North Bethesda, MD, February 8, 2022—The Foundation for the National Institutes of Health (FNIH) Biomarkers Consortium (BC) has launched a project to validate new methods of detecting and quantifying the small amount of cancer cells remaining in the body following anti-cancer therapy, or measurable residual disease (MRD), in patients with acute myeloid leukemia (AML). The use of MRD as a new prognostic and diagnostic biological indicator, or biomarker, will allow for rapid adjustment of treatment in AML patients. According to the National Cancer Institute (NCI), an estimated 20,240 new AML cases were diagnosed in the United States in 2021, and the five-year survival rate is less than 30% across all age groups¹. Long-term, the project hopes to generate important molecular information to better understand this devastating disease and ultimately improve patient outcomes.

For patients with either acute or chronic leukemia, the main obstacle to full recovery is relapse, defined as the return of actively growing cancer after a patient’s disease is stable with ongoing treatment. The presence of MRD is recognized as a strong predictor of relapse in leukemia, but its detection has not been standardized within clinical trial designs or for its association with outcome measures. MRD has been used to guide treatment decisions in chronic myeloid leukemia (CML), and recent collaborative efforts, including those within the FNIH BC, have demonstrated that it is similarly valuable in guiding treatment decisions in acute lymphoblastic leukemia (ALL). For patients with AML, MRD is also strongly associated with relapse, but detection of MRD is technologically more difficult to perform and standardize due to the more variable nature of this cancer type, thereby preventing its use in AML treatment decisions thus far.

This four-year, $6.5M FNIH project brings together critical resources and stakeholders to investigate using genetic tests to improve the accuracy of current MRD detection approaches and help establish MRD as a validated biomarker in AML. Project partners include the NCI, the National Heart Lung and Blood Institute (NHLBI), the U.S. Food and Drug Administration (FDA), 12 private sector partners, and two academic partners. These partners will work collaboratively to test new technologies against established methods for MRD detection and to generate and advance better measures for clinical trial design and drug development, in four key steps:

1. establish a library of reference materials for researchers to use as a benchmark for assay development;
2. create a process and standards through which assays and new technologies can be developed and tested;
3. validate new assays and develop a publicly available MRD toolbox for researchers; and
4. collect data to support MRD as a validated indicator of treatment results in clinical trials.

Validating and standardizing MRD as a biomarker will enable scientists to compare data from multiple clinical trials of various treatments, ultimately speeding development of new therapies for patients with AML. In addition, the critical knowledge gained through this approach could improve real-time patient management and patient care, leading to a higher overall survival rate for patients living with this disease.

For more information about the project click here. To read what our partners are saying about the project click here.

About the Biomarkers Consortium:
The Foundation for the National Institutes of Health’s Biomarkers Consortium (BC) convenes government, industry, patient advocacy groups, and not-for-profit organizations to address one of the most pressing needs in the diagnosis and treatment of disease: the development and the seeking of regulatory approval for disease biomarkers and surrogates. The core operations of the Biomarkers Consortium are supported through the contributing membership program. Organizations representing private industry (including the pharmaceutical, biotechnology, diagnostics, and information technology industries) and not-for-profit organizations (including associations, advocacy groups, trade organizations, and philanthropic organizations) that wish to support biomarkers development are eligible to become contributing members.

About the Foundation for the National Institutes of Health:
The Foundation for the National Institutes of Health (FNIH) creates and manages alliances with public and private Institutions in support of the mission of the NIH, the world’s premier medical research agency. The Foundation, also known as the FNIH, works with its partners to accelerate biomedical research and strategies against diseases and health concerns in the United States and across the globe. The FNIH organizes and administers research projects; supports education and training of new researchers; organizes educational events and symposia; and administers a series of funds supporting a wide range of health issues. Established by Congress in 1990, the FNIH is a not-for-profit 501(c)(3) charitable organization.

¹Data from NCI SEER data sheets.