Examining various methods and treatments currently in clinical and pre-clinical stages .
Pharmacologic, gene therapy, and stem cell therapy approaches to Stargardt’s disease are currently in clinical and pre-clinical development, said Hendrik P.N. Scholl, MD, MA. The FDA recommended a prospective study on the natural history of Stargardt’s disease, he continued. To this end, The Natural History of the Progression of Atrophy Secondary to Stargardt’s Disease (ProgStar) Study was developed.
Several compounds have shown promise as potential medical therapy of Stargardt’s disease, Dr. Scholl said.
“Toxic A2E accumulates in every retina, but at a much higher rate in Stargardt’s disease retinas.
In addition, deuterated vitamin A are may help slow the accumulation of A2E by blocking its formation downstream the visual cycle, Dr. Scholl explained. The effects of deuterated vitamin A are currently in a phase Ia trial.
Gene therapy is also being developed and there is an active clinical trial in phase Ib. The program is being sponsored by Sanofi-Fovea. Pre-clinical data had been convincing to show that delivering the healthy gene to the photoreceptor had a significantly positive impact on A2E accumulation in a mouse model of the disease, Dr. Scholl said.
Stem cell therapy
Stem cell therapy is yet another option for the treatment of Stargardt’s disease that is also currently being studied, Dr. Scholl added.
From the Advanced Cell Technology trial there is a preliminary report on stem cell therapy in age-related macular degeneration and Stargardt’s disease.