Translational and clinical pharmacology considerations in drug repurposing for X-linked adrenoleukodystrophy—A rare peroxisomal disorder

Julianne H. Tieu, Siddhee A. Sahasrabudhe, Paul J. Orchard, James C. Cloyd, Reena V. Kartha

Research output: Contribution to journalReview articlepeer-review

1 Scopus citations

Abstract

X-linked adrenoleukodystrophy (X-ALD) is an inherited, neurodegenerative rare disease that can result in devastating symptoms of blindness, gait disturbances and spastic quadriparesis due to progressive demyelination. Typically, the disease progresses rapidly, causing death within the first decade of life. With limited treatments available, efforts to determine an effective therapy that can alter disease progression or mitigate symptoms have been undertaken for many years, particularly through drug repurposing. Repurposing has generally been guided through clinical experience and small trials. At this time, none of the drug candidates have been approved for use, which may be due, in part, to the lack of pharmacokinetic/pharmacodynamic information on the repurposed medications in the target patient population. Greater consideration for the disease pathophysiology, drug pharmacology and potential drug–target interactions, specifically at the site of action, would improve drug repurposing and facilitate drug development. Incorporating advanced translational and clinical pharmacological approaches in preclinical studies and early-stage clinical trials will improve the success of repurposed drugs for X-ALD as well as other rare diseases.

Original languageEnglish (US)
Pages (from-to)2552-2563
Number of pages12
JournalBritish Journal of Clinical Pharmacology
Volume88
Issue number6
Early online dateSep 23 2021
DOIs
StatePublished - Jun 2022

Bibliographical note

Publisher Copyright:
© 2021 British Pharmacological Society

Keywords

  • X-linked adrenoleukodystrophy
  • clinical pharmacology
  • drug repositioning
  • drug repurposing
  • neurodegenerative diseases
  • rare diseases
  • translational pharmacology

PubMed: MeSH publication types

  • Journal Article
  • Review

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