Children’s Hospital Los Angeles

Children’s Hospital Los Angeles to study “Bubble Boy” syndrome

Tuesday, October 10, 2017

Michael Pulsipher, M.D., of the Children’s Center for Cancer and Blood Diseases at Children’s Hospital Los Angeles, along with co-principal investigator, Sung-Yun Pai, M.D., of Boston Children’s Hospital, has been awarded nearly $9 million from the National Institute of Allergy and Infectious Diseases of the NIH to study a new treatment approach for babies born with severe combined immunodeficiency (SCID), which prevents the immune system from functioning normally. In a multi-site study, investigators plan to determine the lowest dose of chemotherapy needed for babies with 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.

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