A Rational Approach to Drug Repositioning in β-thalassemia: Induction of Fetal Hemoglobin by Established Drugs

Author:

Prosdocimi MarcoORCID,Zuccato CristinaORCID,Cosenza Lucia CarmelaORCID,Borgatti MonicaORCID,Lampronti IlariaORCID,Finotti AlessiaORCID,Gambari RobertoORCID

Abstract

Drug repositioning and the relevance of orphan drug designation for β-thalassemia is reviewed. Drug repositioning and similar terms ('drug repurposing', 'drug reprofiling', 'drug redirecting', ‘drug rescue’, ‘drug re-tasking’ and/or 'drug rediscovery') have gained great attention, especially in the field or rare diseases (RDs), and represent relevant novel drug development strategies to be considered together with the “off-label” use of pharmaceutical products under clinical trial regimen. The most significant advantage of drug repositioning over traditional drug development is that the repositioned drug has already passed a significant number of short- and long-term toxicity tests, as well as it has already undergone pharmacokinetic and pharmacodynamic (PK/PD) studies. The established safety of repositioned drugs is known to significantly reduce the probability of project failure. Furthermore, development of repurposed drugs can shorten much of the time needed to bring a drug to market. Finally, patent filing of repurposed drugs is expected to catch the attention of pharmaceutical industries interested in the development of therapeutic protocols for RDs. Repurposed molecules that could be proposed as potential drugs for β-thalassemia, will be reported, with some of the most solid examples, including sirolimus (rapamycin) that recently has been tested in a pilot clinical trial.

Funder

Agenzia Italiana del Farmaco, Ministero della Salute

Wellcome Trust

FP7-HEALTH-2012

Publisher

F1000 Research Ltd

Subject

General Biochemistry, Genetics and Molecular Biology,Medicine (miscellaneous)

Reference65 articles.

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