"Congreso Mundial de Huntington"
NOVEDADES DE AUSTRALIA

A drug is being developed that scientists hope could slow the progress of Huntington's disease. HWT Image Library

A BREAKTHROUGH by Melbourne researchers is offering new hope to sufferers of devastating genetic disorder Huntington's disease.

There is no effective treatment for the disease, which has been described as a genetic time bomb.
Once symptoms become exposed, sufferers go into a decline that usually results in death in 10-25 years.
But there are hopes a drug called PBT2, being developed by scientists at the Melbourne Mental Health Research Institute at Melbourne University, could slow the deadly progress of the disease.

"The symptoms of Huntington's are an unholy combination of Parkinson's and Alzheimer's," the institute's Dr Robert Cherny said.

"It starts off with tremors and loss of co-ordination and then evolves into this very, very bizarre uncontrolled movement.

"That gets steadily worse and at the same time there is a loss of intellectual function and that evolves into dementia.

PBT2 was designed as a treatment for Alzheimer's and is being expanded to Huntington's, which has many of the same characteristics.

"Not only is there no cure, there is no real treatment available."

Animal trials of the drug have shown positive results, which are being presented in Melbourne today at the world congress on Huntington's.

A human trial is expected to be completed by 2013.

Dr Cherny hoped the results in the animal studies would translate to humans.

"Our hope is that we will slow the progress of the disease. The best outcome will be to slow it to the point where the person does not get the symptoms or that the symptoms are very mild," he said.

The institute and bio-tech company Prana, on the back of positive results in 2008, are proceeding with a human trial in Huntington's patients.

They plan to recruit 100 patients in Australia and the US for the six-month trial.