In normal development, synaptic pruning occurs to get rid of less active synapses to produce more efficient brain action, but this is altered in neurodegenerative diseases such as Huntington’s disease (HD). Dysregulated C1q deposition occurs, and as a result of this deposition and general neurodegeneration, neuroinflammation develops and cell loss occurs. In Alzheimer’s disease, amyloid is one of the triggers for C1q, and in other conditions, it could be cell debris. However, Rajeev Kumar, MD, Rocky Mountain Movement Disorders Center, Englewood, CO, explains that, in HD, they do not know the trigger; they simply know it is present. This is because knocking out C1q improves function, and the use of a C1q antibody also improves function. Targeting C1q is very different from primary genetic therapies as it targets a common phenomenon in other neurodegenerative diseases and could be applied to these as well. Currently, there are Phase II studies in ALS and Phase III studies shortly in Guillain-Barre syndrome, as well as promising preliminary results for the non-neurological disease, cold agglutin disease. This interview took place at the AAN Annual Meeting 2023 in Boston, MA.
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