New Cancer Cure: World’s First Child Treated with Immunotherapy Is Cancer-free 10 Years Later

Emily Whitehead, the first child in the world to receive CAR T-cell immunotherapy, celebrates 10 years cancer free. http://www.chop.edu/immunotherapy-10-years

In 2012, Tom and Kari Whitehead came to Children’s Hospital of Philadelphia (CHOP) looking for a miracle for their 6-year-old daughter, who was battling acute lymphoblastic leukemia (ALL). The standard treatment that works for most children with ALL — the most common form of childhood cancer — did not work for Emily. She relapsed twice, and her disease was so out of control that she was ineligible for a bone marrow transplant to treat it. Their local medical team advised the Whiteheads that it was time to consider hospice care.

Tom and Kari were aware that Stephan Grupp, MD, PhD, a Children's Hospital of Philadelphia (CHOP) pediatric oncologist and a pioneer in the field of cellular immunotherapy, had been working in close collaboration with researchers Drs. Carl June, Bruce Levine and David Porter at the University of Pennsylvania on a completely new way to treat cancer. Called CAR T-cell therapy, the revolutionary approach harnesses the power of a patient’s own immune system by reengineering their T cells so they can find and attack cancer cells.

Dr. Grupp was about to open the first phase 1 trial for CAR T-cell therapy in pediatric patients with ALL, based on early success the Penn researchers had already seen in two adult patients with a different type of cancer.

Emily became the first patient to enroll in the trial, making her the first pediatric patient ever to receive the treatment. Ten years later, with Emily now 17 and in possession of a driver’s license, it is clear the Whiteheads found their miracle. Since receiving the treatment, Emily has remained cancer free. The therapy she received is now an approved drug, and thousands of children around the world have received it.

Emily's path has helped define the field of CAR T immunotherapy.

After she received the CAR T cells, Emily’s immune system went into overdrive, causing her to become critically ill. She was admitted to the pediatric intensive care unit at CHOP. Dr. Grupp and a team of scientists determined that the level of a certain protein had become very elevated as a result of the T cells growing in her body. This same protein is involved in rheumatoid arthritis, and there is a drug for that disease that turns off production of that protein, so the team decided to administer the drug to Emily. The results were dramatic: Her condition improved faster than anyone could have hoped for. On May 2, her 7th birthday, Emily woke up.

This approach to treating the post-infusion illness, now known as cytokine release syndrome (CRS), is now used worldwide for children and adults treated with CAR T cells and other immunotherapies. “Through Emily, we learned how to control CRS when it gets serious in these patients, and that information has been disseminated throughout the world and used by all physicians who treat patients with CAR T-cell therapy,” Dr. Grupp says. The re-engineered T cells did exactly what they were intended to do: they eradicated the cancer and put her into remission “What we learned from Emily changed everything.”