Melanoma. ResearchAlliance Accelerating Progress: Moving Toward a Cure for Melanoma 2008-2011 Report Our First Four Years A period of landmark advances in melanoma research and treatment. “This is clearly the year of melanoma…” George W. Sledge, Jr., M.D., American Society of Clinical Oncology Past President - June 2011 LETTER FROM THE CHAIR AND PRESIDENT n March, an This report offers a immunotherapy called retrospective of the four I Yervoy (ipilimumab) years since the founding became the first drug of MRA, including the approved by the U.S. Food progress we have made, and Drug Administration a highlight of scientific (FDA) in 13 years for findings, and a look to metastatic melanoma. Then the molecularly the future of melanoma research. MRA funding has targeted agent Zelboraf (vemurafenib), an been vital to unlocking the best treatments and inhibitor of a mutated protein found in about continues to offer hope for better therapies and half of melanomas, was approved by the FDA ultimately a cure. in August. These breakthroughs underscore As the pace of progress quickens, MRA is remarkable progress that has been unfolding in re-doubling its efforts to accelerate research. the four years since the founding of the Melanoma With more than $30 million awarded for promising Research Alliance (MRA), a unique organization research, MRA has catalyzed strategic and collabo- whose mission is to find and fund the most rative research efforts to discover new biomarkers promising research worldwide. MRA is working of treatment response, demystify drug resistance to better prevent, detect early and treat deadly mechanisms, and improve effectiveness through skin cancer, toward a day when no one suffers combination therapies. MRA is working with all or dies from melanoma. who care about defeating melanoma, moving from No one person or organization can win the “the year of melanoma” to “the year of the battle against melanoma alone. We are thankful melanoma cure” as quickly as possible. to all who have engaged with us in this fight— DEBRA BLACK, co-founder and chair researchers, supporters, allies, partners, and more. WENDY K.D. SELIG, president and CEO MRA 2008-2011 About MRA 03 Thanks to the ongoing generous support of About MRA its founders, MRA applies 100 percent of Strategic public donations to its research program. under MRA is a public charity formed in 2007 GROWTH OF MRA’S RESEARCH PROGRAM the auspices of the Milken Institute, with generous $35 founding support from Debra and Leon Black. The 2011 $30 mission of MRA is to accelerate the pace of scientific discovery and its translation in order to eliminate $25 2010 suffering and death due to melanoma. Thanks to the $20 ongoing support of its founders, MRA applies 100 2009 percent of public donations to its research program. $15 Funding (Millions) $10 2008 In its first four years, MRA has committed $30.4 million $5 to 73 research programs with the potential to make significant, near-term clinical impact in prevention, 0 diagnosis, staging, and treatment. MRA is the largest pri- MRA research awards provide an important and unique vate funder of melanoma research in the United States. source of funding that addresses the gap in translational science, which is a critical stage in moving scientific TOTAL COMMITMENTS BY AWARD TYPE discoveries into tools and treatments for patients. Pilot/Dev Young ($0.7M) Hallmarks of the MRA research portfolio include: ($2.8M) Partnership ($0.3M) Established • Focus on collaborative team science: Team ($4.2M) Science Awards, which fund multidisciplinary research groups, account for almost three-quarters of research funding to date, followed by a variety of individual Team awards: Established Investigator, Young Investigator, ($22.4M) Pilot/Development Awards, and Partnership Awards. THE FIRST FOUR YEARS MRA has committed $30.4 million to 73 research programs. It is the largest private funder of melanoma research in the United States. $30.4 MILLION Melanoma. www.curemelanoma.org 04 ResearchAlliance Strategic About MRA Strategic • Investment in developing new treatments for metastatic disease: Nearly 80 percent of research funding is directed at melanoma treatment, followed by diagnosis/staging and prevention. In particular, MRA is investing significant resources to improve upon the two new treatment approaches— immune checkpoint targeting and selective BRAF inhibition. • Advances in key scientific and clinical areas: MRA-funded research has made progress in all of the key areas of opportunity identified at its inaugural Call to Action meeting in 2007. Because of TOTAL COMMITMENTS BY SCIENCE AREA the rapid progress that has been made, MRA has Prevention updated and revised this agenda for the next four years. ($2.8) Diagnosis/Staging • Treatment ($3.7M) Support for a diverse group of melanoma ($24.0M) researchers worldwide: MRA is funding 114 Principal Investigators at 56 institutions in 10 countries (Australia, Belgium, Canada, Germany, Israel, Netherlands, New Zealand, Sweden, Switzerland, and the United States). NEARLY 80 PERCENT Nearly 80 percent of research funding is directed at melanoma treatment, followed by diagnosis/ 114 staging and prevention. PRINCIPAL INVESTIGATORS MRA 2008-2011 Strategic 05 Global Presence Location of Principal Investigators leading MRA-funded awards, 2008-2011 I Canada Europe United States 8 G Israel New VZealand Australia B Cs9 F D L Australia Canada Israel New Zealand United States Chicago, IL Cleveland, OH Westmead Toronto Jerusalem Wellington Phoenix, AZ Boston, MA Philadelphia, PA Herston Montreal Ramat Gan La Jolla, CA Cambridge, MA Providence, RI Melbourne Sweden Los Angeles, CA Baltimore, MD Nashville, TN Sydney Germany Netherlands Lund Oakland, CA Ann Arbor, MI Houston, TX Frankfurt Amsterdam Pasadena, CA Detroit, MI Salt Lake City, UT Belgium Heidelberg Groningen Switzerland San Francisco, CA St. Louis, MO Charlottesville, VA Leuven Leiden Zurich Santa Monica, CA Princeton, NJ Seattle, WA Maastricht Stanford, CA Cold Spring Harbor, NY Rotterdam Tampa, FL New York, NY Champaign, IL Rochester, NY Melanoma. www.curemelanoma.org 06 ResearchAlliance Collaborative About MRA Collaborative Collaboration is at MRA's core—from the contributions from an industry partner whose team approaches to research that it funds participation is essential to the project. MRA and the to the way it finds partners who can help partner company will jointly support research with a realize its vision. Relationships built with high likelihood of clinical translation to help patients other nonprofits, corporations, and individuals and those at risk for melanoma. continue to increase: • MRA has co-funded research projects with fellow • MRA and Stand Up to Cancer (SU2C) are jointly cancer research foundations, including the Canadian funding a Melanoma Dream Team of at least $6 Cancer Society, Cancer Research Institute, and million beginning in early 2012. This ground- Melanoma Research Foundation. breaking award represents one of the largest single private grants for melanoma research and the first collaboration of its kind with SU2C. • In its first ever such offering, MRA selected two Academic-Industry Partnership Awards in 2011 that will support projects in conjunction with matched Relative Contribution of Pigmentation and PREVENTION Other Genes to Melanoma Risk Funded by an MRA Team Science Award, Drs. Nicholas Hayward and Nicholas Martin (Queensland Institute of Medical Research) and Dr. Graham Mann (Westmead Institute for Cancer Research and University of Sydney) discovered five new melanoma risk genes that conferred an increased risk of melanoma between 14 and 22 percent. In contrast to other known risk genes, none of these appear to be associated with Relative contribution of pigmentation physical characteristics such as fair and other genes to skin or moles. melanoma risk MRA 2008-2011 Collaborative 07 • Through its growing Allies program, MRA has raised awareness of melanoma and funds for melanoma research with nearly 50 companies who believe in its mission. For example, with the help of key Corporate Allies, including Claire’s, SkinCeuticals, and The Sports Authority, MRA reached as many as 19 million people with information about melanoma while raising nearly $350,000 for melanoma research during Melanoma Awareness Month in May 2011. MRA also leveraged its affiliation with the Milken Institute by hosting an outreach event on the closing day of the 2011 Global Conference in Beverly Hills. The event, supported by Corporate Allies Graff, Montage and Style Network , introduced the work of MRA to dozens of new supporters. Expanded collaborations with additional companies that share the MRA mission, as well as non-profit organizations like the Girl Scout Council of the Nation’s Capital, When longtime member of Bruce Springsteen's E Street Band Danny Federici (center) was diagnosed with melanoma, the Danny Fund are underway. was launched. • When Danny Federici, longtime member of Bruce Through its growing Allies program, Springsteen’s E Street Band, passed away from MRA has raised awareness of melanoma in April 2008, his family and friends melanoma and funds for melanoma received an outpouring of support. Together with research with nearly 50 companies MRA Co-Founders Debra and Leon Black, they brought their Danny Fund under the MRA umbrella, who believe in its mission. MRA working to bring melanoma to the forefront of reached as many as 19 million people public awareness and to fund research around the with information about melanoma world.