USA advisory panel endorses 'breakthrough' gene therapy for child cancer

Novartis dodged deaths from its CAR-T Therapy, but FDA still has safety concerns

An advisory committee to the U.S. Food and Drug Administration voted unanimously to recommend approval of Novartis AG's tisagenlecleucel for treating B-cell acute lymphoblastic leukemia (ALL) in children and young adults who relapsed or failed chemotherapy.

These reprogrammed T-cells are then frozen again at the Novartis plant and sent back to the patient's treatment center, where they are reintroduced via IV drip back into the patient. The panel recommended patients who receive the treatment be monitored for 15 years. "I hope that someday all of you on the advisory committee can tell your families for generations that you were part of the process that ended the use of toxic treatments like chemotherapy and radiation as standard treatment, and turned blood cancers into a treatable disease that even after relapse most people survive".

The treatment does carry several short-term side effects, which include fever and hallucinations.

Novartis said the entire process would take 22 days by the time it is launched.

A single one can destroy up to 100,000 cancer cells, the New York Times reported.

"The majority of calls we are getting are from patients looking for immunotherapies - particularly CAR-T therapies", said Dr. Gwen Nichols, chief medical officer at the Leukemia and Lymphoma Society.

"We believe that when this treatment is approved it will save thousands of children's lives around the world", said Tom Whitehead, the father of a 12-year-old who is now cancer-free after receiving CTL019.

In a unanimous vote on 12 July, the panel determined that the benefits of the treatment, called CAR-T therapy, outweigh its risks. CTL-019 should give kids with ALL a chance for long-term survival, he said.

In May, Novartis signaled its interest in also developing allogeneic auto T-cell therapies, when it entered a nonexclusive license agreement with Celyad to use its USA patents to produce allogeneic vehicle T cells, in a deal that could generate up to $96 million in up-front and milestone payments for the Belgian biotech. An approval decision for the Kite CAR-T is expected on November 29.

"In those settings, the downstream effect [of triggering a secondary cancer] is conceivable", said Maziarz. Shares in the Swiss pharma, higher throughout the meeting, closed roughly 1.5% above Wednesday's open.

Many studies and trials have been conducted on the living drug, most of which have yielded positive results.

Contrary to expectations, the ODAC deliberations focused on relatively minor concerns about the manufacturing of CTL019 and the need to keep tabs on the data with the therapy after approval, and did not dwell for too long on the safety of the approach. The FDA is not required to follow the recommendations of its advisers but typically does so. Beyond that, the company's plans with CAR-T are unclear. Another risk is that there are many other players that are developing treatments in the CAR-T space as well.

Novartis also noted the death of a patient from a cerebral hemorrhage.

On the safety side, the panelists delved into the possible risks of injecting genetically modified cells into patients, including the potential for deadly viral infections, brain toxicity, and, paradoxically, the growth of new tumors brought about by CAR-T cells years after treatment.

Such a complex system for making personalized treatments is likely to drive up their cost, and the next big hurdle (assuming an FDA approval this fall) is to win over insurers.

Kite's therapy also hurt the antibody-producing B cells, but they came back.

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