Shinya Yamanaka. Image credit: Center for iPS Cell Research and Application, Kyoto University

Back in August 2016, Signals celebrated ten years of induced pluripotent stem cells (iPSCs) by hosting its first-ever blog carnival, in which contributors each wrote posts answering the same question: With useful treatments for diseases like heart failure and diabetes yet to be fully realized, “when [will iPSCs deliver] commercial products or new revolutionary medical treatments, and why?”

Some contributors were brave enough to make predictions. Michael Cea named Kyoto University’s Parkinson’s disease program, led by Jun Takahashi, as one to look out for, describing it as the leading iPSC Parkinson’s program and expecting it to enter the clinic in 2017. It got there in 2018, so not far off. Meanwhile, Malgosia Pakulska reported that Gordon Keller, the Toronto stem cell scientist who co-founded BlueRock Therapeutics later that year, expected iPSC-based therapies within the next ten years. David Kent was more cautious about therapies for diabetes, noting that patients may have to wait decades for a treatment that works for them.

Now, with Japan approving the first iPSC-derived therapies this year, and researchers gathering in Kyoto in October 2026 for the 20th anniversary, it feels like a good moment to look back at those predictions and see how they’ve played out.

As Signals readers will likely know, in 2006, Shinya Yamanaka and Kazutoshi Takahashi at Kyoto University discovered that switching on just four genes could reprogram adult mouse cells into pluripotent stem cells, which can become almost any cell type in the body. Human iPSCs followed in 2007, and the discovery won Yamanaka a share of the 2012 Nobel Prize. By the time of the 2016 blog carnival, iPSCs were already earning their keep in disease modelling and drug screening, but using them to replace damaged tissue was still in early clinical testing.

Two therapies reach the market, for now, in Japan

Image credit: Center for iPS Cell Research and Application, Kyoto University

Twenty years on, the first iPSC therapies have been approved where it all started. On March 6, 2026, Japan granted conditional, time-limited approval to two products, one for Parkinson’s disease and one for severe heart failure. It’s a world-first milestone, but not one built on large-scale trials. Japan’s conditional pathway, introduced in 2014, lets regenerative medicines reach patients based on small, early studies showing safety and likely benefit, before larger trials have confirmed it. Companies then have up to seven years to prove the treatment works.

Diabetes isn’t on Japan’s approved list yet, but encouraging signs are emerging, and David Kent was right to be cautious. In 2024, researchers in China reported that a woman with type 1 diabetes no longer needed insulin injections after receiving islets made from her own chemically reprogrammed cells. For the patient, it was life-changing, but for the millions of others living with type 1 diabetes, it’s more of a hopeful first sign than a cure for now. Making it repeatable is where much of the work lies, and some of it was on show in Kyoto, as we’ll see.

Japan’s conditionally approved therapies:

1. AMCHEPRY, from Sumitomo Pharma and Racthera, is for people with Parkinson’s disease whose motor symptoms are no longer well controlled by existing medicines. It contains dopaminergic neural progenitor cells, early versions of the dopamine-producing brain cells that Parkinson’s destroys, and it grew out of the very Kyoto University program Michael Cea tipped in 2016. Its supporting Phase I/II trial treated seven patients. At 24 months, four of the six assessed for efficacy had improved on motor tests taken while temporarily off their usual medication, which is encouraging. With no control group, though, nobody can yet say how much of that improvement was down to the cells, and that’s what larger, controlled trials will need to show.
2. RiHEART, from University of Osaka start-up Cuorips Inc., treats severe heart failure caused by ischaemic cardiomyopathy (where narrowed arteries starve the heart muscle of blood) when standard treatment hasn’t helped enough. It’s made up of sheets of heart muscle cells attached to the heart’s surface, and it’s been covered by Japan’s public health insurance since September 1 at a list price of ¥53.2 million (about CA$475,000). Japan’s income-based caps on medical bills mean patients pay only a fraction of that, though exactly how much depends on their age and income. The first insured transplant was at Osaka Police Hospital on September 29, 2026, for a woman in her fifties.

Gordon Keller’s company, BlueRock Therapeutics, also shows how far the wider field has come since 2016. Ahead of its 10th anniversary in December, its Parkinson’s therapy, bemdaneprocel, is in a pivotal Phase III trial, although it’s made from embryonic rather than induced pluripotent stem cells. Its iPSC-derived retinal therapy, lemiretprocel, is at an earlier stage in Phase I/IIa, but received orphan drug designations in the U.S. and Europe this year.

So, commercial products? Tick, with an asterisk. There’s still a way to go before we have a fully-fledged commercial therapy proven and available at scale.

The road to revolutionary treatments

The International Society for Stem Cell Research’s 20 Years of iPSC Discovery symposium took place in Kyoto from October 20-22, 2026, opening with a keynote from Prof. Yamanaka himself. According to the organizers’ same-day release, he used it to discuss the regulatory milestone of Japan’s two conditional approvals, as well as the scientific and translational challenges that remain. I wasn’t lucky enough to be there, but his open-access March 2026 article marks the 20th anniversary and gives an assessment of how far the field has come. In it, he points to early trials in macular degeneration and Parkinson’s as evidence that iPSC-derived grafts can be transplanted safely, the groundwork Japan’s approvals were built on.

Other highlights from Kyoto included:

• Syntax Bio reported that its CRISPR-based Cellgorithm platform pushed human iPSCs towards pancreatic beta-cell states in just four days, which could make cells for transplant faster and cheaper to produce. Functional testing is now underway.
• Maike Sander of Berlin’s Max Delbrück Center presented lab-grown islet organoids built with blood vessels and supporting cells, which help beta cells work better and make more realistic models for testing treatments. Her team’s 2025 study showed the approach also sped up diabetes reversal in mice.

So, 20 years on, how did the forecasts hold up? On commercial products, the optimists can take a bow: iPSC-derived therapies are conditionally approved, priced and reaching patients, in Japan at least. On revolutionary treatments, there’s still a long way to go, but it’s becoming a question of when rather than if. The next few years of data will show how quickly these first approvals become the foundation of a new kind of medicine, and with artificial intelligence and robotics advances now starting to reach the lab, it could be sooner than any of us predicted. That’s the subject of my next blog, so watch this space. I’m optimistic, and I have a feeling the 30th iPSC anniversary will be quite the celebration.

