Revealing What Was Once Hidden

Published on in Children's View

Cancer cells are experts at cloaking themselves. This is why it can be so difficult to treat cancer that is particularly aggressive or has relapsed: The body’s immune system simply does not “see” the cancer cells.

For years, the tools of cancer treatment were surgery, chemotherapy and radiation therapy. But what if the immune system’s cells could be altered to see — and thus destroy — the cancer cells? This would create a less toxic — and much more effective — option. When the campaign For Tomorrow’s Breakthroughs launched 10 years ago, this idea was still an ambitious dream.

Children’s Hospital of Philadelphia’s Cancer Immunotherapy Program relentlessly pursued that dream. When the researchers attained a promising therapy in 2012, CHOP treated the first pediatric patient with this therapy. It worked.

The team then led national and international pediatric clinical trials to more widely test the therapy — called CAR T-cell therapy — in treating children with advanced acute lymphoblastic leukemia (ALL), the most common pediatric cancer. The therapy re-engineers the patient’s own T cells, a type of white blood cell, into smart fighters able to see and then attack cancerous cells.

Children enrolled in CHOP’s CAR T trials included Nick, who lives in Iowa. His cancer kept coming back, and his family feared they were out of options. Then they learned about the trials at CHOP. In 2016, Nick and his parents, Nicole and Mike Gerdin, journeyed to CHOP to receive the then-experimental therapy. He remains cancer-free. Today, reports Nicole, “Nick is leading the active life of a 12-year-old boy. He loves to play football, basketball, baseball and catch lots of fish, to name a few of his favorite hobbies.”

Now available to children worldwide

Because of the success of the trials that CHOP led, in 2017 CAR T became the first-ever cell therapy to win U.S. Food and Drug Administration approval and is now available to children worldwide. After achieving remission, 70% of these children have remained in remission at two years, an astonishing proportion of children who once faced the direst prognoses. More than 350 patients from around the world have been treated with CAR T therapy at CHOP.

But CHOP’s work is far from over. Researchers and physician-scientists are now tackling immunotherapy for other forms of childhood cancer — and these efforts are fueled by the generosity of the Gerdins, who are determined to help other kids like Nick. “We are so grateful Nick has remained healthy and cancer-free following his CAR T therapy and stem cell transplant,” says Nicole. “Our wish for all families of children going through cancer treatment is they continue to have hope. For this reason, our family chooses to support the CHOP research that gave us hope during a terrifying time and resulted in a cure for our son.”

The family has given a gift through the Gerdin Charitable Foundation to fund two pilot clinical trials. One trial will bring the approved CAR T therapy to new groups of patients with ALL, including ultra-high-risk patients and infants. The other trial targets a different blood cancer called acute myeloid leukemia (AML). Only about 60% of patients with AML are cured with traditional therapies. This trial is one of the first attempts in the world to use cell therapy for this cancer.

CHOP is seeing tremendous interest from families during the initial stage of both trials. “To know the trials we have funded are providing renewed hope for others inspires us and hopefully others to continue to help,” says Mike. Because of philanthropy, children’s lives are being saved.