Last week, the New York Times published a story about a relatively new treatment for babies born without an immune system. The story highlights recent research led by Donald Kohn, M.D., UCLA Distinguished Professor of Microbiology, Immunology & Molecular Genetics; Pediatrics; and Molecular & Medical Pharmacology.
Working with collaborators from across the U.S. and the U.K., Kohn has been pioneering a gene therapy treatment for babies born with severe combined immunodeficiency (SCID) due to a deficiency of adenosine deaminase (ADA).
ADA-SCID is a life-threatening condition that requires extraordinary measures to keep patients alive and well. The latest follow-up study of patients who received gene therapy for ADA-SCID shows the long-term success and great potential of this treatment.
Excerpted from the New York Times article:
With this treatment, babies need only a low dose of chemotherapy, Dr. Kohn said, and since doctors are reintroducing their repaired cells, there’s little-to-no risk of rejection or need for immunosuppression. In the new study, the babies recovered more quickly with fewer long-term side effects compared to what doctors usually see with bone-marrow transplants, he added.
Read the full New York Times piece to learn more about treatments for SCID and how gene therapy treatment for ADA-SCID has changed the lives of patients and their families.