This week Bobbi Conner talks with Dr. Jennifer Joi Jaroscak about new gene therapy treatments for sickle cell disease. Dr. Jaroscak is a Professor of Pediatrics and Director of Non-Malignant Pediatric Stem Cell Transplantation at MUSC.
TRANSCRIPT:
Conner: I'm Bobbi Conner for South Carolina Public Radio with Health Focus here at the radio studio for the Medical University of South Carolina in Charleston. In December 2023, the US Food and Drug Administration approved two new gene therapy treatments for sickle cell disease. Doctor Jennifer Joi Jaroscak is here to provide the details. Doctor Jaroscak is a Professor of Pediatrics, and she's Director of Non-malignant Pediatric Stem Cell Transplantation at MUSC. Doctor Jaroscak, tell us about sickle cell disease.
Dr. Jaroscak: Sickle cell disease is a disease of the red blood cells. And it actually causes the red cells to change shape. And then those sickled red blood cells get stuck in blood vessels and cause pain in patients with sickle cell disease. It can affect lots of organs and it actually shortens the life expectancy for people with the disease.
Conner: Give us an update about this gene therapy treatment that is now available for sickle cell disease.
Dr. Jaroscak: Well, it's super exciting that two gene therapies got approved in December of last year. And both of these gene therapies are very effective in preventing vaso-occlusive episodes or pain crises in patients. The two different gene therapies, one works by inserting a normal hemoglobin gene, and the other works by editing the gene to turn back on fetal hemoglobin. What is amazing for patients is that both of these treatments are about 94% effective in preventing the severe vaso-occlusive episodes that cause people to be hospitalized with a pain crisis.
Conner: And who is a good candidate for this new gene therapy treatment?
Dr. Jaroscak: Most people with sickle cell disease who have vaso-occlusive episodes that require hospitalization and IV pain medicine. So, if you've had four of those episodes in over a two year period, then you would be eligible for this type of treatment. And this treatment is available to patients who are age 12 and up.
Conner: And do we know what the long term impact might be for patients on this new gene therapy treatment?
Dr. Jaroscak: So we don't know if it's going to extend their life. But we do know that the longest patients who got these treatments are currently about six years out from their procedures, and they're maintaining normal hemoglobin levels this whole time. So, we're going to follow these patients out for 15 years after their treatments. But no patients are that far out yet.
Conner: What are some of the challenges related to this new treatment for sickle cell disease?
Dr. Jaroscak: Unfortunately, it takes about nine months to 12 months to get through this process. There's about eight months of preparation because we collect the patient's own cells to create the gene product. And then we have to wait for the gene product to be created. And then we have to give the patient chemotherapy, which can cause infertility. The other drawback is that right now these gene therapy treatments are incredibly expensive. They're somewhere between 2 and $3 million for the tablespoon of cells, that is the gene product. And then patients still have to have a lengthy hospital stay, anticipated about eight weeks in the hospital.
Conner: Doctor Jaroscak, thanks for this update about treating sickle cell disease with gene therapy.
Dr. Jaroscak: You're welcome. Thanks for having me.
Conner: Bobbi from the radio studio for the Medical University of South Carolina in Charleston. I'm Bobbi Conner for South Carolina Public Radio.
Health Focus transcripts are intended to accurately represent the original audio version of the program; however, some discrepancies or inaccuracies may exist. The audio format serves as the official record of Health Focus programming.