A brief primer on opsins

The Nobel win began with green algae: single-celled photosynthetic organisms that detect light and swim toward it for food production. Green algae use a protein called channelrhodopsin (ChR) to do this. As the name implies, ChR proteins are ion channels that open when exposed to light of a certain wavelength. They work with a vitamin A-derived co-factor called all-trans-retinal (ATR), which is already found naturally in mammalian cells and doesn’t need to be supplied by researchers. This availability sets ChRs apart from previous, more complex attempts to control neurons with light, as awesome as they were. ATR changes shape when light hits it, and this causes the entire channel to open up. See the landmark paper on ChR1, and this one on ChR2. Two of the three Nobel winners – Nagel and Hegemann – discovered and characterized these proteins, landing them the prize.

ChR2 is a particularly important player in this story; when blue light hits ATR and the channel opens, sodium floods into the cell. In algal terms, this means it’s heading in the right direction. In neural terms, a flood of sodium into a cell is what allows it to fire electrical impulses. Recognizing this, Deisseroth (the third Nobel winner) engineered the gene for this protein into brain cells. The rest, of course, is history.

Since ChR2, many new opsins – the term we use for light-sensitive protein actuators – have either been discovered or artificially created in the lab. ChR2 was truly just the beginning. Why do we need so many? I explain below; but first, we have to discuss what makes optogenetics different from other modalities.

Life before optogenetics

Neuroscientists were already mapping neural circuits using electrical stimulation in humans and animals. Electrical stimulation is useful because it’s inherently the language of the brain, and it’s temporally precise. Electrical pulses are really quick. You can easily track behaviour as it relates to different types of stimulation patterns and durations. You can also see near-immediate differences between periods of time where stimulation is on versus when stimulation is off. For example, rats will enthusiastically press on a bar (over, and over, and over again) for electrical stimulation to their reward circuitry.

But there’s a problem with this approach: Each part of your brain is a mix of different neuronal cell types – GABA, dopamine, serotonin, and so forth. Electrical stimulation zaps them all at once, meaning it’s not helping to determine the functions and contributions of individual cell types. Neurotransmitter information is critical for drug development efforts. Also, if you want to see what happens when you turn neurons off, in most cases you’d have to create an electrical lesion. Given that lesions are a permanent burn made in the tissue, they’re tougher to accommodate in experimental designs and animal welfare, depending on the region of the brain. It’s also risky in humans for obvious reasons.

Parties more interested in specific, cell-type functions could instead use pharmacological manipulations, including receptor agonists and antagonists, or innovative chemogenetic tools. I recall an era of DREADDs, Designer Receptors Exclusively Activated by Designer Drugs. You could engineer your cell type of interest to express an entirely synthetic lock-and-key chemical system that could selectively activate or inhibit neuron classes without as many off-target effects.

The drawbacks of these approaches, however, are the timescale of action, in addition to the challenges with spatial selectivity. Pharmacological and chemogenetic approaches rely exclusively on brain injections of either a drug or an agent for genetic engineering. It’s tough to inject volumes limited to the brain structure of interest because liquids naturally diffuse as far as physics will take them. In addition, you can’t turn chemicals on and off in a temporally precise way to study activity patterns: Are the electrical impulses evenly spaced? Do they arrive in quick bursts? Are they variable? There’s no way to know.

It was simply down to a large, relatively unnatural flood of chemicals that either activate or suppress activity. You couldn’t turn it off unless you added another chemical to cancel it out again (and that also takes time); otherwise, you waited for the “washout” period, meaning the body would just metabolize and expel the chemical on its own. On the other hand, there were chemical toxins that could lesion an area and allow you to see what happens, but again the permanence of that is not ideal, and the damage could travel farther than intended.

So, in the lead-up to optogenetics, there was a trade-off. Researchers had to choose between controlling when they stimulated the brain and controlling what they stimulated. It’s a bit like making music with a piano: you could either strike groups of adjacent keys at exactly the right moments, or play individual notes without controlling the rhythm. Neither approach let you perform a complete melody.

The new era: Optogenetics, everywhere

Enter the era of ultraprecision. We can now engineer specific types of cells to respond to light; there’s nothing faster than that. Now we have both layers of control at once. You can figure out what neuronal cell type drives a given behaviour or function and what sort of coding language is being used. You can test different firing patterns (as I have!) and control their activity in a reversible, impermanent way. Another powerful improvement is spatial control. Even if your viral infusion for the initial genetic engineering travels farther than intended through the tissue, only the illuminated volume will be affected, and this is easily modelled. Some people are even illuminating just a few cells at a time with holographic optogenetics to try and reproduce specific local firing dynamics.

Earlier, I mentioned that it’s no longer just ChR2 that we have to work with. It was the first opsin protein used, but scientists have since discovered and synthetically created many more new variants to test out different hypotheses. Scientists studying neurons that fire very quickly are better off using ultrafast variants such as the ChETA – my personal favourite. Others require highly sensitive opsins that can be stimulated through the skull. Some users inhibit neurons at the cell body and go with a run-of-the-mill halorhodopsin, while others need inhibition at the axon terminal and choose an eOPN3 opsin from the humble mosquito.

As for matters of application, I’ve written about multiple optogenetic tools engineered to try and replace lost photoreceptor cells of the eye in degenerative eye diseases. This is possible because you have neurons in your eyes, and they allow you to see in cooperation with other front-line, light-sensing cells. If those light-sensing cells are lost, you can make the neurons themselves light-sensitive instead via optogenetic tools. Ongoing Phase I through III clinical trials thus far have yielded highly positive results, ranging from findings of safe outcomes to demonstrated restoration of functional vision.

That being said, optogenetics has now moved beyond the brain. Scientists are looking into how optogenetics can be used for severe, intractable pain conditions. Optogenetic tools are being used in ALS research at the interface of nerves and muscles. I’ve seen papers exploring the use of optogenetics to study cardiac musculature and heart regeneration. Stem cell biologists have used it in the study of differentiation. The field has absolutely exploded as a result of Deisseroth’s groundbreaking success in 2005.

Interestingly, teams are trying to get optogenetics to work in the human brain for therapeutic purposes. Right now, electrical deep brain stimulation (DBS) is approved by Health Canada and the FDA for multiple neurological disorders, including Parkinson’s and essential tremor, and it’s being researched for many more.

While it’s highly effective in its approved indications, the therapy is not perfect for the reasons I mentioned earlier: you can’t choose which brain chemical is affected in a manner that is as precise as optogenetics, though optimized patterning can help activate certain neuron cell types over others. In addition, different regions will react differently to the same electrical pulses, as they all have different properties and, importantly, different network inputs.

