How Gene Therapy is Offering Hope — Once Again

For decades, the idea that you could attack genetic illnesses right at the root — that you could modify people’s genes to treat or cure disease — sounded like science fiction. But in recent years, several new gene therapy treatments have been FDA approved, and many others are showing promise in clinical trials. On this episode, we take a look at what’s new with gene therapy, what’s on the horizon, and the challenges standing in the way. We hear stories about making these treatments more affordable by changing the manufacturing process, what happened when one family tried to enroll their son in a clinical trial under a looming deadline, and one man’s DIY approach to curing his own condition. We also chat with a pioneer in the field of gene therapy about sticking with her research during a time when funding dried up and interest in the field had faded.

ALSO HEARD:

• In 1999, hematologist Kathy High published a study on a promising new gene therapy treatment for hemophilia — one that she hoped could transform patients’ lives. But after a different clinical trial ended in disaster, interest and investment in gene therapy dried up. High knew if she wanted to continue her research, she had to make bold moves.
• Gene therapy may be cutting-edge, but it’s not just happening in multi-million-dollar labs — in fact, some people have started experimenting with it at home. Reporter Grant Hill talks with one researcher about his DIY quest to cure himself of lactose intolerance.
• Could gene therapy, brain surgery, and a group of booze-loving monkeys help treat alcohol use disorder? Reporter Marcus Biddle finds out.