UCSF First in State to Provide FDA-Approved CAR-T for Pediatric Cancer Patients

‘Game-changer’ Therapy Boosts Cure from 10-20 Percent, to 80 Percent

By Suzanne Leigh

UCSF Benioff Children's Hospital San Francisco sign

UCSF Benioff Children’s Hospital San Francisco has been certified as the first medical center in California to provide CAR-T therapy for children and young adults with acute lymphoblastic leukemia (ALL), who have failed standard treatment.

The first pediatric patient at UCSF Benioff Children’s Hospital San Francisco had cells collected on Sept. 14, in the first step of the treatment, which involves turbocharging the patient’s own T-cells – the soldiers of the immune system – to attack the cancer.

CAR-T represents a radically different way of treating cancer. It entails filtering T-cells from the patient’s blood and reprogramming them to harbor a “chimeric antigen receptor,” or CAR, that zeroes in on cancer and develops millions of copies of the soldier cells. These super-potent T-cells are then returned to the patient, where they continue to multiply and fight cancerous cells.

The version of CAR-T therapy is developed by Novartis and marketed as Kymriah. It is approved for children and adults up to age 25 with ALL that did not go into remission or recurred after more than one salvage therapy.

ALL is the most common pediatric cancer and affects approximately 3,100 children and teens each year, according to the National Cancer Institute. The five-year national survival rate is approximately 90 percent for children 14 and under, and 75 percent for older teens. While outcomes for children and young adults with ALL have improved dramatically due to intensive chemotherapy, prognosis for patients that do not respond or relapse after bone marrow transplant is dismal, said Michelle Hermiston, MD, PhD, director of the pediatric cancer immunotherapy program at UCSF Benioff Children’s Hospital San Francisco.

“We are gratified to be the first certified treatment center for CAR-T cell therapy in the state,” Hermiston said. “CAR-T improves the survival rate for these patients from approximately 10 to 20 percent, to 80 percent. This is a game-changer.”

UCSF Benioff Children’s Hospital San Francisco has the largest pediatric bone marrow transplant program in Northern California.

UCSF is one of 14 sites studying CAR-T therapy in a clinical trial for adults with B-cell lymphoma, an aggressive form of non-Hodgkin’s lymphoma. The treatment is expected to be approved by the Food and Drug Administration later in the year. Additionally, the UCSF Helen Diller Family Comprehensive Cancer Center is active in translational and clinical cellular and immunotherapy research. Clinical trials are ongoing for treating solid tumors and hematological cancers in children and adults.