With optogenetics, treatments for neurological diseases could have much stronger specificity. Ensuring that toxicity and immunogenicity are brought under control remains the first priority. Also, human brain structures are larger than their counterparts in rodents, which means getting far more light into the brain (and the dangers of biological tissue heating do grow with light intensity). For this reason, it may be helpful to employ the specialized, highly sensitive opsins that I mentioned above – less light will be required for the tool to work, reducing heat-related side effects.

The last noteworthy element in need of optimization is delivery. Gene therapy in humans isn’t perfect yet, and for a prime example of this, see Sarepta’s recent issues with viral vector delivery and liver toxicity. However, it should be noted that what we learn in animal studies using optogenetics is directly informing DBS and other therapies already. For now, these data provide strong indirect benefits to various clinical interventions.

Long awaited

I hope that I’ve made the breadth and depth of significance associated with optogenetic tools a bit clearer. Though DREADDs and other innovative methodologies were critically useful and highly advanced, they could never quite solve enough problems all at once. As optogeneticists, we’ve been waiting for this moment for years because we experienced the symphonies ourselves, first-hand, and we’re in awe. Indeed, the team swept the awards that typically predict a Nobel. Does the technique still have some way to go before it reaches its full potential? Absolutely. However, the foundation is there, and the paradigm shift is complete.

Blood pressure gauge, courtesy of Pixabay

What is dysautonomia?

Dysautonomia, also known as “autonomic dysfunction,” is the improper functioning of the autonomic nervous system. Rather than a diagnosis itself, dysautonomia is an umbrella term, frustratingly vague in the scope of symptoms and diagnoses it covers. The autonomic nervous system regulates most automatic, unconscious functions, including heart rate, blood pressure, digestion, temperature regulation and circulation, amongst others. So, when things aren’t working as they should with the autonomic nervous system, it can present as a wide variety of symptoms. Some of these include body temperature swings, shortness of breath, brain fog, fast or slow heart rate, fatigue, dizziness and fainting. Some of the conditions are rare, while others are common. This is not even close to a complete list.

The most common symptom is orthostatic intolerance. This is when you stand up and not enough blood reaches your brain, leading to dizziness or fainting. One type of orthostatic intolerance is POTS, a common diagnosis under the umbrella of dysautonomia.

What are some other specific types of dysautonomia?

POTS is the most common diagnosis. But, when experiencing symptoms of dysautonomia, you could also be diagnosed with orthostatic hypotension, vasovagal syncope, inappropriate sinus tachycardia, autoimmune autonomic ganglionopathy, baroreflex failure, familial dysautonomia, pure autonomic failure and multiple system atrophy.

Symptoms frequently overlap, and getting a diagnosis can be difficult. On average, it takes over seven years to get a proper diagnosis. Not to mention, over half of POTS patients were told by a doctor that their symptoms were all in their head. Often, patients are labeled with an anxiety disorder before continuing to seek help and receiving a new diagnosis. Of those with dysautonomia, around 80 per cent are women.

Getting a diagnosis is difficult due to a variety of factors. Testing is complex, as there is no simple biomarker. There is a lack of awareness of the disorder, even amongst doctors. And there are widespread, overlapping symptoms because it affects multiple bodily systems.

Although a diagnosis can feel reductionist to patients experiencing a unique manifestation of symptoms, it can be a useful tool to accurately label and discuss the disease, leading to more awareness both in and out of a clinical setting.

What does it have to do with autoimmunity?

Dysautonomia can be secondary to an autoimmune disorder. Dysfunction of the nervous system can frequently co-exist with mast cell activation syndrome, Sjrogen’s disease, diabetes, and celiac disease. This might be due to the immune system specifically attacking the nervous system, thus making the dysautonomia symptoms a result of the autoimmune disorder. Or they might both be resulting from another cause, like a viral infection. Regardless, they often worsen one another by increasing systemic inflammation. It is interesting to note that 80 per cent of autoimmune diagnoses are also women.

If stem cells can help autoimmunity, can they help dysautonomia, too?

One of my previous blog posts discussed the possibility of stem cell therapies for autoimmune diseases. As I wrote and this study indicates, mesenchymal stem cells (MSCs) have been shown to reduce inflammation and over-reactivity of the immune system. It has been proposed that their immunomodulatory and anti-inflammatory characteristics might be beneficial for dysautonomia, especially when it is secondary to an autoimmune disease. However, the clinical research is incredibly early and experimental.

In 2017, a small cohort study was done at the University of Texas. Two young patients received a specific type of MSCs known as ASCs (adipose stem cells) as an intravenous infusion. The first patient had severe dysautonomia symptoms, as well as elevated autoimmune antibodies. After the infusion, she saw a significant reduction in inflammatory cytokines, and she was able to complete a head up tilt table test without syncope (fainting). These remained three months after the treatment.

The second patient had arthritis, hypotension, tachycardia (abnormally fast heart rate) and dizziness, so severe it prevented him from attending school. Three months after the infusion, his symptoms and arthritic pain were practically nonexistent, allowing him to discontinue his other medications (such as anti-inflammatory agents). He also completed the 30-minute head-up tilt table test without syncope.

Because of the significant reduction in symptoms, this study received a lot of attention, particularly from stem cell banking companies. Further research is needed, with many more patients, before MSCs could be considered a safe and effective treatment for dysautonomia.

Other treatments

Currently, efficacious treatments for dysautonomia are lacking. Most include non-pharmacological approaches, such as lifestyle changes and nervous system regulation. Some are listed below.

• Increase salt and fluid intake to expand blood volume and reduce hypotension
• Eat small meals frequently
• Wear compression garments to prevent venous pooling
• Limit prolonged standing
• Exercise
• Get regular and adequate sleep
• Try yoga, acupuncture, deep breathing, or other stress-relieving activities that activate the parasympathetic nervous system

There are also some pharmacological approaches, but most involve off-label medications. For example, vasoconstrictors and beta blockers are used to ease dysautonomia symptoms but fail to address the root cause. Drugs that lower anxiety or suppress an overreactive immune system are also commonly prescribed.

Despite competing hypotheses, it is unclear what exactly has caused the increased prevalence in autoimmunity and dysautonomia. Studies suggest a combination of environmental factors, like infections, diet, obesity, sleep deprivation, microbiome changes, stress, and air pollution. Dr. Wang at Yale School of Medicine proposes that part of the increase may be chemical use associated with increased sanitization. He says that his patients often experience flare-ups when dealing with stress. If stress plays a role, it is interesting to note the wide gender disparity. It is exciting that fields like regenerative medicine may offer a solution that gets closer to the root cause by alleviating inflammation and immune system over-reactivity.

