Researchers who helped restore sight to blind patients win Sanford Health $1 million award
SIOUX FALLS, S.D. — There was a crash in gene therapy in the 1990s when rapid development led to less support and funding for gene therapy projects.
But the field is booming now.
It was evident on Tuesday night, Dec. 4, in the awarding of the Sanford Health Lorraine Cross Award worth $1 million.
The winners were the team of Dr. Jean Bennett and Dr. Katherine High of the University of Pennsylvania. They were one of three gene therapy pioneers that were finalists for the inaugural award funded by donations from philanthropic families, including Sanford's main benefactor, Denny Sanford
"Gene therapy was a bad word," said Dr. Jim Wilson, one of the finalists in talking about the 1990s. "We had to work harder, and that's what we did."
He went to work on developing a safe , solid foundation for the future of gene therapy by uncovering a new way to deliver gene replacement therapies. His efforts led to a rebirth in gene therapy, leading to more research and potentially life-saving cures that most hope aren't too far away.
The Sanford award was created to award game-changers in medicine. Candidates were identified using a computer algorithm that sifted through medical research globally in search of major discoveries and advancements. An interdisciplinary scientific advisory board identified the finalists just over a month ago.
Then, after the Sanford International Board listened to presentations on the finalist's work earlier in the day and had discussions with the members, they held a dramatic ceremony Tuesday night at a building on the sprawling Sanford research facility in north Sioux Falls where 300 researchers from around the world work..
The board made it known a winner was named by lighting a large blue flame on top of the building where the announcement was made, similar to how the Vatican announces when a new pope is chosen.
Sanford Health CEO Kelby Krabbenhoft told a crowd that included the operation's benefactor, Denny Sanford, that they created the award and do research at its facilities to not have people live forever, but for people to "live life without suffering."
The award, he said, was to inspire people.
Bennett and High won the award for their work with the RPE65 mutation that has reversed an inherited form of blindness.
Bennett and High pioneered the gene therapy, took it to clinical trials and then received FDA-approval for the treatment, the first FDA approval of a gene therapy for a genetic disease.
It started with Bennett testing gene therapy – replacing a missing or mutated gene with a healthy one – to repair vision in dogs with Canine LCA, a genetic condition that eventually causes blindness.
“Ninety-nine percent of the time we saw this remarkable reversal of the blindness,” in dogs, said Bennett. “So we were very excited to test this in humans.," she told Sanford officials. The first human clinical trails were in 2007 with patients seeing dramatic improvement in just days. She helped found Spark Therapeutics, a company founded to develop and distribute the gene therapy drugs.
Here's more on the other two finalists.
- Wilson, director of the gene therapy program at the University of Pennsylvania, has worked mostly on processes, not patients, during his 35 years of innovation in gene therapy. "This has been a journey of mine over three decades, but it's only the beginning of a revolution," he told Sanford officials. "Gene therapy is really the ultimate treatment for patients that have diseases due to single gene defects." The theory behind the treatment is to replace a missing or mutated gene with a cloned one that works and heals the patient from the root of the problem, he said. Rare diseases affect small handfuls of the population, but taken together they affect 350 million people. Most stem from a missing or mutated gene, which Wilson has spend his career trying to replace.
- Dr. Brian Kasper, of Bannockburn, Ill., was the scientific founder of a company called AveXis. He has been working on spinal muscular atrophy, the No. 1 genetic cause of death for infants, leaving them unable to walk, talk, eat or even breathe. Through his research work that he started in 2004, he is giving families new hope with a goal of eliminating the diseases forever. Through his work, children are now living a full life at age 5 when before 93 percent didn't ever reach the age of 2. His drug to help children is hoping FDA approval is just around the corner.
The award is named after the Lorraine Cross, a symbol synonymous for those who take action for their passions. In recent history, Sanford Health has used it to symbolize innovation in health care.
The $1 million prize is one of the top five awards money-wise in the world in medical care advancements.