Retinitis pigmentosa therapy treatments promising but will take time

CHICAGO — Genetic and other therapies for retinitis pigmentosa have shown promise in animal studies, but it may be several years before such treatments are available to clinicians, said John R. Heckenlively, MD, speaking here.

Summarizing current research, Dr. Heckenlively said that there are 94 genes known to cause retinitis pigmentosa. He spoke on the subject here during Retina Subspecialty Day at the American Academy of Ophthalmology meeting.

“We can expect we’ll need a number of different treatments based on the gene involved and the mutation type,” Dr. Heckenlively said.

Numerous treatments are under investigation, the most promising of which is gene therapy, he said. He called stem cell therapy the retinal specialist’s “trump card.”

“Most current studies have not been successful, and the ethical and legal concerns are overstated,” he said. “Currently, stem cell therapy is looking more like a game of Texas hold ‘em than an organized scientific field. The problem is, we don’t have the knowledge to control or direct cells that are used in therapy.”

Pharmacologic interventions for retinitis pigmentosa are also likely to show benefit, Dr. Heckenlively said. In his lab, a clear therapeutic effect has been shown with pharmacologic agents, but he reiterated that gene therapy has been more promising to date.

He advised physicians show restraint when discussing potential treatments with patients, as the process of moving treatment protocols from the lab to human study is “clearly time consuming and involved.”