Early phase stem cell trials show promise in stroke and blindness

Blurring at the center of vision in macular degeneration

The very first time a potential therapy gets tested in people it’s part of what’s called a phase 1 trial, which is very small and is mostly just testing to make sure the drug, cells or device are even safe. Until the start of a trial the potential therapy has generally only been tested in lab animals, which can be quite different from humans.

With that caveat in mind, there’s some hopeful news coming out of a phase 1 trials testing stem cell-based approaches to treating stroke and blindness.

The stroke trial in Scotland is testing a type of neural stem cell to see if it can help people who have had strokes recover function. People in the trial are reporting better grip strength and more coordination. That said, because it’s such a small trial there’s no way of knowing whether some of the improvements would have happened anyway—people do improve over time after a stroke.

A story in the Telegraph quotes Clare Walton from the Stroke Association talking about the results:

“We are very excited about this trial. However, we are currently at the beginning of a very long road and significant further development is needed before stem cell therapy can be regarded as a possible treatment.”

This trial is testing a type of neural stem cell injected directly into the place where the stroke happened. Other groups are working toward trials with other types of stem cells or ways of delivering those cells to the brain. Our stroke fact sheet has more about stem cell research for stroke, including a list of all awards we fund.

Two other trials, led by Advanced Cell Technology, are showing very early positive signs. Both trials are testing cells derived from embryonic stem cells to see if they can replace the function of cells lost in the back of the eye in people with macular degeneration or Stargardt’s macular dystrophy. Macular degeneration is the leading cause of blindness. In very preliminary results, one person went from being effectively blind to having 20/40 vision.

A story in New Scientist quotes Gary Rabin, chief executive officer of Advanced Cell Technology

“There’s a guy walking around who was blind, but now can see. With that sort of vision, you can have a driver’s licence.”

As with the stroke results, it’s too early to know if the cells are responsible for the change, work long-term or are safe. That’s the point of starting slow with phase 1 trials before working up to trials that include more people and will give a better indication of whether the treatment works.

There’s more information about stem cell research for blindness on our fact sheet, including information about our funding for forms of blindness. The groups are testing different type of cells or ways of implanting those cells in the eye to see which approach is most effective for treating various forms blindness.


3 thoughts on “Early phase stem cell trials show promise in stroke and blindness

  1. Where can one find information on the CIRM site pertaining to the amount of funding the CIRM has awarded to Advanced Cell Technology? I only see about 15 applications?

  2. Here's a guy trying to do the best he can for patients…instead of being a lap dog he's putting him self out there…

    Best part of the $SRPT conference today.

    Some days, I come in to work in the media and the twitter feeds and the attention that has been brought to this company and to this accelerated approval decision, sometimes creates distractions and I have to answer a lot of phone calls and I have to address questions that are distracting from just the task at hand of drug development.

    However, I feel the commitment to do everything in my power to get a drug that I believe is efficacious and safe to the patients that need it. And it's very hard to be committed to that and to express how we are trying to do that and avoid the perception of doing something that some believe us unprecedented or aggressive and we don't believe that, we believe in our drug, we understand the regulations and what the authority of the FDA is.

    We believe we are meeting those demands and we hope that FDA does the right thing as it relates to accelerated approval. And that's my job as CEO, I don't want to be the one telling the parents, you know trust me, I know the biotech industry and I know the FDA, so I'm going to decide, we're not even going to try to pursue it and just trust me on this. Guess where their fire is going to be focused? It's going to be on me for being an idiot for not trying to get a drug to them as quickly as possible. So that's why I'm vocal about it and that's why I feel strongly because we believe in the product.


  3. Ethics and the Limits of the Randomized Controlled Trial: Time to Enhance Access to Novel Therapies in Lethal Diseases?
    Les Halpin, founder of Access to Medicine, and himself a motor neuron disease sufferer has argued that for those with life threatening and rare illnesses, current drug approval procedures do not work. He argues in our forthcoming paper that “for such individuals, the “risk-return ratio” is different compared to patients with more benign conditions and drug regulations should be adapted to allow such people the opportunity to try out new combinations of drugs”. He has argued for greater use of new media to track patient progress, and cites the use of the website ‘Patients Like Me’ by MND patients to track their progress on lithium treatment.
    In fact, unrestricted access to medicines may be technically no different to the current relaxed attitude to complementary therapies already used by a high percentage of patients. Nevertheless, there are of course significant challenges to creating a new research method that would be scientifically valid and avoid returning to unregulated , risky innovations that ultimately harm patients. Three physicians in this area suggest in our forthcoming paper in the Journal of Medical Ethics that one answer to the scientific challenges may be to create “data linkages which harness the power of the patient’s own observations to population based disease registries and hospital outcome data”. Given that for this group of patients, receiving a placebo and receiving the new drug may be a matter of life or death, there is a strong imperative to face these challenges head on.

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