to being a researcher at the University Hospital Basel.

He was recently involved in the development of IBM's novel, microfluidic "lab on a chip" technology that uses capillary action to create a potential one-step diagnostic tool, and which could ultimately test for a wide range of diseases and viruses. The chip requires only a small drop of blood, which it draws through tiny channels within the device. The blood reacts with different disease markers to provide accurate diagnoses in about 15 minutes.

Luc represents what I believe is one of the most exciting signs of development in and maturation of the cell therapy industry. Luc's career has included being a Game Developer at Unlikely Games, a Computational Chemistry Developer at Boehringer Ingelheim Pharmaceuticals, and a Quality Assurance Specialist at Steltor. On LinkedIn, he lists "regenerative medicine" as one of his interests.

People with the kind of experience Luc possesses are bringing a world of scientific, technical, and commercial expertise to regenerative medicine and cell therapy from outside the sector. This promises to revolutionize the way we think about, develop, and apply our technologies.

Luc and others like him who are exploding into the regenerative medicine and cell therapy field bring with them the potential for interdisciplinary exploration, the opportunity to draw from lessons already learned in other sectors, and the chance to view our field not just in terms of the incredible potential for new therapeutics which cell therapy represents but how that fits into the broader world in which cell therapy is growing up. A world that includes phenomenal advancements in personalized medicine, diagnostics, theranostics, biomarkers, bioinformatics, the ability to access and interpret personal genomics data, etc.

I have yet to speak to Luc (this was all posted from publicly available information) but I'm hoping to bring you an interview of him shortly not because being the 1,000th member of the LinkedIn Cell Therapy Industry Group is deserving of any particular attention (and certainly will not rank in his list of accomplishments I'm sure) but because I'm curious about what Luc represents.

Stay tuned....

--Lee

Careers in cell therapy & regenerative medicine

noreply@blogger.com (Lee Buckler) — Wed, 29 Sep 2010 15:23:00 +0000

As you will see just below, this post falls under the category of a thinly veiled and somewhat shameless plug which I hope you'll tolerate if I'm transparent about it upfront and I add what may be a little useful commentary along the way.

I won't have to convince "regular" readers of this "irregular" blog that regenerative medicine is one of the fastest growing life science sectors.

Given the pace of the industry's growth, the amount of public money being "invested" in the sector, and the general enthusiasm that the science generates, it is no surprise that is increasingly an area of interest for those seeking a new career path.

One commonly held misconception is that career opportunities in the sector are limited largely to those in early-stage research.

While it is true that the industry is still in the nascent stages of commercial development compared to other more mature segments in the broader biopharmaceutical industry, careers in regenerative medicine span the full research and commercial spectrum from pre-clinical to clinical research in both academic and corporate settings, manufacturing, regulatory, quality, operations, logistics, finance, business development, marketing, sales, communications, and executive management.

As has been discussed here in the past, most analysts agree that in 2008 the industry passed the $1 billion mark in annual revenue generated from sale of approved therapeutic products falling under the regenerative medicine category (see here for more in-depth analysis).

Furthermore, most large multinational life science companies are now investing heavily into the sector on the promise that it will revolutionize healthcare in the coming decades.

I believe the 2008 decision by Pfizer to create a substantial new Regenerative Medicine division will be seen as a seminal turning point in corporate perspectives on the industry. Since that point most life sciences have begun investing in regenerative medicine strategies and the trend has even leaked outside of life science to companies such as Google Ventures which has informally identified regenerative medicine as one of their primary areas of interest when looking at companies in which to invest.

Most major universities have now created both academic and research programs dedicated to the emerging field of regenerative medicine such that there is a new class of graduates now emerging in both scientific and commercial disciplines with regenerative medicine as a primary focus.

However, one of the difficulties with finding a career in regenerative medicine is that it is comprised of what has heretofore been fairly discrete disciplines of stem cell biology, pharmaceutical sciences, biotechnology, tissue engineering, stem cell transplantation, device technologies, cell therapies, etc.

Furthermore, most recruiters and online job sites have very little experience or focus on the industry making recruiting difficult for positions in regenerative medicine companies or departments. The task is further complicated by the fact that the candidate pool is small and diversely spread over a multitude of disciplines, centers, and a multitude of small companies.

Finally there are only now emerging industry publications, organizations, online communities, and websites that truly represent the broad spectrum of regenerative medicine and that can be used as central resources for recruiting.

All this is why my partners and I saw the need for an online recruiting tool that really focused on regenerative medicine in a way that would support the industry's maturation. What we wanted to bring was a very simple solution that would create as much value as possible for those looking to recruit the right talent into their regenerative medicine efforts. This was the genesis behind RegenerativeMedicineJobs.com.

(here comes the shameless part....)

