The FDA has given Advanced Cell Technology (ACT) the go ahead to begin their clinical trial using retinal progenitor cells derived from hESC by clearing the company’s IND application.
The trial will be a combined Phase I/II to treat Stargardt’s Macular Dystrophy and has huge implications for the treatment of blindness more generally.
The FDA approval is a big boost for the small stem cell-based biotech company and in turn gives hope to millions of patients and their families facing blindness.
The study will involve a dozen patients with the primary goal of measuring the safety of ACT’s hESC-derived RPE drug. However, I hope that they also find a measure of efficacy during the study. I think there is reason for cautious optimism. Cell therapy-based approaches to treating blindness together represent a fundamentally new, exciting area.
What this means is that today we have 2 biotechs-ACT and Geron-moving forward with hESC-based therapies, in essence neck and neck, to help patients with previously essentially untreatable diseases that affect millions of people.
You can read ACT’s press release here.
A great day!