Have you heard the exciting news from Spark Therapeutics? Spark is just finishing up its phase 3 clinical trials for their gene therapy treatment (called SPK-RPE65) intended to treat patients with LCA caused by the RPE65 gene mutation. And the results are good!
The highlights of the trial:
There were “no serious adverse events” related to the trial or the procedure. Obviously the FDA is very concerned about safety, so this is excellent news and gets us so much closer to having an approved treatment that is actually available to patients and their families.
It works! As one of the researchers put it, “We saw substantial restoration of vision in patients who were progressing toward complete blindness.”
This comes after over a decade of working on gene therapy approaches to treating blindness, and the end (or should I say the beginning) is really just around the corner. Spark is also announcing that they plan to file a “Biologics License Application” with the FDA in 2016. Yes, you read that right. 2016. As in next year!
Some things to keep in mind:
SPK-RPE65 is designed to treat patients who are losing vision due to mutations in the RPE65 gene (if you haven’t done genetic testing yet, now may be the time to do it).
Even though this therapy only treats RPE65, there are possibilities that the work being done here will have an impact on other blinding conditions in the future.
This is potentially a one-time treatment. If approved, you may only need to have the procedure done once to reap the benefits.
Even though you’ve heard news about gene therapy for years, this really is different. This represents “the first successful randomized, controlled Phase 3 trial ever completed in gene therapy for a genetic disease.” In other words, gene therapy is getting bigger and better… and treatment really could be available soon!
Microphthalmia is a condition where the eye does not fully develop. There’s no cure, but early intervention can improve function for children with poor vision.
Congenital glaucoma is a disease where the pressure inside your baby’s eye is too high. This can damage the optic nerve and can even make your child go blind.
