Identifying and Screening Small Molecules for the Treatment of Neurodegeneration in MS

Start Term
End Term
Funding Amount
$299,385
Affiliation(s)
University of Saskatchewan
Geographic Region(s) / Province(s)
Saskatchewan
Research Priorities
Progressive MS
Impact Goal(s)
Advance Treatment and Care
Understand and Halt Disease Progression

Researcher(s):  Michael C. Levin 

Summary:

  • Damage and death of nerve fibres, a process known as neurodegeneration, drives disease progression in multiple sclerosis (MS). Understanding what causes nerve fibre damage will lead to effective treatments for progressive MS. 
  • Dr. Michael Levin and team discovered that a specific protein called hnRNP A1 does not function properly in MS, causing neurodegeneration. In this study, the researchers will synthesize and screen for small molecule treatments that can target and rescue hnRNP A1 function to reduce neurodegeneration and decrease disease severity in animal models of MS. 
  • This research has the potential to develop new treatments for use in pilot clinical trials for progressive MS.

Project Description: 
Nerve fibre damage and death leads to neurodegeneration, a primary characteristic of MS and is the underlying reason why people living with MS get worse. The source of nerve fibre damage is not well understood, making it difficult to develop effective treatments that target progressive MS.

Dr. Michael Levin and team discovered a protein called ‘heterogeneous nuclear ribonucleoprotein A1’ or ‘hnRNP A1’ that does not function properly in MS. Malfunction of hnRNP A1 causes nerve fibre damage contributing to neurodegeneration, and in turn, disability in people with MS. In this study, the researchers will synthesize and screen for small molecule treatments that can rescue the function of hnRNP A1 and reduce nerve fibre damage in animal models of progressive MS.

Potential Impact: Current MS therapeutics are ineffective at targeting neurodegeneration and treating progressive MS. The findings of this research will provide insights into neurodegeneration in MS and potentially identify new treatments for use in pilot clinical trials for progressive MS.

Project Status: In Progress