Public Release: 

MMRF leads initiative to accelerate development of targeted therapies for multiple myeloma

Collaboration of government, industry and academic researchers would enable simultaneous evaluation of multiple investigational therapies under one 'master protocol'

Feinstein Kean Healthcare


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Credit: MMRF

Norwalk, Conn., Nov. 5, 2014 - The Multiple Myeloma Research Foundation (MMRF) today announced an initiative designed to accelerate the evaluation of new investigational therapies for multiple myeloma (MM).The MMRF, in collaboration with the U.S. Food and Drug Administration (FDA), the National Cancer Institute (NCI), pharmaceutical, biotech and diagnostic industry members, academic center leaders and patients, initiated the formal development of a Master Protocol to allow patients to participate in clinical trials evaluating several investigational therapies at once.

A workshop to advance the MM Master Protocol, attended by more than 80 participants, was held in Washington, D.C. on October 22, 2014. During the meeting, attendees from the FDA, NCI, industry, academia and the patient community endorsed a plan to move forward with the development of the complete master study protocol.

"We have made real progress in the battle against multiple myeloma, but there is still more work to do to bring new, effective, personalized treatments to patients," said Walter M. Capone, President and Chief Executive Officer of MMRF. "The development of a Multiple Myeloma Master Protocol offers a promising solution to accelerate the development of critical targeted therapies to patients through a novel research model."

Complementing the pursuit of novel drug classes and immunotherapies in MM drug development is the promise of identifying new targeted therapies for patients with specific genetic alterations. The MM Master Protocol would enable several, Phase II clinical trials to take place in parallel within a single study design. Patients would be assigned to an appropriate experimental therapy based on an analysis of their genetic alterations. This novel approach - which has recently been applied in other cancers - allows multiple new therapies to be evaluated more rapidly than with a traditional trial design. Decisions to advance an investigational drug to Phase III testing in a specific genetic subgroup, or to test a different drug in this subgroup, can be expedited - with the goal of getting new drugs to patients faster than ever before.

"Myeloma is an incredibly complex, heterogeneous disease. There is an acute need for new therapies, particularly for patients whose cancer has relapsed," said Shaji Kumar, M.D., Mayo Clinic College of Medicine. "The evidence suggests that this is the right approach to accelerate the development of targeted treatments. When finalized, this Master Protocol will be a major landmark for the multiple myeloma community."

In addition to discussing protocol design, meeting participants addressed the application of minimal residual disease (MRD) measurements as surrogate endpoints for MM clinical trials. Multiple reports suggest that MRD is a strong predictor of long-term progression free survival (PFS) in multiple myeloma.

"It is critical that we build the Master Protocol with all stakeholders in partnership," said Dr. Kumar. "MRD measurement, in the context of an innovative trial design like the Master Protocol, is an important tool that can ultimately make drug trials more efficient and less costly, thus speeding the delivery of better care to patients."

The MM Master Protocol is a biomarker-driven, clinical trial that matches patients with relapsed disease with targeted therapies based on the genomic profile of their tumor. This approach builds on the work of MMRF's Multiple Myeloma Genomics Initiative (MMGI), which established novel connections between the disease and important biomarkers and molecular targets. A key example of this is MMGI's discovery, confirmed by other researchers, that the BRAF gene plays a role in a subset of myeloma cases. This type of genomics research has accelerated the ability of scientists to identify specific disease subtypes and design therapies to treat them successfully, particularly in cases where existing therapies are no longer effective.

"The workshop accelerated the development of a Master Protocol and is enabling stakeholders to collaborate and to leverage cutting edge science to bring the right treatments to patients as quickly as possible," said Mr. Capone. "We are honored to work with partners across the spectrum of patient care to drive this concerted effort and work together toward a cure."


About Multiple Myeloma (MM)

Multiple myeloma (MM) is a cancer of the plasma cell. It is the second most common blood cancer. An estimated 24,050 adults (13,500 men and 10,550 women) in the United States will be diagnosed with MM in 2014 and an estimated 11,090 people are predicted to die from the disease. The five-year survival rate for MM is approximately 43%, versus 28% in 1998.

About the Multiple Myeloma Research Foundation (MMRF)

The Multiple Myeloma Research Foundation (MMRF) was established in 1998 as a 501(c)(3) non-profit organization by twin sisters Karen Andrews and Kathy Giusti, soon after Kathy's diagnosis with multiple myeloma. The mission of the MMRF is to relentlessly pursue innovative means that accelerate the development of next-generation multiple myeloma treatments to extend the lives of patients and lead to a cure. As the world's number-one private funder of multiple myeloma research, the MMRF has raised $250 million since its inception and directs nearly 90% of total budget to research and related programming. As a result, the MMRF has been awarded Charity Navigator's coveted four-star rating for 11 consecutive years, the highest designation for outstanding fiscal responsibility and exceptional efficiency. For more information about the MMRF, please visit:

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