FDA panel recommends approval for experimental leukemia treatment

Friday, 14 Jul, 2017

"Any time a new technology crosses the finish line at the FDA, it gets noticed and I think you'll see a rush of investment and see these therapies be improved over time", Loncar said.

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.

A panel of cancer experts unanimously endorsed the leukemia treatment on Wednesday in a 10-0 vote, according to AP.

While the panel's recommendation is not binding, the FDA usually follows the advice of its expert advisory committees.

Scientists are calling the treatment "a living drug" as it harnesses the immune system to beat cancer, according to The New York Times.

Researchers at the University of Pennsylvania developed the treatment, which is now licensed to Novartis.

CAR-T therapy starts with filtering key immune cells called T cells from a patient's blood. In experiments, they found that engineered NK cells taken from healthy cord blood were more efficient at killing CLL cells than were NK cells that were taken from patients' blood and then engineered.

The complication doesn't apply universally across all CAR-T products, and newer generations now in development appear able to avoid the permanent destruction of B cells. Speaking during the public hearing, Megan Polanin, a senior fellow at the National Center for Health Research who specializes in evidence-based mental health interventions, urged the FDA to take it slow in approving the auto T therapy.

There are quite a few risks with the CTL019 treatment by Novartis.

Novartis's CTL019 is created to treat a severe form of leukemia that usually afflicts children and young adults. Of 68 young people receiving it, 52 of them had an excellent response nearly immediately, with their cancer disappearing within the first three months.

The panel on Wednesday recommended approving the treatment for B-cell acute lymphoblastic leukaemia that has resisted treatment, or relapsed, in children and young adults aged three to 25. That happens to more than 600 patients in the US each year.

In drug trials, the immunotherapy results worked far better than chemotherapy and newer types of cancer drugs. In a phase 2 trial, 82% of patients had complete remissions, but 48% experienced cytokine release syndrome. Most patients suffered serious side effects but almost all recovered.

Novartis has developed a set of protocols to deal with the potential side effects, which include limiting it to 30 to 35 medical centers in the USA where oncology departments receive extensive training with the therapy.

Indians will be able to access the treatment at USA centres, which will also accepting global patients. In light of the drug's side effects, the FDA advisory panel viewed this as a favorable risk-benefit balance. The patient numbers were small, but the responses were extraordinary for the patients who responded, with an 83% overall remission rate and 79% overall survivability rate at 12 months - hitting the primary endpoint.

One attendee of the panel's meeting was the first patient from the trials, Emily Whitehead, age 12.

Now Emily's survival has stretched to five years, cancer-free. As such, the treatment is tailored for each patient. It will likely be available within months for children and young adults with a hard-to-treat blood cancer.