If successful, the procedure could open up similar BMT therapies to patients with other auto-immune diseases such as multiple sclerosis, lupus or diabetes that are generally not candidates for BMT currently. They plan to eliminate the bad cells with an antibody, a protein, that very specifically targets and eliminates blood forming stem cells. This team proposes to replace SCID patients’ dysfunctional immune cells with healthy ones using a safer form of bone marrow transplant (BMT). Clinical Stage Programs Stanford School of Medicine (X-linked SCID) These regimens kill many types of cells beyond those intended and result in numerous toxic side effects. Most of the risk of current BMT procedures comes from the radiation or chemotherapy given to patients before the transplant to wipe out the patient’s own stem cells that form immune cells. They are trying to improve the safety of bone marrow transplant (BMT), which essentially uses the stem cells in bone marrow to give the children a new immune system that works properly. Researchers funded by California’s stem cell agency are looking for a better alternative to help these children. Newer gene therapy techniques appear to be safer but have been tried on fewer than 20 patients. Gene therapy has been used to correct the defect in certain types of SCID, but early gene modifying techniques resulted in some patients developing cancer. Because these patients already have a compromised immune system, 10 to 20 percent don’t survive the transplant. Left untreated the children die before the age of 2, and the only readily available treatment involves high-risk bone marrow transplants. SCID – also known as ‘bubble boy disease’ - is a rare genetic disorder, effecting one in 30,000 newborns. Severe combined immune deficiency or SCID is an example of a primary immunodeficiency.
Typically, these diseases have genetic causes and many of them are rare.
Primary immunodeficiencies are disease that compromise or destroy the immune system, leaving patients susceptible to serious infections. CIRM funds many projects seeking to better understand Severe Combined Immune Deficiency (SCID) and other primary immunodeficiency diseases to translate those discoveries into new therapies.
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