Multiple Myeloma (MM) 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 (SMM). These conditions affect ~3% of people over 50 years old and are up to three times more likely to occur in African Americans and in people with a family history of MM. Annually, ~1% of MGUS cases progress into SMM, of which half will then progress to MM within 2 years.

Early therapeutic intervention at the SMM stage has shown improved progression-free survival (PFS) and prevention of morbidity from end-organ damage. Currently, the only ways to assess a patient’s risk of progression from MGUS or SMM to MM require a bone marrow biopsy, which is invasive and presents risk to patients. These evaluations are also relatively simplistic, relying on manual estimates which are limited in accuracy and sensitivity. Consequently, most patients are not evaluated for risk and are not treated until they develop MM. There is an urgent need to accurately define the patient population at risk for developing MM and to intervene early to prevent end-organ damage and improve survival.

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

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

This project will use genetic sequencing of bone marrow and peripheral blood samples to develop prognostic biomarkers and monitoring tools for response to therapy in patients with MGUS and SMM. Using blood-based evaluations in lieu of painful bone marrow biopsies would reduce patient discomfort and disease monitoring cost. This less invasive method is also expected to increase utilization of early assessments of disease progression to generate opportunities for faster intervention, improved options for treatment, and hopefully, subsequent increase in patient response and survival.


  • National Cancer Institute (NCI)
  • U.S. Food and Drug Administration (FDA) 
  • The Broad Institute
  • Dana-Farber Cancer Institute
  • University of Navarra
  • AbbVie
  • Pfizer Inc.
  • Regeneron Pharmaceuticals, Inc.
  • 10x Genomics, Inc.
  • Adaptive Biotechnologies

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