The Food and Drug Administration approved two revolutionary new treatments for sickle cell disease on Friday, ushering in a massive change to how the disease can be treated in the United States.
For the first time, the FDA approved the use of CRISPR, a gene editing tool, in human beings. The agency simultaneously approved a secondary treatment that uses cell-based gene therapy to help patients suffering from the disease.
Sickle cell disease is an inherited blood disorder that causes a protein in the red blood cells called hemoglobin to mutate. The red blood cell becomes “sickle” shaped, restricting blood flow and causing severe pain and even early death. In the United States, sickle cell disease is primarily found in Black Americans. Approximately one in every 365 Black babies born in the United States suffer from the disease.
“Sickle cell disease is a rare, debilitating, and life-threatening blood disorder with significant unmet need, and we are excited to advance the field, especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today,” said Nicole Verdun, M.D., director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research, in a statement.
“Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited.”
Some physicians have warned that accessing these treatments won’t be easy for the average patient. The tools are expensive, not every medical center is set up to use them and the treatment process is incredibly time-consuming. The New York Times reported that the FDA warned that only a fraction of the patients currently suffering from sickle cell disease will be able to use the treatment.
Still, for many in the healthcare field, this announcement is a game changer for the treatment of sickle cell patients. “We are talking for the first time about survivorship,” Dr. Sharl Azar, medical director of the comprehensive sickle cell disease treatment center at Massachusetts General Hospital, told the New York Times.