Published: Thu, August 31, 2017
Medical | By Garry George

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

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

A spokeswoman for Switzerland-based Novartis declined to say how much the drug will cost, though analysts have estimated a price of $500,000 or more.

"There will be no charge for the therapy if the patient doesn't respond within the first month after treatment", Novartis CEO Joseph Jimenez said during a media call.

In another novel development Wednesday, Novartis plans to price the therapy, Kymriah, "based on the clinical outcomes achieved", and specifically only allow for payment when ALL patients "respond to Kymriah by the end of the first month".

This type of immunotherapy, known as a CAR-T cell therapy, was known by the term CTL019 until now.

This first use of CAR-T therapy is aimed at patients desperately ill with acute lymphoblastic leukaemia, which strikes more than 3,000 children and young adults in the USA each year.

This procedure, known as Kymriah (tisagenlecleucel), works to combat acute lymphoblastic leukemia (ALL), the most common childhood cancer in the United States with over 3,000 new cases diagnosed every year.

In clinical trials, CAR-T therapies have shown remarkable efficacy against blood cancers.

The "historic action" by the Food and Drug Administration (FDA) permits a new treatment that uses a leukemia patient's own blood to engineer a method to attack the cancer.

While promising, CAR-T treatments won't be like other drugs that win FDA approval, which can quickly wind up on pharmacy shelves and hospitals. The altered T-cells include a new gene that contains a protein called chimeric antigen receptor, or auto, that directs the T-cells to kill leukemia cells with the antigen CD19.

"We're entering a new frontier in medical innovation with the ability to reprogramme a patient's own cells to attack a deadly cancer", said FDA commissioner Scott Gottlieb. The FDA expanded its previous approval of a rheumatoid arthritis drug, Actemra, to help manage these side effects.

Forty-nine percent of patients in the ELIANA trial experienced grade 3 or grade 4 cytokine release syndrome, caused by an overreactive immune response.

"This is remarkable technology", said Dr. Mikkael Sekeres of the American Society of Hematology.

The market for the breakthrough drug is relatively small; about 600 children a year in the USA may be candidates.

"This is a group of patients for whom current treatments don't do anything", Grupp told HemOnc Today.

Eleven patients who had CR+ CRi relapsed after tisagenlecleucel prior to data cut-off and before any new cancer therapy. Kite Pharma's similar CAR-T brand, developed by the National Cancer Institute, is expected to win approval later this year to treat aggressive lymphoma, and Juno Therapeutics and other companies are studying their own versions against blood cancers including multiple myeloma. "Continued research will also lead to improved manufacturing of large numbers of cells, which is necessary to make this therapy accessible to more patients".

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