Can Early Intervention Prevent Leukemia? New Trial Gives Patients a Choice Beyond the Current 'Wait and See' Approach

Scientists are exploring if early intervention can prevent AML in high-risk patients, shifting the focus from monitoring to proactive prevention.

CAMBRIDGE, Mass., July 10, 2025 /PRNewswire/ -- Break Through Cancer, a Boston-based cancer research foundation, has launched a clinical trial to evaluate the feasibility of early intervention for Clonal Hematopoiesis (CH), a precursor to acute myeloid leukemia (AML). This multi-institutional study focuses on high-risk patients, exploring a new approach to managing this pre-malignant condition and advancing the groundwork for preventing this aggressive blood cancer.

Understanding CH and AML Risk

AML is a malignant blood disorder characterized by the rapid production of abnormal myeloid cells in the bone marrow and bloodstream. This aggressive cancer disrupts normal blood cell production and is associated with severe clinical outcomes, including a five-year survival rate of approximately 30%. CH can occur naturally with aging or as a consequence of chemotherapy or radiation treatment. Before receiving an AML diagnosis, many patients already have CH with AML-associated mutations, making it a pre-malignant condition. While often symptomless, some individuals with CH may have low blood counts—a condition known as Clonal Cytopenia of Uncertain Significance (CCUS). Those with high-risk CCUS have a greater than 50% chance of progressing to AML within five years.

"A lot of patients have already survived one cancer, and it's devastating when they receive a diagnosis of a therapy-related AML. The ability to potentially target the disease before it progresses to that point is what really excites me," said Dr. Kelly S. Chien, Assistant Professor at The University of Texas MD Anderson Cancer Center and a clinician on the study.

A New Approach: Early Intervention

As part of the new interventional trial, patients diagnosed with high-risk CCUS have the opportunity to choose proactive treatment for their precursor condition.

The trial (NCT06802146) will evaluate Inqovi (Astex Pharmaceuticals) in patients with high-risk CCUS, assessing the safety and feasibility of intervention at this precursor stage. Inqovi—an oral drug currently FDA-approved to treat a related blood cancer—works by altering the DNA of abnormal blood cells, thus slowing their growth and reducing the risk of progression to more advanced disease stages.

The two-arm study will enroll patients with high-risk CCUS who opt for one year of treatment with Inqovi or observation only.

"Many patients feel anxious when given a CCUS diagnosis and information about AML risk," says Dr. Lachelle Weeks, Assistant Professor, Dana-Farber Cancer Institute and a clinician on the study. "There are a number of patients who want to do something about it, and this trial could be the first step."

All patients will be observed for three years to assess the effects of Inqovi on AML progression and other health outcomes.

"Patients with high-risk CCUS already have low blood counts, which can significantly affect their quality of life," said Dr. Chien. "We're also looking at whether treatment improves quality of life, stabilizes blood counts, or reduces the burden of comorbidities, like heart disease, that are sometimes associated with certain mutations."

The trial will also assess mutational burden—whether the percentage of cells with mutations increases, decreases, or stays the same with treatment. A comparison of treated and untreated patients will provide insights into potential benefits of early intervention in CCUS.

This trial aims to pave the way for future larger studies testing the efficacy of intervention as part of developing comprehensive AML prevention strategies.

"The ultimate goal is to prevent diagnoses like AML altogether," Dr. Weeks notes. "This trial represents a critical step in understanding CCUS progression and exploring how early intervention could redefine care standards. If successful, it could spare patients and their families the profound challenges of living with and treating AML."

Prioritizing Diversity in Cancer Research

In addition to its scientific goals, this trial centers inclusivity in cancer research. By leveraging Break Through Cancer's model of radical collaboration and tapping into the broad reach of four disparate communities, this trial seeks to recruit a diverse and representative patient population and proactively circumvent long-standing inequities in cancer prevention research.

"By recruiting from community sites and reflecting the demographics of our catchment areas, we're ensuring our findings benefit all populations at risk," says Dr. Weeks.

To achieve this, the trial partners with community-based nurse navigators linked to Break Through Cancer's four partner sites. Together, researchers at Dana-Farber Cancer Institute, Memorial Sloan Kettering Cancer Center, Johns Hopkins Sidney Kimmel Comprehensive Cancer Center and MD Anderson are working to close gaps in care and improve outcomes for all patients.

About the trial

Title: Early Intervention in High Risk CCUS (NCT06802146)

About Break Through Cancer

Founded in 2021, Break Through Cancer empowers outstanding researchers and physicians to both intercept and find cures for several of the deadliest cancers by stimulating radical collaboration among outstanding cancer research institutions, including its founding partners: Dana-Farber Cancer Institute, Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Memorial Sloan Kettering Cancer Center, MIT's Koch Institute for Integrative Cancer Research, and The University of Texas MD Anderson Cancer Center.

The Foundation is supported by a Board of Directors from the five partner institutions and a Scientific Advisory Board of U.S. cancer experts. The Foundation was launched with an extraordinary challenge pledge of $250 million from Mr. and Mrs. William H. Goodwin, Jr. and their family, and the estate of William Hunter Goodwin III.

For further information, please visit the Foundation's website at www.breakthroughcancer.org.

References

1. Surveillance, Epidemiology, and End Results (SEER) Program. Cancer Stat Facts: Acute Myeloid Leukemia (AML). https://seer.cancer.gov/statfacts/html/amyl.html
2. Weeks LD, Ebert BL. Causes and consequences of clonal hematopoiesis. Blood. 2023;142(26):2235-2245.
3. Savona M, Bewersdorf JP, Allen SL, et al. FDA approval summary: decitabine and cedazuridine tablets for myelodysplastic syndromes. Clin Cancer Res. 2022;28(16):3411-3414.

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SOURCE Break Through Cancer