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FDA approves first-of-its-kind 'living drug' for childhood leukemia

FDA approves first-of-its-kind 'living drug' for childhood leukemia

The agency called the decision a "historic action" because the therapy, developed by Novartis, is the first gene therapy treatment approved in the United States.

The first cancer treatment that involves reprogramming a patient's own blood cells to fight cancer has been approved by the US Food and Drug Administration, leading the way for federal approval of other, similar efforts.

In this type of therapy, patients' own immune cells are modified ex vivo to include a gene for the vehicle protein, which directs them to attack leukemia cells carrying the CD19 antigen. It is the first gene therapy of its kind to hit the USA market.

The drug, known as Kymriah (tisagenlecleucel), will be used to treat certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL). "New technologies such as gene and cell therapies hold out the potential to transform medicine". Based on those results, an FDA advisory panel recommended the approval in July.

The treatment is not a pill or a form of chemotherapy.

Short for chimeric antigen receptor T-cell therapy, CAR-T treatment takes a person's own cells, removes them from the body, re-engineers them, and then puts the cells back in the body where they can attack cancer cells.

The Kymriah approval came just over three months after the first patient to receive it, 12-year-old Emily Whitehead of Philipsburg, Pa., celebrated five years cancer-free in May.

The treatment, which is expected to be costly, can cause serious side effects including high fevers and neurological problems.

"Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease", Peter Marks, MD, PhD, direct of the Center for Biologics Evaluation and Research, FDA, said in the release. (Currently, CAR-T companies have been focusing on blood cancers.) "We also want to see if we can expand it to other diseases down the line", he said.

Novartis is also required to conduct a post-marketing observational study involving patients treated with Kymriah, to further evaluate the long-term safety of the CAR-T therapy.

The road to approval for CAR-T has been filled with uncertainty, but Novartis and lead rival Kite Pharma made strides over the previous year, notably with Novartis' recent unanimous FDA panel vote in favor of approval and Kite's $11.9 billion acquisition this week by Gilead Sciences. Made from scratch for every patient, it's one of a wave of "living drugs" under development to fight additional blood cancers and other tumors, too.

On a conference call Wednesday, Novartis executives said the company is working with the Medicaid program and private insurers and expects broad coverage and will offer some financial assistance with such things as copay and travel costs.

In a key study of 63 advanced patients, 83 percent went into remission soon after receiving the CAR-T cells.

Despite its great promise, the FDA warns that Kymriah does have the potential for severe side effects.

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