teknolojiarsivi.com March 19, 2018

Gene therapy on the horizon

14 July 2017, 12:36 | Audrey Hill

FDA Panel Recommends Approval for Gene-Altering Leukemia Treatment

FDA advisers back Novartis' game-changing personalized CAR-T for cancer, approval in sight

If the FDA were to accept the recommendation, which was likely, the treatment would be the first gene therapy ever to reach the market, according to commentators.

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 United States markets. The one-time treatment would be for children and young adults with advanced leukemia.

Despite unknowns surrounding the therapy, "it is hard to argue with the unprecedented clinical success we have seen in this population of patients who do not have other viable treatment options", added Grzegorz S. Nowakowski, MD, of the Mayo Clinic in Rochester, Minn.

Basically, CAR-T therapy involves taking a patient's own immune "killer" T-cells, inserting new genetic code into those cells which turn them into cancer-hunters that can home in on malignant B-cells (another kind of immune cell), and then pumping these specialized leukemia-busting cells back into the patient. The FDA doesn't have to follow advisory panels' recommendations.

The therapy, CTL019, is being tested as a treatment for pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL), which is the cause of one-quarter of cancer diagnoses in the US for children under the age of 15 years old.

The products are made by extracting and isolating a patient's T cells, genetically engineering them to recognize and target specific cancer cells, and then infusing them back into the patient.

The National Cancer Institute's James Gulley said in the panel discussion: "This is a product that has shown patient benefit, and I think our goal here is to try and find ways that we can make sure we have optimal safety and potency". It will vote on whether to recommend FDA approval. The immune cells from the patient's blood are being reprogrammed to focus on cancer cells and completely destroy them without doing any damage to the healthy cells that are around it.

44 % of diagnoses were in children under 15 years of age.

"It's a pretty unbelievable new treatment", Dr. David Agus, director of the USC Norris Westside Cancer Center and CBS News medical contributor, said on "CBS This Morning".

FDA advisers spent half of the 12 July meeting learning about how Novartis has attempted to standardize, to the extent possible, a treatment based on cells taken from individual patients. Success would also help advance a cancer-fighting technique that scientists have been trying to flawless for decades and lift the broader field of cell therapy. He said the treatment is "ushering in a new era".

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.

David Maloney, medical director for cellular immunotherapy at Fred Hutchinson Cancer Research Center in Seattle, said he was elated that the field is moving forward.

Arie Belldegrun, CEO of Novartis' rival Kite Pharma, which is developing a CAR-T therapy that was filed shortly after CTL019, said that during the meeting he would be "Novartis' biggest cheerleader".

Observers crowded into the FDA's presentation room this morning, rubbing shoulders with CAR-T celebs like Penn's Carl June with many, many more looking on online.

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