Seattle Children’s Cancer Study Awarded Top Clinical Trial

May 27, 2025 — A groundbreaking pediatric cancer drug trial co-led by a Seattle Children’s physician-researcher received the prestigious David Sackett Trial of the Year from the Society for Clinical Trials on May 20 at the organization’s annual meeting in Vancouver, British Columbia.

The Children’s Oncology Group AALL1731 clinical trial, which concluded last year, showed adding the drug blinatumomab to a chemotherapy regimen for children with B-cell acute lymphoblastic lymphoma (B-ALL) decreased risk of relapse by about two-thirds. These findings represent the biggest breakthrough in childhood cancer treatment in decades.

Rachel Rau, MD

Since 2008, the Sackett Trial of the Year Award has been awarded to a randomized, controlled trial published in the previous calendar year that provides a basis for substantial change in healthcare and “improves the lot of humankind.”

“Receiving the Sackett Award is monumental,” said study co-leader Rachel Rau, MD, a pediatric hematologist-oncologist at Seattle Children’s with a lab-based research program in the Ben Towne Center for Childhood Cancer and Blood Disorders Research at Seattle Children’s Research Institute. “It really recognizes the significant impact and magnitude of our work, placing it in the context of world health.

“For me, it was a big picture moment. This practice-changing finding will lead to many lives saved, and given that the results apply to children, the years of life ultimately saved is really incredible to think about,” Dr. Rau said.

B-cell acute lymphoblastic leukemia is the most common childhood cancer. While it has a high overall cure rate, relapse of the disease remains a leading cause of death among children.

The AALL1731 study began in July 2019 and ended early, in July 2024, after data from the first interim analysis showed children who received standard chemotherapy had a three-year disease-free survival of 87.9% versus 96% in those who received chemotherapy and blinatumomab — a better outcome than expected. The regimen is now the ideal standard of care for children with B-ALL around the world.

— Colleen Steelquist