About RMJ

At RegenerativeMedicineJobs.com, we believe the growth trajectory of this industry requires a specialized, online recruiting system – one that is more than just a website for posting jobs but also plugged into the large and diverse network of associations, publications, and social media that serves this industry.

RegenerativeMedicineJobs.com is a specialized online job board focused on recruiting for positions in the rapidly growing field of regenerative medicine. The site is simple to use and focused in scope. But this is more than just another website.

RMJ has developed a sophisticated system designed to leverage our extensive network in RM to ensure every post gets maximum attention and attracts the best candidates.

Traffic is pulled to the site and job postings are pushed to a targeted audience by focused social media and marketing campaigns. This is the latest way to recruit for any position in a regenerative medicine department, division, or company. This is regenerative medicine recruiting with a focus.

RegenerativeMedicineJobs.com (RMJ) is a collaboration between CTG Consulting, Co. and Pencilneck Software, Co. The personnel behind this collaboration created Cell Therapy News, Cell Therapy Blog, the LinkedIn Cell Therapy Industry Group, and have produced over a dozen organizational or event-based websites in the cell therapy, stem cell, and regenerative medicine space.

The site has been designed to be simple and intuitive with only one goal in mind: to be the best online tool for recruiting personnel into positions in the regenerative medicine industry.

We don't offer resume posting, career advice, assistance building your CV, hand holding, shoulders to cry on, or job postings outside of regenmed. We believe in the value of specialization.

The site is a listing of regenerative medicine jobs (period). All types of jobs for all types of organizations but only regenmed job (period).

We have only one rule: the jobs posted must be for a position within the regenerative medicine space. We define regenerative medicine as anything involving cell therapy, stem cells as therapies or tools for discovery or toxicity testing, or non-cell based programs/products (e.g., small molecules, biologics, devices, etc) used to replace or regenerate cells, tissues, or organs to restore, repair, or establish normal function.

The site has a topical niche with no regional focus or bias. This is meant to be a global tool equally useful for a company or university in Poland, Taiwan, Brazil, the United States, etc.

The site is also meant to be useful for recruiting any positions scientific or commercial, business or technical, executive or laborer.

We hope you find it useful.

We hope it contributes to the industry's growth and maturation.

We hope you will provide us feedback on what we're doing well and what we can do better.

FDA files injunction again Regenerative Sciences citing Regenexx violates regulations

noreply@blogger.com (Lee Buckler) — Mon, 09 Aug 2010 16:17:00 +0000

For those of you who follow this blog, you'll imagine my surprise to wake up the morning to the following announcement from the FDA:

_____________________________________________________________

FDA NEWS RELEASE

For Immediate Release: August 6, 2010

Media Inquiries: Shelly Burgess, 301-796-4651, shelly.burgess@fda.hhs.gov

Consumer Inquiries: 888-INFO-FDA

FDA Seeks Injunction Against Colorado Manufacturer of Cultured Cell Product

Violations of current good manufacturing practice and labeling requirements cited

The U.S. Food and Drug Administration is seeking an injunction in federal court against Regenerative Sciences LLC, of Broomfield, Colo., citing violations of current good manufacturing practice (cGMP) that cause its cultured cell product to be adulterated. The product is also misbranded due to the lack of adequate directions for use and the failure to bear the “Rx only” symbol.

The company’s cultured cell product is derived from a patient’s bone marrow or fluid surrounding the patient’s joints (synovial fluid). The cells are grown, processed, and mixed with drug products outside the body before being injected back into the patient.

Regenerative Sciences’ cultured cell product is not approved by the FDA, and no adequate and well-controlled studies have been done to demonstrate its safety or effectiveness for any indication.

“FDA recognizes the importance of the development of novel and promising new therapies,” said Karen Midthun, M.D., acting director of FDA’s Center for Biologics Evaluation and Research. “However, when companies like Regenerative Sciences fail to comply with FDA laws and regulations, they put the public’s health at risk.”

The complaint for the injunction was filed Aug. 6, 2010, by the Justice Department on behalf of the FDA in the U.S. District Court for the District of Columbia, against Regenerative Sciences and three of its employees, Christopher J. Centeno, M.D., John R. Schultz, M.D., and Michelle R. Cheever. The injunction would permanently prevent the company and cited individuals from adulterating and misbranding the cultured cell product while the product, or one or more of its components, is held for sale after shipment in interstate commerce.

Regenerative Sciences has agreed to cease production of the cultured cell product while the case is pending.

The FDA warned Regenerative Sciences about its cGMP violations as recently as June 2010. The company failed to make sufficient corrections, and the conduct of the individuals cited in the complaint demonstrates refusal to comply with the law.

Money for regenerative medicine

noreply@blogger.com (Lee Buckler) — Tue, 06 Jul 2010 16:25:00 +0000

This is simple. You can give real money to regenerative medicine without it costing you a dime. JPMorgan Chase & Co's Chase Community Giving program is giving away $5 million to the charities with the most votes. Your vote could mean money for regenerative medicine.

