In a major milestone for pediatric cancer treatment, Children’s Hospital of Philadelphia (CHOP) has treated its 500th patient with CAR T-cell immunotherapy, a “living drug” that harnesses the power of a patient’s own immune system to fight cancer. Pioneered at the University of Pennsylvania and CHOP, CAR T has revolutionized the treatment of cancer across the globe.

In 2012, CHOP researchers led by Dr. Stephan Grupp, Chief of the Cellular Therapy and Transplant Section at CHOP, infused the first-ever pediatric CAR T patient, Emily Whitehead. Emily had acute lymphoblastic leukemia (ALL) that was resistant to all available treatments. CHOP opened the first pediatric clinical trial for CAR T-cell therapy, and Emily became the first patient to enroll, making her the first pediatric patient to receive CAR T and the first patient of any age to receive it for ALL. Eleven years later, Emily is cancer free, as are hundreds of other patients who have gone on to receive the treatment. In addition, as many as 20,000 patients across the globe have received CAR T therapy more safely because of toxicity management protocols that were developed during Emily’s case.

“This milestone shows how far we have come in treating pediatric cancer,” said Dr. Grupp, Inaugural Director of the Susan S. and Stephen P. Kelly Center for Cancer Immunotherapy at CHOP. “We have gone from treating the very first pediatric patient to treating hundreds of other children with cancer, many of whose lives have been saved by CAR T therapy. Thanks to the generous support of Steve and Susan Kelly, we have launched an entire center for cancer immunotherapy, which will allow us to expand and improve the use of these and other gene therapy treatments for hundreds more children.”