|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.