Gene therapy to kill cancer moves a step closer to market

The Novartis therapy involves drawing some blood from the patients genetically modifying a type of immune called T-cells in it and re-infusing it into the patients. These re-programmed cells then target and kill the cancer cells

Elizabeth Smith, SVP of regulatory affairs and quality at Juno Therapeutics, explained the potential for these new treatments in a comment on the Novartis meeting.

The youngest patient to be given the altered cells was 12-year-old Emily Whitehead, who started the treatment aged six.

The treatment would be for children and young adults.

The recommendation of ODAC was based on the evaluation of the CTL019 r/r B-cell ALL development program that included the firm-lead ELIANA study, which is claimed to be the first pediatric global CAR-T cell therapy registration trial.

"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.

Novartis expects to get a decision from the FDA by October.

Only a few thousand people are affected by the B-cell acute lymphoblastic leukemia that proves stubborn in the face of conventional treatments for about 15 percent of cases, majority in children and young adults. That happens to more than 600 patients in the USA each year.

He said in a blog: "With every paper I read, it becomes more and more clear that CAR-T is not a biotech race to be won, but rather a revolutionary therapy to be explored and applied to other cancers". ALL is the most common childhood cancer in the US.


In a test conducted, gene therapy results exceeded chemotherapy, and even newer types of cancer medicines. Approximately 82.5 percent of patients treated achieved remission.

But the drug has side effects that can be fatal, such as cytokine release syndrome or CRS, which "looks like sepsis" and causes blood pressure to drop dangerously low, said Diefenbach. The virus creates a new cell receptor - which is, in Novartis' case, part mouse - that targets another receptor on the cancer cells: CD19.

The treatment destroys not only leukemic B-cells but also healthy ones, which help fight germs, patients then need treatment to protect them from infection. The whole testing process took 16 weeks on average, which FDA advisers noted could be too long for severely ill patients.

"I am amused by the horse-race metaphors that are used to frame the various companies developing CAR-T therapies". Kite Pharma of Santa Monica, Calif., has applied for FDA approval to treat aggressive non-Hodgkin's lymphoma, and a similar Novartis application is close behind. Some estimates have put the treatment costs of CAR-T therapies in the hundreds of thousands of dollars.

The therapy, developed by drug giant Novartis, is recommended for B-cell acute ALL that has either not responded to other treatments, or responded but relapsed.

The treatment's short-term side effects, including fever and hallucinations, are often intense as the body's revved-up immune system goes on the attack. About 85% are cured by chemotherapy, but those whose cancer returns have few options.

Speaking to CNBC, Reni Benjamin, a pharmaceuticals analyst with Raymond James described the field of gene therapy, into which the new treatment falls, as one attracting significant investment and excitement in the medical community. At the University of Texas M.D. Anderson Cancer Center, one team of researchers is turning to umbilical cord blood for inspiration.

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