A new experimental therapy for ALS and fronto-temporal dementia

Journal of Clinical Investigations graphical abstract

Jean-Pierre Julien's team recently published an article in the high impact journal, the Journal of Clinical Investigations, about a new experimental therapy for ALS and frontotemporal dementia based on the use of antibodies that target the abnormal accumulation of a protein called TDP-43 in degenerating neurons. The formation of TDP-43 aggregates is associated with ALS development.

The researchers produced an antibody which targets a region of TDP-43 implicated in abnormal aggregation of the protein.  Administration of this antibody lead to an improvement int the cognitive and motor performances and to reduce the pathological changes in mouse models of ALS and fronto-temporal dementia.  

This study is the first to show it is possible to target aggregation of TDP-43 with an antibody, and opens the door to the development of immunotherapies for these devastating neurodegenerative diseases.  

Read the original research article in the  Journal of Clinical Investigations:

Pozzi S, Thammisetty SS, Codron P, Rahimian R, Plourde KV, Soucy G, Bareil C, Phaneuf D, Kriz J, Gravel C, Julien JP. Viral-mediated delivery of antibody targeting TAR DNA-binding protein 43 mitigates associated neuropathology. J Clin Invest. 2019 Jan 22. pii: 123931. doi: 10.1172/JCI123931.

Read an article in Le Fil de l'Université Laval:


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