A new therapy to cure leukemia approved in the United States

Pharma giant Novartis on Wednesday won highly anticipated United States approval for the first of a new type of potent gene-modifying immunotherapy for leukemia, an exhorbitantly priced treatment that nevertheless marks the start of a potential new treatment approach for some cancers. CAR-T cell therapy is a completely different type of method to strengthen the immune system as compared to the popular immunotherapy drugs called "checkpoint inhibitors" which treat many types of cancers by enabling the natural T cells to spot tumors more efficiently.

Novartis and other companies have been racing to develop gene therapies for other types of cancers, and experts expect more approvals in the near future. In the pivotal Novartis trial, 83 percent of patients achieved remission with a disease that has historically poor outcomes. The new cells would then continue to multiply to fight disease for months or years.

The Kymriah is intended for children and young adults up to 25 who have resisted other therapies against this leukemia or relapse, which occurs in 15% to 20% of cases.

CAR-T therapy was pioneered by Dr. Carl June at the University of Pennsylvania, who has been working on the treatment for nearly three decades.

But for those it does help, the new treatment, called Kymriah, comes with a giant price tag: $475,000 per treatment.

He added: "We believe this is just the first of what will soon be many new immunotherapy-based treatments for a variety of cancers".

Epstein, the former CEO of Novartis' pharmaceuticals division, left in 2016 and now serves as executive chairman at Rubius Therapeutics, a biotech that's also working with cell therapy to develop treatments like Kymriah that don't have to be as personalized.

Over the last few years, CAR-T cell research has exploded, and there are now nearly 300 clinical trials underway experimenting with the treatment.


As a result, the FDA is requiring strong warnings.

The approval was widely expected after an FDA advisory panel last month unanimously recommended the action.

The safety and effectiveness of the Kymriah have been demonstrated in a clinical trial with 63 patients pediatric and young adults who had not responded to other treatments or had relapsed. It was identified in previous reports as CAR-T cell therapy, CTL019 or tisagenlecleucel.

The approval of tisagenlecleucel was based on phase II results from the single-arm, worldwide ELIANA trial of 63 patients who received a single dose of tisagenlecleucel.

The therapy does have side effect for which the FDA has created a treatment.

The price of the therapy is tagged at around $500,000. The cost of the treatment?

The Novartis-Penn Center for Advanced Cellular Therapeutics (CACT) opened in 2016 and hosted Vice President Joe Biden at the launch of his Cancer Moonshot initiative, cementing Penn's role as an worldwide innovator in the development and manufacturing of personalized cellular therapies. Other CAR trials at Penn are exploring the technique for prostate cancer, melanoma, and triple-negative breast cancer.


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