This is a flow chart that shows how to go from discovery biology to a clinical trial. The process of translational research.

Time after time headlines in the news will spotlight a recently developed drug that may potentially provide treatment to patients that suffer from diseases like Alzheimer’s, Parkinson’s and multiple sclerosis. These drugs appear in the news because they have reached the late or near final stages of the drug development process where they can be tested in clinical trials involving humans who are living with these diseases. But again and again it seems that these drugs are discontinued at this late stage and are thwarted out of becoming the medical therapies their developers had hoped to create. Why is this so?

In a recent edition of The Lancet Neurology, an article discussed the problems for drug development in Alzheimer’s disease. And, although the summary only focused on this disease, the problems discussed can explain why drugs being tested for diseases like MS are also failing. One of the reasons that The Lancet Neurology gives to explain drug failure is the animal issue.

Before a drug can be tested in humans, it must go through an animal model that is designed to be as humanlike as possible. This way researchers can observe how the drug will react with the newly developed treatment as if it were being tested in a human system. However, it is almost impossible to create an animal model that will react to a treatment in the same way a human would or one that can “accurately reflect human pathogenesis.” So, after experimentation, what a researcher has actually found is a drug that appears to  cure, alleviate symptoms of and repair damage from a disease in an animal. Yet, when the drug is tested in humans and it does not have the same effect as it did in the animal model, the drug is rendered useless to treat human diseases. Then it’s back to the drawing board.

The term “translational research” is something else The Lancet Neurology questions. It looks easy on paper, but attempting to translate drug response in animal models to human clinical trials is something that rarely seems to pan out. This is why it is important to create animal models that directly model human pathogenesis.

If you would like to read more about this issue, check out the article in The Lancet Neurology here.

When stories about Dr. Paolo Zamboni’s new treatment for MS hit the Internet I, like everyone with MS, breathlessly read the news and watched the videos about CCSVI (chronic cerebrospinal insufficiency).  CCSVI exploded onto the MS scene and grabbed the imagination of MS patients in a way that hasn’t been seen before.  No wonder.  The theory is extremely attractive because if true, the “fix” for the most common forms of MS would be a common outpatient surgical procedure.

And Dr. Zamboni has cred: he is a surgeon and Director of the Vascular Diseases Center at the University of Ferrara in Italy, and has authored two books and more than 300 professional articles.

In today’s blog I give an overview of the issues surrounding CCSVI.  It’s a subject rooted in science, but for us MS-ers it’s also a highly emotional subject.  Of course.  It’s just important that we keep clear heads about it, and I’m hoping that this summary will provide a helpful perspective on the subject.  As always, space limitations prevent me from including all but the highlights, but I have to say this: Please don’t shoot the messenger!

So what is CCSVI?  Simply put, it’s a narrowing or blockage of certain veins and arteries leading from the brain to the heart (jugular vein) and spinal cord (azygos vein).  According to Dr. Zamboni, the slowing of blood flow at these bottlenecks result in a backing up (reflux) or leaking of red blood cells into the brain and spinal cord.  This in turn causes an excess of iron deposits in those locations, and related inflammation of the blood-brain barrier. 

The treatment is the “liberation procedure.”   First, the presence of the narrowing is confirmed by noninvasive ultrasound.  Then a balloon angioplasty is performed—a tube is threaded through a vein in the thigh and widens the narrowing with a balloon, which is then removed.  In some patients a stent may be placed in the vein to keep it open.

I reviewed the abstracts of two of Dr. Zamboni’s most cited studies. The first, published in December 2008, found that almost all of 65 patients with MS had “venous outflow anomalies.”  RRMS and secondary progressive patients had different patterns of anomalies than did primary progressive patients.

The second reported that performing angioplasty on, again, 65 patients, resulted in a “minor and negligible complication rate.”  The improved blood flow resulted in fewer relapses and lesions.  In RRMS and primary progressive patients there was a “significant” improvement in physical quality of life, with a “positive trend” in secondary progressives.  Rates at which narrowing reoccurred suggested a need for improved techniques in certain types of patients.  The study concluded that another control study was warranted.

What hope this gives to all of us with MS!

A layperson—at least this layperson—has to believe that it is enormously significant that virtually all of the research subjects with MS also had CCSVI.  This is especially true when combined with the fact that none of the non-MS subjects had narrowing or blockage.  And the results of Dr. Zamboni’s studies are extremely encouraging, to say the least, in some cases.

But hold the phone.

Even if we all have CCSVI, how successful is the treatment, and how risky?  And for some of us, is any potential benefit worth even the ultimate risk?  Tedious questions all, but they have to be asked.

The fact is that patients who have traveled overseas for the procedure have had widely differing results.

The risks are far from hypothetical.  Earlier this year, Stanford University stopped treating CCSVI after one patient suffered a brain hemorrhage and died, and another had open heart surgery after a jugular stent migrated to the heart.  It’s worth noting that both of these procedures involved stents, and one patient didn’t have MS.  And Stanford is proceeding with grant applications for a CCSVI clinical trial.

