The FDA recently approved the first cure for sickle cell disease based on gene-editing tool CRISPR.
Known as Casgevy, the medicine created by Vertex Pharmaceuticals and CRISPR Therapeutics was approved on Friday. Using the new cure, the bone marrow transplant that was typically needed from a donor during the only cure previously available is now unnecessary.
The CRISPR tool edits DNA in stem cells to erase the gene that causes sickle cell disease, ensuring that sickle-shaped cells are no longer created by the patient’s body.
Although it’s given as a one-time treatment, the process includes blood transfusions, during which the stem cells are extracted, as well as chemotherapy to remove the patient’s bone marrow.
In the treatment’s clinical trial, 29 out of 46 participants had successful results with Casgevy. Previously, biochemists Emmanuelle Charpentier and Jennifer Doudna were given a Nobel Prize in Chemistry for their creation of CRISPR.
“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 the director of the Office of Therapeutic Products in the FDA’s Center for Biologics Evaluation and Research, Nicole Verdun, M.D., per 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.”
Considered an inherited disorder, sickle cell disease affects approximately 100,000 out of all 340 million Americans, per the Centers for Disease Control and Prevention.
While sickle cell disease has been found in people from other demographics, it’s been reported as recurring mostly amongst Black Americans.
Today we approved two milestone treatments – the first cell-based gene therapies to treat sickle cell disease in patients 12 years and older. https://t.co/az8lDdRyz0 pic.twitter.com/7DGkohRoIo
— U.S. FDA (@US_FDA) December 8, 2023
Per the CDC, one out of every 365 Black American babies have sickle cell disease.
If not diagnosed with sickle cell disease, the chance of developing sickle cell is common, as one in 13 Black American babies is born with the sickle cell trait. Overall, approximately eight to ten percent of Black Americans are diagnosed with sickle cell trait.
When diagnosed with sickle cell disease, patients are forced to live with complications such as sickle crisis, acute chest syndrome, strokes and anemia, per Johns Hopkins Medicine.
With the new treatment, these symptoms can be alleviated.
As they continue to monitor the long-term span of Casgevy, researchers are also looking to make the treatment more affordable; currently, the treatment is very expensive, averaging the cost of $2.2 million per patient.
“We really have to make sure that it is accessible,” said pediatric hematologist-oncologist and previous advisor for Vertex, Dr. Rabi Hanna, per NBC. “This could be an equalizer for people with sickle cell because many patients cannot pursue career options because of the illness.”
