FDA Approves Groundbreaking Gene Editing Treatment for Sickle Cell Disease
On Friday, the Food and Drug Administration (F.D.A.) made a historic decision by approving the first gene editing therapy ever to be used in humans for the treatment of sickle cell disease, a debilitating blood disorder caused by a single mutated gene. Additionally, the agency approved a second treatment using conventional gene therapy for sickle cell that does not use gene editing.
For the 100,000 Americans suffering from the disease, most of them being Black, the approvals provide hope for finally living without the affliction that causes excruciating pain, organ damage, and strokes. However, obtaining either of these groundbreaking treatments will not come without challenges and high costs.
Dr. Stephan Grupp, chief of the cellular therapy and transplant section at Children’s Hospital of Philadelphia, referred to the approval as “practically a miracle,” although he remains realistic about the difficulties associated with the treatments.
Obstacles to treatment are numerous, including the requirement for an extremely limited number of medical centers authorized to provide it, the necessity for technicians to edit or add genes to each patient’s cells individually, onerous procedures that not everyone can tolerate, and an expensive price tag.
The two treatments were approved for patients aged 12 and older who experience recurrent episodes of excruciating pain from sickle cell disease. The gene editing treatment, known as Exa-cel and utilizing the brand name CASGEVY, was developed by Vertex Pharmaceuticals of Boston and CRISPR Therapeutics of Switzerland. On the other hand, the other treatment, called Lyfgenia and made by Bluebird Bio of Massachusetts, uses a common gene therapy method to add a good hemoglobin gene to patients’ DNA.
While the newly approved treatments bring hope to patients and their families, the process of obtaining them is complex. It involves the collection of bone marrow stem cells from patients, a sequence of quality checks, extensive chemotherapy to clear the patient’s marrow, and a long hospitalization period.
Dr. Sharl Azar, medical director of the comprehensive sickle cell disease treatment center at Massachusetts General Hospital, expressed that patients and doctors are starting to hope for longer life expectancy, with patients possibly living into their 70s and 80s rather than dying young.
“The F.D.A’s approval of the gene editing therapy for sickle cell disease represents a monumental step forward in medical advancements. However, the logistical and financial hurdles associated with obtaining the treatment remain significant,” said Dr. David Jacobsohn, chief of the division of blood and marrow transplantation at Children’s National Hospital in Washington.
Despite the many unknowns, medical centers are preparing to offer the treatment to interested sickle cell patients, gradually addressing the challenges posed by this groundbreaking therapy. As for now, the approved treatments provide a glimmer of hope for patients and doctors, showing promise in the battle against this debilitating blood disorder.