What about the fact that some the effects of balloon angioplasties can wear off with time, and stents can collapse or migrate?  If you knew you’d have to undergo repeated angioplasties or stent implants, both procedures (the stent in particular) with defined risks that can’t be performed an endless number of times, and that you might end up right back where you started, would you still do it?

These are not minor details; they are life-and-death issues.

A recent study conducted at Humboldt University in Germany found CCSVI in only one of 56 MS patients studied.  So what? you may ask.  If my veins are blocked, I’m willing to take any risks involved.

As angry and frustrated as questions may make MS patients, they are ignored at our peril.  In April 2010, at a forum held by the National MS Society and the American Academy of Neurology,  Dr. Zamboni himself stated, “This is a work in progress,” and noted that his group is planning a controlled clinical trial.  A recording of the entire forum can be seen here.

The medical profession is sitting up and taking notice, as it should.  The University at Buffalo, the State University of New York, has taken a lead in moving the research along in the U.S.   Dr. Robert Zivadinov and his team at Buffalo, in collaboration with Dr. Zamboni, studied 16 MS patients, all of whom were found to have CCSVI.  Another team at Buffalo is now studying whether balloon angioplasty improves MS symptoms or progression.  Dr. Zivadinov has stated, “If we can prove our hypothesis that cerebrospinal venous insufficiency is the underlying cause of MS, it’s going to change the face of how we understand MS.”  Extremely exciting.  Significantly, though, he also stated that media coverage of CCSVI has been “unrealistic.”

What all this means is that the results of Dr. Zamboni’s studies are fascinating—so fascinating that clinical trials are or soon will be in play around the U.S. and Canada.  Let’s hope that their results come in quickly as well.  There is no time to waste.

In the meantime, check out Nadine LeGier’s journey with CCSVI on YouTube.  It will be fascinating to see if her improvement continues.  Many, many other videos of MS-ers who have taken the plunge are available on YouTube.  Their stories are compelling, and run the gamut from not much of a difference if any, to considerable improvement, at least in the short run.

What does this mean for us individually?  Just personal empowerment and the possibility of a revolution in the treatment of MS!  Personally, I plan to wait until we know a lot more.  But that’s easy for me to say now, as I’m doing relatively well (knock wood).  Who can fault anyone who is deteriorating quickly, or is already severely disabled, for trying anything that might help?  The important thing would be for that person to go in fully informed. Some MS patients say they don’t care about the risks, or would rather die than continue as they are.  If you are one of those, I can only hope that you can hold on long enough to do this when safety and effectiveness are more clearly achieved.  If you can’t, we all are rooting for you and are anxious to hear about your experience.

And what does this mean for the work of the Myelin Repair Foundation?  The MRF welcomes new discoveries wherever they come from—of course!  The goal we all share is to end this pernicious disease.  Whatever the cause of MS, even CCSVI, the disease process involves the destruction of myelin, and that myelin needs to be repaired.  So, thank goodness, the vital work of the MRF continues.

Additional sources for this blog and for more information:  “What Causes CCSVI” by Julie Stachowiak, Ph.D.; “CCSVI—A Huge Breakthrough in MS” by Ashton Embry, Ph.D. for New Pathways Magazine; “University at Buffalo Launches Clinical Trial of New Multiple Sclerosis Treatment“.

What do you think about CCSVI?  Are you planning to sign up for clinical trials?  Would you travel overseas for the liberation procedure?  Have you had the procedure?  If so, what is your experience?

SARATOGA, Calif.–(BUSINESS WIRE)–The Myelin Repair Foundation (MRF), a non-profit medical research foundation known for its pioneering Accelerated Research Collaboration™ (ARC™) model, is taking a lesson from online game developers to explore creative ways to accelerate the pace of medical research and shorten the time to market for new medicines.

This fall, the MRF will host two real-time online “idea sourcing” events – Breakthroughs to Cures – an online gaming event designed to engage university scientists, business leaders, healthcare innovators, biopharma executives and R&D professionals, government regulators, entrepreneurs from all walks of life, patients and the general public, in conversations about the barriers that are slowing the pace of medical research and the strategies that may break down these barriers.

“The evolution of the ARC™ model over the last six years has taught us that there are many, many discrete places in the value chain of research and drug discovery that can be accelerated,” says MRF President Scott Johnson. “Reaching out to a broader universe— individuals who work within the current system of medical research as well as others with a bent for problem solving in any arena—for out-of-the-box thinking is certainly in line with our values.”

The idea-sourcing, gaming events are being supported by a grant from the Robert Wood Johnson Foundation’s Pioneer Portfolio, which seeks ideas that break free of conventional thinking, often because they reframe and refocus problems and explore different paths to breakthroughs.

