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Translational and Clinical Pharmacology Considerations in Drug Repurposing for X-linked Adrenoleukodystrophy-A Rare Peroxisomal Disorder
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  • Julianne Tieu,
  • Siddhee Sahasrabudhe,
  • Paul Orchard,
  • James Cloyd,
  • Reena Kartha
Julianne Tieu
University of Minnesota Twin Cities
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Siddhee Sahasrabudhe
University of Minnesota
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Paul Orchard
University of Minnesota
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James Cloyd
University of Minnesota
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Reena Kartha
University of Minnesota
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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 (PK/PD) 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 development. Although there is a good understanding of X-ALD pathophysiology, the absence of information on drug targets, pharmacokinetics, and pharmacodynamics hinders the repurposing of drugs for this condition. Incorporating advanced translational and clinical pharmacological approaches in preclinical studies and early stages clinical trials will improve the success of repurposed drugs for X-ALD as well as other rare diseases.

Peer review status:ACCEPTED

06 Jul 2021Submitted to British Journal of Clinical Pharmacology
06 Jul 2021Submission Checks Completed
06 Jul 2021Assigned to Editor
12 Jul 2021Reviewer(s) Assigned
30 Jul 2021Review(s) Completed, Editorial Evaluation Pending
02 Aug 2021Editorial Decision: Revise Minor
09 Sep 20211st Revision Received
11 Sep 2021Submission Checks Completed
11 Sep 2021Assigned to Editor
11 Sep 2021Review(s) Completed, Editorial Evaluation Pending
11 Sep 2021Editorial Decision: Accept