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

December 7, 2024
3 min read

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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