“The current model used for developing effective, life-saving disease treatments is not keeping pace with scientific discovery,” says Nancy Barrand, Senior Program Officer at the Robert Wood Johnson Foundation’s Pioneer Portfolio (http://rwjf.org/pioneer). “Gaming is the perfect tool to help foster the type of unconventional thinking that is necessary to create radical change in health care and accelerate the speed at which treatments are delivered.”

“Gaming could be the next tool for solving some of our most pressing real-world problems,” says Institute for the Future (IFTF) game designer and creator and producer of the BreakthroughstoCures events Jane McGonigal. “Those who play games have a sense of urgency and abandon when they are engaged in a game scenario. We have seen these behaviors in corporate strategic game play where there are real stakes. The game we are building for the MRF is designed to generate that sort of urgency and unleash creative ideas for finding ways to speed medical research.”

WHO: Participants to include innovators and creative thinkers from within and beyond the medical research enterprise. Those intimately familiar with the industry’s R&D issues and those that from on-high recognize that the current pace of developing new patient treatments or cures for diseases with unmet needs is failing patients.

WHAT: Myelin Repair Foundation will host Breakthroughs to Cures, with funding from the Robert Wood Johnson Foundation’s Pioneer Portfolio and produced in partnership with the Institute for the Future, this online “idea-sourcing” event is designed to engage as many as 600 individuals in conversation threads that are expected to identify a wide range of new strategies for accelerating research. Participants will watch a short (2 minute) scenario video, before sharing a brief 140-character or less idea(s) about the future of medical innovation and research. These ideas spur reactions and brainstorms, and lead to innovative and collaborative ideas about what the future could look like.

THE SCENARIO: The premise of the online gaming scenario involves a widespread contamination, estimating that as many as 100 million people in the United States have been infected. America faces a national health crisis unlike any before and leaders in Washington are meeting to discuss how to address the state of this national emergency. This idea-generating event calls upon its participants to solve a deadly disease sweeping the nation. To view the scenario visit: http://info.breakthroughstocures.org/

WHEN: The game will be played on October 7^th.

HOW TO GET INVOLVED: Breakthroughs to Cures is open to the public. Registration begins September 3. If you’d like to learn more about Breakthroughs to Cures visit breakthroughstocures.org.

MRF’s Johnson adds, “While we think this activity may afford us some additional strategies to enhance the ARC™ model. We also think it’s important to broaden the base of individuals and organizations who understand the problems faced by the industry and may have process ideas for how to improve outcomes for patients.”

About the Myelin Repair Foundation

The Myelin Repair Foundation (MRF) – http://www.myelinrepair.org – is a Northern California-based, non-profit research organization focused on accelerating discovery and development of myelin repair therapeutics for multiple sclerosis. Its Accelerated Research Collaboration™ Model is designed to optimize the entire process of medical research, drug development and the delivery of patient treatments.

About the Robert Wood Johnson Foundation and the Pioneer Portfolio

The Robert Wood Johnson Foundation focuses on the pressing health and health care issues facing our country. As the nation’s largest philanthropy devoted exclusively to improving the health and health care of all Americans, the Foundation works with a diverse group of organizations and individuals to identify solutions and achieve comprehensive, meaningful and timely change. Projects in the Pioneer Portfolio are future-oriented and look beyond conventional thinking to explore solutions at the cutting edge of health and health care. When it comes to helping Americans lead healthier lives and get the care they need, the Foundation expects to make a difference in your lifetime.

About the Institute for the Future

The Institute for the Future (IFTF) is an independent, nonprofit research group with 42 years of forecasting experience. IFTF’s core work is in identifying emerging trends and discontinuities that will transform global society and the global marketplace. IFTF provides insights into business strategy, design process, innovation, and social dilemmas with research that generates the foresight needed to create insights that lead to action. IFTF research spans a broad territory of deeply transformative trends, from health and health care to technology, the workplace, and human identity. The Institute for the Future is located in Palo Alto, CA.

Contacts:

For The Myelin Repair Foundation
Darren Brandt, 212-446-1861
dbrandt@sloanepr.com
or
Sarah Hawkins, 212-446-1890
shawkins@sloanepr.com

At the Myelin Repair Foundation we like to keep up to date with our volunteers, other MS bloggers and news articles throughout the week. So we figured we should share our favorites. And what better day to do it than Friday? Here is the latest edition of Follow Friday, full of great stories, interesting information and captivating blogs we recommend to any of the MRF’s blog readers.  Let us know what you think by leaving comments or finding us on Facebook. Hope to catch you reading every Friday!

1. You have probably heard a lot about it, but for a good reason! Fred Schwartz, one of the MRF’s Ambassadors at Large, is taking to the sky with a skydiving event to raise money for the MRF. Check out his new video! And let us know if you have any ideas for an event like this at a location near you.

2. Wheelchair Kamikaze is a blog written by Marc, a New York City native who was diagnosed with MS in 2003. He takes on unique perspective throughout each of his posts, whether they take video, written or photographic form. Here is his latest post.