Nine months ago, CCRM established DeepTech Bio Lab™ (DTBL) and announced its first partner: IonQ, a global leader in quantum computing. Since December 2025, DTBL has put business, legal, financial and governance infrastructure in place; set up workstreams and developed strategic plans; begun executing projects; developed branding tools and created marketing materials; and recently hosted a workshop and an event, to name just a few accomplishments.

Highlights from “Quantum Leap: A New Era in Advanced Therapies” 

On September 16, 2026, DTBL hosted “Quantum Leap: A New Era in Advanced Therapies,” at BioLabs University of Toronto, in Toronto, Ontario. The event brought together industry leaders, investors and innovators to explore how quantum computing, AI and other emerging technologies can revolutionize advanced therapies.

The program included an overview of DTBL; an introduction to quantum computing by Sumner Alperin-Lea, Quantum City; a discussion of quantum in life sciences by Zheng Cui, IonQ; and a life science use case presented by Evgeny Epifanovsky, IonQ. The final talk was an investor view of quantum by Cynthia Lavoie, CCRM Enterprises Inc., and Steve Tregay, Mission BioCapital. A lively panel discussion followed, and the event ended with a networking reception.

Recognizing the growing importance of data and deep tech solutions, DTBL was established to identify high-value life science challenges and determine where advanced computational technologies can create measurable impact. DTBL is intended to serve as a collaborative and neutral ecosystem builder rather than a technology developer itself. As Michael Israels, CCRM’s Chief Financial Officer, noted in the kickoff presentation, DTBL focuses on practical application rather than technology development for its own sake.

The following slide, presented by Michael Israels, outlines how DTBL could deploy emerging technologies to increase efficiency and reduce the cost of advanced therapies.

Image courtesy of DeepTech Bio Lab

Jessica Tate, CCRM’s Vice President, Vector Platform Technology, also spoke about DTBL and explained how it could optimize the lentiviral vector purification process, which currently takes many months and can involve substantial financial losses and delays if manufacturing fails. To optimize the process, DTBL would use computational modelling, AI, automation and quantum techniques. The objective would be to reduce development timelines, improve yield and decrease manufacturing costs while maintaining viral particle integrity.

Speaking of manufacturing, we heard that biomanufacturing is a major area for computational innovation. Potential applications include process optimization, supply-chain management, advanced analytics (data as a strategic asset was also mentioned), process control, manufacturing simulation (using technology to model, experiment and optimize your production process using digital models of your manufacturing environment) and resource allocation.

When Cynthia Lavoie, President and Chief Investment Officer of CCRM Enterprises, gave her talk, she emphasized that DTBL acts as an evaluation and validation platform for emerging opportunities before investment decisions are made. In the second part of the investment presentation, Steve Tregay, Managing General Partner, Mission BioCapital, noted that approximately one-quarter of emerging companies entering their ecosystem now identify advanced computational capabilities as a core differentiator. His message was clear: start by solving a valuable problem. Technology selection should follow from the problem, not precede it.

Photo: panel discussion with (l-r) Alán Aspuru-Guzik, University of Toronto, Lisa Lambert, IonQ, Kausar Samli, DeepTech Bio Lab and Board Director, Genome BC; and Michael May, CCRM (moderator).

A lively panel

The panel discussion opened with a question about what has changed of late to enable us to focus this conversation around real-world applications of quantum computing. The answers included: improved quantum hardware; better algorithms; more sophisticated software tools; growing talent pools; and stronger industry partnerships. In other words, the conversation has shifted from “What can quantum computers do? to “Which real-world problems should quantum computers solve?”

The panellists emphasized the importance of collaboration among key stakeholders and described the field as a “team sport” requiring continuous collaboration between technical and domain experts.

Repeated again during the panel was the message that commercial success depends on solving meaningful problems, not simply adopting trendy technology. We shouldn’t be distracted by “quantum pixie dust.” Successful companies will identify an important problem, understand customer needs, determine whether quantum methods can create genuine value and build solutions around measurable outcomes.

A central theme throughout the event was that life sciences and advanced therapies face increasingly complex biological, manufacturing and data challenges that traditional approaches may struggle to solve efficiently. We heard that AI/ML, quantum computing, automation and robotics are converging to create new opportunities for innovation across the entire biotechnology value chain.

The panellists expressed optimism that quantum-enabled applications will begin appearing in life sciences workflows over the coming decade and concluded that many of the most important opportunities will emerge through the convergence of quantum computing, AI, advanced analytics and biological expertise.

A little quantum homework

While those of us in the room learned about the fundamentals of quantum computing from Dr. Alperin-Lea, I recommend you watch the videos below rather than relying on this English major to disentangle (quantum pun intended) this topic.

If you want some fun with your physics, the novel Dark Matter, by Blake Crouch, is an enjoyable read. I read it about a decade ago and enjoyed this sci-fi, mystery, suspense/thriller, and felt a little wiser afterward. It has been turned into a series and is now streaming. The other night I watched Season 1, Episode 1, and was amused to see the protagonist teaching his university class about Schrödinger’s Cat. Dr. Alperin-Lea also discussed the famous, fictional and fateful feline in his presentation.

Watch IonQ’s “What is Quantum Computing?” below and the follow-up video, “How does quantum computing work?” at this link: https://www.youtube.com/watch?v=uqBiCvRRVz4&t=28s

It started with this parody song about placebos, by anesthetist Dr. Suman Biswas, which The BMJ posted on X in two parts. I thought maybe it was time for a light parody post, so I was looking for one link that I could share. This led me to The BMJ’s “Creating a healthier world” YouTube page, which features podcasts, explainer videos, webcasts, and more.

And that sent me down a different rabbit hole: how do we know whether the health information we encounter online is trustworthy?

Turning to social media for health information

I was immediately enticed to watch “How the Internet Hijacked Our Health” since this topic dovetails with my interest in misinformation and disinformation. BMJ editor Kamran Abbasi interviewed author Dr. Deborah Cohen about her new book of the same name (Bad Influence: How the Internet Hijacked Our Health), marketed by the publisher as follows: “When did we start trusting influencers over doctors? You used to see a doctor. Now you go online.” The short answer to the question above is: during the COVID pandemic. For a more nuanced answer, read Cohen’s book, shortlisted for the 2026 Royal Society Science Book Prize.

I don’t think it will surprise you to read that tens of millions of Americans now turn to social media to obtain health information, according to the Pew Research Center. Canadians do as well.

The ISSCR’s Stem Cell Reports has covered the rise of health information on social media, in this case focusing on YouTube. Authors Anders Grundtvig, University of Copenhagen, and Megan Munsie, University of Melbourne, write about “Stem cells in your YouTube feed: The complex landscape and unexpected mediators of online information” and rightly point to the concerns that “authority, reliability, and accuracy” may be sacrificed.

