Experimental Chemotherapy and Diabetes Medication Combination Shows Potential In Huntington’s Disease

MedicalResearch.com Interview with:

Audrey S. Dickey, Ph.D. Assistant Professor Department of Neurology, DUMC 2900 Durham, NC  27710

Dr. Dickey

Audrey S. Dickey, Ph.D.
Assistant Professor
Department of Neurology, DUMC 2900
Durham, NC  27710

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

Response: A drug already used to treat certain forms of cancer may also be an effective therapy for Huntington’s disease, according to a new study in the latest issue of Science Translational Medicine. The same study also increases our understanding of how this drug, and other medications like it, may offer hope for other neurodegenerative diseases like Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), and Parkinson’s disease.

Huntington’s disease is a devastating, inevitably fatal disease, with no medications that slow or stop disease progression. In this study, mice with the equivalent of Huntington’s disease became more mobile, recovered from neurodegeneration, and lived longer after being treated with Bexarotene. The same research builds on a 2016 study where Dr. Al La Spada, Dr. Audrey Dickey and colleagues showed that the drug KD3010 is an effective treatment for Huntington’s disease in mice and in human patient neurons made from stem cells.

MedicalResearch.com: What should clinicians and patients take away from your report?

Response: Senior author Al La Spada, MD, PhD, director of the forthcoming Duke Center for Neurodegeneration and Neurotherapeutics, said the study results are exciting not just because these drugs worked, but because of how they worked. “The pathways that these compounds are targeting are relevant to other neurodegenerative disorders and potentially the aging process itself, in addition to Huntington’s Disease.”

MedicalResearch.com: What recommendations do you have for future research as a result of this study?

Response: Audrey S. Dickey, PhD, who lead the research team, found that a combination treatment with low dosages of Bexarotene and KD3010 had an additive effect, higher than treatment by each alone.  Ideally, this would allow the optimal approach with minimal side effects.   We are currently carrying out further research on the underlying mechanisms of neuroprotection in addition to applying this research to other diseases with similar issues of mitochondrial dysfunction and protein quality control, such as Parkinson’s disease, Alzheimer’s disease and others. 

MedicalResearch.com: Is there anything else you would like to add?

Response: The study doesn’t mean that patients with Huntington’s disease or other conditions should rush out to get bexarotene or KD3010. Further research needs to determine how to use these drugs to treat these diseases in human patients. Bexarotene can have difficult side effects at high dosages, and optimal doses aren’t known, while KD3010 has only been tested in human subjects for type II diabetes. Future therapies for Huntington’s disease and other neurodegenerative conditions may take a cue from HIV treatments and involve a “cocktail” approach of combined medications.

MedicalResearch.com: Thank you for your contribution to the MedicalResearch.com community.

Citation:

PPARδ activation by bexarotene promotes neuroprotection by restoring bioenergetic and quality control homeostasis

Audrey S. Dickey1, Dafne N. Sanchez1, Martin Arreola1, Kunal R. Sampat1, Weiwei Fan2,Nicolas Arbez3, Sergey Akimov3,

Science Translational Medicine  06 Dec 2017:
Vol. 9, Issue 419, eaal2332
DOI: 10.1126/scitranslmed.aal2332

Note: Content is Not intended as medical advice. Please consult your health care provider regarding your specific medical condition and questions. 

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Last Updated on December 7, 2017 by Marie Benz MD FAAD