While it has been referred to as gene therapy, the new treatment does not actually change or replace the genes that cause the cancer, rather it alters genes of the immune system to better recognize and eliminate the cancer. In that sense it is the first FDA-approved gene therapy in that it reprograms the patient’s own cells to attack the cancer.
The approval marks a milestone in the advancement toward a cancer cure. Specifically the FDA approved Kymriah (tisageniecleucel) for certain children and young adults with acute lymphoblastic leukemia (ALL). Childhood ALL is a type of cancer in which the bone marrow makes too many immature white blood cells or lymphocytes, which crowd out the mature immune cells that defend the body from infections and cancer.
Kymriah is a genetically-modified T-cell immunotherapy. Each dose of Kymriah is prepared for each patient using the individual patient’s own T-cells genetically modified to include a specific protein, called a chimeric antigen receptor or CAR that directs the T-cells to specifically target leukemia cells that display a specific protein (CD19) on their surfaces.
After the genetically altered T-cells are re-infused into the patient, they continue to be made in the patient’s body making Kymriah the first living therapy that has the potential to provide long-term cancer treatment.
Treatment with Kymriah has the potential to cause severe side effects. It carries a boxed warning for cytokine release syndrome (CRS), which is a systemic response to the activation and proliferation of CAR T-cells causing high fever and flu-like symptoms, and for neurological events.
Consequently, in the same announcement, the FDA issued expanded approval of Actemra (toclizumab) to treat CAR T-cell-induced severe or life-threatening CRS in patients over two years old. Because of the risk of CRS and neurological events, Kymriah is being approved with a risk evaluation and mitigation strategy (REMS), and requires hospitals and clinics that dispense Kymriah to be certified.
Novartis, the drug’s maker said the cost of the drug is expected to be priced at $475,000. Current treatments for ALL include bone marrow transplants that can total from $350,000 to $800,000.
Novartis, the drug’s maker said the cost of the drug is expected to be priced at $475,000. Current treatments for ALL include bone marrow transplants that can total from $350,000 to $800,000.
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