FDA approves first gene-editing treatment for human illness
In a landmark decision, the Food and Drug Administration Friday approved the first gene-editing treatment to alleviate human illness.
The FDA approved two gene therapies for anyone 12 and older suffering from the most severe form of sickle cell disease, a brutal blood disorder that has long been neglected by medical research.
The decisions are being hailed as milestones for treating sickle cell and for the rapidly advancing field of gene editing, which is stirring excitement for treatment of many diseases.
“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 Dr. Nicole Verdun, director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research, in 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.”
“I’m elated, excited, in awe,” Jennifer Doudna of the University of California, Berkeley, who helped discover the gene-editing technique called CRISPR that is used in one of the sickle cell treatments, told NPR in an interview. “It’s an exciting day and the beginning of a new day in medicine.”
For the CRISPR treatment, which was developed by Vertex Pharmaceuticals and CRISPR Therapeutics, both in Boston, doctors remove cells from each patient’s bone marrow, edit a gene with CRISPR and then infuse billions of the modified cells back into patients.
The edited cells produce a form of hemoglobin known as fetal hemoglobin, restoring normal function of red blood cells. While not a cure for the disease, the hope is the therapy, brand name Casgevy, is designed to be a one-time treatment that will alleviate symptoms for a lifetime.
In data presented to the FDA, the treatment resolved the severe pain crises for at least 18 months for 29 of the subjects — 96.7%. The treatment has produced similar results for patients suffering from a related condition known as beta thalassemia.
