Repurposing Approved and Abandoned Drugs for the Treatment and Prevention of Cancer through Public–Private Partnership

The cancer medical community, perhaps more than any other, understands the dire need for new, innovative therapies for the estimated 11,714,000 Americans living with cancer (1). While our understanding of the biology and genetics of cancer has increased dramatically in recent decades, the pace of discovery, development, and registration of new drug therapies for the treatment, prevention, and control of cancer has not. This is especially true for the many rarer forms of cancer. Indeed, many cancers meet the Orphan Drug Act definition of <200,000 prevalence in the United States (2). Understanding cancer in this light and making meaningful inroads into the development of targeted therapies for these rare diseases requires new thinking, new approaches, and new collaborations.On July 6, 2010, leaders from industry, government, academia, and nonprofit organizations participated in a national Town Hall co-sponsored by The University of Kansas Cancer Center (KUCC), the Ewing Marion Kauffman Foundation, the Kansas Bioscience Authority, the Friends of Cancer Research, and the Council for American Medical Innovation (CAMI). This Town Hall meeting, entitled “The New Role of Academia in Drug Discovery and Development,” drew attention to academia's changing role in translational research. Today, academic institutions are poised and challenged unlike ever before to meet society's expectation that breakthrough technologies and biomedical discoveries—funded by the federal government and made within these institutions—be moved through the therapeutic product development process to benefit human health.Several policy leaders participated in the Town Hall meeting. U.S. Department of Health and Human Services (HHS) Secretary Kathleen Sebelius opened the meeting with a keynote address describing HHS's support for this new, powerful challenge. NIH Director Francis Collins and U.S. Food and Drug Administration (FDA) Commissioner Margaret Hamburg joined former U.S. Majority Leader and CAMI Chairman Richard Gephardt to discuss agency perspectives on how to foster innovation and collaboration. In December 2010, the Town Hall co-sponsors released a white paper entitled “The New Role of Academia in Drug Discovery and Development: New Thinking, New Competencies, New Results,” which reflects key recommendations coming out of the event (3).In January 2011, Secretary Sebelius further called for strategic partnerships between industry, government, academia, and nonprofit organizations to build “a stronger foundation for a new century of treatments and cures” (4). Responding to this challenge, three organizations with forward-thinking strategies and unique respective drug repurposing experiences established The Learning Collaborative to advance therapies for blood cancers more efficiently than any of the component organizations could do alone. This collaboration represents the future in cancer drug development—a team effort that brings together unparalleled strengths in the various disciplines needed to advance a treatment from the laboratory to regulatory approval as rapidly as possible. The Learning Collaborative is a dedicated collaboration between the NIH Chemical Genomics Center (NCGC) and its Therapeutics for Rare and Neglected Diseases (TRND) program, The Leukemia & Lymphoma Society (LLS), and KUCC to discover and develop new drug therapies for rare blood cancers. Specifically, The Learning Collaborative combines the disease expertise and network of the LLS, the cancer drug repurposing and drug development expertise of the KUCC, and the drug discovery expertise of the NCGC to create a pipeline of new therapies to treat blood cancers using less traditional forms of drug discovery and development. The partners have a shared commitment not only to discovering and developing new molecular entities (NME) for the treatment of rare blood cancers but also to exploring new uses for approved and abandoned drugs. The Learning Collaborative is advancing blood cancer–focused projects from target identification and validation through clinical proof-of-concept.While The Learning Collaborative is focused on both NMEs and repurposing, this commentary emphasizes drug repurposing strategies for two reasons. First and foremost, repurposing approved and abandoned drugs for cancer represents an opportunity to rapidly advance to patients promising drug therapies by capitalizing on existing data and experience. The same holds true for abandoned or “shelved” drug candidates, agents whose development was discontinued for any number of non-safety–related reasons. NIH has taken notice of the important potential of repurposed and abandoned drugs (5). In April 2011, Dr. Francis Collins convened a meeting with research and development leaders from the pharmaceutical industry in Bethesda, MD entitled