FOUNDATIONS AND DRUG DEVELOPMENT: AN EXAMPLE

The Multiple Myeloma Research Foundation (MMRF), which was founded in 1998, is the largest private funder of multiple myeloma research, raising over $190 million to date. It funds research around the world, builds community among people affected by multiple myeloma, and partners with the Multiple Myeloma Research Consortium (MMRC), which supports research in 16 institutions to determine which drugs can move from preclinical testing into Phase I and Phase II development and which was founded in 2004. Walter Capone of the MMRF and the MMRC described the organizations and the vision behind their approach.

Multiple myeloma is the second most common blood cancer, affecting 64,000 people in the United States and causing more than 10,000 deaths annually. It occurs largely among older adults, often African American, and predominantly male. In 1998 the average survival period with the disease was 3 years and no drugs were in the pipeline. Today, the average survival period is 7 years, 4 drugs for the disease have been approved, and 9 drugs are in Phase III trials.

MMRF’s success has been based on increasingly complex collaborative models to drive drug development. By developing strategic plans and building community, MMRC has accelerated trials and built a tissue bank that includes more than 3,500 samples. Through the Multiple Myeloma Genomics Initiative, more than 80 of these samples have been sequenced and are available through an open-access portal, with plans to sequence 250 samples by the end of 2012. More than 1,000 researchers have accessed the data, and more are expected in the future. Interestingly, while expected mutations were found through the genomic analysis, 4 percent of patients also had activating BRAF mutations, compelling the foundation to examine the use of vemurafenib for treatment of multiple myeloma.

This effort in turn has supported the Multiple Myeloma Personalized Medicine Initiative, which seeks to more fully characterize the range of disease subtypes to enable the development of targeted therapies and potentially curative approaches for patients. Spread across 50 centers and including industry partners, the project combines a 1,000-patient longitudinal study with a companion genomics study that will comprehensively assess the molecular profiles of patient’s tumors throughout disease progression and be correlated to clinical interventions, including treatment regimens. The study design allows 3 years for enrollment and includes 5 years of follow-up, and data will be open access with no intellectual property restrictions.

The MMRF is continuing to expand its programs as the network of stakeholders in the field grows more complex. Today, not just academia, industry, and patients are involved but also regulators, physicians, payers, and diagnostic and platform companies. By working with clinicians,



The National Academies | 500 Fifth St. N.W. | Washington, D.C. 20001
Copyright © National Academy of Sciences. All rights reserved.
Terms of Use and Privacy Statement