Huntington’s disease is a devastating, hereditary neurodegenerative disease affecting about 1 out of every 10,000 people in the USA, Europe and Australia. It typically causes involuntary movements of the face and body and dementia. Symptoms worsen over time, eventually leaving the affected person totally dependent on help from others. There are no effective therapies available today. How might stem cell research lead to new treatments?
Huntington’s Disease (HD) mainly affects nerve cells in the brain called medium spiny neurons (MSNs). MSNs receive and coordinate information from other neurons in the brain to control movement of the body, face and eyes.
In Huntington’s Disease large numbers of MSNs are damaged and destroyed. Some other types of neurons in the brain also appear to be affected, such as cortical neurons. Patients usually first notice symptoms when they are around 35-50 years old, typically weak spasmodic movements of the muscles in the face and limbs.
As the disease progresses, these spasmodic movements become more evident and frequent and other symptoms appear. Patients are affected in different ways, but symptoms may include difficulty speaking and swallowing, dementia or trouble concentrating.