Advanced Cell Technology (ACT) continues to enroll patients in its clinical trial evaluating its human embryonic stem cell-derived retinal pigment epithelial (RPE) cells in patients with various forms of blindness, announcing this month that the fourth patient, and the first patient to receive a higher dose of the stem cells, had been treated in its ongoing clinical trial in the United States for Stargardt’s Macular Dystrophy. In addition, the company announced that received approval to move to increased dosing for its dry age-related macular degeneration (AMD) clinical study. Currently the second patient in the ongoing Stargardt’s disease trial taking place in the United Kingdom has been dosed.
Stargardt’s disease is a genetic disease that causes progressive vision loss, usually starting in children between 10 to 20 years of age, and eventually results in blindness. AMD is the leading cause of blindness in people over 60 years of age in the United States, and dry AMD accounts for 90 percent of all cases. Both Stargardt’s disease and dry AMD are progressive diseases that result in vision loss and blindness due to the thinning of the layer of RPE cells in the patient’s macula, the portion of the retina responsible for central vision.
ACT previously reported that the first patients in each trial had measurable improvements in their vision that persisted for more than four months, with the Stargardt’s patient reporting the ability to start reading letters on the ETDRS visual acuity chart, as early as two weeks post-transplantation. Each clinical trial will enroll 12 patients, with cohorts of three patients each in an ascending-dosage format.
Below is an article from The Wall Street Journal about promising treatments for blindness that includes commentary from Gary Rabin, chairman and CEO of ACT.