Drug Repurposing for Rare Diseases. (April 2021)

• Record Type: Journal Article
• Authors: Roessler, Helen I.; Knoers, Nine V.A.M.; van Haelst, Mieke M.; van Haaften, Gijs
• Is Part Of: Trends in pharmacological sciences. Volume 42:Number 4(2021)
• Page Start: 255
• Page End: 267
• Publication Date: 2021-04
• Subjects: drug discovery -- drug repositioning -- drug repurposing -- genetic diseases -- rare diseases; Pharmacology -- Periodicals; Pharmacology -- trends -- Periodicals; Pharmacologie -- Périodiques; Pharmacology; Electronic journals; Periodicals; 615.1
• DOI: 10.1016/j.tips.2021.01.003
• Languages: English
• ISSNs: 0165-6147

Abstract

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. Highlights: The awareness and interest of the public, media and legislative bodies in the field of rare diseases has been growing consistently. Researchers continue to successfully uncover the molecular and genomic drivers, as well as the clinical course of many rare conditions, due to advances in DNA sequencing technologies and big data analysis, resulting in high expectations for new and optimized treatments. Due to cost effectiveness and a reduced timeline, the process of repurposing drugs for new indications represents an alternative method for finding rare disease treatments with compelling advantages over traditional drug development. The development of systematic approaches to repurpose compounds has led to the identification of promising candidate drugs, some of which are in advanced stages of clinical trials or already approved, with the potential for use in the treatment of rare diseases.