Pioneering Cancer Gene Therapy by Novartis Backed by US Panel

Today a Food and Drug Administration (FDA) panel unanimously recommended that the agency approve a treatment that genetically alters a patient's own cells to fight leukemia.

Novartis AG is now competing with Kite Pharma a developing medical field known as CAR-T, which involves immune cells being taken out and genetically modified to destroy cancer before it returns to the body, according to Bloomberg. "We believe when this treatment is approved, it will save thousands of children's lives around the world". After the modified cells have had time to multiply, they are injected back into the patient where the receptors are free to target and destroy the cancer cells.

Explaining their vote, many advisors were effusive. Timothy Cripe, an oncologist at Nationwide Children's Hospital in Columbus, Ohio, called it one of the most exciting things he has seen in his lifetime.

The treatment does carry several short-term side effects, which include fever and hallucinations. Cancer cells arise from normal cells, so the immune system doesn't always recognize that anything is wrong. While patients on this engineered auto cell therapy have been repeatedly hit by potentially lethal cases of cytokine release syndrome and neurological toxicity with the threat of new malignancies, advisers were clearly willing to accept the risk in order to achieve the potential gains.

Twelve-year-old Emily Whitehead is the success story. This would be a huge treatment option for these children and young adults with B-cell ALL.

"Although this therapy is technologically somewhat complicated and is associated with certain serious side effects, it, indeed, has been proven to be amazingly effective", said Kanti Rai, chief of the chronic lymphocytic leukemia research and treatment program at Northwell Health Cancer Institute in NY. "You have to be a long-term survivor to experience [long-term] toxicity", said Bruce Roth of the Washington University School of Medicine in St. Louis, MO. "Our children deserve this chance", she 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.

But despite the financial promise, the commercial barriers are high for developers of CAR-T therapies.

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And Wednesday's victory for Novartis, however incremental, buoyed the whole CAR-T field.

Variations in quality attributes, potency, and transduction efficiency of Novartis' biological cell therapy CTL019, however, did not translate into changed safety or efficacy, according to statistical analyses.

Other biotech and pharmaceutical companies are developing gene therapies, with some treatments approved internationally, AP reported.

Wednesday's FDA advisory panel took no issue with CTL019's positive effects on patients with acute lymphoblastic leukemia that has persisted despite prior treatment.

In 2012, she was dying from one of the most common forms of leukemia called ALL.

The global CAR-T market is estimated to hit $8.5 billion by 2028, according to a report released in February by Coherent Market Insights. Penn has a next-generation CAR-T under development, dubbed CTL-119, which is not licensed to Novartis.

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

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.

  • Ismael Montgomery