ACT begins trials for macular dystrophy treatment
Advanced Cell Technology announced the dosing of the first patients in each of its two phase I/II clinical trials for Stargardt's macular dystrophy and dry age-related macular degeneration (dry AMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). The patients were treated by Steven Schwartz, M.D., at the David Geffen School of Medicine at UCLA. Robert Lanza, M.D., chief scientific officer of ACT, attended the procedures. Both patients successfully underwent the outpatient transplantation surgeries and are recovering uneventfully.
Both the Stargardt's trial and the dry AMD trial will enroll 12 patients each, with cohorts of three patients each in an ascending dosage format. Both trials are prospective, open-label studies designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation into patients with Stargardt's and dry AMD at 12 months, the studies' primary endpoint.
Dry AMD, the most common form of macular degeneration, Stargardt's and other forms of atrophy-related macular degeneration are usually untreatable. Disease progression of both Stargardt's and dry AMD includes thinning of the layer of RPE cells in the patient's macula, the central portion of the retina and the anatomic location of central vision. With RPE cell death comes the loss of macular photoreceptors and loss of central vision. Currently both conditions are untreatable.
ACT's Stargardt's and dry AMD therapies treat these conditions by transplanting RPE cells in the patient's eyes before the RPE population is lost.