US Approves First Gene Therapy for Young Leukemia Patients

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It is the first gene therapy to be approved by the FDA.

Today, the Food and Drug Administration approved a gene therapy known as auto T-cell therapy that genetically modifies a patient's own cells to help them combat pediatric acute lymphoblastic leukemia (ALL), the most common childhood cancer. The historic action makes the first gene therapy available in the United States.

The price may be high for auto T-cell therapy, but it is important to weigh the costs against the potential benefits for patients, Gwen Nichols, MD, chief medical officer for the Leukemia & Lymphoma Society, said in an interview with OncLive. The treatment was originally developed by researchers at the University of Pennsylvania and licensed to Novartis. 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. Mitchell said to The Guardian that he and his team met with Novartis the day before the drug's approval and price were to be announced, and he claims that the drug company "spent most of the meeting explaining why it needs to charge an astronomical price".

The treatment, called Kymriah, aims to give some patients a second chance after first-line drugs have failed. "Not only does [tisagenlecleucel] provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials".

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Novartis will create a registry to follow patients for 15 years after being treated to monitor their progress and any potential, future side effects.

Each dose of Kymriah contains a patient's own immune cells, which are sent to a lab to be genetically modified using a virus.

Epstein, the former CEO of Novartis' pharmaceuticals division, left in 2016 and now serves as executive chairman at Rubius Therapeutics, a biotech that's also working with cell therapy to develop treatments like Kymriah that don't have to be as personalized.

Novartis has received approval for its chimeric antigen receptor T cell (CAR-T) therapy, Kymriah (tisagenlecleucel) suspension for intravenous infusion, formerly CTL019, from the US Food and Drug Administration (FDA).

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Many patients in the ALL trials experienced a side effect called cytokine release syndrome (CRS) including grade 3 or grade 4, which includes varying degrees of flu-like symptoms, with high fevers, nausea, and muscle pain, and temporary neurologic symptoms, including delirium, but also low blood pressure and breathing difficulties requiring ICU-level care in the most severe cases.

The treatment will only be used once and is made custom for each patient. Eventually, the therapy will be offered at 32 sites, the company said.

Epstein said he envisions cell therapies having much shorter life cycles than traditional drugs. This allows the cells to replicate quickly and zero in on cancer cells, fighting the disease for years.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines, and other biological products for human use, and medical devices. And past year, Juno said five people in its clinical trials had died, all from cerebral edema.

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