A pioneering gene therapy designed to treat leukemia has moved one step closer to becoming the first of its kind marketed in the United States, thanks to a crucial recommendation.
On Wednesday, July 12, an advisory panel from the Food and Drug Administration gave its unanimous recommendation to approve the use of tisagenlecleucel, a drug manufactured by Novartis, for the treatment of leukemia patients aged 3 to 25 years old.
More specifically, the Novartis drug is designed to treat patients diagnosed with relapsed B-cell acute lymphoblastic leukemia (ALL), which is the most common childhood cancer in the country.
The gene therapy, also known as CTL019, uses a new technology called chimeric antigen receptor T-cell therapy, or CAR-T for short. This technology involves the removal of immune cells from the patient's blood and then reprogramming them to recognize and attack malignant cells and injecting them back into the patient.
The Novartis CAR-T cancer therapy is recommended for patients with B-cell ALL that has not responded to other treatments or has responded but relapsed.
Dr. John Maris, a pediatric oncologist at The Children's Hospital of Philadelphia, said the CAR-T cancer therapy will no doubt save the lives of children and young adults who have had no other effective treatment for relapsed ALL.
"This is truly a turning point in the management of this disease," said Maris.
A New Era Of Medicines?
Statistics show that patients with ALL who fail chemotherapy typically only have a 16 to 30 percent chance of survival.
But in a clinical trial that included 63 patients with ALL who had relapsed or failed chemotherapy, 83 percent of them reached complete or partial remission three months following the CAR-T treatment. After 12 months, 79 percent of them were still alive.
"This is a potentially paradigm-changing type of benefit," said Dr. Brian Rini of the Cleveland Clinic Taussig Cancer Institute.
Although the FDA is not obliged to follow the recommendations of its advisory panels, it often does so. The ruling on the use of the CAR-T gene therapy will be announced by the end of September.
The approval of the CAR-T gene therapy will revolutionize the treatment of ALL patients who relapsed or failed chemotherapy. Experts say the approval will pave the way for a new era of gene-based medicines, which has shown promise in treating a wide range of diseases.
Meanwhile, the FDA expressed concern over the side effects of the drug. There might be a risk that it could cause the growth of new malignancies over time, but the advisory panel said the risk was low and that the mitigation proposal presented by Novartis were adequate.