The Emily Whitehead Foundation today presented a check totaling $250,000 to Stephan A. Grupp, MD, PhD, director of the Cancer Immunotherapy Frontier Program, and section chief of the Cellular Therapy and Transplant Program at Children’s Hospital of Philadelphia (CHOP), benefiting cellular immunotherapy research at CHOP.

Her latest check-up reveals Emily Whitehead, the first, and now longest living pediatric patient to receive CAR T-cell therapy, has maintained remission for 6 years since receiving the experimental treatment in 2012. The US Food and Drug Administration (FDA) approved tisagenlecleucel—brand name Kymriah™—in August 2017 for children and young adults up to 25 years of age with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL), making it the first FDA-approved CAR T-cell therapy, as well as the first-ever FDA-approved gene therapy.

“Emily was a very sick little girl who was not responding to her leukemia treatment. It took tremendous courage from Emily and her family for her to be a pioneer for this experimental CAR T-cell therapy, and, in those first days after her infusion, her response was so intense we thought we’d lose her,” said Grupp. “But not only did Emily teach us how to mitigate the side effects of the treatment, she is living proof CAR T-cell therapy can help children achieve long-term remissions. This gives us hope for cures. The grant from the Emily Whitehead Foundation moves us toward helping more children like Emily, who have exhausted all other clinical options.”

CAR T-cell therapy genetically modifies a patient’s immune cells to make them seek out and kill leukemia cells. The approach was developed by a team led by Carl June, MD, of the Perelman School of Medicine at the University of Pennsylvania. That team collaborated with CHOP, the first institution to use the therapy in children with leukemia.

CHOP’s immunotherapy research team is pursuing ongoing clinical trials studying ways to attain a more robust patient response, mitigate serious side effects, prevent relapse, and even use CAR T-cell treatment for other high-risk pediatric cancers, including acute myeloid leukemia.

This is the second gift from the Emily Whitehead Foundation. The Foundation donated $100,000 to CHOP’s Immunotherapy Research Program in 2016.

In Emily’s own words: “Never give up.”