First Gene Therapy For Leukemia Approved By FDA

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

The FDA has just approved Kymriah, a new form of genetically modified T-cell immunotherapy for treating young patients with a type of blood and bone marrow cancer.

"Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease", Dr. Peter Marks, director of the FDA's Center for Biologics Evaluation and Research, said in a statement.

"We delivered engineered T-cell therapy at CHOP for the first pediatric patient in the world, Emily Whitehead, who was only six years old when her leukemia stopped responding to conventional treatments". The customized treatment to be administered at certified medical centers involves drawing a patient's blood, genetically engineering their white blood cells, called T cells, turning them into a "living drug" that fights the disease, before re-dripping them back into the patient.

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An advocacy group called Patients for Affordable Drugs recently met with officials at Novartis to ask it to set a "fair" price for the drug, whose early development was supported by $200 million in federal research grants.

The drug's maker, Novartis, also pledged that it wouldn't charge any patient who didn't respond to the treatment after a month.

There are other severe side effects that can pop up as well that can require hospitalization. Clinical trials of patients treated with CAR-T cells found that 69% had complete resolution of CRS within 2 weeks of one or two doses of tocilizumab. In a key clinical trial of 63 people, 83 per cent of participants were cancer-free after three months.

Along with the excitement surrounding CAR-T cells, which are being developed by several companies, there are worries about pricing-the Novartis drug will cost $475,000, although that is reportedly less than expected. This therapy - known as chimeric antigen receptor T-cell therapy, or CAR-T - gives the cells the ability to recognize and kill the source of the cancer. It carries a boxed warning for cytokine release syndrome (CRS), which is a systemic response to the activation and proliferation of vehicle T-cells causing high fever and flu-like symptoms, and for neurological events. Novartis also considered the cost of bone-marrow transplants, which are now given to many leukemia patients whose cancer relapses.

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"Patients who get this therapy basically have no chance to survive", he said.

The treatment will only be used once and is made custom for each patient.

"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.

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 USA approval of a CAR-T therapy for a different blood cancer.

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There could be side effects. 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. And past year, another company, Juno Therapeutics, said five people in its clinical trials had died, all from cerebral edema.

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