People living with sickle cell disease should have a new option for treatment in the not-too-distant future. In addition to the blood transfusions and bone marrow transplants from healthy donors, a newer procedure that uses the person’s own bone marrow was tested in Paris and the U.S.
A year ago today, it was reported that a Parisian teenager with the disease achieved total remission after receiving this groundbreaking treatment as part of a clinical trial.
More than a year after the end of the treatment cycle, the young man had stopped receiving medication of any kind for his sickle cell and does not display any signs or symptoms of his disease.
Sickle cell disease is an inherited blood disorder where people’s red blood cells do not form properly in the bone marrow. A healthy red blood cell resembles a rounded disc with a depression in the middle of the flat sides. The red blood cell of a person with sickle cell looks like a crescent.
These malformed cells have difficulty carrying oxygen to the various parts of the body, which can cause pain in those areas. The crescent-shaped cells can also clog up narrower veins, denying blood flow to a given body part.
The procedure:
The revolutionary procedure uses bone marrow extracted from the person. After doctors remove the marrow, the stem cells are withdrawn and reprogrammed with instructions to make healthy red blood cells. While this is being done, the person is treated with chemotherapy to kill off the diseased stem cells. After four days of chemotherapy, the reprogrammed stem cells are reintroduced into the system.
Major advantages of this procedure are that there’s no need to find a matching donor. There’s also virtually no chance of rejection of the transplanted stem cells.
Still, much research needs to be conducted concerning this procedure. Clinical trials are being carried out in Europe and the U.S. to determine the viability of this treatment option, not just for sickle cell disease.
