Understanding the Physiopathology of ALS to Develop New Therapies
Amyotrophic lateral sclerosis (ALS) is characterizedby the progressive loss of motor neurons (the neurons that control musclemovement). Most ALS patients die within five years of being diagnosed, and thereis no effective treatment.
Mutations in several heterogenic genes are known to beassociated with the familial form of ALS, but the causes in most sporadic-typeALS cases remain unknown. While not all forms of the disease are caused by thesame genetic anomalies, ALS is marked by the accumulation of a protein called TDP-43in the cytoplasm of the affected neurons.
Dr. Jean-Pierre Julien, Canada Research Chair inNeurodegeneration, is internationally recognized for his work in ALSphysiopathology. The first aim of Julien’s research team is to shine a light onthe mechanisms that contribute to the onset and spread of TDP-43 proteinaccumulations, with the help of unique transgenic mice lines generated in hislaboratory. They are also studying the role of exosomes in the intercellulartransfer of pathogenic factors and the role of the immune response in ALS.
The second aim of the research is to design innovativetreatments for ALS, including therapeutic antibodies targeting the accumulationof specific proteins like TDP-43 and compounds that can mitigate neuroinflammation.
Julien’s work will lead to new therapeutic approaches,offering real hope not only to ALS patients, but to people suffering from otherbrain diseases involving abnormal protein accumulation, such as fronto-temporaldementia, Alzheimer’s disease and Parkinson’s disease.