Tom Whitehead

Tom Whitehead is the co-founder of the Emily Whitehead Foundation, a nonprofit named for his daughter Emily, the first pediatric patient in the world to receive chimeric antigen receptor T-cell (CAR-T) therapy. He has been a public advocate for cell and gene therapy access since 2012, when Emily’s treatment at the Children’s Hospital of Philadelphia became the foundational pediatric case for what is now an FDA-approved class of immunotherapies.

Emily Whitehead was diagnosed with acute lymphoblastic leukemia (ALL) in May 2010, just after her fifth birthday. Standard chemotherapy failed twice across twenty-two months of treatment, and conventional options were exhausted. In April 2012, after the family sought a second opinion at the Children’s Hospital of Philadelphia, Emily was enrolled in an experimental trial and received CAR-T cell therapy, in which her own T-cells were genetically engineered to recognize and kill her cancer. She was cancer-free twenty-three days later and remains in remission more than a decade on.

The therapy Emily received was developed at the University of Pennsylvania and administered by a pediatric oncology team led by Dr. Stephan Grupp. It later became tisagenlecleucel (Kymriah), approved by the U.S. Food and Drug Administration in August 2017 as the first gene therapy of any kind cleared in the United States. Emily’s case is widely cited in the medical literature as the index pediatric case for CAR-T cell therapy.

Tom and Kari Whitehead established the Emily Whitehead Foundation in 2015. The foundation funds research into less toxic, more targeted therapies, connects families with clinical-trial experts, and advocates for equitable access to advanced cell and gene therapies. In 2025 it expanded its mission beyond pediatric cancer to support all individuals with cancer and rare diseases. It has helped tens of thousands of patients reach advanced therapies and has raised more than $4.5 million for research.

Whitehead appeared on Open Door Salon alongside Victoria (Tori) Lee, the tenth pediatric patient to receive CAR-T at the Children’s Hospital of Philadelphia and now a health-policy student presenting at ACRP on the bioethics of cell-therapy access. The conversation centered on the gap between approved CAR-T therapies and the patients who can actually reach them. Whitehead’s recurring point was that the science is no longer the constraint; access infrastructure is. He described manufacturing advances that have shortened production from six weeks to under one and lowered cost toward $50,000 per dose, the movement of trials outside the United States, and the foundation’s work helping families fight insurance denials and find trials.