Eye on the Cure

If you’re going on a cross-country road trip, the highlight of your journey is not the safety check. Putting air in the tires, topping off the brake and power-steering fluids, making sure the front and back lights are working properly and adjusting the mirrors are not cause for raucous celebration.

But without taking these safety precautions, your trip could turn out very badly; you not only might not get where you’re going, you might get injured or worse. Taking a “safety first” approach is important in many of life’s endeavors, including the development of sight-saving treatments for retinal diseases.

That’s why I was pleased to recently learn that Oxford BioMedica’s StarGen™ gene therapy for Stargardt disease and RetinoStat® gene therapy for wet age-related macular degeneration received positive interim safety reviews from a Data and Safety Monitoring Board, an independent panel of science and medical experts, which monitors patient safety and treatment efficacy data for a clinical trial. The Foundation requires a DSMB for all its clinical trials, to ensure that no study participant is unnecessarily put in harm’s way.

After 12 months, eight patients in the Phase I/IIa StarGen clinical trial receiving the lowest dose (dose level 1) treatment have experienced no adverse events, and the DSMB supports treating four additional patients with a higher dose (dose level 2). If the safety profile of StarGen continues to be good, four additional patients will receive the highest dose (dose level 3).

In the Phase I RetinoStat clinical trial, a total of nine patients have been treated thus far with no adverse events. Three patients have been treated for each of three different dosing levels, with the first patients having received the dose level 1 treatment 18 months ago. In addition, the treatments are expressing the therapeutic proteins they were designed to express. The DSMB supports the treatment of nine additional patients in the RetinoStat trial.

The DSMB has not issued a safety review yet for the Phase I/II clinical trial of UshStat®, Oxford BioMedica’s gene therapy for Usher syndrome type 1B. That trial began in March 2012, so I wouldn’t expect a report until next year.

I know for those of you losing vision to retinal diseases, an announcement of safety for a treatment may not be the big news that you hope for, and that is perfectly understandable. You want your vision saved or restored. But please keep in mind that safety is the primary goal of these early-stage studies, and without safety, you have nothing.

Gene therapies, with as much promise as they hold, are still cutting-edge treatment approaches – which is why you can’t currently walk into a doctor’s office and ask for one. But our goal at the Foundation Fighting Blindness is to develop it to the point where your doctor says, “Gene therapy for your vision — no problem.” Safety is an essential step toward getting there.