A word from Dr. Moore, Professor and Clinical Director of Pediatric Hematology/Oncology at UCLA

The Program for Cellular Therapeutics at Mattel Children’s Hospital UCLA works in conjunction with the Gene and Stem Cell therapy and the Pediatric BMT programs to develop innovative safer, less toxic and more effective therapeutic options for children suffering from severe diseases such as leukemia and autoimmune diseases. Its mission is to recruit and enable the cells of the human body to fight cancer and infections, and to correct genetic defects. Researchers from this program are also working in the laboratory development of hematopoietic stem cells and T cells targeting viral infections and chemotherapy-resistant leukemias and lymphomas. During the past year, six patients received gene therapy with hematopoietic stem cells for severe combined munodeficiency (SCID or “bubble boy disease”) who might not have survived past 2 years of age without a bone marrow transplant. With successful gene therapy these children have developed normal immune systems with minimal toxicity. The program has also performed an immune modulating stem cell transplantation for a patient with severe Crohn’s Disease. Clinical trials are now open for cellular therapies for patients with sickle cell disease and adrenoleukodystrophy (ALD), and soon for patients with chronic granulomatous disease (CGD). The Cellular Therapies Program combines extensive clinical expertise, innovative scientific research and genuine care to propose bold therapeutic options for life-threatening diseases.