USA approves first gene therapy treatment

Joy Montgomery
September 1, 2017

US regulators approved a revolutionary new treatment from Novartis that modifies the body's own immune-system cells and reinjects them to treat, and in many cases cure, patients with deadly blood cancers who have run out of other options.

FDA commissioner Scott Gottlieb said the approval ushers in "a new frontier in medical innovation", adding: "New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses". But while the treatment is complex, it offers huge rewards. "It's a great accomplishment".

Those early-stage clinical trials, in which more than 90 percent of patients achieved a complete remission one month after receiving the therapy, led to a global registration trial in 2015, involving 68 children and young adults with advanced ALL treated at 25 centers across the world. "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".

Cancer cells are seen on a large screen connected to a microscope at the CeBit computer fair in Hanover, Germany, March, 6, 2012. Kite Pharma, Juno Therapeutics, and Bluebird Bio are among a growing group of biotech companies working with CAR-T.

"This therapy is a significant step forward in individualised cancer treatment that may have a tremendous impact on patients' lives", said Dr Carl June, the Richard W. Vague Professor of Immunotherapy, director of the Center for Cellular Immunotherapies in Penn's Perelman School of Medicine.

Novartis's new $475000 cancer drug among most expensive ever
However, Novartis also reported that tisagenlecleucel will cost nothing if patients do not benefit within 1 month of treatment. For one, the treatments will have to treat more types of cancer than the one approved on Wednesday.

Epstein left Novartis in 2016 as CEO of its pharmaceuticals divisions.

"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", president of Patients for Affordable Drugs, David Mitchell, told The Guardian. The hope is that one day doctors might be able to prescribe a cell therapy and use it that same day, instead of waiting weeks to get the therapy back. About 3100 people under age 21 are diagnosed with ALL each year in the United States, though most respond to standard therapy. This allows the cells to replicate quickly and zero in on cancer cells, fighting the disease for years. The first patients could be treated within days.

Shares of Gilead Sciences Inc, which this week announced an $11.9 billion deal to buy Kite Pharma, were up 6.2 percent to $80.47 on Wednesday afternoon, as Kite is widely expected to receive the next US approval of a CAR-T therapy for a different blood cancer. They'll also have to be less toxic, so more people can consider it instead of only those who have very few other options. "The lessons learned from the clinical trials of auto T-cell operations will assist us in being able to provide to the larger population".

Over the last few years, CAR-T cell research has exploded, and there are now nearly 300 clinical trials underway experimenting with the treatment.

In clinical trials, CAR-T therapies have shown remarkable efficacy against blood cancers. 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.

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