Sight is something many of us take for granted. For Dr. Jean Bennett and Dr. Katherine High, it’s a precious gift that they’re restoring in dozens of patients. For their work in creating the first FDA-approved drug for genetic treatments for blindness, they won the inaugural $1 million Sanford Lorraine Cross Award last December, sponsored by Sanford Health. But to understand the impact of their work, you must rewind to its beginnings.
Back in the 1990s, gene therapy was just a budding concept. After collaborating at several gene therapy seminars, Dr. Bennett and Dr. High developed a friendship that would last decades.
At the University of Pennsylvania, Dr. Bennett was testing gene therapy — replacing a missing or mutated gene with a healthy one — to repair vision in dogs with Leber’s congenital amaurosis (LCA), a genetic condition that causes blindness.
These studies yielded amazing results, as Dr. Bennett and her collaborators were able to restore the dogs’ sight. But to pursue human clinical trials, she needed financial support.
“Then, one day — I can remember the exact day,” Dr. Bennett says. “One Dr. Katherine High knocked on my office door, sat down and said, ‘Jean, how would you like to run a clinical trial?’”
As Dr. Bennett had been researching LCA, Dr. High had been working in hematology. And though financial support for gene therapy had waned in recent years, Dr. High was convinced it could yield amazing results. She secured funding through the Children’s Hospital of Philadelphia (CHOP) and took the opportunity directly to Dr. Bennett.
The doctors’ first human clinical trial with LCA patients began in 2007 with astonishing results. Patients who were once completely or partially blind were now able to see their families, go to work, and live normal lives as if their symptoms were cured.
“Patients are really the reason we do this work,” says Dr. High. “Their life is limited in a way, but they have talents that they can use to help solve problems in the world. But they need a chance.”
To get this treatment across the finish line, Dr. High and Dr. Bennett formed Spark Therapeutics, a company founded by CHOP to develop and distribute gene therapies. Their drug, Luxturna™, became the first FDA-approved gene therapy drug, opening the door for future breakthroughs.
The Sanford Lorraine Cross Award was founded by Sanford Health to honor medical mavericks who’ve overcome obstacles to push an innovation across the finish line. The prize will be awarded every other year, with nominees filtered through public nominations and a computer algorithm. An interdisciplinary scientific advisory board identified the top four candidates.
For Dr. Bennett and Dr. High, the award was the realization of decades of work. And in terms of the future, the doctors hope this discovery sparks more breakthroughs for scientists around the world through hard work and determination.
“The bottom line is — we would not take no for an answer,” said Dr. Bennett. “We always found a way to navigate around these hurdles. It was just an obvious collaboration.”