A novel immunotherapy that uses a patient’s own cells to fight cancer is now available at only a select group of health care institutions. Monroe Carell Jr. Children’s Hospital at Vanderbilt is the only center in Tennessee approved to provide the new treatment, a CAR T-cell therapy called Kymriah.
Kymriah was approved by the FDA to treat a subset of acute lymphoblastic leukemia (ALL) — pre-B cell ALL that was unresponsive to treatment (refractory) or that has come back after treatment (relapsed).
ALL, which develops from blood-forming cells in the bone marrow, is the most common childhood cancer. Typically, 90 percent of patients with ALL achieve remission with intensive chemotherapy treatment. The remaining 10 percent don’t respond to chemotherapy or relapse and have a poor prognosis.
“We are thrilled to be able to offer this cutting-edge therapy to our patients, particularly when these families may be starting to feel like they have run out of treatment options for ALL,” says Carrie Kitko, MD, associate professor of Pediatrics and director of the Pediatric Stem Cell Transplant Program in the Division of Hematology/Oncology.
CAR T-cell therapy works like this: a patient’s T cells — white blood cells that normally help fight infection — are collected and shipped to the pharmaceutical company Novartis. At Novartis, the cells are engineered to express a chimeric antigen receptor (CAR) designed to recognize the patient’s leukemia cells. Large numbers of engineered CAR T cells are grown in the laboratory and then shipped back to the hospital, where they are infused into the patient.
The CAR T cells recognize and kill the leukemia cells, and send a signal instructing CAR T cells to divide, which produces even more of the cancer-fighting cells.
Because the therapy can have serious and life-threatening complications, the FDA requires that treatment sites have special certification.
“Vanderbilt is ideally suited to care for these patients,” Kitko says. “Our team has been providing cellular therapy to the Middle Tennessee community in the form of stem cell transplantation for many years, and CAR T-cell therapy is the latest and most personalized cellular therapy ever designed.”
In addition to offering Kymriah, Children’s Hospital is an active site for a clinical trial of another CAR T-cell therapy for leukemia.
Marriott International, the longest standing corporate partner of Children’s Miracle Network Hospitals®, has committed $300,000 to pediatric cancer initiatives at Vanderbilt. In doing so, Marriott joins other community partners who are supporting CAR T-cell therapy.
“Marriott International is delighted to support this unique and innovative therapy at Children’s Hospital,” says Nicole Randazzo-Cipriani, president of Marriott’s Nashville Business Council. “We look forward to the advances that Vanderbilt physicians will continue making to improve the treatment and survival of children with cancer.”
– by Leigh MacMillan