New translational research platform launched to study rare cancers

The University of Texas MD Anderson Cancer Center and the Broad Institute of MIT and Harvard today announced the launch of a new translational research platform to study rare cancers and develop a first-of-its-kind resource for the scientific community. The joint initiative will create a catalog of rare cancer models and provide a data resource to accelerate the identification of therapeutics to treat patients diagnosed with rare tumor types.

The National Cancer Institute defines a rare cancer as one with fewer than 40,000 new cases per year. Cumulatively, rare cancers account for roughly one-quarter of all cancer cases and cancer deaths, but the low incidence of each different type of rare tumor presents a significant challenge to efforts to identify effective therapeutic approaches.

"Through this initiative, we hope to overcome some of the challenges that have prevented effective translational research in rare cancers," said Timothy Heffernan, Ph.D., head of oncology research in MD Anderson's Therapeutics Discovery division. "By collaborating with the Broad Institute, we have a tremendous opportunity to create a valuable resource for the entire scientific community that will inspire and catalyze a wave of innovative research to advance impactful new therapies to patients in need."

MD Anderson is the world leader in the diagnosis and treatment of rare cancers; more than 5,000 patients with the rarest diagnoses seek treatment annually. In 2019, MD Anderson established the Rare Tumor Initiative, a multidisciplinary effort to comprehensively characterize rare tumors throughout the course of each patients' care. This new joint translational research platform builds upon MD Anderson's existing commitment to rare cancers with the goal of enabling rapid translation of new therapeutic insights.

This collaborative effort between MD Anderson and Broad will generate models of rare tumors from MD Anderson patients at the Broad's Cancer Cell Line Factory, which works to expand the number and variety (in terms of tumor types and cancer genotypes) of cancer models available for research. These models will be analyzed extensively by the Broad's Cancer Dependency Map project, an effort to systematically detect genetic and pharmacologic vulnerabilities in cancer in order to identify molecular signatures that could inform novel therapeutic approaches.

Treatments for rare cancers have lagged behind common tumors in large part because we as a community lack the tools to study and understand their unique biology in the laboratory. This initiative represents a significant opportunity to close that gap and to start identifying new treatment options for patients with rare cancers. We are thrilled to be able to work closely with our colleagues at MD Anderson towards that goal."

William Sellers, M.D., Director of the Broad's Cancer Program

The initiative aims to generate more than 100 rare cancer models, and to make those models and associated molecular data available to all members of the cancer research community.

"At MD Anderson, our singular mission is to end cancer," said Giulio Draetta, M.D., Ph.D., chief scientific officer at MD Anderson. "This collaboration is an excellent example of our focus on meeting the needs of all of our patients through innovative research approaches and is directly aligned with the themes of our institutional strategy. We are excited to have this opportunity to leverage the exceptional science and expertise at the Broad Institute to accelerate new treatments for patients."