FDA approval brings first gene therapy to the United States

Ray Weaver
September 1, 2017

Welcoming a new era in cancer treatment, the USA health officials have officially approved a new breakthrough treatment for the patients suffering from leukemia where it genetically transforms the patient's own blood cell into an army of assassins which detects and destroys the childhood leukemia. The therapy, marketed as Kymriah and made by Novartis, was approved for children and young adults for an aggressive type of leukemia - B-cell acute lymphoblastic leukemia....

The drug, called Kymriah, is a highly personalized cancer treatment called auto T-cell therapy (CAR is short for chimeric antigen receptor). The U.S. Food and Drug Administration announced the approval Wednesday, weeks ahead of schedule. The FDA on Wednesday expanded the approval of Actemra, a rheumatoid arthritis drug that can also treat cytokine release syndrome.

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.

However, this type of therapy carries risk of severe side effects.

Swiss pharmaceuticals group Novartis has said it will charge $475,000 (CHF458,000) a patient for its new cancer therapy, putting it among the most expensive drugs of all time.

"We have gotten so comfortable with these numbers that are just beyond belief for these agents", Bach said.

Despite Kymriah's success in the clinical trial, Walid Gellad, a doctor and professor at the University of Pittsburgh, suggested to Axios that treatments with undetermined effectiveness-even if they have the potential to save lives-shouldn't cost more than proven measures such as bone marrow or kidney transplants, adding: "This is an fantastic therapy, but there has to be a limit at which point companies can no longer charge desperate patients, or taxpayers, enormous sums". "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.

David Mitchell, president of Patients For Affordable Drugs, said: "While Novartis's decision to set a price at $475,000 per treatment may be seen by some as restraint, we believe it is excessive".

"The only potentially curative option for these pediatric and young adult patients is allogeneic stem cell transplant, which costs $500,000 to $800,000 for the first year", Cooper said. Instead of getting a better, updated therapy for a disease every decade or so, we might begin to see second-generation cell therapies in a few years. The first patients could be treated within days.

Kymriah is the first CAR-T cell therapy to get approved, and there are several more in the works.

Health officials in the United States have approved a revolutionary treatment that uses genetic engineering to combat childhood leukemia. They'll also have to be less toxic, so more people can consider it instead of only those who have very few other options.

Shares of Bluebird Bio Inc, which is developing a promising CAR-T treatment, were up 11.1 percent at $113.73. The patient was a little girl nearly dying and is now five years cancer free.

And to affect more people, the cell therapies would need to go beyond blood cancers.

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