Focusing on stem cell treatments on YouTube, they note that “misinformation already abounds online about unproven stem cell procedures,” so their analysis instead focused on mapping these videos and identifying the mediators and content creators behind them. They collected metadata from 4,481 YouTube videos across 1,734 YouTube channels. There was lots of engagement (comments and likes) and over 134 million views. Read their analysis here.

Drs. Grundtvig and Munsie found that “one influencer, above all others, appeared to have the most impact – Joe Rogan.” I’m not surprised. In 2019, I wrote an open letter to podcaster Joe Rogan about his endorsement of unproven stem cell therapy. In the stem cell space, he is a prominent and problematic advocate. In 2023, I blogged about him a second time. You can read my “interview” with Joe Rogan.

The source matters

Obtaining health information from the internet and social media isn’t necessarily problematic, depending on who you are following and trusting. For example, the College of Physicians and Surgeons of Ontario (CPSO) has developed social media policies prohibiting doctors from posting false or deceptive information. The CPSO also provides advice to Ontario physicians about their use of social media, and outlines what it considers unprofessional behaviour. If doctors are making claims that contradict established, evidence-based public health guidelines without providing reliable sources, that should be a red flag.

Of course, many health and wellness influencers are not doctors, nor do they have to abide by a professional code of conduct. Some have enormous audiences despite having little or no relevant scientific or medical expertise. For example, while some call actress and Goop founder Gwyneth Paltrow a wellness guru, a Toronto-based nutritionist described her as the “reigning queen of problematic wellness misinformation and disinformation.”

Timothy Caulfield, a Canadian professor, health law expert and science communicator known for debunking medical myths and addressing misinformation, went so far as to title his 2015 book Is Gwyneth Paltrow Wrong About Everything: When Celebrity Culture and Science Clash.

More recently, Caulfield and University of Alberta law colleagues Alessandro R. Marcon and Blake Murdoch published an article in the Canadian Journal of Bioethics titled “Influencers and Unproven Health Claims: Curbing Misleading Promotion in the United States and Canada.” They examine the state of legal prohibitions and enforcement relating to misleading advertising – such as for unapproved stem cell treatments – which the authors deem “inadequate,” and they describe the public health harms and how health misinformation erodes trust in public institutions, among other things. They suggest that artificial intelligence could assist with enforcement.

Online sources to keep you healthy

So, what are some reputable sources of health information? There are many! I’ve already mentioned The BMJ’s “Creating a healthier world” above. Naturally, there are medical institutions, hospitals, health charities, government agencies, and other organizations whose mandate is to provide evidence-based information. If you like to watch videos, the Canadian Medical Association (CMA) has its “healthcare for real” site, and it includes videos.  In the stem cell field, visit Dr. Paul Knoepfler’s website. With blogs, videos, and educational materials, his site is a great resource.

Spending time online has its benefits. It turns out that the lighthearted parody song led me to a serious question about whom to trust with our health. In the end, it was a healthy distraction.

The CMA asked physicians to recommend sources for accurate health information. You can watch this video posted to Instagram. Add your own recommendations in the comments below.

Article #1: Raising awareness of endometriosis

A teen from Vancouver, Elize Nocente, is attempting to raise awareness of endometriosis in schools so young people can identify the signs of this complex, chronic disease and seek care earlier. She lives with endometriosis and believes other young people do as well; after all, The Endometriosis Network Canada estimates two million Canadians are affected with this inflammatory disease, in which tissue similar to the lining of the uterus grows outside of it and can cause chronic pelvic pain, painful periods, painful sexual intercourse, bowel and bladder symptoms, and infertility.

For too long, painful periods have been dismissed as something people who menstruate simply have to endure. But severe or persistent menstrual pain is not something that should automatically be normalized. For someone who develops symptoms in adolescence, the years before diagnosis can represent decades of recurring pain, disrupted education and work, and reduced quality of life. According to an article in the Journal of Obstetrics and Gynaecology Canada, a survey found “it takes an average of 7-10 years to receive a diagnosis and treatment for the condition.” That’s not a typo.

Part of the broader problem is that women’s health has historically received insufficient research attention. Researchers from the University of Toronto and the Centre for Addiction and Mental Health reviewed 11 years of grant funding (2009-2020) from the Canadian Institutes of Health Research and found that only 6 per cent of funding dollars went to female-specific research and the number fell to 4 per cent when female-specific cancer research was excluded. A recent report from MaRS Discovery District states that women were omitted from clinical trials in Canada until 1997 and today make up only 30 per cent of participants. Research funding and clinical-trial participation shape which diseases we understand, which treatments we develop and how well those treatments work for different populations.

Treating endometriosis through regenerative medicine

Dr. Peace Chukwu, who blogs for Signals, has written about the therapeutic potential of menstrual blood-derived stem/stromal cells in endometriosis.  In summary, menstrual stem cells (MenSC) may offer a promising new avenue for understanding and treating endometriosis. Researchers are studying how these cells and the substances they release may help identify endometriosis earlier and, in the future, potentially reduce the inflammation, abnormal cell growth and blood-vessel formation that allow endometriosis lesions to develop and persist. While early laboratory research is encouraging, MenSC-based diagnosis and treatments are still experimental and require further research before they can become part of routine care.

A registered clinical study is now investigating menstrual stem cells in endometriosis, illustrating how this field is beginning to move from laboratory research toward clinical investigation.

Article #2: AbCellera looking to solve a common menopause symptom

The second article that caught my attention was about Vancouver-based antibody developer AbCellera and its work on hot flashes, a menopause symptom that up to 80 per cent of women suffer from. AbCellera’s new investigational antibody medicine, called ABCL635, could become, according to AbCellera, “a best-in-class non-hormonal therapy for women in menopause or undergoing hormone-suppressing cancer treatments.” Fingers crossed. You can learn about ABCL635 and hot flashes on AbCellera’s website.

While the results of the trial of 92 women conducted across Canada are very exciting – an investment analyst called the results “practically off the chart on everything measured” – also exciting is the implication that this breakthrough has positioned AbCellera to become Canada’s first homegrown Big Pharma giant. Solving an unmet women’s health problem can become a significant drug-development opportunity.

Why women’s health innovation matters

Closing Canada’s women’s health gap could potentially add $37 billion to Canada’s GDP annually by 2040, according to the McKinsey Health Institute. Further, “Whereas Canada ranks among the top ten countries globally for gender equality and is the ninth-largest economy, it represents the fifth-worst women’s health-related economic gap as a share of its projected 2040 GDP, behind Australia, France, Germany, and Japan. This means Canada has an opportunity to build a stronger economy based on healthier women [bolding is mine].” McKinsey also points out that its $37-billion estimate does not include emerging areas such as endometriosis and polycystic ovary syndrome.

