NIH Grants $9 Million to Children’s Hospital Los Angeles for SCID Research

The National Institutes of Health’s National Institute of Allergy and Infectious Diseases division has awarded nearly $9 million to researchers from Children’s Center for Cancer and Blood Diseases at Children’s Hospital Los Angeles (CHLA) and Boston Children’s Hospital to study the lowest dose of chemotherapy needed for babies with severe combined immunodeficiency (SCID) undergoing bone marrow transplant, the standard treatment for SCID. The goal is to restore the immune system safely and effectively with less toxicity than the higher dose regimens currently in use.

The trial will be randomized for babies to receive a low or moderate dose of busulfan, a type of chemotherapy that acts to suppress the immune system in preparation for the transplant. The investigators propose that a bone marrow transplant can be performed successfully in SCID patients without the higher dose of busulfan typically used due to the patients’ lack of functional T cells. “Our goal is to decrease the possible long-term effects from chemotherapy by determining the lowest doses needed to ensure T- and B-cell function in these infants, restoring normal immune systems that can last throughout their lives,” said Michael Pulsipher, MD, chair of the Pediatric Blood and Marrow Transplantation Consortium, section head of bone marrow transplant at CHLA and professor of pediatrics at the Keck School of Medicine at the University of Southern California.