FDA Relaxes Rules for Alzheimer's Drug Testing

There's potentially good news for future generations of Alzheimer's patients with the Federal Drug Administration allowing for drug testing in patients who are in the earliest stages of the disease, where there are not outward signs of impairment but the characteristic plaques and tangles have begun to form.

If the drugs show promising results with prevention or a slowing progression of the debilitating disease, then the medications could be fast-tracked for approval, according to an article in the New England Journal of Medicine.
Last year the last of a promising group of pharmaceuticals was rejected for failing to work as expected during drug trials. In all cases, the clinical trials involved Alzheimer's patients already showing signs of dementia, the third and final stage of the disease.

Rather than start from scratch on formulas, drug companies instead reintroduced the same medications but in earlier stages of the disease when a patient may be "asymptomatic," that is, not showing any signs they have dementia but already carrying the brain disease. But this also provides a quandary for drug companies since it may be difficult to show benefits in people not yet "suffering" from symptoms of the disease.

According to The New York Times, "The F.D.A. would require companies to study the drugs after they were on the market to show they actually benefited patients. But these studies might not be randomized clinical trials and so would not be as rigorous as the studies that led to approval. Even with less rigorous studies, it might be hard to get the evidence." This could mean it will take millions of patients and many years before companies know if a drug truly works.

Meantime, among the projects underway is one involving a UCSD researcher.

"Dr. Paul S. Aisen of the University of California, San Diego, and Dr. Reisa Sperling of Harvard are leading a federally financed study that will enlist 70-year-olds who seem perfectly normal but are accumulating amyloid plaques in their brains, a sign that Alzheimer’s could arise within about 15 years. Of 1,000 people with plaques, half will get an experimental drug made by Lilly and the rest will get a placebo. The study will also include 500 70-year-olds without plaques, for comparison.

"The researchers will assess the drug by giving subjects several tests, including ones that involve remembering lists of words and paragraphs and tests of an ability to follow instructions to change symbols to letters. Large studies indicate that people who are going to get Alzheimer’s decline faster on these tests than those who will not get the disease. If the drug works, it may slow or stop that decline."