ThinkFest Recap: Dr. Jean Bennett on Curing Blindness
Dr. Jean Bennett at ThinkFest.
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Philadelphia is leading the way in developing gene therapies that could pull off medical miracles like reversing blindness, a Penn medical researcher said Friday at Thinkfest.
“This is really, really an exciting time,” said Dr. Jean Bennett of University of Pennsylvania’s Perelman School of Medicine. She’d helped lead a team that’s in the midst of FDA trials to bring a blindness therapy to market.
Huffington Post summed up the method earlier this year: “The basic strategy for these clinical trials is to inject a harmless virus that inserts good copies of genes that are defective in the individual. These genes are inserted into cells in the retina, the tissue that lines the inner eyeball and senses light.”
Science Magazine reported this year on the first round of trials that began in 2007: “As soon as a few days after the treatment, many patients’ retinas were more sensitive to light, and they were better at navigating a maze, particularly in low light.”
One early recipient of the therapy, Bennett said, was a boy named Corey would was blind. Within a year, he attained some vision — and today, at age 15, has enough sight to go hunting. “This totally transformed his life,” Bennett said.
One lesson learned along the way: The brain is resilient enough that old “circuits” left unused by blindness will become active once the eyes start working. Another lesson? Academic researchers can sometimes achieve with millions of dollars the kinds of research results that come to pharmaceutical companies after spending billions of dollars.
“We’ve proven it is possible for an academic unit to do things in an economically responsible way,” Bennett said.
While researchers proceed through the final phase of trials to win FDA approval, she said, they’re looking at ways to speed up future, similar efforts. Getting this far with this therapy has taken 20 years, she said. “How can we accelerate the progress?”
