Landmark Clinical Trial Results Offer New Hope for Kids with Acute Lymphoblastic Leukemia (ALL)

Transformative breakthrough in pediatric leukemia made possible by decades of St. Baldrick's Foundation funding and support.

SAN DIEGO, Dec. 7, 2024 /PRNewswire/ -- Groundbreaking results from a major clinical trial, supported in part by the St. Baldrick's Foundation, were unveiled today at the 66th Annual Meeting of the American Society of Hematology (ASH) in San Diego, Calif. The Children's Oncology Group (COG) announced transformative findings that are set to redefine the standard of care for children with acute lymphoblastic leukemia (ALL). This breakthrough—hailed as one of the most significant advancements in pediatric oncology in decades—represents a milestone made possible by 25 years of donations to St. Baldrick's head-shaving events, fundraisers, and the generosity of dedicated donors.

As COG's largest philanthropic donor, St. Baldrick's provides vital funding for both groundbreaking discoveries and the clinical trials that bring these innovations to patients. Through the COG's cooperative research network, thousands of children gain access to lifesaving clinical trials each year. At any given time, more than 100 trials are actively recruiting participants. An annual St. Baldrick's grant helps offset the cost of these trials by providing per-patient reimbursements to member institutions, ensuring more children have access to cutting-edge treatments.

This latest trial demonstrates that adding blinatumomab, a targeted immunotherapy drug, to traditional chemotherapy significantly improves disease-free survival (DFS) rates for children with standard-risk (SR) ALL. The results are so impactful that the COG has suspended all ongoing SR ALL trials to incorporate this game-changing treatment into its protocols.

Key Findings
The trial enrolled over 4,200 children and achieved unprecedented results:

• A 96% three-year DFS rate for children receiving blinatumomab compared to 87.9% with chemotherapy alone.
  
  
• Blinatumomab was shown to be well-tolerated, with minimal severe side effects, paving the way for reducing the use of older, more toxic chemotherapy drugs.

"Blinatumomab represents a paradigm shift in how we treat ALL," said Doug Hawkins, M.D., COG Group Chair. "For the first time, we can envision replacing toxic treatments with a safer, targeted therapy without compromising, and even improving, survival rates. We are grateful for the support of so many donors who helped make this discovery possible, including the St. Baldrick's Foundation."

How Blinatumomab Works
Blinatumomab is a type of immunotherapy and monoclonal antibody that targets CD19, a protein found on leukemia cells. By engaging the body's immune system, the drug enables it to recognize and destroy cancerous cells more effectively. Unlike traditional chemotherapy, blinatumomab shows no evidence of causing long-term side effects, an especially critical factor for young patients.

A Milestone Made Possible by Generous Support
The success of this trial underscores the critical role of philanthropic support in advancing pediatric cancer research. To date, St. Baldrick's has granted over $101 million to the COG, empowering the COG to explore cutting-edge treatments like blinatumomab, changing the outlook for children diagnosed with ALL.

"This transformative breakthrough represents a monumental step forward in our fight to conquer kids' cancer," said Kathleen Ruddy, CEO of the St. Baldrick's Foundation. "Thanks to the unwavering support of our donors, volunteers, partners, and advocates, we're not only improving survival rates but also paving the way for safer, less toxic treatments. Together, we are making cures a reality."

A New Era in Treatment
With these results, the medical community is optimistic about the potential to extend this treatment approach to other childhood cancers. Researchers will continue to build on this success, ensuring that children with cancer receive safer and more effective care.

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For more information about the St. Baldrick's Foundation and how you can support lifesaving pediatric cancer research, visit StBaldricks.org.

About St. Baldrick's Foundation
Every 2 minutes, a child somewhere in the world is diagnosed with cancer. In the U.S., 1 in 5 will not survive. The St. Baldrick's Foundation, the largest charity funder of childhood cancer research grants, has awarded more than $354 million to researchers to Conquer Kids' Cancer. When you give to St. Baldrick's, you don't just give to one hospital – you support every institution with the expertise to treat kids with cancer across the U.S. St. Baldrick's ensures that children fighting cancer now — and those diagnosed in the future — will have access to the most cutting-edge treatment, by supporting every stage of research, from new ideas in the lab to the training of the next generation of researchers, to lifesaving clinical trials. St. Baldrick's has played a role in virtually every advancement in the field over the past 25 years and remains essential in advancing progress and fostering innovation in childhood cancer research. Visit StBaldricks.org and help #ConquerKidsCancer.

About The Children's Oncology Group
The Children's Oncology Group (childrensoncologygroup.org), a member of the NCI National Clinical Trials Network (NCTN), is the world's largest organization devoted exclusively to childhood and adolescent cancer research. The Children's Oncology Group unites over 10,000 experts in childhood cancer at more than 200 leading children's hospitals, universities, and cancer centers across North America, Australia, New Zealand, and Saudi Arabia in the fight against childhood cancer. Today, more than 80% of the 15,000 children and adolescents diagnosed with cancer each year in the United States are cared for at Children's Oncology Group member institutions. Research performed by Children's Oncology Group institutions over the past 50 years has transformed childhood cancer from a virtually incurable disease to one with a combined 5-year survival rate of 86%. The Children's Oncology Group's mission is to improve the cure rate and outcomes for all children with cancer.

SOURCE St. Baldrick's Foundation