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Recent therapeutic prospects for Machado-Joseph disease
1Department of Neurology, Michigan Medicine, University of Michigan, Ann Arbor, Michigan, USA.
Current Opinion in Neurology
|July 14, 2020
Summary
New therapeutic strategies are emerging for Machado-Joseph disease (MJD), also known as spinocerebellar ataxia type 3 (SCA3). These promising treatments target DNA, RNA, and cellular pathways for this fatal neurodegenerative disorder.
Area of Science:
- Neurodegenerative diseases
- Genetics
- Drug discovery
Background:
- Machado-Joseph disease (MJD), or spinocerebellar ataxia type 3 (SCA3), is a fatal, inherited neurodegenerative disorder.
- It is caused by a CAG repeat expansion in the ATXN3 gene.
- Currently, no treatments modify the disease's progression.
Purpose of the Study:
- To review recently developed therapeutic strategies for MJD/SCA3.
- To highlight promising treatments that may offer disease modification for this incurable condition.
Main Methods:
- Review of preclinical and early-stage development therapeutic approaches.
- Focus on strategies targeting DNA, RNA, protein quality control, and cellular homeostasis.
Main Results:
- Several novel therapies show potential, including DNA-targeted and RNA-targeted approaches.
- Antisense oligonucleotides, short hairpin RNAs, and citalopram are candidates for clinical trials.
- Pharmacological agents like naphthyridine-azaquinolone and autophagosome-tethering compounds demonstrate therapeutic capacity.
Conclusions:
- A range of innovative therapeutic options are on the horizon for MJD/SCA3.
- These strategies offer hope for future disease-modifying treatments for this fatal neurodegenerative disease.
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