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Updated: Jan 31, 2026
Whole-brain Segmentation and Change-point Analysis of Anatomical Brain MRI—Application in Premanifest Huntington's Disease
Published on: June 9, 2018
Recent advances in the therapeutic development for Huntington disease
1Parkinson's Disease and Movement Disorders Center, Division of Neurology, Department of Medicine, The Ottawa Hospital Research Institute, The University of Ottawa Brain and Mind Institute, Canada.
Insights
Huntington disease (HD) research shows promising gene-targeting therapies, including antisense oligonucleotides (ASO), that may slow neurodegeneration. New symptomatic treatments are also being explored for motor and mood symptoms.
Area of Science:
- Neuroscience
- Genetics
- Pharmacology
Background:
- Huntington disease (HD) is a rare genetic neurodegenerative disorder.
- Genetic diagnosis in HD offers a model for developing neurodegenerative disease therapies.
- Current treatments for HD primarily manage chorea symptoms.
Purpose of the Study:
- To review current and emerging therapeutic strategies for Huntington disease.
- To assess disease-modifying and symptomatic treatments in clinical trials.
- To identify key areas for future HD therapeutic development.
Main Methods:
- Comprehensive literature review of Huntington disease clinical trials.
- Searched major medical databases (PubMed, Web of Science) and registries.
- Included studies focused on disease modification or new symptomatic indications.
Main Results:
- Huntingtin-lowering strategies, particularly antisense oligonucleotides (ASO), show potential for disease modification.
- Early ASO trials indicate safety and reduced mutated huntingtin protein (mHTT).
- Ongoing trials explore novel symptomatic treatments for motor and mood impairments.
Conclusions:
- The future of Huntington disease therapy is promising with novel approaches targeting core disease mechanisms.
- Clinical research platforms like Enroll-HD are crucial for advancing HD clinical trials.
- Gene-targeting and symptomatic therapies offer hope for improved patient outcomes.
Introduction:
Huntington disease (HD) is a rare genetic neurodegenerative condition. The availability of a genetic diagnosis makes HD an attractive model for the development of therapies that can delay or, at best, halt the progression of neurodegenerative conditions. Tetrabenazine and deutetrabenazine are the only treatment options with a formal indication (chorea) for this patient population.
Methods:
Literature review on HD and clinical trials using the medical databases Pubmed, Web of Science, and clinical trial registries. Recent clinical trials conducted with the goal of disease-modification or new symptomatic treatment indications were included. Non-pharmacological interventions were excluded.
Results:
Therapeutic approaches aiming at disease-modification include huntingtin-lowering strategies, the modulation of huntingtin homeostasis and neuroinflammation. Huntingtin-lowering strategies are of particular interest by targeting the mRNA of the huntingtin (HTT) gene at the core of HD biology. Antisense oligonucleotides (ASO) are the only huntingtin-lowering strategies in clinical development. The initial results suggest that the first non-allele specific ASO was safe and associated with a reduction in the levels of mutated huntingtin protein (mHTT). Other clinical trials for disease-modification in HD have generated negative results or are ongoing. Assays to measure CSF mHTT and brain nuclear imaging specific to HD can support the rational development of these therapies. Novel symptomatic treatment indications explored in clinical trials include motor disability, irritability and apathy.
Conclusions:
The years ahead are promising for novel and revolutionary therapies aimed at core disease mechanisms in HD. Clinical research platforms such as Enroll-HD are expected to potentiate the conduction of clinical trials in HD.
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