University of Pennsylvania’s Personalized Cellular Therapy Receives FDA’s Breakthrough Therapy Designation

The U.S. Food and Drug Administration has designated an investigational therapy, referred to as CTL019, as a Breakthrough Therapy, making it the first personalized cellular therapy to receive that federal award. The method, which involves removing a patient’s T-cells and genetically reprogramming them to attack tumor cells, was developed by a research team at the University of Pennsylvania.

A staggering 89% of leukemia patients who participated in early-stage clinical testing went into complete remission after being given CTL019. None of the participants had been responding to the conventional treatment for relapsed and refractory adult and pediatric acute lymphoblastic leukemia. Now, the research team has begun trials on patients with non-Hodgkin lymphoma and myeloma, and they are investigating the potential for the therapy’s use on other forms of cancer, including ovarian, breast, and pancreatic cancer, as well as mesothelioma, which is primarily caused by asbestos exposure and is almost always fatal.

“Our early findings reveal tremendous promise for a desperate group of patients, many of whom have been able to return to their normal lives at school and work after receiving this new, personalized immunotherapy,” said Carl June, MD, the research team leader. June explained that the recognition was “an essential step” toward expanding the therapy “to patients across the world who desperately need new options to help them fight this disease.”

In fact, bringing newly developed therapies to patients with life-threatening illnesses is the primary goal of the FDA’s Breakthrough Therapy designation, which looks to expedite the process of developing and reviewing new medicines so that they are more quickly available to those in need.

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