In a Breakthrough, FDA Backs Gene Editing Therapy

Two gene therapies for sickle cell disease received government approval on Friday, one of which could represent a major turning point in medical treatments. To combat the blood disorder, the Food and Drug Administration for the first time endorsed gene editing therapy for humans, the New York Times reports. The other sickle cell treatment approved uses conventional gene therapy. The development brings hope to 100,000 American patients, their families, and their doctors. "I've been taking care of kids with sickle cell for over 30 years, and I've been waiting for something like this to happen for a long, long time," said Lewis Hsu, chief medical officer of the Sickle Cell Disease Association of America, per the Washington Post.

One treatment, called Lyfgenia, uses a harmless virus to place a gene in a patient's stem cells. It's approved for patients 12 and older with repeated instances of pain. The risk of cancer means the treatment will come with the FDA's top-level warning, a black box. The gene-editing treatment, Exa-cel, uses the brand name Casgevy. In that method, the Nobel Prize-winning gene editing tool, CRISPR, is used on bone marrow cells. The patients later receive the edited cells, which produce fetal hemoglobin. There are obstacles to the treatments for the patients, most of whom have African ancestry, dealing with a disease that can cause great pain, strokes, and organ damage.

"It is practically a miracle that this is even possible," said another expert, adding, "I am very realistic about how hard this is." The companies that developed the treatments estimated their wholesale prices at $2.2 million and $3.3 million. That's against the lifetime average of $1.7 million spent on patients with the disease who have commercial insurance, and the $44,000 the patients pay out of pocket. The editing process could be a breakthrough for treating other diseases, as well. "We are very gratified and celebrating," Hsu said, "but at the same time we have to roll up our sleeves and figure out how to make it happen." (More sickle cell disease stories.)