Current status of drug repositioning in hematology

ABSTRACT

Introduction

Drug repositioning (DR) is defined as determining new therapeutic applications for existing drugs. This approach is advantageous over de novo drug discovery in accelerating clinical development, in terms of lower costs, a shortened development period, a well-known action mechanism, a feasible dosage, and an acceptable safety profile.

Areas covered

This work was aimed at reviewing agents with successful DR in hematology.

Expert opinion

Thalidomide and plerixafor have been successfully repositioned for treating multiple myeloma and harvesting peripheral blood stem cells, respectively. The former was originally developed as a sedative and the latter as an anti-HIV drug. Currently, the feasibility of repositioning various agents is being explored (e.g. an anti-influenza virus drug oseltamivir for primary immune thrombocytopenia, an anti-HIV drug abacavir for adult T-cell leukemia, and a macrolide antibiotic clarithromycin for multiple myeloma). Furthermore, bosutinib for chronic myeloid leukemia or the antiplatelet drug cilostazol have been suggested to have clinical benefits for the management of amyotrophic lateral sclerosis and ischemic stroke, respectively. To promote DR, effective application of artificial intelligence or stem cell models, comprehensive database construction shared between academia and pharmaceutical companies, suitable handling of drug patents, and wide cooperation in the area of specialty are warranted.

KEYWORDS:

• Drug repositioning
• thalidomide
• drug reprofiling
• artificial intelligence
• stem cell model

Article highlights

• Drug repositioning (DR) is an attractive approach for efficient clinical development as it is associated with lower costs and shorter periods.

• Compared to conventional drug discovery, DR is advantageous to clinical development for rare malignancies, the market size of which is small.

• Representative DR agents are thalidomide for multiple myeloma, plerixafor for harvesting peripheral blood stem cells, cilostazol for ischemic stroke, and zidovudine for adult T-cell leukemia.

• The DR approach is currently shifting from unexpected finding of novel action mechanisms as observed in the case of thalidomide to comprehensive drug reprofiling.

• The application of artificial intelligence and stem cell models, solution of problems related to patents, and close cooperation across the area of specialty are essential steps for future DR development.

Acknowledgments

We would like to thank Editage (www.editage.com) for English language editing.

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was not funded.