Pioneering stem cell treatment for sickle cell is a UK first
A pioneering stem cell transplant treatment used for sickle cell patients has been successful in curing 23-year-old Remmy Kamya of this crippling disease. The treatment, which is a UK first, was led by Dr Mickey Koh, director of stem cell transplants and consultant haematologist at St George’s Hospital in London.
Sickle cell anaemia is an inherited, lifelong disease and is the most common of the hereditary blood disorders. Red blood cells are produced by stem cells within the marrow found inside the bones. Healthy red blood cells are biconcaved discs which can bend and flex easily. In those with sickle cell disease, faulty stem cells produce red blood cells that are crescent shaped. These are rigid, unable to squeeze through smaller blood vessels, and prone to causing blockages that deprive parts of the body of oxygen, leading to periods of intense pain.
Management of sickle cell disease has always focused on treating symptoms with the only cure up until now being a stem cell transplant, where the abnormal red blood cells are replaced with new healthy ones from a donor. To do this, high levels of chemotherapy are given to the patient to kill off the unhealthy blood cells and to ensure that the donor’s tissue is not rejected. For this reason these transplants are often restricted to children as the levels of chemotherapy may be too toxic for an adult, especially if their organs are already affected from the disease.
The new procedure is novel because it does not use any chemotherapy in the transplant regimen. Instead, it relies on low dose radiotherapy and antibody which modulates the immune system. This makes the treatment tolerable to the patient while minimising potential serious complications from the transplant. This treatment was initially pioneered in America.
Mickey said: “This is an exciting development as it opens up the possibility for more patients affected by this disease to be potentially cured by a transplant. This novel chemotherapy-free regimen was well tolerated and successful in terms of graft acceptance and the absence of serious post transplant complications.”
After his referral, Remmy’s family was tested for a suitable donor and his brother came up as a match. He spent around a month in hospital for the transplant as his immune system was quite low. Following the transplant, the new stem cells from the brother have grown well and tests have revealed that his red cells are being replaced by his brother’s.
Six months on from his transplant, Remmy feels like he leads a normal life now. He said: “I am less tired and have not suffered a sickle cell crisis since!”
Notes to editors
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