A major celebration rocked the Perelman Center after the FDA’s landmark decision was announced.

Robert Vonderheide, MD, DPhil (l), director of Penn’s Abramson Cancer Center, and Carl June sport the special t-shirts created for the occasion. Staff from across Penn Medicine received these t-shirts.

In a landmark decision, the U.S. Food and Drug Administration in August approved chimeric antigen receptor T-cell (CAR T) therapy developed by researchers at Penn to pediatric and young adult patients with relapsed acute lymphoblastic leukemia (ALL), who have run out of convenional treatment options. This personalized cell therapy – which uses a patient’s own immune system to battle a disease – is the first of its kind to receive FDA approval .

“This is a turning point in the fight against B-cell ALL that opens up opportunities for patients across the world who desperately need new options,” said Carl June, MD, director of the Center for Cellular Immunotherapies in Penn’s Abramson Cancer Center, who led the development of the therapy with Stephan Grupp, MD, PhD, chief of Cell Therapy and Transplant at Children’s Hospital of Philadelphia, where the first clinical trials of the therapy in children were conducted. “We hope the momentum behind the technology builds as we continue to investigate the abilities of personalize cellular therapeutics in blood cancers and solid tumors to help patients with many other types of cancer.”

The new treatment modifies patients’ own immune T cells to potentially seek and destroy their leukemia cells. As part of the therapy, the patients’ T cells are first collected and reprogrammed, and then infused back into their bodies. These newly built “hunter” cells both multiply and attack, targeting cells that express a protein called CD19. Tests reveal that the army of hunter cells can grow to more than 10,000 new cells for each single engineered cell patients receive, producing high remission rates in completely refractory leukemia – and can survive in the body for years. This CAR T therapy , known as Kymriah, is manufactured by Novartis. In 2012, Penn licensed the therapy, then known as CTL019, to Novartis, and entered into ainto a global collaboration with the international pharmaceutical company to further research, develop and commercialize Kymriah and other CAR-T cell therapies for the treatment of cancers.

Media throughout the country reported on the FDA’s approval, including the Washington Post, the Wall Street Journal, Time magazine, and ABC World News Tonight. In a New York Times article, June said that while he once thought it “improbable” that this type of gene therapy would be approved, now “I think the cancer world is forever changed.”   

To read more about this landmark approval, go to the Penn Medicine news release.

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