As part of our series on Alzheimer’s Disease, Real World Health Care spoke with Creighton Phelps, Ph.D., acting director, Division of Neuroscience, National Institute on Aging, National Institutes of Health. Dr. Phelps discusses the challenges facing AD researchers and how the NIA is working to overcome them.
Real World Health Care: In terms of comparative effectiveness research for Alzheimer’s disease, where is the NIA focusing its efforts and why?
Creighton Phelps: Currently, because we do not have treatments to delay or prevent Alzheimer’s, comparative effectiveness research is not really an option. That said, Alzheimer’s disease is a complicated disorder and we are funding research into genetic, behavioral, and environmental factors that may all play a role in disease onset and progression. As a result, not one, but many interventions may be needed. For this reason, we can’t leave any stone unturned. NIA is funding drug-discovery projects, trials using pharmacological interventions, preventative lifestyle interventions, and caregiving interventions.
RWHC: What are some of the biggest challenges researchers and industry face in developing AD therapies?
CP: Clinical trials focused on finding a prevention or cure are imperative to Alzheimer’s disease and related dementias research. But a big challenge is that it is often difficult to recruit participants into trials. We need participants with cognitive impairments, as well as those without; we need ethnic minorities; we need people age 65 and older, and also younger adults. This point of comparison helps us to determine which changes in the brain are specifically related to Alzheimer’s disease and which can be attributed to aging. Increased participation in clinical trials will really hasten our search for effective therapies. Additionally, participants with dementia need a study partner to assist the participants during the trial, which adds another level of burden for families and loved ones. But without the generous participation of clinical trials volunteers, we won’t find a cure for Alzheimer’s.
RWHC: How is the NIA helping to overcome these challenges?
CP: NIA is working hard to overcome these and other challenges, and we are grateful that both public and private organizations, and the general public, also place a high priority on dementia research.
Congress just boosted federal funding for Alzheimer’s disease and related dementias research by $350 million dollars. It is anticipated that this increased budget will accelerate investigator-initiated discovery after years of smaller budget increases. The budget increase will enable new, highly collaborative initiatives.
In 2011, President Obama signed into law the National Alzheimer’s Project Act. This called for the creation and maintenance of an integrated National Plan to overcome Alzheimer’s, with the ultimate goal being to find a prevention or cure by year 2025. As mandated in the National Plan, NIA hosted Alzheimer’s Disease Summits in 2012 and 2015, bringing experts from pharma, academia, and advocacy groups together to advance the research agenda. NIA also worked with the National Institute of Neurological Disorders and Stroke to hold two Alzheimer’s Disease-Related Dementias Summits in 2013 and 2016 to examine the issues central to the other dementias that sometimes overlap with Alzheimer’s.
This spirit of collaboration can also be seen in the groundbreaking Accelerating Medicines Partnership (AMP). AMP is a bold new venture among the NIH, 10 biopharmaceutical companies, and several nonprofit organizations that aims to transform the current model for developing new diagnostics and treatments for chronic diseases. AMP-AD, which applies this innovative model to Alzheimer’s disease, will enable the rapid sharing of large biomedical datasets that may lead to speedier discovery of therapeutic targets.
RWHC: Can you please provide an overview of some of the more promising therapeutic targets (particularly those moving out of the lab and into human studies) on which the NIA is focused? Where you can, please explain why those have become a priority.
CP: NIA is funding, often in collaboration with others, prevention trials testing drugs that may clear amyloid protein—a hallmark of the disease—in cognitively normal volunteers at high risk for developing the disease due to genetics or who show abnormal amyloid levels in their brains, as visualized by PET Scans. The hope is that by treating the disorder earlier in the disease process that we may delay or even prevent the disease.
RWHC: Where does the NIA see the greatest need for research into Alzheimer’s disease: diagnosis, symptom management, stopping/slowing the progression of the disease, or prevention of AD? Why?
CP: Currently, about 5.2 million Americans age 65 and older are living with Alzheimer’s disease; many thousands more are diagnosed with related disorders, such as Lewy body or frontotemporal dementia. An even greater number are in the pre-symptomatic stages, sometimes called Mild Cognitive Impairment (MCI) or prodromal disease. These numbers are expected to more than double by 2050 unless we find one or more treatments. Alzheimer’s and other forms of dementia are expected to cost the United States $236 billion this year. Of course, the ultimate goal is to treat and prevent the disease. But these staggering statistics reveal that any discoveries—whether in diagnosis, symptom management, stopping/slowing disease progression, or preventing the disease—could help the millions currently afflicted.
RWHC: Besides translational and comparative effectiveness research, what other initiatives does the NIA support in relation to Alzheimer’s disease? Any highlights to share in terms of non-pharmacological intervention research?
CP: With these additional financial resources, we are able to fund many new and exciting projects. The additional $350 million will enable: the development of new human cellular models of Alzheimer’s that may enable rapid screening of hundreds of thousands of molecules as potential therapeutic agents. It will also allow us to establish translational centers that will develop and apply cutting-edge approaches to drug discovery and development. Other upcoming projects include population studies of trends in the incidence and prevalence of dementia, the development of novel interventions to support dementia caregivers, and clinical trials of therapies in people at the highest risk of dementia.