Spinocerebellar ataxia clinical trials: opportunities and challenges

Sarah M. Brooker, Chandrakanth Reddy Edamakanti, Sara M. Akasha, Sheng Han Kuo, Puneet Opal*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

3 Scopus citations

Abstract

The spinocerebellar ataxias (SCAs) are a group of dominantly inherited diseases that share the defining feature of progressive cerebellar ataxia. The disease process, however, is not confined to the cerebellum; other areas of the brain, in particular, the brainstem, are also affected, resulting in a high burden of morbidity and mortality. Currently, there are no disease-modifying treatments for the SCAs, but preclinical research has led to the development of therapeutic agents ripe for testing in patients. Unfortunately, due to the rarity of these diseases and their slow and variable progression, there are substantial hurdles to overcome in conducting clinical trials. While the epidemiological features of the SCAs are immutable, the feasibility of conducting clinical trials is being addressed through a combination of strategies. These include improvements in clinical outcome measures, the identification of imaging and fluid biomarkers, and innovations in clinical trial design. In this review, we highlight current challenges in initiating clinical trials for the SCAs and also discuss pathways for researchers and clinicians to mitigate these challenges and harness opportunities for clinical trial development.

Original languageEnglish (US)
Pages (from-to)1543-1556
Number of pages14
JournalAnnals of clinical and translational neurology
Volume8
Issue number7
DOIs
StatePublished - Jul 2021

ASJC Scopus subject areas

  • Neuroscience(all)
  • Clinical Neurology

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