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

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
  • Michael Smith Foundation for Health Research
  • Genome BC
  • Pacific Alzheimer Research Foundation (PARF)

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.