The U.S. Food and Drug Administration has approved the use of a new gene therapy, Kymriah, also known as CTL109 or tisagenlecleucel, for one of the most common and lethal forms of cancer in children and young adults -- acute lymphoblastic leukemia, or ALL. OHSU Doernbecher Children’s Hospital, in Portland, Oregon, is the first hospital in the Pacific Northwest – and only one of a handful of certified treatment centers in the nation - to offer this urgently needed therapy.
“It is not uncommon for ALL patients to undergo multiple treatments, including bone marrow transplant, chemotherapy or radiation throughout their medical journey,” said Eneida Nemecek, M.D., M.S., M.B.A., director of the pediatric bone marrow transplantation program at OHSU Doernbecher Children’s Hospital. “Unfortunately, these options are not effective for all patients, and nearly 20 percent of the 3,500 pediatric and adolescent patients diagnosed with ALL every year in the United States relapse or don’t respond to these conventional treatments.”
Kymriah, the first gene transfer therapy available in the U.S., is manufactured and marketed by Novartis. It uses chimeric antigen receptor (CAR) T-cell therapy, a form of immunotherapy in which the patient’s own blood cells are collected, genetically engineered to attack B-cell leukemia cells, then infused back into the patient.
In a recent clinical trial, a single dose of Kymriah resulted in 83 percent of participants becoming cancer-free after three months. This innovative therapy is now approved for the treatment of children, adolescents and young adults up to age 25 with B-cell precursor ALL that is refractory to treatment or in second or later relapse.
Nemecek, a clinical researcher with the OHSU Knight Cancer Institute and an endowed professor of pediatrics and medical oncology in the OHSU School of Medicine, was one of the principal investigators for the CTL109 clinical trial that lead to approval of the drug.
“The results of the phase 2 CTL109 studies were incredible, with many patients achieving sustained remission of their leukemia. The FDA approval of this novel therapy will help usher in a new era of personalized immunotherapy treatment for patients, allowing OHSU to expand its already successful pediatric and adolescent cancer care to patients in need, across our region and beyond.”