Huntington’s disease is an inherited progressive form of dementia that affects motility, personality and memory as the disease advances. Huntington’s affects about 30,000 Americans with a prevalence of 1 in every 10,000 people. The prevalence of the disease is between 3-7 per 100,000 in populations of Western European decent. Huntington’s is a genetic disease with an autosomal dominant trait and can be inherited from either parent. The general onset of the disease typically occurs between the ages of 30 to 45 and is characterized by changes in movement, moods and decline of mental abilities. Because the disease is progressive, the quality of life for patients steadily declines as their ability to think, speak and walk is diminished. There is no cure for Huntington’s and treatment focuses on reducing symptoms to help control emotional and movement issues.
Researchers at the University of Michigan have developed small molecule agonists of Heat Shock Protein (HSP70). Heat shock proteins are cellular chaperones that play a pivotal role in maintaining protein homeostasis by facilitating re-folding or degradation of mis-folded intermediates. Huntington’s disease is characterized by neuronal aggregation of the Huntingtin protein and the cells inability to handle the abnormally folded protein. HSPs are the first line of defense against the accumulation of the disease protein and are a potential therapeutic target for Huntington’s disease. The small molecules could potentially be useful for other diseases characterized by protein aggregation such as Alzheimer’s disease.
Applications and Advantages
- Potent agonists of HSP70 as a therapeutic-nl-for Huntington’s Disease
- Potential therapeutic for other diseases-nl-associated with protein accumulation-nl-(i.e. Alzheimer’s disease)
- Potentially a long-term therapy with minimal side effects