The US Food and Drug Administration is studying the first CRISPR gene-editing treatment that may treat sickle cells
The U.S. Food and Drug Administration is reviewing a cutting-edge treatment called Exa-Cell that could treat people with sickle cell disease, a painful and fatal disease with no universally successful treatment. “If approved, exa-cel, made by Boston-based Vertex Pharmaceuticals and Swiss company CRISPR Therapeutics, will be the first FDA-approved treatment to use genetic editing called CRISPR,” CNN reported. From the report: CRISPR, or clustered regularly interspaced short palindromic repeats, is a technology used by researchers to selectively modify DNA, the carrier of genetic information that the body uses to function and develop. (…) A new exa-cel treatment under FDA study could use the patient’s own stem cells. Doctors modify them using CRISPR technology to fix the genetic problems that cause sickle cells, and then the modified stem cells are returned to the patient in a one-time injection.
In the company’s studies, the treatment is considered safe and has “very positive risks for patients with severe sickle cell disease,” Dr. Stephanie Krugmeyer, vice president of global regulatory affairs at Vertex Pharmaceuticals Incorporated, told the committee. Thirty-nine out of 40 people tested with the treatment did not have a single vaso-occlusive crisis, meaning that misshapen red blood cells impede normal circulation and can cause moderate to severe pain. It’s the main reason sickle cell patients go to the emergency room or are hospitalized. Before treatment, patients experienced about four of these painful crises per year, resulting in about two weeks spent in the hospital.
The FDA requested the independent panel’s advice, in part, because this would be the first time the FDA has approved a treatment using CRISPR, but Dr. Fyodor Ornov, a professor in the Department of Molecular and Cell Biology at the University of California Berkeley, reminded the committee that CRISPR has been around for 30 years ago, and during that time, scientists have learned a lot about how to use them safely. “The technology is, in fact, ready for use during peak times,” Urnov said. Using this type of gene editing, scientists can inadvertently make a change to a patient’s DNA that is off target, and the treatment could harm the patient. (…) The US Food and Drug Administration is expected to make an approval decision by December 8th.