Huntington’s disease (HD) is an incurable, fatal neurodegenerative disease that typically develops in the third to fifth decade of life. Although HD is rare, its pathology is strikingly similar to other more common neurodegenerative diseases including Parkinson’s and Alzheimer’s disease. No effective treatment exists currently for HD, but using a chemogenomic approach University of Pittsburgh researchers have demonstrated that synergy in neuroprotective drug activity can be a more effective therapy for HD than any single drug therapy in current clinical trials. This discovery has the potential to reduce symptom severity in HD patients and halt progression of the disease, effectively prolonging lifespan. It may also be applicable to the development of treatments for other incurable neurodegenerative disorders.
Description
University of Pittsburgh researchers employed a well-established cellular model for HD and a cutting-edge chemogenomics approach to identify existing drugs and molecular pathways that protect neurons from HD-related cell death. They identified new indications of existing drugs based on their effects on the protective molecular targets, pathways, and networks identified. Researchers tested combinations of existing drugs with diverse molecular targets and associated pathways. They identified specific combinations that demonstrated synergistic activity, which resulted in up to 80 percent of cells having protection from toxicity.
Applications
· Individuals with neurodegenerative diseases
· Patients with Huntington’s disease
Advantages
· Treatment shows more efficacy than single-drug treatments for Huntington’s disease
· Discovery may help with the development of treatments for other incurable neurodegenerative disorders
Invention Readiness
In vitro data
IP Status
https://patents.google.com/patent/US11253594B2
