On Wednesday, the Food and Drug Administration (FDA) approved the first cell-based gene therapy in the United States.
The FDA called the new therapy historic, and FDA officials called U.S. Georgia Senator Johnny Isakson on Wednesday to relay the news of the agency’s approval of the groundbreaking therapy.
Georgia U.S. Senator Johnny Isakson worked on the legislation that allowed for the innovation of new treatments under the Advancing Hope Act.
Isakson’s commitment to helping find effective treatments for rare pediatric diseases began in 2004, following a visit from Alexa Rohrbach, a young Georgian suffering from a rare cancer who came to see Isakson to ask for his help in finding a cure. Alexa passed away in 2008, but Isakson said that her memory is “a reminder of why getting a result on bills like this one is as critical as anything we could ever do in the United States Senate.” He shared Alexa’s story on the Senate floor in 2016 to urge his colleagues to support an extension of the priority review program.
The legislation extends the Rare Pediatric Disease Priority Review Voucher Program and was included in the 21st Century Cures Act, signed into law in December 2016.
The FDA approved and innovative therapy called Kymriah. Scientists refer to this new therapy as a “living drug” because the drug genetically modified immune cells and reprograms patient’s own cells to attack their cancer.
“This type of therapy is exactly what we had in mind when I began working for the Advancing Hope Act, which was ultimately approved and extended in last year’s 21st Century Cures legislation,” said Isakson. “When I heard this wonderful news directly from the FDA yesterday, I thanked them and told them to get it on the market, because it’s time to start saving kids’ lives.”