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New cutting-edge treatment for type of pediatric leukemia offered at Monroe Carell Jr. Children’s Hospital at Vanderbilt 

Sep. 18, 2017, 2:54 PM

Monroe Carell Jr. Children’s Hospital at Vanderbilt is part of a select group of health care institutions recently chosen to offer a new FDA-approved immunotherapy for a subset of pediatric acute lymphoblastic leukemia (ALL).

Only 32 health care 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 pre-B cell ALL that has come back after treatment (relapsed) or unresponsive to treatment (refractory). This therapy, called Kymriah, takes a patient’s own immune cells, white blood cells that normally help fight infection, and reprograms the cells to recognize and destroy the patient’s leukemia cells.

ALL is a cancer of the bone marrow, and is the most common form of pediatric leukemia. About 3,000 young children and adolescents are diagnosed with ALL in the U.S. each year.

“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, M.D., 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.

“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 their ALL,” said Carrie Kitko, M.D., associate professor of Pediatrics and director of the Pediatric Stem Cell Transplant Program in the Division of Hematology/Oncology. “The Vanderbilt Pediatric Stem Cell Transplant Program is ideally suited to care for these patients. Our team has been providing cellular therapy to the Middle Tennessee community in the form of stem cell transplantation and CAR T-cell is the latest and most personalized cellular therapy ever designed.”

The treatment works like this: 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.

Because there are potential severe complications involved with this type of treatment, the FDA is requiring that all treatment sites have special certification acknowledging that the center is able to handle these risks. The most severe and potentially life-threatening side effects include high fevers, low blood pressure and difficulty breathing that may require care in the intensive care unit.

“Vanderbilt is uniquely well-suited to provide this treatment,” Friedman said. “We have the specialized laboratory to collect and process the patient’s T cells, expertise on our team with CAR T-cell therapy from previous clinical trials and exceptional oncology and comprehensive subspecialty services and intensive care facilities, all located within our hospital.”

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