It Feels 'Like Being Stabbed All Over.' A New Cure Is Closer

Panel clears CRISPR gene-editing product for sickle cell disease patients; FDA to weigh in
By Jenn Gidman,  Newser Staff
Posted Nov 1, 2023 12:25 PM CDT
This Treatment Could Be a Big First for Genetic Diseases
This microscope photo shows crescent-shaped red blood cells from a sickle cell disease patient in 1972. The disorder affects hemoglobin, the protein in red blood cells that carries oxygen.   (Dr. F. Gilbert/CDC via AP)

Sickle cell disease is a painful, debilitating, and possibly deadly inherited disorder that currently can only be remedied with a bone marrow transplant. Now, Boston's Vertex Pharmaceuticals hopes exa-cel, its treatment developed with CRISPR Therapeutics, will soon be approved by the FDA following a thumbs-up from a panel of experts on Tuesday who say it's safe for patients with the chronic condition, reports the New York Times. If the FDA gives the green light by Dec. 8, which is now expected, exa-cel would be the first treatment for a genetic disease using CRISPR's gene-editing process.

MarketWatch notes that CRISPR Therapeutics' stock spiked 17% in premarket trading on Wednesday following the announcement. Sickle cell disease is described by the National Heart, Lung, and Blood Institute as "a group of inherited red blood cell disorders that affect hemoglobin, the protein that carries oxygen through the body." A gene mutation causes blood cells in patients with this condition to warp into crescent shapes, which then end up getting stuck in blood vessels. "It's horribly painful," Dr. Allison King, a professor at St. Louis' Washington University School of Medicine, tells the AP. "Some people will say it's like being stabbed all over."

The AP explains how the exa-cel treatment works: Stem cells are removed from the patient's blood, and CRISPR is used to snip out the mutated gene before the stem cells are returned to the patient. The body will then revert to producing a fetal form of hemoglobin that doesn't form the crescent shape. But as the Times notes, it's not an easy undertaking, requiring chemotherapy and more than a month spent in the hospital. There's also the fear that CRISPR could accidentally snip DNA in the wrong place, though that didn't happen in the Vertex trial.

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This might not be the only possible cure for sickle cell disease coming down the pike: Bluebird Bio, which already has gene therapies for thalassemia and adrenoleukodystrophy (ALD), has a sickle cell disease treatment it's hoping to have approved by the FDA by Dec. 20. It's not clear how much either treatment will cost, though a recent evidence report by the Institute for Clinical and Economic Review suggests a per-patient price tag of $2 million. Still, lifelong care for the disease is also "enormously expensive," per the Times—research puts the treatment figure through age 65 at $1.6 million for women and $1.7 million for men. (More CRISPR stories.)

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