U.S. approves first gene therapy for cancer

Novartis' CAR-T gene therapy, the first approved by FDA, to be priced based on cancer patients' outcomes

In the pivotal Novartis trial, 83 percent of patients achieved remission with a disease that has historically poor outcomes.

The therapy was evaluated in clinical testing involving 63 children and young adults with relapsed or refractory B-cell ALL. The patient's T-cells are collected and sent to a manufacturing center where they are genetically modified to include a new gene that contains a specific protein (a chimeric antigen receptor or CAR) that directs the T-cells to target and kill leukemia cells that have a specific antigen (CD19) on the surface. According to Children's of Alabama, more than 190 children in Alabama are diagnosed with various forms of cancer each year, including ALL.

This first use of CAR-T therapy is aimed at patients desperately ill with acute lymphoblastic leukaemia, which strikes more than 3,000 children and young adults in the United States each year.

Novartis has not yet announced the cost for the therapy, but British health authorities have said a price of $649,000 for a one-time treatment would be justified given the significant benefits, according to a Kaiser Health News report last week. Kymriah is intended for people with B-cell ALL whose cancer hasn't responded to or has returned after treatment, the FDA said.

Juno Therapeutics Inc previous year reported a handful of patient deaths during trials of its CAR-T therapy for leukemia.

It's too soon to tell how widely applicable CAR-T will be - it's especially suited to leukemia since infusing patients with T-cells is relatively simple. In June, a clinical trial in China showed promise in treating another blood cancer, multiple myeloma, using reprogrammed T-cells in much the same way Kymriah does. Kymriah will carry a boxed warning for cytokine release syndrome, a potentially lethal systemic response to the activation and proliferation of CAR-T cells, causing high fever and potential for neurological problems. The drug's maker, Novartis, is required as a condition of approval to conduct a longer-term study of Kymriah's use, the agency said.

Other genetic therapies for cancer are also in the research pipeline.

"Five years ago, we began collaborating with the University of Pennsylvania and invested in further developing and bringing what we believed would be a paradigm-changing immunocellular therapy to cancer patients in dire need", Novartis CEO Joseph Jimenez said in a statement. More than half were children and teens.

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