Gene therapy for AMD safe in phase 1 trial

Injecting a gene directly into the eye to inhibit blood vessel growth was safe and well-tolerated in a phase 1 clinical trial, investigators announced. The therapeutic approach holds promise for halting the progression of wet age-related macular degeneration, the researchers said.

Michael Klein, MD, and colleagues at Oregon Health & Science University used the gene therapy technique in six patients as part of a multicenter study that included 28 people with advanced stages of AMD. Their results were announced in a press release from the university.

The researchers placed the pigment epithelium-derived factor (PEDF) gene in an adenovirus and then injected it into the participants’ eyes. Participants received either a single low dose or a single high dose of the adenovirus with the PEDF gene, and they were followed for 1 year.

Although phase 1 studies are not designed to test only for safety, not for efficacy, the press release noted that patients who received a high dose of the treatment had “no growth in their macular lesions at 6 and 12 months post-therapy, while those who received a low dose saw some increase in their lesion size.”

A phase 2 efficacy trial is being planned, the release said.

The study is published in the February issue of Human Gene Therapy.