Gene Therapy Cancer Treatment Given Federal Approval

Kymriah's maker, Novartis, hasn't provided a price for the drug.

According to this technique, a treatment is created for each patient whose immune cells are collected and frozen before being sent in a lab where they are genetically engineered to attack leukemia. "It's shown a massive response rate in people with these cancers".

Kymriah works by training a patient's cells to recognize the cancer and attack it, CNN reported. It is extremely pleasing that the patient's own cells can be reprogrammed to destroy cancer.

While that is the only time the treatment's given, it's not the end of the road.

Kymriah is expected to be available through a network of certified treatment centers throughout the United States. It carries a boxed warning for cytokine release syndrome (CRS), which is a systemic response to the activation and proliferation of auto T-cells causing high fever and flu-like symptoms, and for neurological events.

Gottlieb said the FDA approval was expedited, coming just seven months after the agency received the initial application.

This side-effect can be controlled with drugs.

According to studies, about 83 percent of patients responded to CAR-T cell therapy went into remission after three months. But while the treatment is complex, it offers huge rewards.

"Of course, we have to talk about pricing from a national standpoint", he said. "It's a great accomplishment".

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"Patients who get this therapy basically have no chance to survive", he said. The patient was nearing death, but has now been cancer-free for five years.

Cancer cells are seen on a large screen connected to a microscope at the CeBit computer fair in Hanover, Germany, March, 6, 2012.

For more on childhood acute lymphoblastic leukemia, visit the U.S. Kite Pharma's CAR-T drug Axi-Cel is now under priority review by the FDA with results to come out in November. Kite's decision date at the FDA in refractory aggressive non-Hodgkin lymphoma is set for November. "The established path to commercialization taken by Novartis will provide considerable motivation for cell therapy scientists and development companies as well as investors looking to benefit from the ongoing success in the industry".

The high cost of the therapy reflects the fact that the CAR-T procedure is fairly intensive.

Epstein said he envisions cell therapies having much shorter life cycles than traditional drugs. The reason is that the therapy requires individual manufacturing of the therapy using the patient's own T cells.

Those updates could potentially expand the number of patients cell therapies are able to treat.

Devoe also said that while the positive results are exciting, many questions remain about how long these novel cancer treatments - developed mainly in the past decade or so - will last. Other CAR trials at Penn are exploring the technique for prostate cancer, melanoma, and triple-negative breast cancer.

"CAR T-cells are not worse than these".

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.

  • Ismael Montgomery