Drug Discovery in Hematology

Advancing novel therapeutic agents for the treatment of malignancy into the marketplace is an increasingly costly and lengthy process. As such, new strategies for drug discovery are needed. Drug repurposing represents an opportunity to rapidly advance new therapeutic strategies into clinical trials at a relatively low cost. Known on-patent or off-patent drugs with unrecognized anticancer activity can be rapidly advanced into clinical testing for this new indication by leveraging their known pharmacology, pharmacokinetics and toxicology. Using this approach, academic groups can participate in the drug discovery field and smaller biotechnology companies early-stage drug discovery projects. Here, several scientific approaches used to identify drug repurposing opportunities are highlighted with a focus on hematologic malignancies. In addition a discussion of the regulatory issues that are unique to drug repurposing and how they impact developing old drugs for new indications is included. Finally, the mechanisms to enhance drug repurposing through increased collaborations between academia, industry and nonprofit charitable organizations are discussed.