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
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.
MedicalResearch.com: What should readers take away from your report?
Response: I think there are three main messages:
a. Deuteration of small molecules for pharmaceutical use is feasible for CNS drugs.
b. We can safely and effectively reduce hyperkinetic movement disorders, such as chorea, over 12 weeks. Longer studies are currently underway to examine the longer term safety and efficacy.
c. Use of deutetrabenazine resulted in improvements in quality of life and overall improvement beyond chorea.
MedicalResearch.com: What recommendations do you have for future research as a result of this study?
Response: This study was over 12 weeks and a longer term study is underway to further examine the safety and efficacy of treatment with deutetrabenazine. Clinicians, patients and families will need to consider how this emerging potential therapy fits in with other commonly used treatments in Huntington Disease.
MedicalResearch.com: Is there anything else you would like to add?
Response: We are grateful to the patients and families who participated in this study since research in Huntington Disease would not be possible without them. We are also hopeful that this drug, if approved by the FDA, provides another treatment option making Huntington Disease an increasingly treatable condition.
MedicalResearch.com: Thank you for your contribution to the MedicalResearch.com community.
Effect of Deutetrabenazine on Chorea Among Patients With Huntington Disease A Randomized Clinical Trial
Huntington Study Group
JAMA. 2016;316(1):40-50. doi:10.1001/jama.2016.8655
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