Don't be silly. Just click here and vote.

The changing face of PR and why it matters to regenmed

noreply@blogger.com (Lee Buckler) — Fri, 11 Jun 2010 19:13:00 +0000

This may seem a little off-topic for those who don't know me but for those of you who do, you'll know this is a little mix of many of my primary loves - cell therapy/regenerative medicine, communications/public relations, social media, and all things internet/technology.

This is a reprint (with permission) of an article I was invited to write for the 2009 World Stem Cell Report.

______________________________________________________________________

If you’re breathing, you’re in PR [1]
Some Do’s and Don’ts and 2.0s for orgs/co’s in cell therapy & ReGEN

You may be one of them. The scientist who can’t get the job or promotion you want. The company not securing customers fast enough. The investigator not finding collaborators. The company not finding quality employees. The company failing to secure much needed investment. The big company not effectively penetrating new, niche markets like cell therapy-regenerative medicine. The executive or investigator not getting the speaking invites to create the buzz needed to help move things forward.

Whatever the challenge, consider a healthy dose of marcom. [2]

Ok, I’m biased and it certainly isn’t a single panacea for all these problems, but seriously. A good profile (personal or corporate) – built from a solid reputation and strong relationship network – can help address almost any challenge you or your company is facing.

INTRODUCTION

I know. You don’t believe me. Humor me and answer this one question. If you could double the number of people that know about your company or technology, the number of people that learn, hear, or read something about your research, product or service in the average 30–day period, and/or the number of people that interact with one of your employees or colleagues every week, would that – on the whole – likely help or hinder you in addressing at least some of the primary challenges you are currently facing?

See where I’m going with this? Even if your challenges are deeply scientific or technical, is it possible to imagine that if only you knew the right person …?

Your existence depends on human interaction. Your success depends on how well you interact and the impressions you make. The impressions are not solely or even primarily driven by the brilliance of your technology or the compelling weight of your data. This is likely more true than you may think. For those of you who have read Malcolm Gladwell’s book, Blink, you will appreciate just how much people’s perceptions are influenced not by logic, data, or comprehensive consideration but by the judgments of the subconscious in a matter of seconds.

The PR (public relations) I discuss in the following pages is not spin and it’s not advertising. It’s about establishing and maintaining relationships that result in profile among your various kinds of constituents be they your collaborators, peers, investors, employers, employees, customers, or potential strategic partners.

Successful people and companies use effective PR to create relationships, turn them into interactive networks, and then leverage them to help solve the challenges they face.

This has always been true. What are so radically changing today are the tools which people and companies can now employ to conduct effective PR, create relationships, and build networks all at a fraction of the cost, with greater returns, and performed by individuals or small companies not previously capable of affecting global impact. These tools are, of course, online.

A successful strategy for PR is now increasingly determined by how well online tools are employed to engage in conversation, create relationships, and build profile.

As it turns out, there are, in my opinion, too few people in this industry who, as yet, understand the power of PR and fewer still who understand the potential of web2.0 to change their fate.

What I will share with you in the next couple pages is:

1. why if you’re breathing, you’re in PR - if you can’t avoid it (and I don’t recommend you try), you should learn to embrace it;

2. the cost of dodging the embrace; and

3. how you can embrace it.

POINT #1: YOU’RE IN PR. REALLY.

There are a zillion reasons why you underestimate the importance of PR in your career or to your company. There are a zillion other reasons why you think online social media is irrelevant to what you do. Let’s look at five.

1. My company is a research-based, early-stage company conserving burn-rate, with few employees, and nothing to sell. Ergo we don’t have a need for PR stuff like marketing, communications, or profile building.

There is nothing new but certainly nothing outdated in the adage: Everyone is selling something.

Don’t pretend you have no one to impress, you have nothing to sell, or that content is the only thing that impresses in the science business. If you thing you have nothing to market, you just haven’t figured out what it is yet.

Are you looking to recruit quality people? Do you anticipate needing to find and impress new investors sometime this year or next? Would you be interested in engaging in discussions from parties potentially interested in licensing or acquiring some of your IP? Are you looking to impress potential academic or corporate collaborators? Do or will you need to recruit patients for a clinical trial? Do you need to start educating doctors about the potential of your therapeutic or engage them in giving advice about your trial or product? Would you benefit from increased government funding for cell therapy and regenerative medicine? Do you believe there are policy changes that could be made that would benefit this industry?

A better profile and a wider network of contacts can’t help but help you address these needs. If you have anyone to impress (and everyone does), then you need to be talking with them. Will a bigger and better profile hurt your efforts to find good employees, seduce investors, recruit patients, identify collaborators, secure partners, etc? It’s hard to imagine how.

