Bone marrow transplantation has been used for 60+ years to treat >1,000,000 patients suffering from many types of blood or immune diseases, and this therapy is the best current treatment for FA patients experiencing blood problems. This procedure relies on replacing sick blood-forming stem cells in patients with healthy ones from a donor, resulting in a completely new blood system. However, this currently requires use of irradiation and/or chemotherapy which can be extremely harmful, especially to FA patients whose cells cannot fix DNA mistakes caused by these agents. We think we can make this procedure safe and eliminate all of the bad side effects through use of antibodies that can target specific cells of the body, and together allow safe turnover of the bone marrow and eliminate bone marrow disease in FA patients. This type of therapy could not only be used to treat blood problems in FA patients, but also prevent them all together if used upfront in recently diagnosed patients.
Researchers: Agnieszka Czechowicz