Peptide Inhibitor of VCP/Huntingtin Interaction to Treat Huntington’s Disease

Background

Mitochondria primarily play a role in the regulation of energy metabolism in cells, but have also been implicated in several human diseases including heart failure, diabetes, cancer and neurodegenerative diseases, such as Huntington’s Disease (HD). HD is a fatal inherited disease for which there is currently no cure. Available treatments are aimed at providing symptomatic relief, but do not alter the path of the disease.

Technology Overview

Researchers at Case Western Reserve University have identified a protein, Valosin‐containing protein (VCP), which interacts with the Huntingtin gene (mtHtt) on mitochrondria. A peptide inhibitor has been developed that selectively blocks the protein‐protein interaction between VCP and Htt, which has shown efficacy in a variety of HD models.

Further Details:

‑ Peptide Treatment Corrected Neurite Degeneration

‑ Treatment corrected mitochondrial dysfunction and neuronal cell death.

Benefits

• Reduced neuropathology of HD
• Increased body weight and survival of HD mice
• Reversed motor deficits (long‐term treatment)
• Corrected biomarkers of mitochondrial dysregulation (in mouse plasma)