Pediatric Bone Marrow Transplant (BMT), Cellular & Gene Therapy

Advanced therapies for childhood diseases

Bone marrow transplantation, cellular therapies and gene therapies are transforming how we treat childhood diseases. At UCSF, we offer these therapies to children with a wide range of complex conditions, including:

• Blood cancers

• Noncancerous blood disorders

• Brain tumors and other solid tumors

• Immune system disorders

• Inherited metabolic disorders

Excellence in bone marrow transplantation

A bone marrow transplant, also called a stem cell transplant, may be part of your child's treatment plan. During this procedure, we replace diseased or damaged blood-forming stem cells with healthy cells from a closely matched donor.

A transplant may be recommended for children and young adults with:

• Leukemia or lymphoma

• Sickle cell disease

• Thalassemia

• Aplastic anemia

• Bone marrow failure syndromes

• Autoimmune diseases

• Immunodeficiency disorders

• Inherited metabolic disorders, including lysosomal storage diseases

UCSF excels in every aspect of the transplant process, achieving excellent outcomes. Our comprehensive approach involves:

• Thorough pretransplant evaluation to ensure children are good candidates

• Stem cell collection through apheresis, a procedure that collects blood components and returns the remaining blood to your child's body

• Personalized chemotherapy dosing to prepare children for treatment

• Long-term follow-up care and a robust survivorship program

Pioneers in cellular therapy

UCSF is at the forefront of cellular therapies, such as CAR T-cell therapy, a type of immunotherapy. We were the first in California certified to provide CAR T-cell therapy to children with leukemia outside of a clinical trial. We're also one of only a few centers in Northern California offering the first FDA-approved CAR T-cell therapy, Kymriah.

CAR T-cell therapy uses a child's own immune cells, which are collected and modified in a laboratory to recognize and attack cancer cells. This advanced therapy may be used to treat children with B-cell acute lymphoblastic leukemia (B-ALL) when other treatments haven't worked or cancer returns after treatment.

Gene therapy for transfusion-dependent blood disorders

UCSF was among the first to offer gene therapies for children with sickle cell disease and beta thalassemia. These inherited blood disorders often require frequent blood transfusions and ongoing medical care.

Gene therapies are also available for inherited immune system and neurological disorders, including metachromatic leukodystrophy, Wiskott-Aldrich syndrome and Artemis-deficient severe combined immunodeficiency. Our program offers FDA-approved gene therapies, as well as access to clinical trials studying emerging gene treatments.

Gene therapy works by modifying a child's stem cells to correct the underlying genetic cause of the disease. For eligible patients, gene therapy may reduce or eliminate the need for transfusions and greatly improve their quality of life.