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Preclinical development of a disease modifying small molecule therapy for Alzheimer disease

Project Overview

No medications exist that can stop or even slow the progression for Alzheimer Disease (AD). Several dramatic late stage clinical failures have heightened recognition that novel approaches need to be quickly pursued in order to develop useful treatments for the aging population. The two pathological hallmarks of AD are protein aggregates deposited in the brain that are known as tangles and plaques. These aggregates form from inappropriately modified forms of the microtubule associated protein tau and peptide fragments, known as Aβ which are generated by cleavage of the amyloid precursor protein. Dr. Vocadlo and team have recently pioneered a new potential approach that has been shown to block disease progression in animal models of AD by blocking the toxicity of both of Aβ and tau. Their approach centers on a specialized sugar unit that is found attached to nuclear and cytoplasmic proteins, including both tau and APP. The multidisciplinary team now aims to address the key remaining challenges that would clear the way for a promising new therapeutic target to advance to the clinic. These findings will enable the rapid advance of these optimized molecules into formal toxicology studies and downstream trials.

Principal Investigator

David Vocadlo , Simon Fraser University

Team Members

Gideon Davies, York University

Sharon Gorski, Simon Fraser University

Leonard Foster, University of British Columbia

Cheng-Xin Gong, Institute for Basic Research in Developmental Disabilities

Ian Mackenzie, University of British Columbia

Howard Feldman, University of British Columbia

Michael Silverman, Simon Fraser University

Ging-Yuek Hsiung, University of British Columbia

Robert Britton, Simon Fraser University

Cheryl Wellington, University of British Columbia

Partners and Donors

Michael Smith Foundation for Health Research

Genome BC

Project Ongoing

Preclinical development of a disease modifying small molecule therapy for Alzheimer disease

  • Program Type

    Team grants

  • Area of research

    Neurodegeneration

  • Disease Area

    Alzheimer’s

  • Competition

    BC Alzheimer's Research Award Program

  • Province

    British Columbia

  • Start Date

    2015

  • Total Grant Amount

    $1,410,000

  • Health Canada Contribution

    $705,000

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

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