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“We knew there were things offered at CHOP that were cutting-edge — newer things that weren’t being tried anywhere else,” Emily’s mom, Kari, recalls. On April 17, 2012, the 7-year-old received an infusion of her own immune cells, re-engineered in a lab to attack cancerous cells. The experimental therapy, called CART19 or CTL019, had shown remarkable results in adult patients. Emily was the first child to be treated.
The vial of cells represented years of work by a team at CHOP, led by physician-scientist Stephan Grupp, M.D., Ph.D., in collaboration with teams from the University of Pennsylvania.
“When people talk about big science or team science, this is what they’re talking about,” Grupp says. “It’s an amazing machine that you have to put together to be able to develop these new treatments, and an environment like CHOP is the only place where you can do that.”
Meet Emily, her parents and Dr. Grupp in this video.
And Emily’s response represents the very best hope of those many, dedicated teams: She has experienced a complete remission. A test of her bone marrow three weeks after this single treatment showed no cancer cells, and those results have been maintained. It’s a story that has garnered world-wide attention since the results were published in December.
“CTL019 was the only option left for Emily,” Kari says. “We had a lot of hope. All along we said, it just has to work, it has to work for Emily — and it did.”
Scientists are hopeful CTL019 will in the future be an effective therapy for certain leukemias and lymphomas. Nine of 12 pediatric and adult patients have experienced a partial or complete response (absence of cancer) after the therapy, as of December 2012. However, many more must be treated to determine the success rate. And other questions remain, such as whether the cells provide long-term disease control and whether similar cells can be applied to other cancers.
A picture of good health: Emily seven months after the experimental therapy.
The Whiteheads returned home to central Pennsylvania in June. Emily spent a happy summer, relaxing and enjoying her puppy, Lucy. And in September, she reached another milestone her parents feared might never come: She boarded the school bus to start second grade.
“She is a kid who has just thrived after having the burden of her disease lifted from her,” Grupp says. “To see her go from leaving the Hospital and starting to recover, to going to school and playing soccer and looking like every other kid is just wonderful. It’s the best thing about doing what I do.”
Learn more about the CTL019 clinical trial.