3. As many you have probably read on our blog from author and MRF volunteer Gali Hagel, living with MS or getting diagnosed with MS means you start asking a lot of questions you never had to ask before. Healthline has a community for MS and posts useful information like common medications that are prescribed for MS.

4. Lisa Emrich is another blogger we continue to follow. Her ability to share such personal information about her life with MS is inspiring to say the least. Check out one of her most recent posts about having a chronic illness and dealing with the fear it invokes. Her message is always a powerful one and is strengthened with each post. Check it out here.

If you have any recommendations for blogs or articles to read, please feel free to post a comment or to send them to megan@myelinrepair.org

When confined to a wheelchair, world experiences can seem out of reach. But not to Fred Schwartz. Fred is one of the Myelin Repair Foundations volunteer Area Directors who has had MS since 1994 and has been limited to life in a wheelchair for the last 10 years. But now, Fred is taking himself and the new MRF Ambassadors program to the sky.

Armed with the goals of, not only supporting the MRF, but also escaping the limitations of the wheelchair, Fred Schwartz has put together a skydiving event where he will take to the sky, along with other participants, as they hope to raise funds for MRF research. This skydiving event is one of many, we hope, that will be part of the new MRF Ambassadors Program. If you want to know more about the event, please check out the new video Fred has posted or visit his website.

MRF Ambassadors is a newly developed volunteer program that will help people from all over the world gain the skills to help raise money for and give their time and effort to the MRF. The program will consist of a toolkit that is accessible on our website, and a series of videos that will give people, interested in becoming an MRF Ambassador, the knowledge on how to plan an event, how to get people to participate in and donate to their event and to raise money for the Myelin Repair Foundation to further our goal of getting a myelin repair treatment to market.

The Ambassadors program hopes to launch this September and will be led by our Ambassadors at Large (ALs for short), Bob Gregory and Fred Schwartz. The ALs will work with the volunteers to guide them in their fundraising endeavors and lead with example by holding events like skydiving, to name one of the many.

To Fred, the opportunity to skydive may have always seemed to lie outside of his reach because of his wheelchair confinement. But knowing what it could do for the MRF has pushed him to doing the impossible — jumping out of a plane in a wheelchair. We hope that all of our volunteers and new members of the MRF Ambassadors program will think outside of the box to create programs that will raise money for MRF’s research and awareness about myelin repair and the impact it could have on people with multiple sclerosis or other neurodegenerative diseases.

When scientists talk about their research on diseases like Parkinson’s, Alzeheimer’s or multiple sclerosis, it seems that the only thing that people want to know is, “When is there going to be a cure?” Scientists, in return, answer with matter of fact figures like 15, 20 or even 50 years. But in the grand scheme of scientific research and medical discovery, how many cures have actually been found by their predicted deadline? Not many, according to Emily Yoffe, from Slate Magazine.

In her most recent article titled The Medical Revolution, Yoffe discusses the types of promises that have been made due to technologies like stem cells and genomics. But instead of focusing on how these new medical practices may be the answer to our medical problems, she writes about how they might be the cause.

A decade ago the human genome was finally unraveled. It was at that time that we were supposed to understand how and why we are affected by such devastating diseases like those mentioned above that rid of us of our memories, our motility and most importantly the ability to lead a completely normal life free of complication. But that did not happen. Instead, Yoffe explains, scientists found that the human genome is much more complicated than they anticipated. Still, however, researchers use the fact that they have mapped the human genome to over promise cures to wishful patients. They do this not only because to discover anything in biology one has to be continually optimistic, but also to receive funding for their research. This money goes into the discovery portion of the drug development process but it does almost nothing to cover the cost of bringing the new drug to clinical trial – the critical step to making a drug target become a treatment.

Another broken portion of the drug discovery process is that the researchers are so focused on discovering a cure for a certain disease, and promising their patients that cure, that they forget to mention the target validation process which is required to catch the wandering eye of pharmaceutical companies. Most discovery biology gets trapped in the moat that surrounds the target validation process because of the money it takes to push a target through. This moat is called the valley of death in the medical field. That is because small molecules that show promise to become a treatment, by ridding animal models of disease completely, go there to die when they are not picked up by big pharmaceutical companies or they lose funded research.

Some may argue that the problem with the medical system is that there are not enough cures or treatments coming out of all of the funded research. But take a step back. Maybe scientists are just promising too much when they understand too little. That is one of the reasons that the MRF is traveling down the road less traveled of drug target validation. By having an Accelerated Collaborative Research model, our drug targets will be able to bridge the valley of death and successfully land on the side of clinical trials.

If you would like to read more about The Medical Revolution, go to the article written in Slate by Emily Yoffe here.

Scott Johnson, Myelin Repair Foundation President and Founder … waiting for a cure since 1976

This week, we released the third Myelin Repair Foundation video that has been produced since our founding in 2003. For those who have seen videos #1 and#2, you will see a marked contrast in this third video. This is because over the last few years we’ve grown as an organization and evolved the ARC model in ways that are bringing us much closer to the first myelin repair clinical trial.

