With September being National Sickle Cell Awareness Month, a U.S. Food and Drug Administration (FDA) recently approved sickle cell disease gene therapy is giving hope of a cure to patients who struggle with a lifetime of agony, pain and hospitalization.
Dr. Resa Caivano, a physician who works at the Kaiser Permanente Sickle Cell Center of Excellence in Inglewood, noted gene therapy is a game changer in efforts to this previously incurable disease.
“Gene therapy for sickle cell disease takes a patient’s own blood stem cells, genetically modifies them so they can make healthy hemoglobin, and returns them to the body,” Dr. Caivano said. “Approved by the FDA in December 2023, this groundbreaking treatment has helped many patients avoid painful sickle cell crises and offers the likelihood of a long-term cure.”
How it works
People with sickle cell disease inherit a gene that causes their red blood cells to become hard, sticky and sickle-shaped. These abnormal cells can block blood flow, causing severe pain episodes, organ damage and other serious complications.
This groundbreaking gene therapy is giving patients great hope for a future free of this debilitating disease, however. This is how it works:
- Doctors collect blood stem cells from the patient.
- In a laboratory, those cells are genetically modified.
- The patient receives chemotherapy to make room in the bone marrow.
- The modified cells are infused back into the patient’s body.
- The cells begin producing healthy or healthier hemoglobin, which helps red blood cells stay round and flow normally.
“Gene therapy is a game changer,” Dr. Caivano said. “It can be a one-time treatment that addresses the root cause of the disease rather than simply managing symptoms. It’s a likely cure for a disease for which people have a life expectancy of about 45 years. And now, through this process, we’re able to extend that life expectancy. Furthermore, our patients don’t have to live in agony any longer.”
What impact has gene therapy had on patients?
The impact has been remarkable for many patients, Dr. Caivano said. “Not only does gene therapy offer the potential for a functional cure, but it also allows many of our patients to live more normal lives with fewer interruptions from severe pain and medical emergencies.”
Kaiser Permanente offers valuable information about life with sickle cell disease.
