First Critical Clinical Tests of the Amyloid Theory of Alzheimer’s Disease

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In 1991, researchers currently at the Roskamp Institute published a paper in the highly prestigious Nature journal entitled “Early Onset Alzheimer’s Disease Caused by Mutations of the Amyloid Gene”. This and related findings were historic milestones in the understanding of the causes of Alzheimer’s disease. Today, seventeen years later, Roskamp Institute researchers are delighted to hear that Elan, one of the largest pharmaceutical companies, is having success with an approach which targets the amyloid protein.

The early findings by Roskamp researchers placed amyloid at center stage as the cause of the disease. Now Elan’s vaccine, which is targeted to soak up the amyloid protein, may be a verification that reducing amyloid accumulation is a way to tackle the disease. Although some researchers remain skeptical of the validity of this approach, the early results from Elan’s Phase II study, published this week (June 17) indicate that reducing amyloid levels with the anti-amyloid vaccine has slowed the rate of progression of the disease in some patients. Interestingly, the vaccine approach has been helpful in cases of the disease that do not have a high genetic risk loading. Although there was a hint that all cases of Alzheimer’s (even those with high genetic loading might benefit from the vaccine), there was not a statistically significant difference between placebo group and those individuals that carry an APOE4 gene. This gene is known to increase the likelihood of developing Alzheimer’s early in life and may represent a particularly aggressive form of the disorder. Nevertheless, the fact that there were trends in a treatment improvement in the high risk group and statistically significant improvement in income in the low genetic risk group, strongly suggest that targeting amyloid will be a therapeutically useful strategy.

For Roskamp Institute researchers the new data from Elan is very important as Roskamp Institute researchers have developed drugs that do the same thing as the vaccine, i.e., lower the accumulation of amyloid levels. One of those drugs is in clinical trials already in Europe and trials are set to begin in the US pending FDA approval.

For Alzheimer’s sufferers and Alzhiemer’s researchers alike, the news from Elan is very hopeful.