September 19, 2020

The Niche

Knoepfler lab stem cell blog

FDA approves ACT’s second stem cell trial for blindness

Advanced Cell Technology (ACT) announced that the FDA has approved their second human ES cell-based clinical trial, a combined Phase I/II, for the treatment of blindness.

As a result, soon ACT will have two active, human ES cell-based clinical trials, both using the same human ES cell-based drug.

The aim of the 2nd trial is to treat Dry Age-Related Macular Degeneration, a condition that affects tens of millions of people.

The primary objective of both trials is to determine the safety of the stem cell based drugs. Because in these early trials typically patients with severe disease are involved, information on efficacy may not be obtained.

While we are still years away from treatments potentially getting to patients, this news is still a step forward and it provides more hope for patients suffering from blindness and their families. In the long run, cell therapy (whether of the kind ACT is testing or others) offers the best possibility for treatments or cures of some types of blindness.