Fellowship Current News

August 30, 2017

History in the Making: FDA approves first gene therapy in the United States

The FDA approved Kymriah (tisagenlecleucel) today, a cell-based gene therapy, that has produced unprecedented results in patients with Acute Lymphocytic Leukemia (ALL). The Novartis therapy is approved for pediatric and young adult patients up to 25 years old with B-cell precursor ALL that is refractory or in second or later relapse.

Kymriah is a genetically-modified autologous T-cell immunotherapy. Each dose is a customized treatment created using an individual patient’s own T-cells. The patient’s T-cells are collected and sent to a manufacturing center where they are genetically modified to include a new gene that contains a specific protein (a chimeric antigen receptor or CAR) that directs the T-cells to target and kill leukemia cells that have a specific antigen (CD19) on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells.

The price tag is $475,000 for a course of treatment - and analysts say it's a bargain.

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