The Menopause Foundation of Canada says that unmanaged symptoms of menopause cost the Canadian economy an estimated $3.5 billion per year through health care costs, absenteeism, lost productivity ($237 million) and other impacts.

Canada’s femtech sector is rapidly emerging as the world’s third-largest femtech ecosystem, driven by over 170 companies (tracked by Femtech Canada) addressing critical gaps in women’s health. According to an article in Canadian Healthcare Technology, the ecosystem has created more than 1,200 jobs across Canada and “generated over $250 million in private-sector investment.” Femtech Canada says the ecosystem has filed 75 new patents (as of December 2024).

These numbers suggest that women’s health is not simply an underserved health care market. It is becoming an important innovation sector.

Women’s biology has created opportunities for entirely new diagnostics, therapeutics, devices and care models.  Watch the TEDx Talks below, by Loulia Kassem, on “Why women’s health is the future of healthcare.” Kassem is the Co-founder and CEO of Rea Diagnostics, a Swiss medtech startup that has developed the first at-home, self-test for preterm births.

Her talk focuses on the need for modern medical technologies for women (not ones created in 1845!), painless ones (think uncomfortable mammogram equipment), and dignified (she references endometriosis). She reminds us that the women’s health gap is not only a health care problem. It is also a research gap, a data gap, a clinical-development gap and an innovation gap. Canada has both a problem to solve and a growing ecosystem capable of solving it.

This blog first appeared on CCRM Australia’s website and is reposted here, with permission. It contains some minor edits from the original.

CCRM, a leader in developing and commercializing regenerative medicine-based technologies and cell and gene therapies (CGTs), has long supported early-stage technologies. One of the key ways for doing this has been through a program of technology evaluation and incubation, with the end goal of creating companies that can propel promising advanced therapies into the clinic.

Traditional models of company creation often begin with a promising technology. While this approach has produced many notable successes, it is also resource‑intensive, inefficient at scale, and yields only a low number of compelling investable ventures. In a nutshell, starting with technology and then searching for a market can be a risky strategy.

CCRM’s venture studio model, Venture by Design, was developed to complement existing frameworks by introducing a more explicitly market‑driven entry point. The process differs in that it begins with clearly articulated unmet needs (which could be technical, clinical, manufacturing, or operational), and then deliberately assembles the science, intellectual property (IP) and capabilities needed to address them. This framing shifts attention from developing specific individual technologies to building the overall configuration required for a viable company.

From technology push to company readiness

In the few years since we launched our venture studio, we’ve witnessed firsthand that proto companies that originate from clearly defined bottlenecks and problem statements meet commercialization readiness standards both faster and more often. Viable ventures are those designed with credible answers to questions such as: How might we best solve the problem? Who will adopt this approach? How will it be paid for? Can it be made at scale? And why this solution now?

Our shift from asking “Is the science interesting and can it be applied to a certain problem?” to “Does this configuration support a workable company?” has proven to be one of the most consequential aspects of the model.

Emerging patterns across venture studio cycles

Running repeated venture studio cycles has revealed additional patterns that separate viable company creation from stalled efforts:

1. Market pull is non‑negotiable.
   Companies born from the desire to address clear unmet needs (e.g. underserved patient groups, manufacturing constraints, delivery gaps, etc.) create more compelling propositions than those built around unanchored science. Early end-user and partner conversations should be used as design inputs.

2. Initial de‑risking must happen before incorporation.
   Wet‑lab diligence, application-driven feasibility work, manufacturability assessments and freedom‑to‑operate thinking are most powerful when done before a company is set up. This early exploratory work enables the concept and strategy to be refined in an expedited, capital-efficient manner. CCRM’s specialized laboratories and academic collaborator network are hugely beneficial for this.

3. Early external engagement is not optional.
   Programs that engage pharma, clinicians, or health systems early don’t just validate the need, they also shape the product. Even tentative early engagement can benefit downstream development. It may be surprising to some, but these stakeholder groups are predominantly very willing to engage and advise, for which we are very grateful.

4. IP strategy must be intentional, not reactive.
   Successful Venture by Design projects treat technology components and IP as design variables. Being overly prescriptive or fixated upon specific solutions early in the concept-building stage can stall progress.

5. Teams matter earlier than you may expect.
   A recurring challenge is the availability (and bandwidth) of leadership in pre‑financed companies. Ventures that allocate a leader to drive fundraising, partnerships and execution, even if only in the interim, outperform those trying to manage the early stages by committee. 

Reflections on the Venture by Design model

One surprising aspect of working in this model is how much of company creation is about trying to remain objective about emerging concepts, and how challenging this is. Venture by Design is as much about pivoting from ideas early as it is about nurturing them. That can be difficult when accustomed to championing novelty, but it’s entirely necessary.

Another lesson is that incubating concepts inside a larger organization has drawbacks. Our deep infrastructure, expertise and networks are powerful accelerants, but they can also slow decision‑making if not managed deliberately. The ventures that progress fastest are the ones that selectively decouple to some degree and adopt startup‑appropriate processes early while still leveraging institutional strengths.

Further, we’ve found from running our model that process and infrastructure alone are not the advantage, but rather their orchestration is. Access to manufacturing, capital, talent and partners only creates value when it’s deliberately sequenced. The Venture Studio’s strength is not just in structured ideation but also in its integration. 

Why Venture by Design works

Venture by Design is not simply an ideation workshop; it’s a company formation system. It integrates:

• An ideation process grounded in market need
• Structured, stage-gated diligence with objective scoring
• Early partner and customer signals
• Intentional governance, IP, and incubation design
• Capital‑efficient paths to value inflection

The belief is that this combination will increase the probability that a spun‑out entity is more financeable, partner‑ready and resilient to occasional pivots. For the regenerative medicine sector, where timelines are long and capital efficiency matters, an ordered and disciplined “bench to bedside and back again” approach is a necessity. If we want more companies to reach patients, we must spend time designing the company as well as developing the science.

Venture by Design works because it treats company creation as a system, integrating science, diligence, partners and capital into a coherent whole. As our venture studio continues to evolve (incorporating quicker early validation, stronger external engagement and deeper advanced computing), I’m convinced this type of model will be shown to be one of the most effective paths for translating complex biological innovation into durable, scalable companies.

