We’re closer than ever to finding effective treatments for Alzheimer’s disease

Alzheimer’s research has seen significant progress in recent years, but its future depends on federal funding.

More than five million Americans are living with Alzheimer’s disease. Without medical breakthroughs, that number could be as high 16 million by 2050. So just how close are we to effective treatments and preventions? We speak with Keith N. Fargo, Director of Scientific Programs and Outreach for the Alzheimer’s Association, about the state of Alzheimer’s research and what we need to sustain it for the future.

ResearchGate: What is the current state of Alzheimer’s research?

Keith Fargo: The first thing to understand is that Alzheimer's disease is more than just memory loss – it is a fatal neurodegenerative disease. There are a few drugs currently available, but they only treat symptoms of the disease. They have not been shown to slow the disease process in the brain. A person who takes these medications still develops amyloid plaques and tau tangles, loses brain cells, experiences dementia and, unfortunately, their disease still ends with death. The "holy grail" in Alzheimer's research at this time is to discover or create medications or other interventions that effectively treat or prevent the underlying disease process.

Anyone who follows the news or the scientific literature knows that there have been many challenges in achieving this goal. However, with additional focus and resources, we can expand the number of therapeutic targets and strategies, identify at-risk people earlier and get the right people into clinical trials. We believe this will significantly raise the likelihood of success.

The Alzheimer's research world is in a productive state of scientific revolution. Not long ago, it was impossible to visualize Alzheimer's-related brain changes during life until they reached a relatively advanced stage. Now, with the advent of PET imaging for beta-amyloid, and more recently for tau protein as well, researchers can visualize some of the key Alzheimer's-related processes in the living brain, well before dementia symptoms become apparent. Many of the large clinical trials incorporate these imaging techniques to help them identify appropriate participants and track how well the drug candidates are working.

“The Alzheimer's research world is in a productive state of
scientific revolution.”

RG: How close are we to finding effective treatments?

Fargo: With drug development, you're not done until you're done – in other words, until compounds are rigorously tested, approved and on the market. That said, there is a tremendous sense of optimism in the Alzheimer’s field right now. Many researchers believe that we are closer to finding effective treatments than we have ever been before. Several large clinical trials with promising drug candidates are slated to report out in the next few years. The Alzheimer’s Association International Conference (AAIC) is often a platform for these reports.

In addition, there are multiple efforts underway to evaluate non-drug therapies. For example, in Finland, there is a large clinical trial: the Finnish Geriatric Intervention Study to Prevent Cognitive Impairment and Disability (FINGER). It’s currently examining the effects of exercise, cognitive and social stimulation, and controlling vascular risk factors in preventing or delaying the onset of cognitive decline. Researchers in the US are launching a large clinical trial of exercise: the EXERT study. The study will examine the effects of aerobic exercise on cognition, functional status, brain atrophy and blood flow, and cerebrospinal fluid biomarkers of Alzheimer’s in 300 adults with amnestic Mild Cognitive Impairment.

Another factor driving this optimism is the recent trend toward the federal government acknowledging the importance of Alzheimer's disease and allocating significant new funding through the NIH to expand Alzheimer’s and dementia research efforts. For example, last year the NIH funding for Alzheimer's and dementia research was raised by $350 million, a historic increase of 60 percent. A similar increase has been proposed for the next fiscal year’s budget.

RG: Can we expect improvements in early diagnosis to implement those treatments?

Fargo: Early and accurate diagnosis is critical, not only for the quality of life of people who have the disease, but also for scientific progress. One of the things that we have learned from amyloid PET imaging is that people begin to develop amyloid plaques in their brains a decade or more before dementia symptoms begin. This powerful tool is now being used in clinical trials to find people who are in the early stages of the disease and may benefit most – particularly from anti-amyloid treatment.

However, PET imaging is expensive and not very portable. There are many efforts now underway to find other laboratory tests to identify people who have the disease or who are at high risk for the disease. These include potential blood tests, analysis of cerebrospinal fluid, and saliva tests. In fact, a newly-published special issue of one of the Alzheimer’s Association journals focuses on the latest progress in Alzheimer’s blood-based biomarker discovery.

In addition, scientists are actively pursuing many other techniques, including analyzing sensory organs and abilities – such as identifying odors – that appear to offer early hints that a person may be developing brain or cognitive changes that may lead to Alzheimer's disease.

“Early and accurate diagnosis is critical, not only for the quality of life of people who have the disease, but also for scientific progress.”

RG: What about preventing the onset of the disease altogether?

Fargo: We are living in the "era of the prevention trial." There are now five large-scale, Phase III clinical trials aimed at preventing – or at least delaying – the onset of Alzheimer’s dementia symptoms. As with many chronic diseases, the best outcomes might be achieved when Alzheimer's is identified and treated early, even before symptoms begin. The good news here is that, as mentioned above, amyloid PET imaging has revealed that amyloid plaques are present many years before dementia symptoms. This opens a window of potential prevention, a window that researchers are working very hard to exploit. An effective prevention could potentially save a half a million lives a year in the US alone, and even more in the future as the population grows.

While there is still much work to do in this area, there are things people can do now to reduce their risk for cognitive decline as they age. In 2015, the World Dementia Council asked the Alzheimer's Association to conduct a review of the scientific literature on modifiable risk factors – what might be called lifestyle risk factors. The resulting article helped generate a campaign to educate the public about ways they can reduce their risk, including getting regular exercise, quitting smoking, practicing lifelong learning, and making good food choices.

RG: What’s needed to ensure continued progress in Alzheimer’s research?

Fargo:  Increasing Alzheimer’s disease research funding at the NIH has been a longstanding legislative priority for the Alzheimer’s Association and its advocates. In order for us to change the current trajectory of the disease so that we can save lives and money, we need consistent and meaningful investments in Alzheimer’s research from the federal government.

Today, more than 5 million people are living with Alzheimer’s, and, without medical breakthroughs, by 2050 this may be as high as 16 million people at a cost of $1.1 trillion per year to Medicare and Medicaid. With the number of Americans living with Alzheimer's set to triple in just over a generation and no way yet to stop or even slow the progression of this fatal disease, research is the only option to one day prevent and effectively treat Alzheimer's.

Although the US federal government has recently stepped up its investment significantly, the current budget is still less than half of what scientists tell us is needed, which is at least $2 billion per year. That may sound like a lot, but Alzheimer's disease is the 6th leading cause of death in the United States, and the only cause of death among the top ten that does not yet have effective treatments or prevention strategies. It is also the most expensive disease in the US, costing the country $236 billion per year.

Another challenging obstacle is the shortage of participants for research studies, including clinical trials. There are more than 150 clinical trials underway for Alzheimer's disease and dementia, and more than 70,000 volunteers are needed for these studies. To address the problem, the Alzheimer's Association has created a free, easy to use matching service called TrialMatch that helps people find clinical trials that may be a good fit for them. This is an excellent way to get involved directly in the fight against Alzheimer's. Anyone who wants to get involved in this or other ways, or learn more about scientific progress in Alzheimer's disease and dementia, can visit the Alzheimer's Association website. 

In the 2016 election, Alzheimer’s disease research has received attention from both the Clinton and Trump campaigns. Read our interview with Robert Egge, Executive Director of the Alzheimer’s Impact Movement, for his analysis of why both candidates consider Alzheimer’s a priority.

Featured image: a neuron with Alzheimer's Disease. Credit: National Institute on Aging, NIH