Revolutionizing Cancer Treatment

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Children's View

Paulina’s situation was dire: Her cancer kept coming back. Her family feared they were out of options. “After the third time her leukemia relapsed,” says her father, Santiago Villanueva, “we thought, ‘What are we going to do now?’”

Then they learned about Children’s Hospital of Philadelphia’s Cancer Immunotherapy Program. At the time Paulina’s family contacted the program, the Immunotherapy team was conducting experimental studies on a cell therapy for treatment-resistant forms of acute lymphoblastic leukemia (ALL), the most common type of childhood cancer.

Paulina smiling Immunotherapy patient Paulina, 16 Paulina, now 16, and her family journeyed to CHOP from their home in Mexico to participate in the trial. The Immunotherapy team collected her T cells, a type of white blood cell, and reprogrammed them so that they could kill her cancerous cells. That was three years ago; today she remains cancer-free. Because of the amazing success of the trials, in August the U.S. Food and Drug Administration (FDA) approved the use of this immunotherapy. It’s the first-ever cell therapy available as an option for children whose cancers relapse.

Since treating the first pediatric patient in 2012, CHOP has treated more than 170 others. “We’re the most experienced cell therapy center in the world,” says Stephan Grupp, MD, PhD, the program’s director.

And there’s more to come. Immunotherapy team members are already aggressively pursuing similar treatments that will fight other forms of cancer.

Fueled by philanthropy

The Cancer Immunotherapy Program was the featured beneficiary of CHOP’s 2017 Carousel Ball — where For Tomorrow’s Breakthroughs made its public debut. Gifts to the campaign have played a crucial role in making the immunotherapy trials possible. Donations help cover travel and lodging when desperate families like Paulina’s want to participate but live far away. In other cases, donor funds pay for the cost of reprogramming the cells. Every gift helped CHOP develop the first cell therapy to receive FDA approval — a wondrous example of turning a visionary idea into a lifesaving therapy.