FNIH Biomarkers Consortium Announces Biomarker Project to Better Identify Patients at Risk for Progression to Multiple Myeloma

More accurate, less invasive prediction methods could lead to earlier treatment and better outcomes in those with precancerous conditions.

North Bethesda, MD, June 26, 2024— The Foundation for the National Institutes of Health (FNIH) Biomarkers Consortium (BC) announces the launch of MMyeRisk, a project to evaluate tumor and immune biomarkers to predict progression from early precursor conditions to multiple myeloma and identify patients likely to benefit from earlier treatment.

Multiple myeloma is the second most common hematologic malignancy in the US and is almost always preceded by precancerous conditions, including monoclonal gammopathy of undetermined significance (MGUS) and smoldering multiple myeloma. These conditions affect approximately 3% of people over 50 years old and are up to three times more likely to occur in African Americans and people with a family history of multiple myeloma. Annually, about 1% of MGUS cases progress into smoldering multiple myeloma, of which half will then progress to multiple myeloma within two years.

Early therapeutic intervention at the smoldering multiple myeloma stage has shown improved progression-free survival and prevention of morbidity from end-organ damage. Currently, the only way to assess a patient’s risk of progression from either condition to multiple myeloma requires a bone marrow biopsy, which is invasive and holds risks for patients. These evaluations are also relatively simplistic, relying on manual estimates that have limited accuracy and sensitivity. As a result, most patients are not evaluated for risk and are not treated until they develop multiple myeloma.

“There is an urgent need to accurately define the patient population at risk for developing multiple myeloma and to intervene early to prevent end-organ damage and improve survival,” said Stacey Adam, PhD, Vice President, Science Partnerships at FNIH.

This three-year, $4.5 million FNIH project brings together critical resources and stakeholders to develop prognostic biomarkers and monitoring tools for response to therapy in patients with MGUS and smoldering multiple myeloma. Project partners include the National Cancer Institute, part of the U.S. National Institutes of Health (NIH); the U.S. Food and Drug Administration (FDA); five private sector partners; and three academic partners. These partners will work collaboratively to accomplish two key aims:

• Develop a blood-based assessment of disease burden and tumor biology, as well as a prognostic biomarker for patients with MGUS/smoldering multiple myeloma.

• Validate the blood-based biomarkers to be used for identifying the high-risk population that will develop myeloma.

The development of blood-based evaluations that can be used in lieu of painful bone marrow biopsies is expected to reduce patient discomfort and disease-monitoring costs. This less invasive method is expected to increase utilization of early assessments of disease progression to generate opportunities for faster intervention, improved options for treatment, and hopefully, a subsequent increase in patient response and survival.

Public-sector partners
National Cancer Institute (NCI)
U.S. Food and Drug Administration (FDA)

Academic partners
The Broad Institute
Dana-Farber Cancer Institute
University of Navarra

Private-sector partners
Pfizer Inc.
Regeneron Pharmaceuticals, Inc.

Technology Collaborators
Adaptive Biotechnologies
10x Genomics, Inc.

Learn more at the MMyeRisk project page.

About the Biomarkers Consortium:
The Foundation for the National Institutes of Health’s Biomarkers Consortium leads cross-sector efforts to validate and qualify biomarkers that accelerate the development of new therapeutics and health technologies. The core operations of the Biomarkers Consortium are supported through its contributing membership program, which includes the NIH, the FDA, private industry, and not-for-profit organizations.

About the Foundation for the National Institutes of Health:
The Foundation for the National Institutes of Health (FNIH) builds public-private partnerships that connect leading biomedical scientists at the National Institutes of Health (NIH), life sciences companies, foundations, academia, and regulatory agencies, including the Food and Drug Administration and European Medicines Agency. Through team science, we solve complex health challenges and accelerate breakthroughs for patients, regardless of who they are or what health challenges they face. The FNIH accelerates new therapies, diagnostics, and potential cures; advances global health and equity in care; and celebrates and helps train the next generations of scientists. Established by Congress in 1990 to support the mission of the NIH, the FNIH is a not-for-profit 501(c)(3) charitable organization. For more information about the FNIH, please visit fnih.org.