Throughout our efforts to promote the advancement of medical technology and research, the Finker-Frenkel Family Foundation has partnered with a variety of innovative organizations, including the Bascom Palmer Eye Institute. We’ve written about Bascom Palmer in the past, and their track record is impressive—bringing new treatments to the field of ocular medicine and revolutionizing older treatments to the benefit of patients. The organization is routinely noted as a worldwide leader in its field.
It’s no surprise that Bascom Palmer has turned its attention to potential applications of gene therapy technology. Many diseases, common and rare alike, are the result of genetic factors often outside of an individual’s control. As a result, many of these diseases are difficult to treat and manage. In the case of rare disease, some treatments may not even exist due to small patient populations and insufficient research efforts. It’s a tragedy that affects thousands of Americans each year, making gene therapy potentially important for managing or eliminating these conditions.
Earlier in April, Bascom Palmer trialed new gene therapy techniques aimed at restoring sight to children born with a rare genetic disorder. The condition, known as Lebert Congenital Amaurosis, affects just one in 40,000 children and eventually renders them blind. Such was the case with five-year-old Ro’Nylah Cummings, who loves dancing but suffered from degenerating vision for her entire life until it was no longer possible.
However, Bascom Palmer had the answer in the form of an FDA-approved procedure known as Luxturna, conducted by Spark Therapeutics. The procedure involves attacking the defective genes that impede the eye’s ability to produce a certain protein. Mere days after the procedure, patients have experienced the restoration of sight.
For Ro’Nylah, her recovery is nothing short of miraculous. Her mother has commented on how quickly she has felt empowered to complete tasks that, up until recently, felt impossible. However, it comes with a substantial price—$425,000 per eye. Though prohibitive, future advances in gene therapy technology could provide a cut to the cost and make treatments like these more accessible to the general public.
More research still needs to be completed to assess the long-term viability of gene therapy—children like Ro’Nylah can potentially help pave the way for something bigger in the future.
At the Finker-Frenkel Family Foundation, we support innovation in medicine and are happy to partner with Bascom Palmer and other organizations looking to rethink the way we approach disease.