Alzheimer’s disease is a debilitating, progressive neurodegenerative disease affecting close to five million Americans. The Alzheimer’s Association estimates the disease is responsible for at least 60%, and perhaps as much as 80%, of all dementia cases, and afflicts more than 10% of Americans over age 65, and one-third of those over age 85. Worldwide, some estimates put the number of people with Alzheimer’s as high as 40 million.
While not observable in the living, autopsies show microscopic growth of abnormal clumps of beta-amyloid protein plaque and neurofibrillary tangles within and between brain cells of Alzheimer’s patients. While these are associated with the disease, it is still not known whether they cause it, or result from some other cause.
Since family history is the second greatest risk factor, trailing only age, Alzheimer’s also appears to have a genetic factor. Alzheimer’s is usually first identified in patients age 65and up – most commonly by difficulties in assimilating or remembering new information, impaired reasoning, personality changes or other mental or physical irregularities. In less than 5% of cases, onset comes much earlier, and a specific genetic link has been identified for this form of the disease.
Some current treatments can bring temporary easing of symptoms, but thus far no treatment has been discovered that reverses or stops the progression of Alzheimer’s. The disease’s prevalence, virulence and costs – the annual costs of treating an American with Alzheimer’s is somewhere around $50,000 — has drawn $565 million in government research this year seeking its causes and better treatments, and numerous private companies are seeking to develop effective remedies.
In hopes of discovering the first new treatment in a decade, a Phase II clinical study, bearing the label NOBLE, is now underway at about 50 U.S. hospitals hopes to enroll 450 people between the ages of 55 and 85 with mild to moderate Alzheimer’s. It aims to assess whether a new experimental drug, T-817MA, helps prevent or slow the loss of brain nerve cells.
The NOBLE trial is funded entirely by the drug’s developer, Toyama Chemical Ltd., part of Japan’s Fujifilm Group. Running the clinical trial is the Alzheimer’s Disease Cooperative Study, a National Institutes of Health-supported clinical trials consortium based at the University of California San Diego. The lead researcher is Dr. Paul Aiser, a leading Alzheimer’s authority.
All participants in the trial are already taking the leading current treatment drug (donepezil, also known by the trade name Aricept), and some are also taking memantine (trade name Namenda), another treatment of a different type. The trial will randomly divide participants into three groups. One group will receive a placebo; the other two groups will receive the experimental drug, in one of two dosage levels. All participants will be tested for memory, other mental functions and overall ability to function, and will receive regular physical exams, blood tests and brain scans.
Toyama has been working on the drug since 2005. In 2008-2011, a more limited trial in the U.S. and Canada using only one dosage level suggested T-817MA has the potential to slow down cognitive and functional decline in Alzheimer’s patients.
Begun in March 2014, the NOBLE trial is scheduled to last until March 2016. New participants are still being sought.