Prana Biotechnology Limited announced today that researchers are planning recruitment for a phase II clinical trial of the experimental drug PBT2 in Huntington’s patients. There are currently no treatments for the genetic, neurodegenerative disease; Australia is increasingly seen as a leader in the development of therapies for neurologic disorders. Huntington’s often affects young adults and progressively impacts the body, mind and emotions. The disease causes incapacitation and death about 15-25 years after onset.
“Compared to other diseases, there are relatively few researchers working on treatments for Huntington’s, so we are very pleased to be conducting this trial,” said University Melbourne Associate Professor Robert Cherny, who is also Prana’s Head of Research. “PBT2 initially showed great results in patients with Alzheimer’s, which attracted the attention of many leading scientists working in the Huntington’s field around the world because the two diseases have quite a few similarities. This treatment, developed in Melbourne, has demonstrated both the ability to protect and restore function to damaged brain cells, which is very promising for those with Huntington’s.”
In human clinical trials of Alzheimer’s patients, the experimental drug improved the cognition of patients which is the most important aspect of behavior to be affected by Huntington’s. Scientists believe the drug affects the distribution of naturally occurring, biological metals in the brain which appear to play a role in protein aggregation. This protein aggregation is believed to be the cause of cell death in neurodegenerative diseases. This research on biological metals is led by Australian scientists but has generated international attention and is strongly featured at the World Congress on Huntington’s Disease being held currently in Melbourne.
Professor of neurology Rudolph E. Tanzi from Harvard Medical School, who worked on the team that discovered the Huntington's disease gene and is a frequent advisor to the US Congress on neurological disease, research and treatment says, "I believe PBT2 has a very high chance among all current candidate therapies to be the first effective disease-modifying treatment for Alzheimer's Disease and we are very hopeful we will see similar results with Huntington's Disease, a terribly devastating disease in dire need of a cure."
Researchers conducting the trial will need to recruit approximately 100 people with early stages of the disease across Australia and the United States, at 14-16 sites. Patients will be followed over 6 months. Researchers will assess safety, tolerability and efficacy of PBT2. Final results are expected in 2013.
Prana will also soon hold clinical trials for PBT2 in Alzheimer’s. The company is also developing another drug, PBT434, for Parkinson’s which recently received research funding from the Michael J. Fox Foundation.
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