The Gene Therapy 'Quagmire:' Multimillion- Dollar Costs and Untreated Patients with Rare Diseases

Jason Mast is a former English teacher who now focuses on such things as multimillion-dollar gene therapies and whether they ever reach patients.

Mast writes for STAT, the respected online biomedical news service. On Monday, he encapsulated the “gene therapy crisis” in 115 words:   

  “Jennifer Puck has successfully treated 10 children with a gene therapy for a fatal disorder that decimates their immune system. But she has no idea how to get her drug approved and frankly is running out of ideas.

“‘I wish I had a clue about where to go from here,’ said Puck, an immunologist at University of California, San Francisco…. 

“The problem is simple: Size,” Mast wrote. “Puck’s therapy is for a disease, Artemis-SCID, that affects just two to three new U.S. patients every year — far too few for a company to generate a profit, or to even run the kind of studies regulators usually demand before approving drugs.”

All in all, Mast wrote: “a quagmire born of the field’s own success.”

The 115 words were just a start in a lengthy piece...