Multiple Myeloma Research Foundation Announces Publication of First Whole-Genome Sequence Analysis for Multiple Myeloma

Report in the journal Nature a critical step

Norwalk, CT — March 23, 2011

The Multiple Myeloma Research Foundation (MMRF) today hailed a new publication in Nature as a major step forward in the organization’s efforts to advance a personalized medicine approach in multiple myeloma. Researchers at the Eli and Edythe L. Broad Institute of Harvard and MIT today published an analysis of 38 multiple myeloma genome sequences – the largest such data set ever published on multiple myeloma. The authors uncovered novel connections between multiple myeloma and important molecular targets, at least one of which could have immediate clinical significance. Drugs against this target are already showing promise in melanoma, underscoring the potential importance of these findings for cancer types beyond multiple myeloma.

The work was conducted as part of the Multiple Myeloma Genomics Initiative (MMGI), a genome-mapping program supported by the Multiple Myeloma Research Foundation (MMRF) to significantly advance biological understanding of the disease. The analysis was conducted using a combination of whole-genome sequencing and whole-exome sequencing, performed on patient samples gathered via the Multiple Myeloma Research Consortium (MMRC), a network of 16 collaborating academic medical centers, and organized into a centralized tissue bank. Funding for the project, which is ongoing, comes from the MMRF.

“It is critical to improve our understanding of the complex biology of multiple myeloma in order to accelerate the development of next-generation treatments, and eventually find a cure,” said Kathy Giusti, Founder and CEO of the MMRF and MMRC, and a multiple myeloma patient. “Our vision is that upon diagnosis, patients will have access to a range of treatments, chosen based on individualized insights from a patient’s molecular profile. Today’s publication is an important step toward that goal, and a foundation upon which the scientific community can build to help improve the treatment of patients with multiple myeloma and other cancers.”

The Nature paper describes connections between multiple myeloma and several molecular pathways, including those associated with protein production and epigenetic regulation (histone methylation). Additionally, oncogenic mutations were seen in the kinase BRAF, which was not previously associated with multiple myeloma, but which is a therapeutic target in clinical trials for melanoma. A follow-up analysis revealed that 4% of 161 multiple myeloma samples harbored a BRAF mutation, including the specific BRAF mutation that is the target of the drug being developed in melanoma by Roche and Plexxikon (PLX4032). Beyond the specific implications for multiple myeloma, the results underscore the valuable role that large sample cancer genome sequencing can play in uncovering new insights into cancer not anticipated by existing knowledge.

Todd Golub, M.D., Director of the Broad Institute’s Cancer Program and Charles A. Dana Investigator in Human Cancer Genetics at the Dana-Farber Cancer Institute and principal author on the study, said: “The vast majority of the discoveries highlighted in our paper today would not have been possible by sequencing only a single multiple myeloma genome, underscoring the vital role that this initiative is playing in elucidating the processes involved in multiple myeloma. These findings are a testament to the vision and effectiveness of the MMRF’s efforts in genomics and personalized medicine, which have rapidly advanced to become an invaluable resource to the broad scientific community.”

About the Multiple Myeloma Genomics Initiative
The MMGI is a $12 million, six-year program that comprises several research and discovery efforts spanning the spectrum of genome science. The MMRC contributes patient samples from its 16 collaborating academic members via a centralized Tissue Bank; the MMRF provides funding for the project. The comprehensive genomic survey of MMRC samples is conducted in collaboration with the Eli and Edythe L. Broad Institute of Harvard and MIT and the Translational Genomics Research Institute (TGen). The MMGI comprises several research and discovery efforts spanning the spectrum of genome science. Array comparative genomic hybridization (aCGH), gene expression profiling (GEP), DNA methylation analysis and DNA sequencing are among the assays performed on hundreds of patient multiple myeloma tumor tissue. Data from the MMGI and other multiple myeloma genomics efforts is available to the scientific community through the Multiple Myeloma Genomics Portal, the world's only myeloma-specific repository of genomic data (www.myelomagenomics.org).

To continue the momentum of these study findings, the MMRF has granted Biotech Investment Awards to two organizations focused on epigenetic targets. Altogether, the MMRF is investing more than $5 million in programs to drive the rapid translation of these findings into effective treatments for multiple myeloma.

About Multiple Myeloma
Multiple myeloma is an incurable blood cancer. The five-year relative survival rate for multiple myeloma is approximately 38 percent, one of the lowest of all cancers. In 2010, approximately 20,000 adults in the United States will be diagnosed with multiple myeloma and approximately 11,000 are predicted to die from the disease in 2011.

About the Multiple Myeloma Research Foundation
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 over $160 million since its inception to fund more than 130 laboratories worldwide. An outstanding 90% of funds raised go toward research and related programming. The MMRF has supported 70 new compounds and approaches in clinical trials and pre-clinical studies and has facilitated 30 clinical trials through its sister organization, the MMRC. For more information about the MMRF, visit www.themmrf.org.

About the Multiple Myeloma Research Consortium
The Multiple Myeloma Research Consortium (MMRC) is a 509(a)3 non-profit organization that integrates leading academic institutions to accelerate drug development in multiple myeloma. It is led from MMRC offices in Norwalk, Conn., and comprises 16 member institutions. Baylor Charles A. Sammons Cancer Center at Dallas, City of Hope, Dana-Farber Cancer Institute, Emory University's Winship Cancer Institute, the John Theurer Cancer Center at Hackensack University Medical Center, H. Lee Moffitt Cancer Center & Research Institute, Mayo Clinic, Ohio State University, Mount Sinai School of Medicine, Sarah Cannon Research Institute, University Health Network (Princess Margaret Hospital), University of California-San Francisco, University of Chicago, University of Michigan, Virginia Cancer Specialists, and Washington University.

The MMRC was founded in 2004 by Kathy Giusti, a myeloma patient, and with the help of the scientific community. The MMRC is a sister organization to the Multiple Myeloma Research Foundation (MMRF), the world's leading funder of multiple myeloma research. The MMRC is widely recognized as an optimal research model to rapidly address critical challenges in drug development and to explore opportunities in the today's most promising research areas in genomics, compound validation, and clinical trials. The MMRC is the only consortium to join academic institutions through membership agreements, customized IT systems, and an integrated tissue bank. For more information, please visit www.themmrc.org.

Multiple Myeloma Research Foundation

Anne Quinn Young, MMRF, 203-652-0212
Lynn Blenkhorn, Feinstein Kean Healthcare, 508-851-0930
Greg Kelley, Feinstein Kean Healthcare, 617-461-4023