FDA Advisory Committee Unanimously Votes to approve Drug Saving Terminal Pediatric Leukemia Patients

The Federal Drug Administration (FDA) advisory committee unanimously voted to recommend a Chimeric Antigen Receptor T-cell (CAR-T) therapy for approval to treat relapsed or refractory (r/r) B-cell acute lymphoblastic leukemia (ALL) in terminally ill young adults and pediatric patients.

Patients that received this miraculous treatment did so because there were limited options for those diagnosed with ALL and the prognosis was poor. Results from this therapy, published in June, showed that 83% Complete Remission (CR) rate in 72 patients. It was after reviewing data collected in June that the FDA unanimously recommended to approve Novartis’ CAR-T therapy, CTL019.

“The results, including relapse-free survival findings at six and 12 months, reaffirm our confidence in CTL019 to potentially become an effective treatment for pediatric and young adult patients with r/r ALL in need of more options.” Said Vas Narasimhan, MD, Global Head of Drug Development and Chief Medical Officer, Novartis

This therapy will have an immediate impact on those suffering from ALL, a disease that accounts for 25% of cancer diagnoses among children under 15 years old and is the most common childhood cancer in the US. Data evaluating 63 patients over the course of 6 months demonstrated relapse-free outcomes in the majority receiving the CAR-T therapy.

Though the results are promising, the treatment doesn’t come without risks. Cytokine Release Syndrome (CRS), a common side effect associated with CAR-T therapies, has resulted in deaths of patients in other trials. CRS is a known complication in the investigational CAR-T therapies that may occur when the engineered T cells in the body are activated. In this trial 47% of patients experienced grade 3 or 4 CRS, though no deaths were observed.

The future of CAR-T therapy to combat cancer is promising. Novartis has several ongoing clinical trials to treat other leukemias and Kite Pharmaceuticals released promising clinical trial results in April to treat patients with Non-Hodgkin’s Lymphoma. Scientists are hoping to apply CAR-T therapy in the future to treat solid tumors like lung cancer. Learn the details of CAR-T.

Cover photo: T-cell-red-NIAID


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