Monroe Carell Jr. Children's Hospital at Vanderbilt has been selected as part of a select group of healthcare institutions to offer a new FDA-approved immunotherapy for a subset of pediatric acute lymphoblastic leukemia (ALL). Only 32 healthcare institutions in the United States will be certified to offer the therapy. Vanderbilt is only one of two centers in the state, and the only one located in the Middle and East Tennessee.

This novel therapy, a personalized treatment known as chimeric antigen receptor (CAR) T-cell therapy, is an innovative treatment designed for children and young adults with relapsed or refractory pre-B cell ALL. This therapy, called Kymriah, takes a patient's own immune cells and reprograms the cells to recognize and destroy the patient's leukemia cells.

"It is an honor and privilege to be selected as one of the first 10 centers in the country to offer this innovative therapy," said Debra Friedman, MD, director of the Division of Pediatric Hematology/Oncology, associate professor of Pediatrics and E. Bronson Ingram Professor of Pediatric Oncology. "This is in recognition of our expert oncology and stem cell transplant teams and the multispecialty care that we can provide here. We welcome referrals from around the region for this exciting new therapy to offer new opportunities for more children and young adults."

Typically, 90 percent of ALL patients are cured with intensive chemotherapy treatment. The remaining 10 percent of ALL patients don't respond to chemotherapy or relapse and have a very poor prognosis. Patients up to age 25 who have these high-risk features may be eligible for this innovative CAR T-cell therapy. With Kymriah, a patient's T cells are collected and shipped to the pharmaceutical company, Novartis, where the cells are engineered to express a chimeric antigen receptor designed to recognize the patient's leukemia. The engineered CAR T-cells are grown in the laboratory and then shipped back to the hospital where they are infused into the patient. Each time a CAR T-cell recognizes a cancer cell it is activated to kill the leukemia cell. Once this happens, a signal is sent to the to the CAR T-cell to divide, producing even more of the cancer fighting cells.

The FDA approved the CAR T-cell therapy on Aug. 30 following multicenter clinical trials at several sites around the country that demonstrated an 83 percent rate of remission in these very high-risk patients, and these remissions are long lasting in many patients.