I was very excited to learn that another clinical trial using stem cells for the potential treatment of a retinal disease — in this case, dry age-related macular degeneration (AMD) — is now underway. StemCells, Inc. (got to love the company’s name) announced last week that its first patient was treated at the Retina Foundation of the Southwest in Dallas.
While the 16-person Phase I/II study will evaluate the transplanted neural cells’ ability to preserve vision, the primary goal for now is to demonstrate safety. If the Phase I/II trial is successful, later stage studies will take a closer look at efficacy.
When I hear about groundbreaking studies like these, I always wonder who the courageous patient was who agreed to be the first to receive the treatment. I don’t know if I’d personally have the chutzpah to be the first. The people who volunteer to be in these early-stage human studies for cutting-edge treatments are the true pioneers and heroes.
In addition to receiving a potential treatment with very little (or no) track record in people, they have to make numerous clinical visits with extensive retinal exams and vision tests. Not only are they taking a risk, but clinical trial participants make a big commitment of time and energy.
I’d be remiss if I didn’t acknowledge the indispensible role of the clinicians and researchers who run the trials and perform the related procedures. They, too, are true pioneers. In the case of StemCells, Inc., Dr. David Birch is the principal investigator, with Dr. Rand Spencer performing the surgeries.
A researcher funded by the Foundation Fighting Blindness, Dr. Birch is no stranger to clinical trials. He currently serves as the principal investigator on human studies of valproic acid for the treatment of autosomal dominant retinitis pigmentosa (RP) and docosahexaenoic acid (DHA) for X-linked RP.
In recent years, he also led clinical trials of Neurotech’s encapsulated cell technology and Second Sight’s “bionic” retina. He clearly has a penchant and talent for living on the cutting edge of retinal research.
While the first results from the StemCells, Inc. study probably won’t be reported for a while, I look forward to reporting on them when they are announced. Stay tuned.
Pictured above: Dr. Rand Spencer, left, and Dr. David Birch, surgeon and lead investigator, respectively, in the StemCells, Inc., clinical trial.