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Funded Grants

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Gangliosides in Huntington’s disease: from bench to clinics and back

Project Overview

Huntington disease (HD) is an inherited disorder that causes progressive malfunctioning and death of parts of the brain that control movement, reasoning and emotions. HD patients also suffer from depression and personality changes.  There is no cure for HD. Symptoms progress steadily until patients can no longer conduct a normal life and eventually die. The cause of HD is known: people with HD have a mutated protein called huntingtin which becomes toxic. One of the reasons why mutated huntingtin is toxic is that it causes the brain to produce insufficient amounts of a molecule called GM1 that is important for healthy brain function. Supplementation of GM1 to mouse models of HD dramatically improves the symptoms of the disease and slows down the underlying neurodegenerative process. This suggests that GM1 could be a potential treatment for HD.  In order to facility clinical use of GM1 or similar molecules, the first goal of our proposal is to determine how exactly GM1 works.  We will look at thousands of proteins in brain cells from mice with HD and from HD patients, and determine which proteins are beneficially affected by GM1. This will help us to understand how GM1 works and whether there are additional molecules that could be exploited for HD therapy. We will also determine whether GM1 exerts its therapeutic activity by regulating the function of the immune cells of the brain and inflammation. A second goal of the proposal is to determine whether the levels of GM1 (and related molecules) can be monitored in the blood and brain fluid of HD patients to gain information on disease and brain damage progression. This information would help in clinical trials, by showing whether the drugs tested are working decreasing mutated huntingtin toxicity.

Principal Investigator

Simonetta Sipione , University of Alberta

Team Members

Jonathan Curtis, University of Alberta

Sylvain Chouinard, CHUM Research Centre, University of Montréal

Oksana Suchowersky, University of Alberta

Justyna Sarna, University of Calgary

Richard Fahlman, University of Alberta

Partners and Donors

Huntington Society of Canada

Project Ongoing

Gangliosides in Huntington’s disease: from bench to clinics and back

  • Program Type

    Team grants

  • Area of research

    Neurodegeneration

  • Disease Area

    Other

  • Competition

    Brain Canada-Huntington Society of Canada Creating HD Clinician-Scientist-to-Patient Virtual Networks Multi-Investigator Research Initiative (MIRI)

  • Province

    Alberta

  • Start Date

    2016

  • Total Grant Amount

    $950,000

Contact Us

1200 McGill College Avenue
Suite 1600, Montreal, Quebec
H3B 4G7

+1 (514) 989-2989 info@braincanada.ca

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