At the very minimum, as a member of the life sciences industry you have an obligation to get out of your lab and communicate to the world around you about the science you are so passionate about. As President Obama encouraged in his address to the April 27, 2009 National Academy of Sciences [3], we all have a duty to do a better job of engaging the people around us in a discussion about the importance of science and the science we’re doing.

2. My company is a big, public, multi-national company with departments that handle marketing, communications, PR, and advertising. Ergo I don’t need to pay attention to these things because they do that.

Firstly, unless you have tenure, chances are between now and retirement you may want/need/be recruited for another job. A strong, personal “brand” (profile) is certainly a huge asset in terms of ensuring you are successful in finding that job and negotiating the right remuneration.

Secondly, if you are ever let out of the lab/office or given internet access, you have the capacity to (and likely already) influence your company’s profile and brand. You are either complementing or distracting from your company’s primary messages and brand.

Thirdly, if you care about your company’s success, don’t assume your company’s department or external marcom firm is doing what needs to be done. Investor relations, marketing and communications firms or departments may be good at investor relations, marketing, and communications but that doesn’t necessarily mean they are good at the kind of profile building that is going to attract and support meaningful business development opportunities or potential investigators, customers, licensees, collaborators, employees, or clinical trial patients.

Finally, too many big companies haven’t yet figured out how or how to successfully use Social Media. If you’re using Social Media (e.g., Facebook, Twitter, FriendFeed, Digg, etc), you might be able to play a leadership role in being the face of your company on these platforms – just don’t do it without permission!

3. My company is a science-based company with a technical sale to scientists who are not receptive to glitzy marketing, advertising, PR, and-the-like. Ergo there is little ROI on spending money on these kinds of things. We don’t “do” marketing.

You may be in a company that believes putting out press releases is an unnecessary distraction from the business of science; or the company that believes that communications is something done only big companies, public companies, stock hustlers, or companies with something to sell.

Wrong. First you’re working on the assumption that all marketing looks like a drug ad.

Second your target audience is human. Humans are susceptible to having their perceptions influenced via all the usual means. Even if you don’t want to look at it so crassly, you would have to agree they are susceptible to forming more positive impressions of your company or product if information about it is available, packaged nicely, and easily accessible. You would also have to agree that any impression or sale is easier to make when there is a personal relationship/contact.

Your network is one of your most valuable assets to you and to your company. Building a global network and industry profile is marketing under what ever name you want to call it.

4. All this web 2.0 and social media stuff is for tech geeks and kids. They are not my target market and don’t have a critical mass of the kinds of people I need to building profile among.

Let’s assume for a moment, this is true. If you’re looking to be an attractive employer to potential, young employees you may want to reconsider.

Secondly, young people grow up and become industry leaders and executives. Is it too early to start shaping their perceptions?

Thirdly, there is the occasional executive that is actually occasionally influenced by input from their younger employees. I know – it’s crazy!

Furthermore, it’s not true. For example, I have a very high percentage of c-level executives on my LinkedIn Cell Therapy Industry Group, Jim Till who proved the existence of the stem cell in his ground-breaking nature paper in 1963, is an active poster on Twitter, and the average age of social media users is much older than you believe - check your data.

5. Twitter-schmitter. Today’s social media sites are tomorrow’s relics. They’re all hype when they launch and then they fall out of favor and are no longer useful. I’m a serious business professional with no time or need for these gimmicks that have no real business applications or executives like me on them.

If you think LinkedIn, Twitter, or Facebook (my three favorite and most used social media sites) are not going to be around and successful for long enough to have a real impact on your business, you’re definitely one to bet against the odds.

If you think serious business is not being done and useful business connections being made on these sites (see the last paragraph in the point above), you’re dead wrong. I’ve found customers, employees, and collaborators for my customers using social media in addition to getting them speaking engagements and highlighted in publications.

The point? Everyone has some kind of PR (marketing, communications, profile building, networking, advertising, community participation, publishing, speaking, etc) to do. Your potential audience, network, and impact are all much bigger than likely believe to be true.

My guess? You have more than likely significantly underestimated the value of engaging in PR. And, you have likely significantly overestimated the time and financial cost of engaging in PR.

That brings me to Point #2.

POINT #2: THERE IS A REAL COST TO YOU AND YOUR COMPANY IN PRETENDING YOU DON’T DO OR NEED TO DO PR.

All I should really have to do here is refer you back to point #1. The benefits of engaging in PR are the costs for not. Nevertheless, I will briefly expound.

Your next job, employee, collaboration, investor, customer, patient, etc will be influenced by the profile of you and/or your company. While your CV – comprised of its peer-reviewed publications, presentations, and collegial references – is still an important driver in people’s perceptions in our industry what often makes someone or some company stand out among the others (perhaps even imperceptibly) is the overall profile one has created. One’s profile is increasingly judged online.

