FDA approves first genetically modified 'living drug' for cancer treatment

Emily Whitehead is seen in May five years after she became the first pediatric patient in the world to receive an experimental therapy at Children's Hospital of Philadelphia that has put her leukemia into long-term remission

"This is the first-ever true gene therapy treatment made available to the U.S. population and will help accelerate the speed at which we will see even more gene-based therapies come to fruition".

A rapidly emerging immunotherapy approach is called adoptive cell transfer (ACT): collecting and using patients' own immune cells to treat their cancer.

A brand new way of treating leukemia is on its way to the US market, and it could change how doctors fight cancer.

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.

What is proposed in the Kymriah, this is a single treatment, with a remission rate of 83% in the three months following treatment.

The new therapy, which would cost just under €400,000, turns a patient's cells into a "living drug" and trains them to recognise and attack the disease. The illness is the most common cancer for children in the USA with the National Cancer Institute estimating around 3,100 patients less than 20 years of age diagnosed each year. Novartis said it hopes to have an initial network of 20 treatment centers within a month with plans to expand that to 32 by the end of the year.

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Doctors remove T cells from each cancer victim and ship the material to its factory in Morris Plains, New Jersey. The genetic changes wrought through the customized process creates coding for a protein known as a chimeric antigen receptor, or vehicle. Penn and Novartis are also investigating the next generation of auto therapies for multiple myeloma, and for solid tumors, through trials in glioblastoma, mesothelioma, and ovarian and pancreatic cancer. Along with the Kymriah approval, the FDA also expanded the approval of a drug named Actemra (tocilizumab) used to offset the side effects like these, related to a cytokine release syndrome.

In CAR-T cell treatment the Patients own blood cells or immune cells are filtered out and sent to lab where they get reprogrammed into a CAR (chimeric antigen receptor) which makes the T cells super active or turbocharge and after that the T cells are again infused into the body so that they could combat with the cancerous cells more efficiently. Indication-specific pricing is an approach pharmacy benefits manager Express Scripts has been pushing for other cancer drugs. According to him, a price of $475,000 per treatment was "excessive".

Despite the high price tag, it's worth remembering that other leukemia treatments, like bone marrow transplants, can be even more expensive.

For now, Kymriah is only available to a small subsection of cancer patients.

Other side effects include serious infections, low blood pressure (hypotension), acute kidney injury, fever, and decreased oxygen (hypoxia). Novartis CEO Joseph Jimenez, however, says developing the treatment cost more than $1 billion. In a pivotal trial, one-quarter of patients who responded initially later saw their disease progress.

"We're at the very beginning of what's going to be a big new field of medicine", David Epstein, who helped license Kymriah in from the University of Pennsylvania while at Novartis.

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