UChicago Medicine certified to offer breakthrough CAR T-cell gene therapy

The University of Chicago Medicine is among the first sites in the Midwest certified to offer a breakthrough gene therapy for pediatric acute lymphoblastic leukemia (ALL), which was approved Wednesday, Aug. 30 by the U.S. Food and Drug Administration.

Chimeric antigen receptor T-cell, or CAR T-cell, therapy, targets two common forms of cancer and has been used in clinical trials at UChicago Medicine to treat both ALL and diffuse large B-cell lymphoma (DLBCL) in adults.

"CAR T-cell therapy has revolutionized the treatment of acute lymphoblastic leukemia," according to cancer specialist Sam Tinaglia, 18, of Park Ridge was one of the first 100 children in the country to participate in clinical trials of CAR T-cell therapy for acute lymphoblastic leukemia. Diagnosed in 2003 at age 5, Tinaglia relapsed for the first time five years after initial treatment. Over the next seven years, doctors tried more rounds of chemotherapy and a bone marrow transplant. With each recurrence, his chances of survival lessened.

"We thought we had reached the end, " said Sam's mother, Suzie. "But then Dr. Cunningham told us about the CAR T-cell clinical trial that was giving hope to kids like Sam, who had no other options."

In 2015, during his junior year of high school, Cunningham referred Sam to Children's Hospital of Philadelphia to receive CAR T-cell therapy. Although he experienced some of the serious side effects, he recovered after treatment in a pediatric intensive care unit. "A few weeks later, they sent me home and that was pretty much it," said Sam, who just started his freshman year of college. "I'm obviously very happy, because my cancer, hopefully, is gone forever.

Sam will continue his care at Comer Children's, where he received most of his treatment for ALL and is now followed by experts in the Childhood Cancer Survivors Center.

"For Sam, CAR T-cell therapy was the exact treatment he needed," said Suzie. "And we are so thankful it worked out. We never thought we'd get to this point."