That brings me to revise Point #2 to the following:

There is a real cost to you and your company in pretending you can/should ignore the PR power of web2.0’s social media.

I’m going to steal now from my October 2008 blog posting on the subject. [4]

Only a few minutes ago (relatively speaking in the passage of time) most companies didn't think they needed to have a website or a domain name strategy. This seems as foolish now as ignoring social medial will seem in a few minutes from now.

In 1995 - a mere 14 years ago - I was a young associate in a downtown law firm making a pitch to a skeptical management committee at a weekend retreat that the firm needed a website and an email "system". It was not an easy sell. They "knew" lawyers would never send their own emails and certainly not use it to communicate with clients. Similarly they were convinced a website would likely not bring in any new clients and existing clients would not likely find a website useful.

How quickly they were proven wrong.

We are at a similar technological threshold. Most companies are ignoring the importance of "social media" to their corporate strategy. Most consider using social media an unnecessary "luxury" in exactly the same way websites, domain names, and email were considered by people in their positions only a few years ago. They do not understand how social media is already changing the way they do business, the importance of staking position/profile early, nor the pain they will feel in the very near future for having failed to be proactive in building and leveraging online communities.

This is very understandable. In times of fiscal constraint one tends to focus on that which hurt you yesterday or causes you the most pain today. Nonetheless, the companies that will succeed tomorrow are looking at tomorrow now and preparing for it. Tomorrow's mainstream business activities involve social media as much as today's involves website and email.

For those thinking that this will be true only for businesses that deal direct with the retail consumer, they’re dead wrong. If you have a constituency that you need to keep informed about and actively engaged in your company, then this applies to you. Whether its business (B2B) customers, investors, media, patients, recruits, employees, or collaborators, they are online and engaging in social media. If you're not there, they're listening to and engaging with someone else.

LinkedIn now has 42 million members with 1,945,047 members who identify themselves in biotech, pharma, or healthcare. Some 300,000+ of them are in biotechnology. There are 293 biotechnology groups in LinkedIn and 375 biotechnology groups on Facebook. The LinkedIn cell therapy industry group has 400+ members engaging in active discussions and exchanges of information in the public forum and using the platform to make and strengthen connections offline.

Bottom line? There are so many different kinds of social media out there that can be used to reach so many different kinds of audiences at a fraction of the cost of traditional media that it's just good business to figure out how to use it to your advantage. In fact, you may be feeling the pain of ignoring it already and just don't know it...

Which brings me to point #3.

POINT #3: WEB 2.0 HAS PUT THE ABILITY TO DO EFFECTIVE, LOW-COST PR IN YOUR HANDS. THERE ARE NO MORE GATEKEEPERS.

The internet has blown the sense of community up and redefined it. Gone are the days when it was feared the web would destroy “community”. Instead web 2.0 is all about creating more and larger communities than we ever thought possible eliminating all kinds of barriers and cutting across all kinds of definitions.

It used to be (before web 2.0) that corporate profile, marketing, communications, PR, advertising was all quite carefully controlled by managing the one-way push of information to the open receptacles we called “readers” or “audience”. If you wanted a global profile, you needed a global PR firm.

Just like the internet has taken the magic out of the business of travel agents, web 2.0 has forever changed the grip that marketing, communications and PR firms had on the business of managing global corporate profiles. Similarly, a global profile is no longer only the purview of companies sufficiently large to retain the services of such firms or have big, internal PR departments.

Just as the information on how to build long-range missiles is no longer the exclusive domain of rocket scientists, with the empowering tools of web 2.0, successful marcom can now be effectively done by subject-matter experts who are not big-budget marcom professionals.

It’s why newspaper are dying and bloggers are thriving. It’s why small companies are building niche profiles that beat out their Fortune 500 competitors. It’s why startups can have a global profile in weeks on next-to-no budget.

All of that is a rather lengthy introduction to primary point of this article:

In this new world of interconnectedness and easy-to-access information, it is easier than ever for everyone and anyone to influence the profile of a company and what it is offering to the world. That’s the good news. The bad news is that you are expected to. All of which leads to one not-so-original observation of the impact of web 2.0 on companies.

If you’re breathing with an internet connection, you can and should have significant and immediate impact on the awareness and perceptions of your company among people around the globe.

If you’re not creating global awareness and positive perceptions, it may be assumed you’re terminally short of breath.

Like it or not. You can’t help it and you certainly can’t avoid it. And the people forming these perceptions are not just the potential customers of companies with something to sell – they’re current or potential investors, partners, collaborators, patients, purchasers, journalists, regulators, policy-makers, employees, employers, etc.

LIGHTS, CAMERA, ACTION

So what can you do to begin leveraging the power of the PR tools at your fingertips and turn a bungalow profile into a chateau; a respectable list of contacts into a vibrant, enviable network of relationships?

