FDA approves the first immunotherapy drug for childhood leukemia

by Jared Lewis August 31, 2017, 5:28
FDA approves the first immunotherapy drug for childhood leukemia

United States regulators approved the first cancer drug that uses a patient's own cells to fight cancer.

The FDA on Wednesday approved a chimeric antigen receptor (CAR) T-cell therapy for treatment of certain pediatric and young adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia. Instead of helping the body's T cells better spot cancer, the CAR-T treatment enables them to fight those cancer cells better.

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.

FDA approved the Novartis drug, called Kymriah (tisagenlecleucel), for children and adults up to age 25 with B cell ALL who have not responded to conventional therapy or who have relapsed.

"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".

In addition to Kymriah's approval, FDA also announced the expanded approval of Roche's Actemra (tocilizumab) to treat auto T-cell-induced severe or life-threatening cytokine release syndrome in patients 2 years of age or older. According to Children's of Alabama, more than 190 children in Alabama are diagnosed with various forms of cancer each year, including ALL. Experts say it could lead to high fever, flu-like symptoms, and neurological events that can be life-threatening.

The drug company also said in the statement that it was collaborating with Medicare on a plan in which the government would only pay for the treatments if patients responded to them by the end of the first month.

The road to approval for CAR-T has been filled with uncertainty, but Novartis and lead rival Kite Pharma made strides over the past 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. The drug will treat acute lymphocytic leukemia, the most common type of childhood cancer in the US. Each dose of Kymriah is a customized treatment created using an individual patient's own T-cells, a type of white blood cell known as a lymphocyte. And while Novartis' success was widely expected, the FDA's decision to rapidly clear the drug underscores the excitement that doctors and the life sciences community feel about such therapies, which have been shown to eliminate all signs of aggressive blood cancers in some patients after just one treatment in clinical trials. Novartis and other companies are racing to develop T-cell therapies for other blood disorders, as well as for solid tumor cancers, which have so far defied this immune-boosting approach. It's shown very encouraging results in clinical trials, but the price tag will be hefty: Analysts say it will cost "a fortune," or maybe $700,000 for one course of treatment. Plus, there can be serious side effects, such as inflammatory problems that have the potential to be fatal.

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