Drug Repurposing for Rare Diseases

Helen I Roessler; Nine V A M Knoers; Mieke M van Haelst; Gijs van Haaften

doi:10.1016/j.tips.2021.01.003

Drug Repurposing for Rare Diseases

Trends Pharmacol Sci. 2021 Apr;42(4):255-267. doi: 10.1016/j.tips.2021.01.003. Epub 2021 Feb 6.

Authors

Helen I Roessler¹, Nine V A M Knoers², Mieke M van Haelst³, Gijs van Haaften⁴

Affiliations

¹ Department of Genetics, Center for Molecular Medicine, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands.
² Department of Genetics, Center for Molecular Medicine, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands; Department of Genetics, University Medical Center Groningen, Groningen, The Netherlands.
³ Department of Clinical Genetics, Amsterdam University Medical Center, Location AMC, University of Amsterdam, Amsterdam, The Netherlands; Department of Clinical Genetics, Amsterdam University Medical Center, Location VUMC, VU University Amsterdam, Amsterdam, The Netherlands.
⁴ Department of Genetics, Center for Molecular Medicine, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands. Electronic address: G.vanHaaften@umcutrecht.nl.

PMID: 33563480
DOI: 10.1016/j.tips.2021.01.003

Abstract

Currently, there are about 7000 identified rare diseases, together affecting 10% of the population. However, fewer than 6% of all rare diseases have an approved treatment option, highlighting their tremendous unmet needs in drug development. The process of repurposing drugs for new indications, compared with the development of novel orphan drugs, is a time-saving and cost-efficient method resulting in higher success rates, which can therefore drastically reduce the risk of drug development for rare diseases. Although drug repurposing is not novel, new strategies have been developed in recent years to do it in a systematic and rational way. Here, we review applied methodologies, recent accomplished progress, and the challenges associated in drug repurposing for rare diseases.

Keywords: drug discovery; drug repositioning; drug repurposing; genetic diseases; rare diseases.

Publication types

Research Support, Non-U.S. Gov't
Review

MeSH terms

Drug Repositioning*
Humans
Orphan Drug Production
Rare Diseases* / drug therapy