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FDA advisory committee recommends Novartis' CAR-T therapy for young leukemia patients

FDA advisory committee recommends Novartis' CAR-T therapy for young leukemia patients

Kite Pharmaceuticals has another CAR-T drug up for FDA priority review for the treatment of lymphoma. It will vote on whether to recommend FDA approval. Approval in pediatric ALL now seems all but assured; the FDA rarely bucks the advice of its advisory committees.

Novartis' experimental product, CTL019, is being recommended for children and young adults aged 3 to 25 who have hard-to-treat (or recurring) forms of the rare blood cancer B-cell acute lymphoblastic leukemia (ALL). The one-time treatment would be for children and young adults with advanced leukemia. "When other organizations, including the NIH, considered gene therapy too risky, ACGT believed in the science and funded us when no one else would", said Dr. June. Approximately 82.5 percent of patients treated achieved remission. He said the treatment is "ushering in a new era". But many of those participants have remained cancer free for months or years.

Explaining their vote, many advisors were effusive.

"This is a major advance", said panel member Dr. Malcolm A. Smith of the National Cancer Institute.

But the therapy poses serious risks.

Most patients in the Novartis study experienced something called cytokine release syndrome, which causes fever and flulike symptoms that can range from mild to extremely severe, said Stephan Grupp, an oncologist at the Children's Hospital of Philadelphia who led the Novartis trial.

But there have been no deaths from CRS in CTL019 trials so far, and the panel was satisfied that expert clinicians administering the therapy would be able to deal with CRS effectively.

More news: Top striker says goodbye to Chelsea squad ahead of summer move

Neurological problems such as seizures and hallucinations were also relatively common but temporary, the Novartis team reported. The scientists also figured out how to embed a "suicide gene" that shuts the cells down if they start to trigger cytokine release syndrome-a unsafe inflammatory response that has raised concerns during CAR-T clinical trials conducted by Novartis and its rivals.

It would also advance a technique scientists have been attempting to ideal for decades and help lift the entire field of cell therapy.

CAR-T therapies also present a gnarly regulatory challenge for the FDA: how do you assure the potency and purity of a complex, living drug that must be made fresh for each patient? First, a process specific to each patient is conducted where millions of immune system T-cells are removed and frozen at an approved medical center. To generate a batch of tisagenlecleucel, white blood cells are purified from a sample of a patient's blood and shipped to a central processing centre.

The FDA previously approved Amgen's T-VEC, which injects a modified herpesvirus into melanoma cells, causing them to rupture.

A year ago, the family was preparing for a bone-marrow transplant when they learned about the cell treatment, which Connor then underwent at Duke University. These include the possible risk that the virus used to engineer the cells could acquire the ability to replicate, or that improper insertion of the foreign gene could turn the T cells cancerous. It was noted that patients infused with T-cells might see a change in the purity of the cells that they are infused with. The long-term side effects of the treatment are unknown.

There was also some questioning related to the chances of the viral vector replicating itself in patients, although this has not been seen to date in any of Novartis' clinical trials.

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