10 Ways to a Better Profile for RegenMed and Cell Therapy Organizations

I’ll start with some rules that apply regardless of the PR platform you’re employing (e.g., traditional ones like conference presentations or journal publications or web2.0 ones like blogging or social networking).

1. You need to have a communications strategy and then mandate and empower your people to communicate your message/story to your audiences.

This doesn’t have to be complicated. It’s about defining your primary/secondary audiences, messages, keywords, and tactics to accomplish your primary/secondary goals.

2. Talk about what you’ve done not about what you’re going to do.

It is very rare that news about what you are going to do is really newsworthy. Don’t be tempted to put out a press release that’s not news just because you haven’t put one out in awhile. For example, a press release that your executive is going to speak at a conference is not news unless they are presenting new data or they are the keynote at the biggest conference of the year. That doesn’t mean you can’t post it on your website or let your network know in other ways but the press release should be used judiciously.

3. Make sure you tell the market story not just the science story. Similarly, be clear about what problem your technology solves not just how brilliant it is.

4. Be upfront about the limitations of your product/service. Don’t try to pretend you have the perfect product/service. Don’t try to be everything to everyone – defining who you are (your company and its product/service profile) is just as much what about what it is than what it is not.

5. Avoid the temptation to promise when you will do something by (e.g., begin or end your trial, submit your IND, launch your product, etc) unless it’s the subject of a contractual obligation. There are too many unknowns that may prevent you from meeting the expectation you created. The ensuing disappointment will hurt your profile and perceptions of your company and its product/service and management.

6. Don’t over communicate your regulatory interactions with the FDA – this is one area where the less said is often better.

7. Wait on that hiring announcements just a little while. It’s critically damaging to put out a press release that now has a very, long online life outside of your control announcing the hire of a key executive only to have him/her not last beyond the first few weeks.

8. Understand that communications, marketing and profile building are very different from advertising. Act accordingly.

9. Value the untapped potential of your existing customers and prospects to spread your gospel.

One of the real discoveries from social media is the alarming willingness of people to zealously proselytize on behalf of companies and products win which they believe. Find ways to actively engage them in doing so. [5]

10. Engage online.

The 8-step Plan to Enhance your Online Profile and Network

1. SEO.

If you’ve decided your profile matters, then your online profile really matters. The fastest way to an impressive online profile is Search Engine Optimization. There are a lot of tricks up the SEO tricksters sleeve and I’m not about to give them all away but suffice it to say that SEO is a multi-pronged attack and there are many arrows in the quiver each with a different use to the same end: higher rankings on the major search engines = increased traffic to the website = greater profile = whatever your end goal is (sales, investors, collaborations, changing the world, etc).

2. Listen & Monitor.

Whether you employ Google Alerts, Twitter search tools, or simple subscriptions to topical news services or blogs, there is a lot of content and discussions being added to the internet every day. You will likely be amazed how much (or how shockingly little) your company and product/service is being discussed and who is behind the discussions. Listening to and reading what’s going on the internet will get you intel about your competitors, new ideas for potential products or studies, new leads for potential collaborators or customers, etc.

3. Follow/Subscribe.

Find your favorite content providers, writers, resources, publications, etc and follow or subscribe to the content their pushing out and discussions they are leading.

4. Join

There’s no harm in joining. People join for all different reasons. There’s no problem in being an observer. In fact it’s likely best you just observe for a while after you join to assess the flavor of the group and discussion so when you do participate you don’t offend.

5. Participate

Once you’re comfortable, start to participate. Engage in discussion, exchange information, and above all make sure you are providing value to the other participants not just talking about yourself. This will be punished.

6. Connect (yourself and others)

There’s a reason why these are called online networks or communities. Use them to connect with people not just for the sake of building a bigger ‘friends’ list but because you have something in common and want to begin building a relationship.

7. Create

Be original. Create something of value you can share. That might just be facilitating a channel in which 3rd party information is exchanged. It might be you writing original content. It might be facilitating introductions.

8. Enjoy and don’t offend.

Most importantly find the online networks that you enjoy, that give something of value to you, then contribute value in return and ensure you don’t offend the rules. Nothing slaps harder than an online, viral slap from a community you’ve offended online. [6]

CONCLUSION

Don’t pretend you have no one to impress. Everyone needs to engage in PR.

Even in the business of science, man cannot live on data alone. You need more than just solid content to affect the kind of impressions that will be critical to your success. Don’t let your data or career speak for itself – it can’t talk.

Web 2.0 has made it exponentially easier – if not expected - to engage in PR and profile building.

What’s missing from this article is the web 2.0 tools I’d recommend you use to put the 8-step plan in place. For that, watch here for further blog postings on the subject. See what I did there?

