Breakthrough gene therapy for cancer approved in US

Endpoints News

Despite these negatives, FDA approval of gene therapy is a big step for the treatment both in the United States of America and around the globe.

Since its founding in 2001, ACGT has allocated approximately $29 million in grants to USA and Canadian cancer gene therapy research projects.

Amazingly, of the 63 patients who received CAR-T therapy, 83 percent were in complete remission within three months. Patients often undergo multiple treatments including chemotherapy, radiation, targeted therapy or stem cell transplant, yet less than 10 percent are alive after five years.

More than 3,000 patients aged 20 or younger are diagnosed with ALL every year, according to the National Cancer Institute.

But the new therapy doesn't come cheap: Developed by Novartis Pharmaceuticals and the University of Pennsylvania, the one-time infusion is priced at a reported $475,000.

The Food and Drug Administration (FDA) has called this a historic moment because it is considered to be the first gene therapy hitting the USA markets.

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

He said the hospital was also developing a different set of CAR-T cells which aim to one day treat people with brain tumours and bone cancers.

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The price per person is likely to be much higher when accounting for additional costs, such as drugs to prepare the patient for the treatment and medicines to control potential side-effects.

Professor Joe Trapani, the executive director of cancer research at the Peter MacCallum Cancer Centre, was involved with the very first CAR-T clinical trial on patients in Australia in 2010.

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

To customize Kymriah for individual patients, white blood cells called T cells will be removed from a patient's bloodstream at an approved medical center, frozen, shipped to Novartis in Morris Plains, N.J., for genetic engineering and multiplying, frozen again and shipped back to the medical center to be dripped into the patient. Of those, about 600 patients whose disease relapses or doesn't respond will be eligible for the Novartis treatment, according to the company. Indication-specific pricing is an approach pharmacy benefits manager Express Scripts has been pushing for other cancer drugs.

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.

While Kymriah's price tag will grab headlines, it pales in comparison with some other gene therapy treatments. Salvage therapies for children with relapsed ALL are highly toxic and only work in about 50 percent of relapsed patients.

Novartis will create a registry to follow patients for 15 years after being treated to monitor their progress and any potential, future side effects. But speaking earlier this year to The New York Times, drug industry analysts estimated that individualized therapies could cost more than $300,000.

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