Amid Giddiness Over First-Ever Gene-Silencing Drug’s Approval Is An Acknowledgment Of Its Limitations
Right now, the RNAi drug is limited to cells that go through the liver, which is -- in relative terms -- easy to target. Getting the drug to other tissue, like the skin or brain, is more challenging. “It’s always been the same problem. And it’s delivery, delivery, delivery,” Steven Dowdy, a cancer biologist at the University of California, San Diego’s school of medicine, tells Stat. “It’s always been the 800-pound gorilla in the room.”
The Future Of RNAi Medicine Is Exciting — But Incredibly Uncertain
On Friday, the Food and Drug Administration approved the first-ever drug to rely on a Nobel-prize-winning technique that mutes disease-causing genes — a watershed moment for that field of research, and a starting pistol for the race to find a way to use the therapy for other debilitating genetic diseases like ALS or Huntington’s. But already, a major question looms: What if the technique won’t work in other parts of the body? The first-ever RNAi drug, patisiran, targets the liver. So, too, do similar, already-approved therapies in the broader category of “sequence-based drugs.” There’s a simple reason for that: The liver is an easy target. The liver is the body’s filter; if something is in your blood, it will eventually pass into the organ. (Sheridan, 8/13)
Meanwhile, a new therapy could provide hope for patients with hemophilia —
The New York Times:
They Thought Hemophilia Was A ‘Lifelong Thing.’ They May Be Wrong.
Scientists are edging closer to defeating a longtime enemy of human health: hemophilia, the inability to form blood clots. After trying for decades to develop a gene therapy to treat this disease, researchers are starting to succeed. In recent experiments, brief intravenous infusions of powerful new treatments have rid patients — for now, at least — of a condition that has shadowed them all their lives. (Kolata, 8/13)