FDA announces first U.S. gene therapy approval for cancer treatment

Novartis has developed the ground-breaking leukaemia therapy

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

The Food and Drug Administration on Wednesday approved the first-ever gene therapy in the US.

Dr. David Maloney of the Fred Hutchinson Cancer Research Center being greeted by Ken Shefveland, whose lymphoma was successfully treated with CAR-T cell therapy.

Novartis, the Swiss pharmaceutical company who helped develop the treatment, announced they are "so proud to be part of this historic moment in cancer treatment".

Then, the cells are taken to Novartis's manufacturing facility in New Jersey, at which point the cells are reengineered to recognize cancer cells and wipe them out.

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. The new treatment can act as a lifesaver for those patients who did not recover through first line drug treatments.

The one-time treatment isn't cheap: Novartis, which makes Kymriah, on Wednesday said the price would be $475,000, which is lower than many expected.

The therapy does have side effect for which the FDA has created a treatment. Auto T-cell therapies can damage healthy immune cells, including the cells that produce the antibodies that fight disease. "There are also over 800 cell therapy clinical trials now underway, and a considerable pool of research and pre-clinical work right across the cell therapy sector", said Bruce Levine, PhD, ISCT Commercialization and Immuno & Gene Therapy Committees, Barbara and Edward Netter Professor in Cancer Gene Therapy, University of Pennsylvania Perelman School of Medicine. "Emily's cancer remains in remission, and in larger trials, we're seeing overall remission rates over 80 percent, which is a remarkable improvement upon previous treatment success rates", said lead investigator of the CHOP and global trials of the therapy, Stephan Grupp, MD, PhD, the Yetta Deitch Novotny Professor of Pediatrics at Penn and director of the Cancer Immunotherapy Frontier Program and chief of the section of Cell Therapy and Transplant at CHOP.

Until recently, the use of auto T-cell therapy has been restricted to small clinical trials, largely in patients with advanced blood cancers. The organisation forecasts that considerable investment will be made in the cell therapy field, particularly as there are other companies in the process of submitting applications to regulatory bodies for similar therapies.

Most patients with ALL recover through other treatments such as radiation, chemotherapy and stem cells. 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. Instead of getting an updated therapy that works better on a disease every decade or so, we might begin to see second-generation cell therapies in just a few years. Penn and Novartis are also investigating the next generation of vehicle therapies for multiple myeloma, and for solid tumors, through trials in glioblastoma, mesothelioma, and ovarian and pancreatic cancer.

Other drugmakers have been developing Car-T products, including Kite Pharma, which was snapped up for $11.9 billionby Gilead Sciences earlier this week.

With the treatments available now, fewer than 10 percent of patients with the relapsed, hard-to-treat leukemia are alive five years after diagnosis.