Our staff now includes seasoned drug hunters who are guiding the basic research and retaining our focus on clinical relevance. Engaging this level of industry expertise on our staff also gives us the necessary horsepower to manage the translation of the discoveries made in our funded labs and deliver to pharma what they want and need to enter partnerships with the MRF and move our therapeutic targets forward to the clinic.

I think you will be impressed with these new additions to our scientific management team as well as their level of commitment to developing a myelin repair treatment. I feel honored that these individuals have chosen to join us.

While we have our own plans for using the video and a new companion brochure, we thought you would find the video useful in telling your friends and family members about the MRF.

Please take 12 minutes to watch the video.

Then share it with your friends. Help us tell our story and share with others why you support the MRF — for its singular focus on myelin repair as the next generation MS therapy and for its model that is accelerating the pace of medical research and drug development.

We continue to make great progress in our discovery biology program and are expanding our translational medicine research program in ways that we think will be easily replicated for qualifying each new target identified in our funded laboratories. I look forward to keeping you updated on our progress.

Thank you for your commitment to our work. Without you it would not be possible.

Scott Johnson
President & Founder
Myelin Repair Foundation

Every week our good friend and MRF volunteer, Gali Hagel, writes a phenomenal blog about her personal experiences with MS. Her advice is priceless and holds our community together. So this week we wanted to bring back one of her old posts. If you have not read every week, here is your chance to start back at the beginning! Let us know what you think by leaving comments below.

My MS – A Story by Gali Hagel

A diagnosis of a progressive, incurable disease would make anyone feel helpless, hopeless and angry.  At least, that’s how I felt when I discovered at the age of fifty that I had MS.  I was an active and successful corporate attorney who wasn’t used to having to accept the unacceptable.

After the initial shock wore off and I was able to face my situation more directly, I did my homework.  I had already discovered that none of the ABC drugs would halt the disease.  Of course the good news was that they would slow the progression, but this was—and is—not enough.  I was shocked to discover the glacial pace of research on neurological diseases.  So when I heard about the Myelin Repair Foundation I called Scott Johnson, the founder and CEO of the MRF.  Suddenly I had hope.  His explanation of the genesis, philosophy and goals of the MRF, its revolutionary cooperative research model, and his own obvious determination, inspired me to volunteer as the Area Director for Georgia.

As people familiar with MS know, fatigue is a huge factor in MS.  I wouldn’t be devoting my energy to helping the MRF if I weren’t convinced that it will succeed.  Besides, I know the folks at the MRF, and they are the best—a dedicated, focused, talented and compassionate team.

What I have heard first-hand from the MRF research scientists about their progress has long since convinced me that, with the donations necessary to support their research, the MRF will achieve its goal and timeline for bringing to market a drug that will repair the myelin sheath.

Since I started volunteering with the MRF, I no longer feel helpless or hopeless. The MRF’s vital research, and the opportunity for me to make even a small contribution to its efforts, have given me a very real sense of personal empowerment.  I am still angry, and when my legs aren’t working right, or I can’t button a blouse, or I don’t have the energy to go to dinner with friends, I keep my eye on the ball that the MRF has put in play: a drug that will help real people, people like me and hundreds of thousands of others with MS and other neurological impairments, regain a measure of normal function.

I’ll be blogging more about my personal experience with MS and related issues.  To make my blog posts as interesting and helpful as possible, I’d love to hear what topics you are particularly interested in, any questions you might have for someone with first-hand experience of MS, and anything else you might want to share.  Please e-mail me at glhagel@gmail.com with any and all thoughts!

The Boy who Cried MS

So tell me, have you ever been in a place where you were telling the truth but no one believed you? Kinda like the boy who cried wolf but in this story, the boy never lied.

Early in my teen years, I experienced changes in my body. Oh sure, puberty does some weird things to you at that age… but not fatigue, memory loss, or bladder problems. In school, I started forgetting my homework and kept falling asleep in class. Those are pretty common things  in high school freshmen right? At least that’s what all my teachers and family thought, assuming I was just lazy and apathetic about my grades.

MS is one of the most misunderstood diseases out there. MS is often referred to as an “invisible disease”. Meaning just because you don’t see it, doesn’t mean it’s not there. Some if not all MS patients, or “MSers” as I call them, are different. Some have vision problems and some have 20/20 vision. Some use a wheelchair, and some can run a city marathon. The fact is everyone has been around someone who has MS, its just you never know by looking at them. Without physical evidence, it is hard for other people to believe someone has MS.

On Sunday morning in February of 2003 I was at a past girlfriend’s church. I was sitting in one of the pews near the front with my her sitting beside me. A man stood up behind me to speak. I turned around to look at him when I noticed he was blurry. I haven’t woken up that early in a long time and figured I had sleep dust in my eyes still. But I noticed that when I looked at my girlfriend, my vision wasn’t blurry. When I drove home that afternoon I noticed every time I looked left my vision was blurry.

