Children’s Center for Cancer and Blood Diseases at Children’s Hospital Los Angeles and Boston Children’s Hospital have been granted almost $9m 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).
SCID is a condition preventing the immune system from functioning normally.
In a multi-site study, investigators – Michael Pulsipher of the Children’s Center for Cancer and Blood Diseases and Sung-Yun Pai of Boston Children’s Hospital, intend to find the lowest dose of chemotherapy required for babies sufering with SCID and undergoing bone marrow transplant, which is the standard treatment for SCID.
The aim is to restore the immune system safely and effectively with less toxicity than the higher dose regimens presently used.
Babies suffering with SCID usually die of infection before their second birthday unless they have a bone marrow transplant (BMT). The transplanted cells are injected into the blood stream where they become healthy white blood cells that restore immune function.
According to the investigators, BMT can be performed successfully in SCID patients without the higher dose of busulfan typically used, due to the patients’ lack of functional T cells.
Given the already reduced immune system, the investigators believe that giving a lower dosage of busulfan will be effective and safe for patients.
Pulsipher said: “Our goal is to decrease the possible long-term effects from chemotherapy by determining the lowest doses needed to insure T and B-cell function in these infants, restoring normal immune systems that can last throughout their lives.”
The trial was organised as a joint project between the PBMTC and the Primary Immune Deficiency Treatment Consortium (PIDTC), a group of over 40 centres in North America dedicated to improving therapy for children with immune deficiencies.
As CHLA is the primary recipient of the NIH award, it will serve as coordinating centre for the trial, which will involve 50 centres in the US and Canada. Besides CHLA and Boston Children’s Hospital, other institutions receiving funding to conduct the study include the Medical College of Wisconsin, University of California, San Francisco, University of Pittsburgh, the CHU Sainte-Justine Research Center of the University of Montreal and the National Marrow Donor Program in Minneapolis.