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FDA approves first-ever gene therapy treatment for acute lymphoblastic leukemia

31 August 2017

The Food and Drug Administration (FDA) on Wednesday approved a gene-therapy treatment that has the potential to aid young patients suffering from an aggressive form of leukemia.

"We are so proud to be part of this historic moment in cancer treatment and are deeply grateful to our researchers, collaborators, and the patients and families who participated in the Kymriah clinical program", added Bruno Strigini, CEO of Novartis Oncology.

Kymriah will carry a boxed warning because of the treatment's potential to cause deadly side effects, including neurological complications and what's known as cytokine release syndrome, a systemic reaction triggered by the destruction of the cancer cells. Instead, in a complicated process developed at the University of Pennsylvania, white blood cells called T cells are extracted from the patient and sent to a manufacturing center to be modified to include a new gene that directs the T cells to target and kill leukemia cells.

In this type of therapy, patients' own immune cells are modified ex vivo to include a gene for the auto protein, which directs them to attack leukemia cells carrying the CD19 antigen.

The immunotherapy now can be used in children and young adults with B-cell ALL that will not respond to other therapies, the FDA announced. Under the arrangement, Novartis will only collect payments from CMS when patients respond to treatment by the end of the first month. Researchers filter those cells from a patient's blood, reprogram them to harbor a "chimeric antigen receptor" or vehicle that zeroes in on cancer, and grow hundreds of millions of copies.

"Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease", said Peter Marks, M.D., Ph.D., director of the FDA's Center for Biologics Evaluation and Research, in the news release.

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The drug has shown promising remission and survival rates in clinical trials, the FDA said. When the body's immune system kicks into high gear to fight the cancer, extremely high fevers can occur as well as the production of proteins that produce inflammation.

Patients' collected immune cells will be frozen and shipped to a Novartis factory in New Jersey that creates each dose, a process the company says should take about three weeks.

"This is a brand new way of treating cancer", said Dr. Stephan Grupp of Children's Hospital of Philadelphia, who treated the first child with CAR-T cell therapy - a girl who'd been near death but now is cancer-free for five years and counting. Kymriah's maker, Novartis, hasn't provided a price for the drug.

Analysts have speculated the treatment could cost several thousand dollars.

"The theory is they should attack the tumor and continue to grow to become a long-term monitoring and treatment system", Lichtenfeld said.

For more on childhood acute lymphoblastic leukemia, visit the U.S.

FDA approves first-ever gene therapy treatment for acute lymphoblastic leukemia