Concerned about my vision, I visited an optometrist. At first he thought I had diabetes, but a blood test proved that false. From there I went to an ophthalmologist and he said I had optic neuritis. Optic neuritis is a common symptom that shows up as a first sign that an individual has Multiple Sclerosis. Soon I was sent to a neurologist to get checked out. An MRI and lumbar puncture later, I was diagnosed with Multiple Sclerosis on March 3rd, 2003 at the age of 19. Finally, after all those years of crying wolf, I had evidence. And here it is.

This is a scan of Stephens brain showing evidence of multiple sclerosis.

Courtney Carver Supporting the MRF

Previously we blogged about Courtney Carver’s efforts to raise money for the Myelin Repair Foundation. This past month she put together a booth for a biking event to publicize the Myelin Repair Foundation. Here’s Courtney’s update on her project:

Last week was the Tour of Utah cycling event and I knew that would be a great place to display cycling images and bring awareness to how The Ride Project is supporting The Myelin Repair Foundation.

The Ride Project booth was a great success. We met many people with MS or that knew people with MS, but very few people that knew about the work that the Myelin Repair Foundation is doing. It was great to talk about the cutting edge research that is going on and to really inspire hope and excitement. I shared with them DVDs, brochures and talked to them about the progress they were making in myelin repair — a treatment that could help people with all types of multiple sclerosis.

Most of the people that stopped by the booth were surprised to learn that I had MS. They wanted to know about the things I did to stay healthy and to learn more about why I thought myelin repair was essential in terms of a cure for MS. Almost everyone that stopped by mentioned that they had either donated money or done fundraising for MS. They were shocked when we talked about the fact the the MRF dedicates 80% of their fundraising to research and I think that made them realize that not all organizations are the same in how they spend their dollars.

The Ride Project is my commitment to donate $1,000,000 to the Myelin Repair Foundation, through the sale of limited edition fine art photography. I know this is a lofty goal, but it is achievable. It may take time, but I am patient.

Ways you can help me…

1. Tell your friends about therideproject.com

2. Follow me on Twitter Click here!

3. Join The Ride Project Facebook page Click here!

4. Ask me about Guest Blogging or selling my photography on commission!

5. I would love to display images in your favorite bike or surf shop or even a great restaurant that is looking for some color! Email me contact info and recommendations: courtney@therideproject.com. There is no cost to the business you recommend to feature an image from therideproject.com.

6. Buy a signed, numbered limited edition print for yourself or someone you love. Click here!

7. Tell your Neurologist about The Ride Project and ask him/her to buy a print for their office waiting room to support MS research.

8. Blog about The Ride Project or include a link to the website!

9. Tell me what you think! Send me your feedback and great ideas.

A drug target is usually a small molecule that is specific to a disease condition. These two cartoon characters are line drawn molecules.

If you click through the Myelin Repair Foundation’s website, you will eventually happen upon the term “drug target.” And though these two words might seem straight forward separately, put together they have an entirely new definition that most people are unaware of as well as a critical role in drug development and design.

Contrary to what the name implies, a drug target is not the final goal put in place by a research team when they begin the process of finding a new drug. Instead, a drug target is usually a tiny molecule that is involved in some sort of process in a human cell. The process it is involved with can be anything from metabolic (having to do with metabolism) to signaling (taking a chemical signal and turning it into a physical response). However, what makes these processes significant is that they are specific to a disease condition, like the destruction of the myelin sheath with multiple sclerosis.

Take for example, a tree in the rainforest. This tree appears very healthy and lush from faraway; blocking harsh rays of sun by playing its part in the dense canopy of the jungle. But from a closer view, tiny lines appear to have been carved into the thick trunk of a tree. They run up and down and back and forth, crisscrossing so many times that it is clear that the tree, though it appears healthy from a distance, is actually terribly ill. These lines can represent a disease condition, almost like a symptom. When conditions like these are observed in a patient, it is then that it becomes important to investigate what is causing this condition. This is because one might be able to discover what is behind the illness and find a treatment.

Let’s continue the example. With further investigation it might become apparent that the cause of the carved lines on the surface of the tree was carpenter ants. These ants, with their sharp pincers, take the bark of the tree back to their colony to build with and use for energy. They carve and consume, scarring the tree and making it diseased. The ants in this example represent the drug target. They are the tiny molecule, or organism in this case, that is responsible for the disease. So by identifying the drug target, a researcher would be able to investigate its properties and then hypothesize how to stop, reroute or differentiate this pathway to keep the disease condition from occurring.

For MS, drug targets could be small molecules that initiate differentiation in oligodendrocyte precursor cells (OPCs) to oligodendrocytes (OLs) so that they can then make myelin. It is critical that research teams, like the ones that work with MRF, identify and pursue potential drug targets because they can ultimately lead to a treatment and an understanding of why the disease condition happened in the first place.

