Published: Fri, September 01, 2017
Medicine | By Daryl Nelson

FDA Approves First 'Living Drug' for Childhood Leukemia

FDA Approves First 'Living Drug' for Childhood Leukemia

Each dose of Kymriah is a customized treatment created using the patient's own T-cells.

An FDA advisory committee had recommended the therapy for approval in July to treat the relapse of a blood cancer known as B-cell acute lymphoblastic leukemia, or ALL.

"We're entering a new frontier in medical innovation", Food and Drug Administration Commissioner Scott Gottlieb said in a statement.

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.

More than 3,000 patients aged 20 or younger are diagnosed with ALL every year, according to the National Cancer Institute. The individualized approach involves harvesting cancer patients' immune cells, genetically engineering them, then returning them to patients' bodies.

Those arguments didn't assuage David Mitchell, founder of Patients for Affordable Drugs, and Jamie Love, director nonprofit Knowledge Ecology International, who believe the drug should be more affordable given that taxpayers funded early research. The engineered T-cells can lead to what is called cytokine release syndrome, a life-threatening condition that is triggered by an overactive immune system.

Those reprogrammed cells are sent back to the patient and administered, at which point the new cells try to kill off the cancer cells.

Dr. Kenneth Anderson is president of the American Society of Hematology, which focuses on blood cancers.

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A spokeswoman for Switzerland-based Novartis declined to say how much the drug will cost, though analysts have estimated a price of $500,000 or more.

Those updates could potentially expand the number of patients cell therapies are able to treat. Right now, that's where most of the big successes have come from, but one day that could expand out to solid tumors, and maybe even auto-immune diseases like type 1 diabetes, Epstein said. "I'm going to predict this technology will spread to other diseases over the next few years".

However, Anderson stressed that "this approval only pertains to a small population of children [with ALL]". The overall remission rate within three months of treatment was 83 percent, the company said.

The arrangement would see Novartis provide the drug free of charge if patients did not respond to treatment within a month.

This of course results in a very expensive drug price.

However, Kymriah comes with the potential for severe side effects. The therapy also can cause neurological events, serious infections, low blood pressure and acute kidney injury.

"The FDA is requiring that hospitals and their associated clinics that dispense Kymriah be specially certified", it added. Novartis say the drug will cost $475,000. Other CAR trials at Penn are exploring the technique for prostate cancer, melanoma, and triple-negative breast cancer. "While payers are developing their coverage policies for [tisagenlecleucel], we will offer an access program in the United States for eligible uninsured or underinsured patients". "We can deliver on the original promise". "The disease progresses quickly and is the most common childhood cancer in the US", the FDA said. "ISCT will continue to drive to support all elements of the sector to make sure this happens". We have a little favor to ask.

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