The takeaway is simple: while great science and technologies are of course necessary, viable, high-impact companies happen by design.

Peptides are chains of molecules called amino acids that link together; they are smaller versions of proteins. Thousands of naturally occurring peptides have been identified in the human body. They perform a wide variety of biological functions, although scientists continue to discover new peptides and learn more about their roles. Many peptides act as signalling molecules, allowing cells to communicate with one another, while others are hormones that bind to receptors on cells and regulate processes such as appetite, metabolism, growth, inflammation and tissue repair. Insulin is widely recognized as the first peptide hormone to be isolated and became the first peptide drug used successfully in modern medicine.

Why does it feel like they are suddenly everywhere? Between advertising and marketing campaigns, celebrity endorsements and social media, peptides are having a moment. Ozempic, a semaglutide used to treat diabetes, hit the market in 2017, following approval by the U.S. Food and Drug Administration (FDA). Health Canada and the European Medicines Agency approved it in 2018. Before semaglutide received formal approval for chronic weight management, some physicians were prescribing it off-label for obesity. In 2021, the FDA and Health Canada approved the higher-dose formulation (Wegovy) specifically for weight management. Since then, awareness has grown. Celebrities like Oprah Winfrey, Rosie O’Donnell and tennis star Serena Williams, and many others, have spoken openly about using semaglutides for weight loss. Semaglutide mimics a natural gut hormone named glucagon-like peptide-1 (GLP-1).

Hailey Bieber and Gwyneth Paltrow have launched skincare products that feature peptides to keep them and us looking young. Jennifer Aniston and Kate Hudson like to drink their peptides by adding collagen peptide supplements to their daily smoothies to keep their skin looking young and glowing.

Peptides have important functions in the body but, like stem cells, they are not a magic bullet for all that ails us. In the case of using collagen peptides for skincare, Joe Schwarcz, at McGill University’s Office for Science and Society, says their efficacy is “debatable.” He writes, “The contention is that once these are absorbed, they will assemble to produce of [sic] collagen in the skin. Evidence for this is sketchy, but in any case, collagen peptides are effective moisturizing agents so they can improve the appearance of the skin.”

Despite mild to sometimes serious side effects from taking GLP-1 medications for weight loss, they are approved medications with well-established safety profiles when prescribed appropriately. Likewise, collagen peptides are generally considered safe, although evidence for many of the cosmetic claims remains limited. You can read about the many uses of peptides in this WebMD article.

Peptides are being used in other ways that may have dangerous side effects. Dietary supplements* and other products marketed for fitness purposes may promise muscle growth and injury recovery, or make anti-aging claims. Not all of these wellness products are regulated or have completed the rigorous clinical testing required for authorized medications.

Health Canada is warning Canadians against injecting peptides bought online. Products labelled “For Research Use Only – Not for Human Consumption” are ones to watch for. “Unauthorized drug products are illegal in Canada and have not been assessed for safety, efficacy, and quality by Health Canada. They have not been demonstrated, through scientific trials, to provide the health benefits that they claim.

Consumers taking unauthorized peptide drugs are exposed to a variety of risks, including, but not limited to, hormonal imbalance, mood swings, blood sugar imbalance, liver or kidney damage, blood clots, and growth of cancerous tumours.”

Health Canada lists examples of unauthorized injectable peptide drugs, additional possible side effects, and what you should do if you have used one and feel unwell. Find more information on Health Canada’s website.

At the top of Health Canada’s list is BPC-157, a synthetic peptide originally isolated from human gastric juice that is popular among athletes. While BPC-157 has shown regenerative properties across animal models, human data are “extremely limited.” This means data regarding safety and associated side effects are also limited. In 2022, the World Anti-Doping Agency first banned BPC-157 under the S0 Non-Approved Substances category, prohibiting its use in both competition and training due to its lack of human clinical approval. It is still on the list, as of January 2026.

As interest in peptides continues to grow, it’s worth remembering that not all peptide products are created equal. Some are evidence-based medicines that have transformed patient care, while others remain unapproved products with little or no evidence of safety or effectiveness.

CBC’s The National went undercover to investigate the use of, and how to access, unauthorized peptides. Watch Idil Mussa’s report, which includes an interview with Timothy Caulfield, University of Alberta.

*People should exercise caution when taking any supplement and discuss the potential benefits and risks with a doctor beforehand.

A scientist at work in CCRM’s Centre for Advanced Therapeutic Cell Technologies. Photo credit: CCRM

“Regulatory approval does not translate to dollars for a company, nor does it lead to patients being treated. Without reimbursement, all of the efforts of translation and development are lost.”

My former colleague, Mark Curtis, wrote these words back when he blogged for Signals and worked as a business development analyst for CCRM.  At the time, he was reflecting on a significant obstacle facing regenerative medicine: getting innovative therapies to patients after they receive regulatory approval. A quick search of topics we have covered on Signals shows that reimbursement has remained a recurring theme for well over a decade. In that time, how much has actually changed?

In May 2026, Bettina Hamelin, President & CEO of Innovative Medicines Canada, wrote “The Cost of Red Tape: Why Canadians Have Slow and Limited Access to New Medicines.” Hamelin says that Canadians now experience the longest wait among G7 countries to obtain new and innovative medicines, a gap she believes will continue to widen as global companies prioritize markets with more predictable pricing and reimbursement environments.

Hamelin goes on to explain that how prices are determined in Canada often undermines “the financial case for bringing new medicines to Canada.” Following Health Canada approval, most medicines undergo health technology assessment and separate price negotiations before provinces decide whether to publicly fund them, creating multiple sequential steps between approval and patient access. While rigorous review is essential, the process is so laborious that “even scientifically promising therapies can become commercially unsustainable to launch here.”

The consequences of these policy and pricing challenges extend well beyond industry investment decisions. They can affect whether patients are able to benefit from emerging treatments in a timely manner.

This delay plays out in ways that hurt Canadian patients. Kelly Grant recently wrote in The Globe and Mail that “there is a 2 ½-year gap, on average, between when new medications are approved by the U.S. Food and Drug Administration [FDA] and when they’re publicly funded in at least one Canadian province.”

Grant was writing about Evan Armit, a 22-year-old with metastatic melanoma, who died in 2025 while the tumour-infiltrating lymphocyte (TIL) therapy that might have been an option for him was still undergoing regulatory review in Canada. The FDA approved Iovance Biotherapeutics’ Lifileucel, to treat metastatic melanoma, in 2024. Health Canada’s authorization came after he passed away.

