The panel recommended that the Food and Drug Administration approve the drug, tisagenlecleucel, for patients ages 3 to 25 with relapsed B-cell acute lymphoblastic leukaemia (ALL), one of the most common form of childhood cancer.
A novel cell treatment that saved the life of 9-year-old Austin Schuetz was given the green light by U.S. regulatory advisers on Wednesday and doctors hope it can save the lives of more children with the most common type of childhood cancer.
The CART-T therapy developed by Novartis involves extracting special immune system cells from the patient, then reprogramming them.
Dr. Grupp and the Whitehead family were among those testifying before the FDA advisory panel, hoping to get the treatment approved. Speaking during the public hearing, Megan Polanin, a senior fellow at the National Center for Health Research who specializes in evidence-based mental health interventions, urged the FDA to take it slow in approving the vehicle T therapy. It's a process where white blood cells are extracted from the patient's body, then genetically programmed to find and destroy cancer cells, before being put back into the patient's body. If CTL019 is approved, the Swiss pharmaceutical giant plans to dole it out from about 30 preapproved sites, each trained in the multi-step process of harvesting cells, handling the product, and treating patients for the feverish and often life-threatening immune response that usually accompanies CAR-T therapy. The vote comes as the agency considers whether to issue its first approval of a CAR-T therapy - a drug called tisagenlecleucel that's manufactured by Novartis of Basel, Switzerland. The deal covers Celyad's rights to U.S. Patent No. 9,181,527 related to allogeneic human primary T cells that are engineered to be T-cell receptor deficient and express a auto.
But the overall effectiveness of the drug and the lack of other options seem to have won the committee over: About 89% of the patients in the small trial survived at least six months, and 79% survived at least a year. "While I have some concerns about late toxicity, you have to be a long-term survivor to experience late toxicity, and I think that's what this drug gives us".
Each patient would have a copy of the drug suiting their body, their cells used in the manufacturing of their own drug.
Side effects, however, can be severe, and include spiking fever and crashing blood pressure.
Austin Schuetz, one of a handful of CAR-T patients to achieve multi-year remission of his cancer, requires biweekly infusions of immunoglobulin to replace B-cells wiped out by the engineered cancer-killing T-cells. The cells would be altered to seek out the cancer and attack it.
The complication doesn't apply universally across all CAR-T products, and newer generations now in development appear able to avoid the permanent destruction of B cells. The company hasn't disclosed how many patients in its trials needed immunoglobulin after treatment. Echoing comments parents and a patient advocate made during the public hearing, the panel's patient representative, Gianna McMillan, said the education and monitoring results should be extended to the patient's caregiver, who should be seen as part of the health care team.