Drug repositioning in sarcomas and other rare tumors

Alex T.J Lee,Paul H Huang,Seth M Pollack,Robin L Jones

doi:10.1016/j.ebiom.2016.03.021

Abstract

Drug repositioning in sarcomas and other rare tumors

Highlights

The exploration and identification of new anti-cancer therapeutic indications for an approved drug already in use, or for a drug that was shelved for non-safety reasons in early clinical development, represents an attractive alternative to the costly, time-consuming and inefficient process of developing novel anticancer agents
The most significant example of drug repositioning in oncology has seen thalidomide and its analog lenalidomide approved for the treatment of multiple myeloma.(Engelhardt et al, 2014) Notoriously developed as a sedative and anti-emetic in the 1950s, thalidomide was decades later seen to have anti-angiogenic effects in animal models, research that translated into significant clinical activity in myeloma, acute myeloid leukemia and myelodysplastic syndrome
In many cases, lack of effective systemic therapies indicate a compelling case for drug repurposing studies in sarcoma

Summary

Introduction

The exploration and identification of new anti-cancer therapeutic indications for an approved drug already in use, or for a drug that was shelved for non-safety reasons in early clinical development, represents an attractive alternative to the costly, time-consuming and inefficient process of developing novel anticancer agents. Drug repositioning or repurposing can bypass the discovery and early clinical phases of cancer drug development, potentially reducing the hundreds of millions of dollars and more than a decade typically required to see a novel agent through to regulatory approval.

Results

Conclusion