Blood Cancer United Launches Innovative Sub-Study in Beat AML® Master Trial

Blood Cancer United Embarks on New Sub-Study in Beat AML® Master Clinical Trial



Blood Cancer United®, formerly known as The Leukemia & Lymphoma Society, is making significant strides in the fight against acute myeloid leukemia (AML). On January 14, 2026, the organization announced that the first patient has received treatment in a groundbreaking sub-study of its prominent Beat AML® Master Clinical Trial. This new research initiative, in partnership with AVEO Oncology—an LG Chem company—focuses on evaluating the effects of ficlatuzumab, a monoclonal antibody that targets hepatocyte growth factor (HGF), in combination with venetoclax and azacitidine for adults aged 60 and above who are newly diagnosed with untreated AML.

This latest sub-study is currently being conducted at the University of California San Francisco and The University of Kansas Medical Center. Dr. Lore Gruenbaum, Chief Scientific Officer at Blood Cancer United, emphasized the need for innovative therapies, stating, "The shift five years ago to venetoclax plus azacitidine as a standard of care for older adults with AML was practice-changing; however, many patients still experience relapse with poor long-term outcomes. Therefore, our active pursuit of new triplet therapies reflects our commitment to improving survival rates and the quality of life for patients."

Advancing the Beat AML Clinical Trial



The newly launched sub-study is one of four active studies under the expansive umbrella of the Beat AML Master Clinical Trial. This pioneering trial aims to test various treatments across different subtypes of AML, utilizing genomic testing to match patients with the most suitable targeted therapies based on their individual genetic profiles.

As of now, more than 1,850 patients have undergone genomic testing within seven days of their diagnosis, with over 650 participants enrolling in one of the precision treatment sub-studies. The Beat AML initiative prides itself on its collaboration, having established partnerships with numerous clinical sites across the United States, including recent additions at the University of Pittsburgh Medical Center and West Virginia University.

Blood Cancer United's Beat AML collaborative is a unique integration of leading blood cancer specialists, pharmaceutical companies, genomic testing firms, and data experts, all working together to accelerate discoveries that are centered around patient needs.

Recent Milestones and Updates



Additionally, two other sub-studies within the Beat AML trial have achieved notable milestones:

  • - Revumenib-Based Triplet Sub-Study: The addition of the menin inhibitor revumenib to the standard venetoclax and azacitidine combination has shown promising results. This early-phase sub-study has enrolled adults aged 60 and above with NPM1 mutations or KMT2A rearrangements, leading to high rates of complete remission and impressive median overall survival rates. Patients receiving this innovative triplet therapy have experienced a median overall survival of 15.5 months, compared to just 2.5 months with traditional treatment methods.

  • - Opti-AML Sub-Study: Launched in June 2023, this sub-study assesses whether a reduced duration of venetoclax treatment—14 days instead of the FDA-approved 28 days—coupled with azacitidine can maintain effective complete remission rates in newly diagnosed AML patients aged 60 and above. Early expectations suggest that this shorter treatment cycle could promote faster blood count recovery while minimizing risks of infections, hospitalizations, and the need for transfusions. All patient enrollment for this sub-study concluded in September 2025, with results anticipated later in 2026.

Community Efforts and Future Outlook



Dr. John Byrd, Chief Medical Officer of Beat AML and Director of the UPMC Hillman Cancer Center, expressed optimism about the future of the trial, mentioning, "Beat AML has surpassed our expectations, evolving into a robust network of innovative therapeutic trials. Our patients have seen improved survival rates and a better quality of life compared to those undergoing standard chemotherapy options. As we approach ten years since the trial's inception, we are committed to pushing the boundaries of precision medicine in AML."

The Beat AML Master Clinical Trial (NCT03013998) has grown to become the first collaborative precision medicine trial in blood cancer, harnessing advanced genomic technologies to match patients with the most appropriate targeted treatments based on their genetic mutations. Spanning multiple therapies and sub-studies, Beat AML continues to demonstrate strong results, reflecting a commitment to enhancing survival and quality of life for patients battling AML.

For more information and updates on Blood Cancer United and the Beat AML Master Clinical Trial, visit BloodCancerUnited.org and for specific details on the Beat AML trial, navigate to bloodcancerunited.org/beataml.

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