A groundbreaking gene therapy called Casgevy, which utilizes the Nobel Prize-winning Crispr technology, has shown promising results in treating sickle cell disease. Developed by biotech company CRISPR Therapeutics, Casgevy modifies patients’ cells to produce healthy hemoglobin. This involves extracting stem cells from a patient’s bone marrow, editing them in a laboratory, and then reintroducing the edited cells into the patient’s bloodstream. In a clinical trial, 29 out of 31 patients remained free of pain crises for at least a year after receiving a single dose of their edited cells. This approach could potentially offer a cure for sickle cell disease, as previous treatments like stem cell transplants are limited to a small number of patients and can have life-threatening risks.
In addition to Casgevy, the US Food and Drug Administration (FDA) has also approved a second gene treatment for sickle cell called Lyfgenia. Developed by Bluebird Bio, this therapy adds a therapeutic gene to cells to enable the production of healthy hemoglobin. In a two-year trial, 28 out of 32 patients experienced the elimination of pain crises between six and 18 months after treatment with Lyfgenia. However, the FDA has placed a black box warning on Lyfgenia due to severe safety risks, including the development of blood cancer in some patients.
Doctors and researchers are optimistic about the potential of these gene therapies to transform the lives of individuals affected by sickle cell disease. Alexis Thompson, chief of hematology at Children’s Hospital of Philadelphia, expresses that these therapies allow for conversations about the possibility of a child being cured of sickle cell, something that would have been unimaginable just a few years ago. While the first commercial patients to receive Casgevy will likely have to wait until early next year, the development of these innovative treatments brings hope to patients who have been waiting for effective solutions for a long time.