[1] In this article, I use “PR” not to mean “public relations” as you may have come to understand it used but rather simply to mean any kind of relating to the public via marketing, communications, writing, profile building, networking, advertising, community participation, publishing, speaking, proselytizing, lobbying, etc. It’s a not-so-dirty little secret that we all do it in one form or another or several all at once.

[2] Marketing & communications

[3] See http://www.youtube.com/watch?v=k5-MgZD5IMc at around the 30 minute mark.

[4] Oh, I haven’t mentioned my blog? It’s at http://www.celltherapyblog.com/. Of course if you are a subscriber, this will be a refresher. For those who haven’t read this on my blog, you would have benefited from reading these same thoughts back in October 2008 if you had been a blog subscriber, or if you followed me on Twitter (I’m @celltherapy) or if you were connected with me on LinkedIn (http://www.linkedin.com/in/celltherapy). I’m just saying…

[5] One the flip side these same people will use these same tools to punish companies mercilessly for offending them or the rules of social media.

[6] Just ask Johnson & Johnson about the power of mommy bloggers. Look it up.

Biotech tax credit appears perfectly designed for cell therapy companies to recoup research dollars spent in 2009-10

noreply@blogger.com (Lee Buckler) — Sat, 22 May 2010 04:34:00 +0000

Stewart Lyman of Lyman BioPharma Consulting posted a great article in today's Xconomy summarizing some key points and links to more information about the rules governing the Therapeutic Discovery Project Credit which have now been released by the US Treasury Department. Today, a detailed fact sheet was released about the tax credit program and it seems almost perfectly designed for most cell therapy companies.

Lyman points out a few important details about the application schedule including:

1. The Formal IRS applications (Form 8942) will not be available until June 21st or thereabouts.

2. The application period opens on June 21 and ends on July 21. The postmark on the application is deemed to be the date of delivery. Preliminary review of the applications is to be completed by Sept. 30; this is to ensure that applicants are eligible taxpayers and that their applications are complete. Applicants will receive determinations as to whether or not they qualify for credits and/or grants, and how much they will receive, by Oct. 29.

By way of a little more background, the following is excerpted from a March Forbes.com article by Dean Zerbe:

What does the credit cover?

The credit/grant covers research in tax years beginning in 2009 and 2010. The taxpayer is provided a 50% credit/grant for qualified investments in "qualifying therapeutic discovery projects." What expenses count as qualified investments? The aggregate amount of costs paid or incurred in the taxable year for expenses necessary for and directly related to the conduct of a qualifying discovery project. What doesn't count? The pay of employees covered by 162(m)(3) of the tax code--think CEOs--doesn't count. Other excluded items: interest expenses; facility maintenance expenses (e.g. mortgage or rent payments, insurance, utility and maintenance and costs of employment of maintenance personnel); and certain indirect costs (basically general and administrative costs) as defined in the Treasury Regulations at 1.263A-1(e)(4).

What is a qualifying therapeutic discovery project?

According to the legislation, it's a project designed to do one of three things:

--Treat or prevent diseases or conditions by conducting pre-clinical activities, clinical trials and clinical studies, or carrying out research protocols for the purpose of securing federal government approval by the FDA.

--Diagnose diseases or conditions or to determine molecular factors related to diseases or conditions by developing molecular diagnostics to guide therapeutic decisions.

--Develop a product, process or technology to further the delivery or administration of therapeutics.

Finally, to qualify, a venture may not have more than 250 employees in all businesses of the taxpayer--meaning a small biotech project at a big company wouldn't qualify.

Which biotech companies might benefit?

Those that are investing significant resources in pre-clinical or clinical studies, which may take years to come to fruition to ultimately satisfy FDA requirements, could now recoup a significant portion of their expenses. Additionally, biotech start-ups focusing on the development of diagnostic assays or applications to advance therapeutics and treatments can also benefit. Finally, companies currently engaged in basic or applied research which may ultimately contribute to curing caner within the next 30 years may also be excellent candidates. Along these lines, companies studying signal transduction pathways, gene therapy and stem cell research seem like prime candidates.

The Cell Therapy Group will be collecting more information about the tax credit and service providers who might be recommended to assist in the application if needed. Contact CTG for more details or watch here for more information.

Google to Invest in Regenerative Medicine

noreply@blogger.com (Lee Buckler) — Wed, 05 May 2010 19:35:00 +0000

While the US government prints money to shore up failing and broken business models which no one likes but are considered simply too big (not too important or significant or even useful) to fail, Google is making money and investing it in start ups who expect nothing less than to create "disruptive, even world-changing technologies".

No suggestion here that GOOG is being altruistic, just that this is the way the new entrepreneur and investor class thinks. Opportunity and money are to be found in technologies that improve the way we live, work, play, eat, and think... and perhaps even improve the world.

