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‘Breakthrough’ Phase 3 Study Changes Standard of Care for Childhood B-cell Leukemia

Acute lymphoblastic leukemia, which affects white blood cells, is the most common type of cancer among children.
Most children newly diagnosed with B-cell acute lymphoblastic leukemia (B-ALL) in the United States can now receive an immunotherapy as part of their standard treatment plan, following the results of a Phase 3 study co-led by researchers at Children's Hospital of Philadelphia.
When paired with chemotherapy, the medication blinatumomab improved pediatric patients' three-year, disease-free survival rate from 88% to 96%, according to a new study published in the New England Journal of Medicine and presented at the 66th American Society of Hematology Annual Meeting & Exposition in December.
As a member of the Children's Oncology Group (COG), a multisite clinical trials group supported by the National Cancer Institute, CHOP was the largest enrolling site for the Phase 3 trial.
"The study was a home run," said study co-author David Teachey, MD, who chairs COG's Acute Lymphoblastic Leukemia Disease Committee and is the Director of Clinical Research at CHOP's Center for Childhood Cancer Research. "It changes the way that we treat children with leukemia across the globe and is one of the biggest breakthroughs in childhood cancer treatment in our lifetime."
ALL, which affects white blood cells, is the most common type of cancer among children. About 3,000 pediatric cases are diagnosed in the U.S. annually. Most diagnoses are B-ALL, which affects B cells; T-ALL, which affects T cells, is diagnosed less frequently. Chemotherapy can effectively treat childhood leukemia; however, it can take up to 3.5 years to complete, and 10% of pediatric patients diagnosed with ALL do not survive five years after treatment.
In 2017, the U.S. Food and Drug Administration (FDA) granted approval for blinatumomab to be used as an addition to chemotherapy for children and adults with relapsed B-ALL. The immunotherapy — which is administered continuously as an intravenous infusion for up to a month at a time — is a bispecific monoclonal antibody that binds to CD19 and CD3 proteins found on the surface of B cells and T cells.
In 2019, COG launched the AALL1731 clinical trial to test blinatumomab as a frontline therapy for pediatric patients with B-ALL. Researchers evaluated whether adding two, 28-day cycles of blinatumomab to chemotherapy would improve disease-free survival in children who were at least 1 year old. The Data and Safety Monitoring Committee halted the trial halfway through enrollment upon analysis of initial data that showed a significant improvement in disease-free survival rates with the addition of the immunotherapy.
In June 2024, the FDA approved blinatumomab as a frontline therapy for adult and pediatric patients one month and older who were newly diagnosed with B-ALL.
"It's a safe, low-side-effect medication that is going to save a lot of kids' lives," Dr. Teachey said. "The study also provides further evidence that immunotherapies can be moved to the frontline to treat kids with cancer."
Dr. Teachey and the ALL team at COG are now launching clinical trials to study whether adding blinatumomab to a treatment plan could reduce cycles of chemotherapy, and potentially eliminate the need for chemotherapeutics altogether.
This study was co-authored by Stephen Hunger, MD, former COG ALL Disease Committee Chair and Chief of CHOP's Division of Oncology. It was supported by grants from the National Institutes of Health/National Cancer Institute (U10CA180899; U20CA180886) and the St. Baldrick's Foundation.