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Rogue researcher offers new hope for sufferers of Alzheimer's disease

Robin Wulffson MD's picture
Alzheimer's disease, dementia, TauRx, tau protein, research, cure

Approximately 36 million people worldwide suffer from Alzheimer's disease or dementia. Although many researchers are of the opinion that the buildup of amyloid plaques in the brain is the underlying cause of Alzheimer’s disease, Claude Wischik, MD, PhD has long backed a minority view regarding its cause. He feels that the condition is due to a protein in the brain called tau. The company he co-founded 10 years ago, TauRx Pharmaceuticals Ltd., has developed an experimental Alzheimer’s medication that it will begin testing in the coming weeks in two large clinical trials.

Dr. Wischik has been studying Alzheimer’s disease for decade. When he was a young PhD student at Cambridge University in the 1980s, he began collecting brains. It was a daunting task because few organ banks kept entire brains. In his search for an Alzheimer’s cure, he needed to examine brain tissue from Alzheimer’s patients soon after death. That required obtaining family approvals and enlisting mortuary technicians to extract the brains. In a little over a decade, Dr. Wischik collected more than 300 brains. While collecting his specimens, he embraced a concept that, if he is right, could fundamentally transform Alzheimer’s research and raise new hopes for individuals suffering from Alzheimer’s disease or dementia.

Dr. Wischik believes that a protein called tau, which forms twisted fibers known as tangles inside the brain cells of Alzheimer’s patients, is primarily responsible for the condition. His concept is bucking a current premise for the case of the disease. For two decades, billions of dollars of pharmaceutical investment has supported a different theory that a different protein, beta amyloid, which forms sticky plaques in the brains of sufferers is responsible. However, a number of experimental drugs designed to attack beta amyloid have failed recently in clinical trials, including two this summer from Eli Lilly and a partnership involving Pfizer Inc., Johnson & Johnson, Elan Corp.

Dr. Wischik notes that he and other tau-focused researchers have been shouted down over the years by what he calls the “amyloid orthodoxy,” a group of researchers who believe passionately that beta amyloid is the chief cause of the disease. In addition, his research has been hampered by inconclusive research. A small clinical trial of TauRx’s drug in 2008 produced encouraging, but mixed, results.

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In the mid-1990s, Dr. Wischik discovered that a drug sometimes used to treat psychosis dissolved tangles in a test tube. He tried to form a company to develop the drug as a treatment for Alzheimer’s; however, he found that American and British venture capitalists wanted to invest in amyloid projects, not tau. By 2002, Dr. Wischik had amassed approximately $5 million from Asian investors with the help of a Singaporean physician who was the father of a classmate of Dr. Wischik’s son in Cambridge. TauRx is based in Singapore; however, it conducts most of its research in Aberdeen, Scotland.

A turning point occurred in 2008, when Dr. Wischik presented the TauRx results at an Alzheimer’s conference in Chicago. The study found that, after 50 weeks of treatment, Alzheimer’s patients taking a placebo had fallen 7.8 points on a test of cognitive function, while people taking 60 mg of TauRx’s drug three times a day had fallen one point; this marked an 87% reduction in the rate of decline for individuals taking the TauRx drug. However, TauRx did not publish a full set of data from the trial, causing some skepticism among the scientific community. During this time, drugs designed to attack beta amyloid continued to be disappointing.

Currently, both scientists and investors are focusing more attention on tau. With its new clinical trial program under way, TauRx is the first company to test a tau-targeted drug against Alzheimer’s disease in a large phase 3 clinical trial.

According to the World Health Organization, Alzheimer’s disease is the leading cause of dementia in the elderly; the cost of caring for dementia sufferers totals about $600 billion each year world-wide. The disease was first identified in 1906 by German physician Alois Alzheimer, who studied the brain of a deceased woman who had suffered from dementia and documented the plaques and tangles that riddled the tissue. The following decades brought little advancement in understanding the disease, in part because of the difficulty of studying the human brain, which unlike other tissues cannot be biopsied and examined until after death.

Reference: TauRx

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