Improved way of treating sickle cell disease with stem cells

June 19th has been designated as World Sickle Cell Awareness Day to try and raise public awareness about this disease. So it’s rather appropriate that the Science Daily is carrying a story about a Chicago woman who has been cured of her sickle cell disease using a bone marrow stem cell transplant.

Bone marrow transplant is currently the only way of curing sickle cell disease, but the procedure itself can be deadly so it is only used as a last resort. Traditionally, before a person receives the new bone marrow stem cells their own bone marrow is eliminated through high doses of chemotherapy. That procedure itself is risky, and leaves the person without an immune system until the new bone marrow takes over and begins producing immune cells. So, it’s no wonder the technique is only used in critical cases.

The doctors treating the Chicago woman took a different approach. They didn’t completely eliminate her own bone marrow, lessening the risk of infection. If this type of less risky bone marrow transplant becomes widely available, it could open up the possibility of bone marrow transplants to treat multiple sclerosis and other diseases of the immune system.

CIRM-funded researchers are also working on a similar approach to treating sickle cell disease. However, instead of taking bone marrow from a donor Dr. Donald Kohn and his team at UCLA are using the patient’s own bone marrow that has been genetically modified to replace the defective gene that causes the disease. They then give the patient a full dose of chemotherapy to destroy the defective bone marrow and replace it with the corrected version.

Dr. Kohn is now working with other CIRM-funded researchers at USC, and Children’s Hospitals in Los Angeles and Oakland to move this this therapy into clinical trials. You can read about their work here. We also have a video of researchers talking about their approach to treating sickle cell disease.

It’s an example of how researchers around the US are taking different approaches to reach the same goal, curing a disease that affects 80,000 Americans. 

K.M.