To Google Ventures this has already meant wind farms, carbon emission reduction systems, green vehicles, and medical cures. To former Microsoft chief scientist Nathan Myhrvold and his high-level think tank, Intellectual Ventures, this means creating TerraPower - a company intending to revolutionize the nuclear power by developing reactors run on waste uranium - and also actively looking at regenerative medicine technologies.

Having formed the fund a little over a year ago, Google is only now starting to make a splash with the fund. Officially the fund has no specific industry focus saying on the Google Ventures website FAQ:

We are interested in a wide range of industries, including (but not limited to) consumer Internet, software, hardware, clean-tech, biotech, health care and others. First and foremost, we're looking for entrepreneurs who are tackling problems in creative and innovative ways, with the potential for significant financial return.

Unofficially and yet not so quietly, Google has named a few broad areas of interest. An article in Monday's New York Times quoted Google Ventures' managing partner, Bill Maris as saying that while they were not going to name particular investment themes, a few broad ares of interest include:

regenerative medicine, bioinformatics, cloud storage, companies that use large data sets, online monetization and mobile.

There it is. Regenerative medicine right there front and center.

In typical Google tradition, Maris, who looks all of 30 years old on the website, has a successfull and multidisciplinary track record. He was involved in founding Web hosting pioneer Burlee.com (now part of Web.com), where he built much of the key computing, network and technological infrastructure.Prior to that, Bill was a biotechnology and healthcare portfolio manager for Stockholm, Sweden-based Investor AB. Bill’s background also includes research at the Duke University Medical Center, Department of Neurobiology.

Google Ventures is said to be aiming at investing about $100 million a year. Any portion of that for regenerative medicine is more than welcome.

While traditional VC money remains reticent to back RM in any signifant way, Google's move confirms a trend we've been seeing and talking about at the Cell Therapy Group for the past 12 months or so. The multinational lifescience, biopharmaceutical, and healthcare companies along with strategic investors all now have regenerative medicine on their radar. They are all quietly and not-so quietly developing internal and external regenerative medicine strategies.

Please join us in welcoming regenerative medicine to the radar screen. It's bound to be an exciting ride ahead.

Latest Cell Therapy Approval by FDA. Dendreon's Provenge.

noreply@blogger.com (Lee Buckler) — Thu, 29 Apr 2010 17:39:00 +0000

It has been a long-time coming. It has been hyped and scoffed, bet against and hoped for, but now none of that matters. It's here. Dendreon has brought Provenge to market. Here, in the word's of the FDA...

FDA NEWS RELEASE

For Immediate Release: April 29, 2010

FDA Approves a Cellular Immunotherapy for Men with Advanced Prostate Cancer

The U.S. Food and Drug Administration today approved Provenge (sipuleucel-T), a new therapy for certain men with advanced prostate cancer that uses their own immune system to fight the disease.

Provenge is indicated for the treatment of asymptomatic or minimally symptomatic prostate cancer that has spread to other parts of the body and is resistant to standard hormone treatment.

Prostate cancer is the second most common type of cancer among men in the United States, behind skin cancer, and usually occurs in older men. In 2009, an estimated 192,000 new cases of prostate cancer were diagnosed and about 27,000 men died from the disease, according to the National Cancer Institute.

“The availability of Provenge provides a new treatment option for men with advanced prostate cancer, who currently have limited effective therapies available,” said Karen Midthun, M.D., acting director of the FDA’s Center for Biologics Evaluation and Research.

Provenge is an autologous cellular immunotherapy, designed to stimulate a patient’s own immune system to respond against the cancer. Each dose of Provenge is manufactured by obtaining a patient’s immune cells from the blood, using a machine in a process known as leukapheresis. To enhance their response against the cancer, the immune cells are then exposed to a protein that is found in most prostate cancers, linked to an immune stimulating substance. After this process, the patient’s own cells are returned to the patient to treat the prostate cancer. Provenge is administered intravenously in a three-dose schedule given at about two-week intervals.

The effectiveness of Provenge was studied in 512 patients with metastatic hormone treatment refractory prostate cancer in a randomized, double-blind, placebo-controlled, multicenter trial, which showed an increase in overall survival of 4.1 months. The median survival for patients receiving Provenge treatments was 25.8 months, as compared to 21.7 months for those who did not receive the treatment.

Almost all of the patients who received Provenge had some type of adverse reaction. Common adverse reactions reported included chills, fatigue, fever, back pain, nausea, joint ache and headache. The majority of adverse reactions were mild or moderate in severity. Serious adverse reactions, reported in approximately one quarter of the patients receiving Provenge, included some acute infusion reactions and stroke. Cerebrovascular events, including hemorrhagic and ischemic strokes, were observed in 3.5 percent of patients in the Provenge group compared with 2.6 percent of patients in the control group.

Provenge is manufactured by Seattle-based Dendreon Corp.