Reach2HD, a Phase II study in Huntington's disease, launched

June 7, 2012

The Huntington Study Group (HSG), under the leadership of Ray Dorsey, M.D. with Johns Hopkins Medical and Diana Rosas, M.D. with Massachusetts General Hospital, is conducting a clinical trial in Huntington's disease (HD) throughout the United States and Australia, "A randomized, double-blind, placebo-controlled, study to assess the safety and tolerability, and efficacy of PBT2 in patients with early to mid-stage Huntington's disease" comparing a 100 mg dose or 250 mg dose versus placebo. The HSG is a not-for-profit group of physicians and other clinical researchers who are experienced in the care of HD patients and dedicated to clinical research of the disease. This trial is sponsored by Prana Biotechnology Limited (Melbourne, Australia) and is being managed by the University of Rochester Medical Center.

Huntington's disease is an inherited neurodegenerative disease which affects over 30,000 people in both the United States and Australia. HD is characterized by that usually begins between the ages of 30 to 50, and results in motor, cognitive and behavioral signs and symptoms. While there are medications to help relieve some of the disease symptoms, there is no known treatment to address the cognitive impairment associated with HD.

Research has shown that normally occurring metals in the brain play a significant role in diseases such as Alzheimer's disease and more recently, HD. Researchers at Prana Biotechnology are identifying drugs designed to interrupt interactions between these biological metals and in the brain, to prevent deterioration of . One of the , called PBT2, has shown in animal models, and as well as in a small group of patients with Alzheimer's disease, that it may improve cognition. There is some indication in animal models of HD, that the drug may improve motor function and control, increase life span and reduce the amount of brain cell degeneration. Based on these results, Prana is investigating whether the drug will have similar effects with HD patients.

Reach2HD will evaluate how safe and well tolerated PBT2 is at a dose of 100 mg or 250 mg a day compared to a placebo over six months. The trial will also measure whether there is an effect on cognitive abilities as well as other HD symptoms including motor and overall functioning of individuals with HD.

"We are excited to work with Prana to investigate the safety and tolerability of an interesting and innovative experimental treatment for Huntington's disease, PBT2," said Dorsey. "We have few treatment options for Huntington disease, and none for cognition. We hope this is a step to addressing this large unmet need for patients and their families."

Explore further: New drug seems well-tolerated and merits further investigation in patients with Huntington's disease

Related Stories

New drug seems well-tolerated and merits further investigation in patients with Huntington's disease

November 7, 2011
A novel drug (pridopidine) that stabilises dopamine signalling in areas of the brain that control movement and coordination, appears well tolerated and warrants further study in patients with Huntington's disease (HD), a ...

Striatal brain volume predicts Huntington disease onset

April 26, 2012
Huntington disease (HD) is an inherited neurodegenerative disorder caused by a defect on chromosome four where, within the Huntingtin gene, a CAG repeat occurs too many times. Most individuals begin experiencing symptoms ...

Researchers restore neuron function to brains damaged by Huntington's disease

May 29, 2012
Researchers from South Korea, Sweden, and the United States have collaborated on a project to restore neuron function to parts of the brain damaged by Huntington's disease (HD) by successfully transplanting HD-induced pluripotent ...

Recommended for you

Scientists block evolution's molecular nerve pruning in rodents

July 27, 2017
Researchers investigating why some people suffer from motor disabilities report they may have dialed back evolution's clock a few ticks by blocking molecular pruning of sophisticated brain-to-limb nerve connections in maturing ...

Scientists become research subjects in after-hours brain-scanning project

July 27, 2017
A quest to analyze the unique features of individual human brains evolved into the so-called Midnight Scan Club, a group of scientists who had big ideas but almost no funding and little time to research the trillions of neural ...

In witnessing the brain's 'aha!' moment, scientists shed light on biology of consciousness

July 27, 2017
Columbia scientists have identified the brain's 'aha!' moment—that flash in time when you suddenly become aware of information, such as knowing the answer to a difficult question. Today's findings in humans, combined with ...

Social influences can override aggression in male mice, study shows

July 27, 2017
Stanford University School of Medicine investigators have identified a cluster of nerve cells in the male mouse's brain that, when activated, triggers territorial rage in a variety of situations. Activating the same cluster ...

Researchers reveal unusual chemistry of protein with role in neurodegenerative disorders

July 27, 2017
A common feature of neurodegenerative diseases is the formation of permanent tangles of insoluble proteins in cells. The beta-amyloid plaques found in people with Alzheimer's disease and the inclusion bodies in motor neurons ...

Mother's brain reward response to offspring reduced by substance addiction

July 27, 2017
Maternal addiction and its effects on children is a major public health problem, often leading to high rates of child abuse, neglect and foster care placement. In a study published today in the journal Human Brain Mapping, ...

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.