FDA approves breakthrough treatment for dire cancers

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Image credit: Mskcc.org " This is a brand new way of treating cancer", Dr. Stephen Grupp of Children's Hospital of Philadelphia, who treated the first kid with CAR-T cell therapy, told ABC News.

The treatment, called Kymriah, was approved for patients up to 25 years of age who have relapsed or not helped by prior treatment for B-cell acute lymphoblastic leukemia (ALL).

"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer", FDA Commissioner Scott Gottlieb said in a news release following the approval.

To better ensure patient safety, the FDA is requiring Novartis to offer CAR-T therapy only through medical centers specially trained and certified to handle the complicated treatment.

The treatment is not a pill or a form of chemotherapy.

In 2012, Novartis and Penn launched their global collaboration to further research, develop, and commercialize vehicle T-cell therapies, including Kymriah, for the investigational treatment of cancers. It progresses quickly, and is the most common childhood cancer in the United States, affecting an estimated 3,100 patients aged 20 years and younger annually.

Novartis Pharmaceuticals announced the price for its one-time infusion would be $475,000, but noted there would be no charge for patients who didn't show a response within a month of receiving the treatment.

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Kymriah treatment also has the possibility of serious side effects, including cytokine release syndrome (CRS), which consists of high fever and flu-like symptoms and can be life-threatening, and neurological events, which are also life-threatening.

Eleven patients who had CR or CRi relapsed after tisagenlecleucel prior to data cutoff and before any new cancer therapy.

The FDA is including a boxed warning for CRS and, in an unusual move, also approved a new indication for the rheumatologic drug tocilizumab (Actemra) as a treatment for CRS.

But, Novartis is supposedly working with Medicare-Medicaid on a unique plan where reimbursement would only be made if patients responded to treatment within one month. Firms developing CAR-T therapies say their expense is justified because they replace management of otherwise chronic or fatal conditions. But already scientists are studying whether vehicle T cells can be effective in other blood cancers and lymphomas, as well as in solid cancers like breast, prostate, ovarian and lung cancers.

The company did say that it's made an agreement with the USA government to pay for the drug only when pediatric or young adult patients with the cancer respond to treatment by the end of their first month. Also called Kymriah, the treatment isn't actually a drug in the traditional sense, but a collection of the patient's own cells that have been removed from the body and genetically engineered to recognize and destroy cancer cells circulating in the blood.

Manufacturing and delivery are more complex in CAR-T therapies than for a typical drug.

Novartis representatives said they calculated a "cost-effective price" for the therapy that fell between $600,000 and $750,000.

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