The First FDA Approved Therapy to Fight Leukemia is Here

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The FDA has approved a gene therapy treatment for use in the U.S to Novartis Pharmaceuticals Corp. that may give many young patients with leukemia hope.

The treatment is a procedure called Kymriah (tisagenlecleucel) that uses a patient’s own cells to combat a particular type of leukemia. The FDA has approved it to be used only for children and young adults up to age 25.

Kymriah works to combat acute lymphoblastic leukemia (ALL), which is the most common childhood cancer in the US. There are over 3,000 new cases diagnosed annually.

The procedure extracts a patient’s autoimmune T-cells and then sends them to a facility. At the facility they are modified to include a new gene that directs the T-cells to target particular leukemia cells. Then the genetically-modified cells are sent back to be re-inserted in the patient.

Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research (CBER), in the FDA’s post said, “Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease.”

“Not only does Kymriah 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.”

The FDA cited one study that treated 63 children and young adults with Kmyriah. The study saw an 83% remission rate after three months.

Disclaimer: We have no position in Novartis Pharmaceuticals Corp. (NYSE: NVS) and have not been compensated for this article.