The number of potential drug targets that the MRF scientists have found has been increasing every year since the MRF was founded in 2002. As of late, thanks to the hard work of Kumar Hari, we have reached a total of over 150 drug targets. This is an extraordinary number of potential routes to take on the drug development path as we pursue a treatment for MS via myelin repair and could not have been done without your continued support and donations.

Things I wish I’d known when I was diagnosed:

There is life after diagnosis.  It will be different, some of it will be hard, but much of it will be great.

You can take your time learning about MS.  If there is something that scares you, ask a friend to read up on it and educate you.  It can be a lot easier to hear if it comes from someone you trust and knows how to share the information with you.  When you’re ready, if it’s your thing you’ll be doing plenty of your own research.

Your friends and family are being affected in ways you can’t imagine.  Don’t underestimate what they are going through.  Give them time to grieve with you and for you, assess the impact on their own lives as well as yours, absorb and adjust.

All your relationships will change.  Take it as it comes.  As some people go out of your life (whether your choice or theirs), wonderful new friends will come in.  They will be the ones who are there for you, who support you, who know how to listen, and who appreciate a good laugh.

It’s important to get a second opinion regarding both the diagnosis and treatment protocols.   I have a friend who, six years after her diagnosis and starting treatment, just found out from a new neurologist that she doesn’t have MS after all.  (Amazing, wonderful news for her.  Unfortunately, this doesn’t happen too often.)  Yes, protocols are fairly standard.  But advice concerning the choice of A, B, C or R drugs can differ.  As we all know, doctors often seem to have relationships with different drug companies, and this seems to affect their drug of choice.  Other protocol choices include whether you should be on regular steroid treatments, Tysabri, antidepressants, and other medications.  Gather all the information you can from the best doctors you can find and use your best judgment.

Look into other modalities that will complement your medical treatment.   Massage can help keep you loose and minimize spasticity.  Yoga, chi gong and tai chi are all wonderful tools to destress, stretch, and keep yourself as limber as possible.  Meditation has been shown to benefit the immune system in all kinds of ways.  (See my August 2^nd blog, “What, Me Worry?  Stress and MS.”)

Stick to your regular routine as much as possible.  You’ve had enough of a shock for a while.

While you’re going about your regular routine, plan adventures to look forward to.  Whatever rings your bells—a weekend trip or gathering of old friends or a trip around the world.   Don’t expect to be as active or energetic on the adventure as you might have been in the past; plan downtimes and a realistic schedule.  Then, if you are feeling good on the trip, you can do more—and that’s the icing on the cake.  Start planning the adventure after that before you embark on the first one.

If you don’t have a solid support system, get thee into therapy.  Even if you do have a great support system, having a professional to share your most private thoughts and fears with for as long as you need to is priceless.

Hire a professional to help you plan your financial future.  Be as realistic as possible.  It’s a cliché, and a good one: plan for the worst and hope for the best.   Work as long as you can because it provides income, keeps your mind occupied and sharp, gets you out of the house and among people.  Apply for disability when the time comes, but don’t count on it to see you through.  You need your assets and your options.

It’s okay to cry and be afraid.   It’s okay to say to a friend, “This is the pits.”  If you need reassurance, ask them for it.  If you don’t need that comfort, ask them to just be with you in that emotional place as long as you need them to be.  At the same time, don’t lose sight of the possibility that help is on its way, or that your MS will turn out to be manageable, or the probability that life still holds good things for you.  Don’t lose hope.  If you feel yourself losing hope, ask for help.  It is there.  You don’t have to go through this by yourself.

People who love you don’t love you because you’re healthy.  They love you because of who you are.  At the same time, MS can be as much of a challenge and frustration for them as for you.  Give them space if they need it.

Cut yourself a lot of slack, but also expect much of yourself.

There are researchers out there working on effective treatments, researchers like those at the Myelin Repair Foundation.  There is hope and it is up to all of us who care about finding those treatments to support the research organization of our choice.  If not us, then who will do it?

Keep a sense of humor.  MS isn’t funny, but trust me, eventually life will give you reasons to smile and laugh again.  With luck, in savored moments, you will even find joy.

What do you wish you’d known when you were first diagnosed?

Neil Buckholtz, chief of the Dementias of Aging Branch at the National Institute of Aging, in the National Institutes of Health.

The New York Times reports on a successful collaboration, the Alzheimer’s Disease Neuroimaging Initiative, which created a platform for sharing data on the biological markers (or biomarkers) of Alzheimer’s:

The key to the Alzheimer’s project was an agreement as ambitious as its goal: not just to raise money, not just to do research on a vast scale, but also to share all the data, making every single finding public immediately, available to anyone with a computer anywhere in the world.

There were many barriers they had to overcome to get scientists to  share their data. In the end, they managed to get scientists, government agencies, pharma companies, universities and nonprofits to work together to better diagnose Alzheimer’s and measure the  progression of the disease.

It’s great to see yet another example of a collaboration working in medical research.

Read the rest here.

