Novartis Gets FDA Nod For Leukemia Gene Cell Therapy

US clears first 'living drug' for childhood leukaemia

In a key study of 63 advanced patients, 83 percent went into remission.

An FDA advisory committee had recommended the therapy for approval in July to treat the relapse of a blood cancer known as B-cell acute lymphoblastic leukemia, or ALL. The T-cells are collected then sent to a manufacturing center where they are genetically modified before being infused back into the patient. "In clinical trials in patients treated with CAR-T cells", the FDA reported, "69% of patients had complete resolution of CRS within two weeks following one or two doses of Actemra".

"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer", said FDA Commissioner Scott Gottlieb, M.D.in Wednesday's announcement.

"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".

Novartis officials said it is working with 20 hospitals to provide Kymriah within a month.

Numerous pharmaceutical companies are investing in gene-therapy projects.

Novartis is based in Switzerland. British health authorities have said a price of $649,000 for a one-time treatment would be cost-effective given Kymriahs significant benefits.

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"At Novartis, we have a long history of being at the forefront of transformative cancer treatment", said Joseph Jimenez, CEO of Novartis. But he said he doubts their efforts will persuade Novartis to set a reasonably affordable price.

Among concerns, sometimes leukemia can develop resistance, and sometimes patients worsen while waiting for their new cells, said Sekeres, who directs the Cleveland Clinic's leukemia program and wasn't involved with CAR-T testing. Some children whove received Kymriah have already received a transplant.

CAR-T treatment uses gene therapy techniques not to fix disease-causing genes, but to "turbo-charge" T cells, immune system "soldiers" that cancer can often evade.

United States biotech Gilead bought another pharmaceutical firm developing a CAR-T therapy, Kite Pharma, for nearly $12bn earlier this week, further demonstrating the excitement around the fast-emerging field. Novartis already is facing pressure from patient advocates about the expected cost.

Experts have noted that hidden costs could further add to patients financial burdens. CAR-T cell therapy gives patients stronger T cells to do that job. However, there were some severe side effects observed in study. When the body's immune system kicks into high gear to fight the cancer, extremely high fevers can occur as well as the production of proteins that produce inflammation.

Doctors dont yet know what the full range of long-term side effects will be.

Unlike traditional gene therapy, the new treatment doesn't replace disease-causing genes with healthy ones.

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