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FDA approves new gene therapy treatment for leukemia developed by Penn Medicine and CHOP

PHILADELPHIA — The U.S. Food and Drug Administration approved a first-ever treatment developed by the University of Pennsylvania and the Children’s ...
Emily Whitehead,12, spends time with her father, Tom and mother, Kari. Emily was The first child treated by Penn-CHOP and has been in remission for four years.

PHILADELPHIA — The U.S. Food and Drug Administration approved a first-ever treatment developed by the University of Pennsylvania and the Children’s Hospital of Philadelphia that genetically alters a patient’s own cells to fight cancer, according to an announcement from Penn Medicine and Lancaster General Health.

The new treatment modifies patients’ own immune T cells, turning them in to “hunter” cells that multiply and attack the disease. It is designed for patients up to 25 years old, who suffer from B-cell precursor acute lymphoblastic leukemia in refractory or in relapse.

The treatment was first developed by Penn, which later worked in collaboration with the pharmaceutical company Novartis. The therapy will be marketed as Kymriah, and is the first therapy based on gene transfer approved by the FDA.

In the new therapy, the patient’s T cells are collected and reprogrammed at the Novartis manufacturing facility to seek and destroy the patient’s leukemia cells. The reprogrammed cells are infused back into the patient’s body, where they multiply and attack, targeting cells that express a specific protein called CD19.

Tests reveal that the reprogrammed cells can grow to more than 10,000 new cells for each engineered cell the patient receives, producing high remission rates in leukemia. The new cells can survive in the body for years.

Investigators at Penn’s Perelman School of Medicine and CHOP, who together led research, development, and clinical trials of the new therapy in collaboration with Novartis, hailed the FDA’s approval as a game changer for the treatment of younger patients battling the aggressive blood cancer and a pivotal milestone in this new era of cellular therapies that treat cancer with a patient’s own immune system.

“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, the Richard W. Vague Professor in Immunotherapy in the department of Pathology and Laboratory Medicine in Penn’s Perelman School of Medicine and director of the Center for Cellular Immunotherapies in the Abramson Cancer Center.

“We’re excited and proud to have moved this CAR therapy, in collaboration with Novartis and CHOP, through all phases of development and clinical trials, established its efficacy, and now extended its reach to children across the country under this FDA approval,” he added. “We hope the momentum behind the technology builds as we continue to investigate the abilities of personalized cellular therapeutics in blood cancers and solid tumors to help patients with many other types of cancer.”

Kymriah is expected to be available through a network of certified treatment centers throughout the United States.

The first patient to receive the therapy was Emily Whitehead, a resident of Londonderry Township in Lebanon County. She began treatment at six years old, when her leukemia stopped responding to conventional treatments. Now 12, Whitehead’s leukemia remains in remission, according to Penn Medicine.

“In larger trials, we’re seeing overall remission rates over 80 percent, which is a remarkable improvement upon previous treatment success rates,” said lead investigator of the CHOP and global trials of the therapy, Stephan Grupp, MD, PhD, the Yetta Deitch Novotny Professor of Pediatrics at Penn and director of the Cancer Immunotherapy Frontier Program and chief of the section of Cell Therapy and Transplant at CHOP.

Kymriah treatment can include a side effect called cytokine release syndrome, which includes varying degrees of flu-like symptoms, with high fevers, nausea, and muscle pain, and temporary neurologic symptoms, including delirium, but also low blood pressure and breathing difficulties. The FDA’s approval requires hospitals and doctors to be specially trained and certified to administer it, and that they stock the immunosupressant drug tocilizumab or short courses of steroids to combat the symptoms.

Novartis will create a registry to follow patients for 15 years after being treated to monitor their progress and any potential, future side effects.

Other trials with CTL019 therapy are also underway in the Abramson Cancer Center for adult leukemia patients and those with CLL and non-Hodgkin lymphoma. Penn and Novartis are also investigating the next generation of CAR therapies for multiple myeloma, and for solid tumors, through trials in glioblastoma, mesothelioma, and ovarian and pancreatic cancer.

Other CAR trials at Penn are exploring the technique for prostate cancer, melanoma, and triple-negative breast cancer.


Adult patients who are interested in T cell therapies at Penn Medicine can call 215-316-5127 for more information. For information about the Cancer Immunotherapy Program at CHOP, please call 267-426-0762.