The California Institute for Regenerative Medicine (CIRM) board awarded $5.74 million to City of Hope to fund a phase 1 clinical trial testing a novel blood stem cell transplantation procedure for adult patients with severe sickle cell disease (SCD).
The treatment involves induction of mixed chimerism. This refers to transplanting blood-forming stem cells from a healthy half-matched family member donor into a patient who has received a milder, less toxic chemotherapy treatment that removes some but not all of the patient’s diseased bone marrow stem cells. The donor blood stem cell transplants are depleted of some immune cells called CD4+ T cells prior to transplantation. This approach allows the donor stem cells to engraft and create a healthy supply of non-diseased blood cells without causing graft-versus-host disease, a side effect of bone marrow transplantation.