Repurposing Approved and Abandoned Drugs for the Treatment and Prevention of Cancer through Public-Private Partnerships

Abstract

The Learning Collaborative (TLC) 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. TLC is creating a pipeline of new therapies to treat blood cancers using less traditional forms of drug discovery and development. The Learning Collaborative is focused on both the discovery of new blood cancer treatments as well as identifying new uses for approved and abandoned drugs, termed drug repurposing. 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. TLC's first project, which is also a pilot project of the TRND program, focuses on repurposing an existing small molecule drug, auranofin, initially approved to treat rheumatoid arthritis in the mid-1980s. Auranofin is being evaluated by The Learning Collaborative as a treatment for relapsed chronic lymphocytic leukemia (CLL). In just two years after the identification of auranofin's activity in CLL cells, this project has entered clinical trials. The Learning Collaborative will engage a for-profit partner to advance auranofin through late stage drug development, registration and commercialization. TLC has the unique ability to "de-risk" promising drug therapies, like auranofin, using an innovative approach to quickly determine proof-of-concept. The partnership is named The Learning Collaborative because this model is scalable, applicable across a wide range of therapeutic areas, and replicable by organizations committed to innovative models of collaboration. TLC continues to explore solutions to barriers that include addressing regulatory science issues (with particular focus on repurposing off patent and abandoned drugs for rare and neglected diseases) and defining exclusivity path(s) to interest future for-profit partners.