An experimental gene therapy could benefit young boys with a rare genetic disease, despite considerable concerns that treatment may cause cancer, a panel of Food and Drug Administration advisers unanimously agreed in a high-profile meeting Thursday.
The therapy, an infusion of genetically modified stem cells called eli-cel, is designed to treat cerebral adrenoleukodystrophy, or CALD, which causes severe disability and death, typically in the second decade of life. It’s the product of years of research by Massachusetts drugmaker Bluebird bio, which asked the FDA for approval last year.
