Alzheimer’s disease research has reached a major crossroads. The genetic causes of rare forms of the disease have been identified and, in every case, these genes point to buildup of the substance called ‘amyloid’ as the initiating event. If these genes are inserted into a mouse, the mouse develops brain amyloid buildup and forgetfulness. This is a scientific fact.

 

So, why is there controversy about amyloid and Alzheimer’s? The reason is simple: these gene mistakes only occur in about three out of every 100 Alzheimer’s patients. Where does the disease begin in the other 97? This is not yet known with certainty.

 

A century after Dr. Alzheimer reported on his first patient, we are on the verge of having major insight into how and why the brain fails and why our lives and those of our loved ones are increasingly threatened. In the U.S., half of those over age 85 have dementia, and worldwide cost already exceeds $300 billion. No government will be able to sustain itself against the economic devastation that Alzheimer’s will wreak in the coming decades – unless we act now to better diagnose, treat and prevent Alzheimer’s.

 

The answer to this problem is to develop better treatments and prevention strategies for Alzheimer’s. It is absolutely crucial that additional federal funding be allocated for Alzheimer’s research. We’ve made progress in recent years, but there is a lot more work to do, and that starts with research dollars. Maintaining the current inadequate funding levels – while the prevalence of Alzheimer’s disease will quadruple by mid-century – is unacceptable.

 

There exist two possible scenarios in how Alzheimer’s develops. In the first scenario, the final pathway to all Alzheimer’s leads to amyloid accumulation, and all nerve cell injury is caused by amyloid. In this case, if amyloid accumulation is reversed or prevented, no clinical disease will occur. This is the scenario that was encountered in heart disease, where cholesterol accumulation turned out to be the final pathway. As cholesterol accumulation has become controlled or abolished, heart disease prevalence has significantly decreased. Could something like this happen in Alzheimer’s? Yes.

 

In the second scenario, something else causes the brain damage seen in Alzheimer’s and amyloid buildup comes later. In this scenario, if we prevent amyloid buildup, we might partially reverse or prevent the disease, but the primary injury would persist.

 

How will we sort this out? The most promising strategy involves clinical trials with anti-amyloid drugs. By using the new, amyloid-revealing brain scans, we can both follow subjects clinically to see whether cognitive function improves and scan them to see if the medicines are removing the amyloid. We must prove that we have purged the brain of amyloid before we can say that we have adequately tested the ‘amyloid hypothesis,’ as it is called among researchers.

 

When will we know? Drug trials in the United States pass through three phases, the last of which must be successful in order for the U.S. Food and Drug Administration to approve a medicine as effective and eligible to be put into the marketplace. Ninety percent of test drugs that enter early-stage clinical testing fail, so a late-stage clinical trial is  the “home stretch.” Several anti-amyloid therapies are currently in this home stretch.

 

Recruiting and retaining clinical trial participants is now one of the biggest impediments to developing new and better therapies for Alzheimer’s disease. If we can get more people to participate in trials, then we can speed the progress for the benefit of all those affected by the disease.

 

The Alzheimer’s Association has launched a pilot clinical studies initiative in five cities to educate the medical community about the critical shortage of volunteers and dispel any misinformation that may be impacting their willingness to refer patients, inform the public about clinical trial opportunities, and educate minorities about clinical research and encourage their participation.

 

As baby boomers enter the age of greatest risk, Alzheimer's will become the public health crisis of the 21st century. Americans living with Alzheimer’s will explode from as many as 5 million today to as many as 16 million by 2050.

 

Everyone wants better treatments and a cure for Alzheimer’s. Research investment has brought us to the point where we can envision a world without Alzheimer’s disease. That said, the need for more resources in this fight is clear. The Alzheimer’s Association is confident that we will defeat Alzheimer’s disease. How fast we do it is directly related to the level of investment.