15 Jul Small Molecule Shows Therapeutic Potential in Huntington’s Disease Model
MedicalResearch.com Interview with:
Dr. Aleksey Kazantsev
Aleksey G. Kazantsev, PhD
Associate Professor in Neurology,
Harvard Medical School
Drug Discovery Laboratory
MassGeneral Institute for Neurodegenerative Disease,
Massachusetts General Hospital
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: There is no cure (disease modify therapy) for any neurodegenerative disorders (ND), most common Alzheimer’s and Parkinson’s or orphan like Huntington’s diseases. Numerous studies show that the pathologies of neurodegenerative diseases at molecular levels are similar but highly complex. No single neurodegenerative mechanism has emerged as predominant, slowing a development of efficient therapy.
While gene and cell-based therapeutic approaches are still evolving, we relay on discovery of small molecule drugs as essentially the only strategy with approved track record in human subjects. However, so far a traditional approach of targeting single cellular pathway was unsuccessful for CNS drug development.
The current study demonstrated a novel approach of using small molecule with multiple putative neuroprotective activities, which is essentially a combinatorial approach to use compound distinct activities to ameliorate/bock/prevent not one, but a few neurodegenerative pathways.
MedicalResearch.com: What should readers take away from your report?
Response: The study shows a feasibility to identify a small molecule with distinct and independent putative neuroprotective activities, which are expected to confer cumulative or maybe even synergistic therapeutic benefits for human subjects with Huntington’s Diseases and other CNS indications.
MedicalResearch.com: What recommendations do you have for future research as a result of this study?
Response: MIND4 is a great starting template for drug development, and we have promising preliminary results in two mouse models of neurodegneraive. Nevertheless, we need to optimize the pharmacology to meet FDA requirement for a next generation of analogs and identify a clinical candidate to be tested in human subjects.
MedicalResearch.com: Is there anything else you would like to add?
Response: Since one of identified compound activity is activation of NRF2 signaling, a major defense mechanism against oxidative and environmental stress and inflammation, it is expected that the discovered novel class of small molecules can be used as protective agents in a broad range of human diseases and healthy aging and not limited to neurodegeneration.
MedicalResearch.com: Thank you for your contribution to the MedicalResearch.com community.
Citation:
IRT2- and NRF2-Targeting Thiazole-Containing Compound with Therapeutic Activity in Huntington’s Disease Models
Luisa Quinti, Malcolm Casale, Se´ bastien Moniot, …, Donald C. Lo, Leslie M. Thompson, Aleksey G. Kazantsev
Cell Press: Quinti et al., 2016, Cell Chemical Biology 23, 1–13 July 21, 2016 ª 2016 Elsevier Ltd. http://dx.doi.org/10.1016/j.chembiol.2016.05.015
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Last Updated on July 15, 2016 by Marie Benz MD FAAD