Scientists today raise the first hope of a cure for Huntington's disease after unlocking the secrets of what goes wrong in the brains of sufferers. The scientists suggest that a drug being developed for use to treat breast cancer in America could be adapted for use in Huntington's patients to slow or even halt the progressive deterioration in their brains and the devastating emotional and physical consequences.
Patient groups cautiously welcomed what they called "early findings" and said it was particularly exciting as the drug is already being trialled for use.
Huntington's disease is an inherited condition which is usually first noticed in patients after the age of 30 when damage to the nerve cells in parts of the brain causes increasing behavioural and physical symptoms. What starts with forgetfulness and small tremors can deteriorate to involuntary jerking, rapid weight loss, deep depressions and violent outbursts over years or decades.
The research by Leeds University with scientists in Milan, America and King's College London, looked at the molecular pathways in the brain of people with Huntington's and found that the protein that wreaks havoc in their nerve cells is responsible for knock-on effects in 57 separate genes. The findings suggest that this is why the disease has such wide-ranging and catastrophic emotional and physical symptoms.
Scientists have now begun the painstaking work of matching the defective genes to some of the disease's symptoms. "This is a really exciting breakthrough," said researcher Lezanne Ooi.
"It's early days, but we believe our research could lead to radical changes in treatment for Huntington's sufferers. The fact that these cancer drugs have already been through clinical trials should speed up the time for this research to impact directly on patients.
"This is the first step to identifying the molecular pathways which may be important in Huntington's. This will help us develop other treatments. It's early stages but it could be incredibly important and offers an explanation as to why the symptoms of this disease are so broad."
The drug they are most interested in is trichostatin c, which scientists are working on to analyse its effects for a number of cancers. But the drug is so wide ranging that there have been concerns about serious side effects. The symptoms of Huntington's can be managed through medication, but there is no definitive treatment. This research provides a first step in developing a treatment that may even halt the onset of the disease, researchers said.
Cath Stanley, head of care services at the Huntington's Disease Association, said: "It is a devastating illness that affects whole families. Those who know they've inherited the faulty gene live in a shadow of uncertainty over how long [until] their symptoms start to develop."
The research was published in the Journal of Neuroscience.
