Brain cholesterol therapy for Huntington's disease – Does it make sense?

The article discusses the potential use of cholesterol as a therapy for Huntington's disease (HD). HD is a neurodegenerative disorder that currently lacks disease-modifying treatments. Cholesterol is crucial for neuronal function and membrane integrity in the brain, and studies have shown that cholesterol biosynthesis is reduced in the HD brain. Various strategies, such as direct infusion, gene therapy, and nanoparticle delivery, have been explored to raise cholesterol levels in the HD mouse brain and have shown promising results. Clinical trials to restore cholesterol biosynthesis in HD patients have been initiated but paused until 2025. However, challenges such as dosage, delivery methods, and reliable biomarkers need to be addressed before cholesterol therapy can be implemented in human trials. Overall, the article suggests that the rationale and evidence for exploring cholesterol therapy in HD are strong, and it is now appropriate to move forward with early phase clinical trials. [Extracted from the article]