Are immunotherapies for Huntington’s disease a realistic option?


There is compelling evidence that the pathophysiology of many neurodegenerative diseases includes dysregulation of the immune system, with some elements that precede disease onset. However, if these alterations are prominent, why have clinical trials targeting this system failed to translate into long-lasting meaningful benefits for patients? This review focuses on Huntington’s disease, a genetic disorder marked by notable cerebral and peripheral inflammation. We summarize ongoing and completed clinical trials that have involved pharmacological approaches to inhibit various components of the immune system and their pre-clinical correlates. We then discuss new putative treatment strategies using more targeted immunotherapies such as vaccination and intrabodies and how these may offer new hope in the treatment of Huntington’s disease as well as other neurodegenerative diseases.

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FC is a recipient of a Researcher Chair from the Fonds de Recherche du Québec en Santé (FRQS) 35059 providing salary support and operating funds, and receives funding from the Canadian Institutes of Health Research (CIHR) MOP326050 to conduct her HD-related research. FL holds a Joseph Demers scholarship award from Université Laval and HLD a Desjardins scholarship from the Fondation du CHU de Québec. We would like to thank Mr. Gilles Chabot for artwork.

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Correspondence to Francesca Cicchetti.

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Denis, H., Lauruol, F. & Cicchetti, F. Are immunotherapies for Huntington’s disease a realistic option?. Mol Psychiatry 24, 364–377 (2019).

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