…I’d like to tell you a story about one evening in the life of an MS patient.   There’s no real moral to the story—at least I’m not sure what it is if there is one, other than perhaps to say “m-u-l-t-i-p-l-e  s-c-l-e-r-o-s-i-s” and not “MS” when speaking to medical professionals (all will become clear below), but maybe you’ll find it mildly entertaining.

One night I was walking my dog and tripped on the uneven sidewalk, falling like a timbered tree and meeting the concrete with the side of my face.  (This had nothing to do with MS; it had everything to do with general clumsiness.)  For some reason, perhaps the shock of the fall, the hit to my head or the stiffness that sets into my muscles when my adrenalin is flowing, I couldn’t get up.  In fact, I couldn’t move at all.

A Good Samaritan came to my rescue, calling an ambulance and my friends Rick and Suellen on my cell.  He also took my dog, promising to keep him safe in his condo until I could come get him.  (You better believe I got every piece of information about him there was to be had short of his social security number.)  The ambulance came and I was lifted onto a gurney and placed inside, flat on my back and strapped to said gurney.  Then something strange happened.  We just sat there.  And sat there.  And sat there.

“When are we going to get going?” I asked.

“In a minute.  I gotta fill out this paperwork,” the ambulance driver said while the medic took my pulse.

“I have MS,” I said to the medic.  It seemed vaguely important that he understand this.

“What was that?” he asked.

Before I could answer, the driver asked me, “Have you done this before?”

I couldn’t make any sense out of his question.  “Done what before?”  No answer, so I turned back to the medic.

“I said, I have MS.”  He nodded with a blank look on his face.

“Why would you want to do such a thing?” the driver asked.

“What are you talking about?”

“Well, trying to kill yourself.”

“What?  Why would I want to kill myself?”

“That’s what I’m asking.  This came in as a suicide call.”

I felt as though I was receding, probably because of the surreal conversation he was forcing me into. 

“A suicide call?  Are you insane?”

“No ma’am.  That’s what they told me, and I’m supposed to find out why you did it.  I gotta put it on the forms here.”

“This isn’t a suicide call, it’s a trip-and-fall, and I can assure you that if I ever do decide to commit suicide, it won’t be by throwing myself face first on the sidewalk.  Now can you please just get me to the hospital!”

“Well, they told me you were trying to do yourself in.”  If we didn’t start moving soon, I was going to do him in.  “But if you say so…”  His tone indicated he had definite doubts, but he revved the engine and finally we set out.

At the ER there were the usual delays.  They wanted to do a CAT Scan of my head, and while I was waiting I showed my MedicAlert necklace to the nurse.  I’d bought it before a trip to Europe the year before, thinking it might do some good if there was emergency and I was knocked out or otherwise  unable to speak.  When you have MS you think about these things.  The pendant said I had MS and gave an 800 number to call.  The person at the other end of the line would have my entire medical history as well as contact information for all my doctors.

“It just occurred to me,” I said.  “No one in the ambulance or here at the ER has checked for this necklace.  MedicAlert told me that emergency heathcare professionals all over the world are trained to look for it before anything else.”

“Huh,” she said as she turned it over in her hand.  “I think I’ve seen these advertised on TV.  You know, on those late-night infomercial things.”

A few minutes later the medic from the ambulance appeared.  I was touched by his thoughtfulness in stopping by.

“I just want to wish you the best with the mass in your head,” he said.

“What?  What mass in my head?”  Suddenly I was back in the surreal world of the ambulance.

“You know, the mass.  The mass you kept telling me about.”

“Oh-h-h-h!  You mean the MS?”

“Yeah, the mass.” 

“Well, thank you.  That’s very kind of you.”

In the meantime Rick and Suellen appeared and, thankfully, took over.  About an hour later I was wheeled off for the CAT scan.  An hour after that a nurse appeared and said she wasn’t supposed to tell me but the scan was normal, and the doctor would be in soon.  An hour after that, at my request Rick  informed the head nurse that the doctor was taking too long and I was leaving.  Miraculously the doctor immediately appeared.

“Do you have any questions for me?” he asked, clearly irritated.  Maybe it was just me, but I kind of thought he would come in with information, not questions.

“Yes, as a matter of fact.  Do you have anything to tell me?”

“Yes.  Your CAT scan was normal.”

“Is there anything else I should know?”

“Put ice on your face.  It’s turning purple.  Sign the forms on your way out.  Good-bye.”

And that’s the story of my fall and visit to the ER. 

Have any good stories to share?  Let us in on them!  But please, no e-mails defending doctors or ERs or ambulance drivers.  There are so many wonderful ones—I don’t even want to think what we’d do without them!

Hopefully you have seen the MRF’s new video that was released earlier this week (and if not, you can check it out here). Now you can see what the video means to one of our regular bloggers, Gali Hagel. Gali shares with us about how the video will effect the MRF community and what it means to her personally.

Check out the video and also feel free to post your video response to our Making Connections.