Emily Whitehead/Stephen Grupp, MD, PhD
Emily Whitehead was just 5 when she developed troubling symptoms, including nosebleeds, bruising, and knee pain. Her doctor diagnosed her with acute lymphoblastic leukemia, the most common type of blood cancer -- and one that is 85% curable. "They told us it was a ‘garden variety' cancer in the beginning," says her father, Tom Whitehead. "But from the beginning, things didn't go the way they should."
Emily went into remission after chemotherapy but relapsed twice, making her ineligible for a bone marrow transplant. Then in 2012, with Emily just days from kidney failure, Stephen A. Grupp, MD, PhD, of the Children's Hospital in Philadelphia, told the Whiteheads of a clinical trial -- one in which doctors would genetically engineer Emily's own infection-fighting T cells to fight the cancer cells.
Only three adults had received the treatment -- – no children. It nearly killed Emily; at one point, she was given a one in 1,000 chance of surviving. But Grupp's team found a drug to stop her adverse reaction, and when Emily awoke from a two-week coma, her cancer was gone.
Emily is now 8 and healthy. Grupp has treated 17 more children with the procedure (80% of whom are in remission). Emily's family has twice traveled to Washington, D.C., with her oncologists to urge legislators to fund pediatric cancer research, and they have helped Children's raise money for its cancer programs. "We are so grateful to these doctors who spend their lives curing cancer," Whitehead says.