TIL therapy is a cellular immunotherapy that harnesses a patient’s own immune cells to seek out and destroy cancer. It illustrates both the promise and problem of advanced therapies. It is highly personalized, complex to manufacture and potentially transformative for individuals with advanced cancers, which are characteristics that make existing reimbursement and health system pathways difficult to navigate. Laszlo Radvanyi, President and Scientific Director of the Ontario Institute for Cancer Research (OICR), and the founding Chief Scientific Officer of Lion Biotechnologies before it became Iovance Biotherapeutics, says that TIL therapy “is truly a life-saving therapy, and it will help make metastatic melanoma a survivable disease.” (Read the full interview with Dr. Radvanyi on OICR’s website.)

But breakthrough science alone isn’t enough if proven treatments aren’t available to people like Evan.

Jon Draper, Vice President, Research & Training with the Stem Cell Network, argues that our system is the bottleneck and “delivery systems are the defining challenge for regenerative medicine today.”

Dr. Draper says that we are looking at a global coordination problem. Other countries are also trying to adapt to the regulatory and reimbursement issues posed by very expensive cell and gene therapies that are more complicated to produce and distribute, particularly if you reside in less densely populated regions and low- and middle-income countries.

As I wrote in 2022, one reason the industry continues to face coverage hurdles is that payer landscapes vary considerably across countries. Health care systems differ in how funding decisions are made, the evidence required to demonstrate value, and the organizations responsible for reimbursement.

For example, a country with a predominantly publicly funded health care system, such as Canada, faces different reimbursement considerations than the United States, where payment decisions are made across a fragmented mix of public and private insurers. As well, traditional reimbursement pathways were designed around chronic therapies with ongoing costs, making it difficult to evaluate and fund one-time treatments with high upfront costs, like cell and gene therapies, whose benefits may extend over decades or could be “one and done.”

There will be no one-size-fits-all solution.

Four years on, progress has been slower than many anticipated. While reimbursement frameworks have evolved and payers have gained experience with advanced therapies, many of the fundamental challenges identified in 2022 remain. You can read “Unpacking talent and reimbursement issues at Advanced Therapies Week 2022” on Signal’s website. It mentions payment models that were emerging at that time, such as treatment milestones and subscription-based plans. Canada has made relatively limited use of managed access agreements and conditional or time-limited reimbursement arrangements compared with some other jurisdictions.

In Canada and around the world, we have an expanding number of transformative cell and gene therapies; now we need to build health systems that can deliver them to patients.

The Stem Cell Network is spearheading a “Made-in-Canada designation and prioritized pathway for advanced therapeutics” that would integrate all the steps into a predictable process, encompassing the regulatory review, health technology assessment and reimbursement model. Efforts like this suggest that workable solutions are beginning to emerge, with collaboration from regulators, payers, industry, clinicians, patients and governments. Success will ultimately be measured by whether patients can access effective advanced therapies in a timely, equitable and sustainable way.

Actor Sam Neill, well-known for his role in the Jurassic Park franchise, announced in April 2026 that he was cancer-free after undergoing CAR T-cell therapy, a form of cancer immunotherapy, to treat his stage-three blood cancer. He had angioimmunoblastic T-cell lymphoma, which he said he’d been living with for five years and was treating with chemotherapy.

Eventually, the chemotherapy stopped working and the Australian actor was enrolled in a clinical trial that was the focus of his type of lymphoma. Now Neill is advocating for Australia’s state and federal governments to expand the availability of CAR T-cell therapy for more cancer patients. He is working with the Snowdome Foundation, a not-for-profit.

Neill is not the only actor whose health journey has recently drawn attention to a serious disease. In May 2026, actor Russell Andrews (Grey’s Anatomy) revealed he has amyotrophic lateral sclerosis (ALS). Andrews’ co-star, Eric Dane, passed away in February 2026 from ALS. He was 53. There is currently no cure for ALS, although gene therapy is a promising area of research for the 20 per cent of cases arising from genetic factors. As well, researchers from the Perelman School of Medicine at the University of Pennsylvania and Children’s Hospital of Philadelphia have published research in Nature Communications demonstrating that a gene therapy significantly slowed motor function loss in mice with ALS.

Celebrities being diagnosed with chronic and even fatal illnesses is nothing new, but they can have an unexpected benefit. Recall the attention around actor Bruce Willis and aphasia, and the announcement 10 months later by his family that he had frontotemporal dementia, a degenerative brain disease. Awareness of the rare neurodegenerative condition increased substantially. As one research paper noted, “the Willis family’s disclosure was a courageous act that helped bring much-needed attention to this disease.”

Celebrities also contribute directly by fundraising, donating and establishing charitable foundations. Their public profile and large followings can attract donors, corporate partners and media attention, benefiting health charities, hospitals, universities, research institutions and patient advocacy organizations.

Perhaps the most influential celebrity-led medical charity is the Michael J. Fox Foundation. Since being diagnosed with Parkinson’s disease at age 29, Fox has transformed his personal experience into one of the world’s largest private funders of Parkinson’s research. The foundation has committed more than US$2.5 billion toward developing better treatments and ultimately a cure, including research into cell and gene therapies.

Another example is Pearl Jam’s Eddie Vedder, who, with his wife Jill Vedder and others, co-founded the EB Research Partnership (EBRP) in 2010 after learning that a friend’s son had epidermolysis bullosa (EB), a devastating genetic skin disease that affects children from birth. (Signals’ bloggers have written about EB many times.)

So far, the EBRP has raised US$80 million. EBRP funds research into gene-editing approaches, including CRISPR-Cas9, that aim to correct the genetic mutations responsible for certain forms of EB. Early studies are advancing rapidly and offer considerable promise, although further clinical research is needed before these therapies become widely available.

While celebrity health news often begins as entertainment headlines, its impact can be far-reaching. Public disclosures, such as Angelina Jolie’s revelation that she had a preventative double mastectomy, can encourage earlier diagnosis and screening, reduce stigma, stimulate research funding, influence health policy and accelerate support for patients and families. Combined with the work of scientists, clinicians and advocacy organizations, celebrity voices can help bring promising treatments and, ultimately, cures closer to reality.

Learn more about EB and the work of EBRP in the video below. You will hear from CEO Michael Hund, Eddie Vedder, Drs. Anthony Oro and Jean Y. Tang, and celebrities Olivia Rodrigo and Tom Holland. As an aside, Tom Holland has his own charity – The Brother’s Trust (TBT) – that raises funds and awareness for charities that “struggle to be heard” among other better-known not-for-profit organizations. With his brothers, TBT runs special events to “create memorable and distracting experiences for children suffering with overwhelming physical and mental hardships.” EBRP seems like an ideal fit.