Gene therapy to kill cancer moves a step closer to market

Gene therapy to kill cancer moves a step closer to market

Gene therapy to kill cancer moves a step closer to market

The vote was heralded by the Alliance for Regenerative Medicine as a major milestone.

Aiman Shalabi, chief medical officer of the Cancer Research Institute, viewed the positive tone of the meeting and unanimous recommendation as a similarly momentum-generating event for CAR-T and immunotherapy more broadly. The production process is complex, and shortages can develop.

The next step is up to the FDA. Ultimately, it is up to the FDA to decide if it should approve the treatment or not.

This advisory committee hearing was the last major regulatory milestone before the agency decides in September whether or not to approve the treatment, which would make Novartis' CAR-T therapy the first-ever gene therapy treatment approved by the FDA in USA markets.

ODAC members were convinced that Novartis, of Basel, Switzerland, was peddling something more than hope for a disease that has few options.

In 2016, a Novartis exec told MM&M that the company plans to use a medical device-like sales model to market its CAR-T cell technology, which would rely more heavily on education for physicians than on detailing.

Twelve-year-old Emily Whitehead is the success story. The treatment nearly killed her, but she recovered and today is cancer free. Last month, updated results from the phase 2 ELIANA trial showed that 83% of pediatric patients achieved complete remission (CR) or CR with incomplete blood count recovery within the first 3 months of treatment.

According to the Times, severe, nearly lethal, side effects emerged in Whitehead when she was treated with CTL019 at age 6, but she survived and has remained cancer-free since.

Not everyone at the meeting was sold on Novartis' data. If approved by the FDA, the drug will be a new avenue for some leukemia patients whose first-line drugs have failed.

But the M.D. Anderson approach did present one big challenge: NK cells don't persist in the body for more than two or three weeks, limiting their potential utility as cancer treatments.

All Novartis requires, in order to start selling the treatment, is the stamp of approval from the FDA, which it is most likely to get. Kite Pharma also has a CAR-T therapy under FDA review and Juno Therapeutics and others are in late stages of testing. Any lifting of the reservations on the long-term side effects of such support. "As an investor I've never seen anything like it". 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.

Next, Novartis plans on making additional filings for the drug in the United States and European Union later this year. Eventually, it will expand to other centers.

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

Though the drug carries some serious side effects, such as interfering with production of antibodies, the experts considered that its benefits outweigh the risks.

Novartis hasn't seen any examples of deaths caused by cerebral oedema, and the panel was happy with its strategy to reduce the risk of CRS, which includes making the drug available through a limited network of specialist clinical centres, and focused more intently on how the company ensures the CAR-T cells it administers to patients are free of other cell types that might be transformed by its viral vector.

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