Neuroprotective treatment promising for macular telangiectasia
CNTF found effective in slowing vision loss from photoreceptor cell death in animal models
Take-home message: The class of molecules called “neurotrophic factors” has been demonstrated to reduce the rate of photoreceptor cell loss during retinal regeneration.
La Jolla, CA—Neurotrophic molecules may prove useful in treating macular telangiectasia type 2, according to research.
“While we don’t have statistically significant efficacy results yet, data from the phase I trial show that the safety with encapsulated cell-based delivery of ciliary neurotrophic factor (CNTF) is good, and anecdotally, outcomes appear better in treated eyes than untreated eyes,” said Martin Friedlander, MD, PhD.
The possibility arises from an 11-year-old collaborative research effort—the MacTel Project—encompassing natural history, genetics, imaging, and laboratory studies of the disease, said Dr. Friedlander, chief of retinal services, Scripps Clinic, and professor, Department of Cell and Molecular Biology, The Scripps Research Institute, La Jolla, CA.
An administrative interim analysis of the phase II clinical trial will take place in May. If there are no safety concerns, final analysis of the phase II trial will take place 1 year later. If the results of this trial and a subsequent phase III trial demonstrate efficacy, CNTF could become the first treatment for macular telangiectasia type 2, he noted.
Research into the proposed treatment stems from recent discoveries that the disease results from deterioration of photoreceptor cells in addition to the vascular changes, and that the cells may be damaged rather than destroyed and, thus, might be revived.
“It’s really a game-changer in terms of treating this disease,” Dr. Friedlander said. “You’re talking about restoration rather than resurrection.”