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Dr. Biglan[/caption]
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.
Dr. Samuel Frank[/caption]
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.
