Investigational Deutetrabenazine Reduces Chorea in Huntington Disease

MedicalResearch.com Interview with:

Samuel Frank, MD Director of the Huntington’s Disease Society of America Center of Excellence Beth Israel Deaconess Medical Center Boston, MA 02215

Dr. Samuel Frank

Samuel Frank, MD
Director of the Huntington’s Disease Society of America Center of Excellence
Beth Israel Deaconess Medical Center
Boston, MA 02215

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Huntington Disease is a hereditary, progressive neurodegenerative disease characterized by involuntary movements (chorea and dystonia), cognitive dysfunction and psychiatric symptoms. Deutetrabenazine is a novel molecule containing deuterium, which attenuates CYP2D6 metabolism, increases active metabolite half-lives leading to stable systemic exposure. We found that deutetrabenazine significantly reduces chorea. There was also an overall improvement in participants’ condition based on patient and clinician measures and improvement in a quality of life measure. There was no worsening, but also no improvement in balance. The improvements in Huntington Disease were seen with a remarkably good safety and tolerability profile.

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Clinical and Genetic Associations of Patients At Risk of Huntington Disease

Kevin M. Biglan, M.D., M.P.H Professor of Neurology and the Associate Chair for Clinical Research Department of Neurology and the Center for Human Experimental Therapeutics University of Rochester School of Medicine and Dentistry Rochester, New York

Dr. Biglan

MedicalResearch.com Interview with:
Kevin M. Biglan, M.D., M.P.H
Professor of Neurology and the Associate Chair for Clinical Research
Department of Neurology and the Center for Human Experimental Therapeutics
University of Rochester School of Medicine and Dentistry
Rochester, New York 

Medical Research: What is the background for this study? What are the main findings?

Dr. Biglan: A therapeutic goal of research in Huntington Disease (HD) is the identification of treatments that delay the progression of disease and onset of illness in individuals at risk for developing manifest HD. Designing such efficacy trials is challenging. A major hurdle is the lack of practical primary outcome measures to assess the effect of an intervention on delaying disease onset. Use of the dichotomous endpoint of clinical diagnosis as the primary outcome requires large sample sizes and long duration of follow up in order to show a significant therapeutic effect on delaying disease onset. Continuous measures that can reliably distinguish cytosine-adenine-guanine (CAG) expanded individuals in the pre-manifest period may allow for the identification of potential disease modifying therapies using relatively smaller cohorts followed for shorter periods of time.

The Prospective Huntington At-Risk Observational Study (PHAROS) represents the largest observational study to clinically evaluate pre-manifest Huntington Disease wherein both research participants and investigators were unaware of Huntington Disease mutation status. Accordingly, PHAROS was uniquely designed to address, in an unbiased manner, those clinical features most associated with the CAG expansion during the prodromal phase in  Huntington Disease.  The identification of continuous outcome measures that are associated with HD in the pre-manifest period may facilitate the design and powering of future studies of potential disease modifying therapies prior to traditional motor diagnosis.

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Huntington Disease Affects Driving Skill Even in Early Stages

Hannes Devos, PhD Assistant Professor Assistant Director Georgia Regents University Driving Simulator Lab Department of Physical Therapy College of Allied Health Sciences Georgia Regents University Augusta, GA 30912MedicalResearch.com Interview with:
Hannes Devos, PhD
Assistant Professor
Assistant Director Georgia Regents University Driving Simulator Lab Department of Physical Therapy
College of Allied Health Sciences
Georgia Regents University Augusta, GA 30912

MedicalResearch.com: What are the main findings of the study?

Dr. Devos: We compared on-road driving performance between 30 active drivers with Huntington disease and 30 age- and gender- matched control drivers. We found that Huntington disease affects all levels of driving skill due to motor and cognitive deficits and leads to unsafe driving, even in the early stages of the disease. Fourteen (47%) drivers with Huntington disease failed the road test compared with none of the controls.

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