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A 2012 image of Novartis headquarters. Photo: lucarista / Shutterstock

Soon young Americans with a cancer of the bone marrow and blood will have their white blood cells extracted from their blood, shipped to a manufacturing center, genetically altered at the level of a single protein translation, and readministered back into the patient’s body, with a tiny DNA tweak designed to destroy the cancer.

The gene therapy called Kymriah, approved by the U.S. Food and Drug Administration today, is the first of its kind – and nothing short of a “new frontier in medical innovation,” according to the regulatory agency.

“New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses,” said Scott Gottlieb, the FDA Commissioner. 

Kymriah, marketed by Novartis, was granted several special designations, including “fast track,” “priority review,” and “breakthrough therapy” consideration.

The therapy, also known as tisagenlecleucel, is a suspension intended to be used one time, with a patient’s T calls. The genetic changes wrought through the customized process creates coding for a protein known as a chimeric antigen receptor, or CAR. The T cells are thus targeted at the leukemia cells with an antigen known as CD19 on their surface.

The treatment is now approved for all patients up to the age of 25 who have already been diagnosed with B-cell precursor acute lymphoblastic leukemia, or ALL. (ALL is the most common childhood cancer in the United States, and it is aggressive). Kymriah is intended for the B-cell subset of the disease – and a group of patients who do not respond to initial treatments (between 15 and 20 percent of patients overall).

The clinical trials in 63 young patients previously showed the safety of the treatment – as well as a remission rate of 83 percent at the three-month mark. However, the treatment can have serious side effects like infections, low blood pressure, kidney injury and other effects. Along with the Kymriah approval, the FDA also expanded the approval of a drug named Actemra (tocilizumab) used to offset the side effects like these, related to a cytokine release syndrome.

The Kymriah research and development was made possible by a deal struck between Novartis and a team at the University of Pennsylvania in 2012.

"Tisagenlecleucel is the first CAR-T therapy to demonstrate early, deep and durable remission in children and young adults with relapsed or refractory B-cell ALL," said Stephan Grupp, Yetta Deitch Novotny Professor of Pediatrics at the Perelman School of Medicine at Penn, also Director of the Cancer Immunotherapy Frontier Program at Children's Hospital of Philadelphia (CHOP). "We've never seen anything like this before and I believe this therapy may become the new standard of care for this patient population."

A widening array of immunotherapies, which involve personalized treatments on a patients’ cells, have been approved by the FDA. But they have not altered the actual DNA of the patients’ cells like Kymriah is touted to do.

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