Panel recommends gene-altering leukaemia treatment to United States drug agency

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Food and Drug Administration advisers on Wednesday enthusiastically endorsed a first-of-its-kind cancer treatment that uses patients' revved-up immune cells to fight the disease, concluding that the therapy's benefits for desperately ill children far outweigh its potentially risky side effects.

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 dramatic effect of the treatment, known for years as CTL-019, was never questioned at the meeting. In a small group of patients who have received the therapy, 83% were in complete or partial remission three months later.

Many of the FDA's advisers were effusive in their praise.

What are the side effects?

Novartis believes it can turn around a finished CAR-T product 22 days from receipt of leukapheresed material to final packing and shipping. This is in stark contrast to some other CAR-T trials that have, over the past year, reported the death of several patients from severe brain swelling. Children with cancer are closely monitored for years in clinical practice, said Timothy Cripe, chief of the hematology/oncology division at the Nationwide Children's Hospital. Novartis plans further filings for CTL019 in the USA and EU later this year, including applications with the FDA and European Medicines Agency (EMA) for the treatment of adults with r/r diffuse large B-cell lymphoma (DLBCL).

The committee members said it was hard to assess the long-term risk, but in patients with no other treatment options, the near-term benefits of CAR-T therapy more than tipped the scales. But the young patients who would receive it have few alternatives, said Bruce Roth, an oncologist at Washington University School of Medicine in St. Louis, Missouri, and those alternatives carry risks of their own.

How does CAR-T therapy work?

Studies have shown that CAR-T therapies can produce lasting remissions in such cases. "This is an entirely new way of treating cancer and I think these cellular therapies are only in their first chapter".

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

By modifying immune cell DNA, this method could, in theory, lead to other cancers - a longtime concern for gene therapy.

The FDA is not required to accept the recommendations of its advisory panel, but it usually does.

Novartis is also testing the drug in diffuse large B-cell Lymphoma, the most common form of non-Hodgkin lymphoma, as is Kite.

CAR-Ts can also pose serious risks, including a potentially life-threatening inflammatory condition. Another risk is that there are many other players that are developing treatments in the CAR-T space as well. Once re-engineered they are placed back into the patient, and the T-cells go to work combating against the cancer.

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.

The drug enables patients' own immune cells to recognize and kill the source of the cancer: a different immune cell gone awry.

Auto T-cell therapy, made by Novartis, retrains a patient's immune cells so they target and attack cancer; the cells are removed from the body, sent to Novartis' plant so they can be genetically modified, and then shipped back to the patient for infusion.

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