In January 2011, President Barack Obama signed the National Alzheimer’s Project Act (NAPA), which called for an aggressive and coordinated national plan to attack Alzheimer’s disease and improve care and services. To provide a living framework for this initiative, and as directed under the legislation, the U.S. Department of Health and Human Services (HHS) unveiled the National Plan to Address Alzheimer’s Disease in May 2012, with an update in June 2013. The Plan addresses the major challenges presented by Alzheimer’s disease and outlines and tracks the various goals and activities—from advancing scientific collaboration to improving patient care—now undertaken with increasing collaboration within the Federal Government and between the public and private sectors.
The Plan’s overarching research goal (Goal 1) is to “prevent or effectively treat Alzheimer’s disease by 2025.” While the Plan is a national effort involving public and private stakeholders, the National Institutes of Health (NIH) play a lead role in identifying and supporting the research needed to fulfill the Plan’s goals. The challenges are many; Alzheimer’s is a complex disorder and research dollars are shrinking. But NIH leadership, staff, scientists, clinicians, and grantees—as well as the many volunteers who participate in studies and make research possible—are fully committed to a future free of this dread disease.
This section lists the strategies and highlights some of NIH’s 2012-2013 activities under Goal 1. These actions are not an end in themselves. Rather, measured in specific milestones, they reflect the momentum achieved under the Plan’s comprehensive and cohesive framework.
Goal 1: Prevent and Effectively Treat Alzheimer’s Disease by 2025
Strategy 1.A: Identify Research Priorities and Milestones
Regularly Convene an Alzheimer’s Disease Research Summit to Update Priorities
A second NIH-hosted Summit is planned for February 9-11, 2015, to evaluate and build on these priorities, milestones, and timelines.
Convene a Scientific Workshop on Other Dementias in 2013
The National Institute of Neurological Disorders and Stroke, also part of NIH, with other NIH Institutes led the Alzheimer’s Disease-Related Dementias: Research Challenges and Opportunities workshop in May 2013. The meeting featured Alzheimer’s disease-related dementias such as Lewy body dementia, frontotemporal dementia, and vascular dementia—debilitating conditions that impair memory, thinking, movement, and everyday functioning, primarily in older adults. Recommendations from the meeting will guide scientific research for the next 5 to 10 years.
To better understand Alzheimer’s in people at high risk for developing the disease, NIH supported the Advancing Treatment for Alzheimer Disease in Individuals with Down Syndrome workshop in April 2013. Co-hosted by the National Institute on Aging (NIA), the Eunice Kennedy Shriver National Institute of Child Health and Human Development, and the National Institute of Neurological Disorders and Stroke, as well as the Down Syndrome Research and Treatment Foundation and Research Down Syndrome, the meeting resulted in research recommendations focused on this special population. These recommendations include developing animal models of Down syndrome and Alzheimer’s to better predict response to therapies, and sharing existing data and development of new data sets that follow study participants with Down syndrome as they age.
Strategy 1.B: Expand Research Aimed at Preventing and Treating Alzheimer’s
Adding to baseline budget funding of Alzheimer’s research projects, in fiscal year 2012 NIH allocated $50 million to speed discovery in specific, promising avenues of Alzheimer’s research. This funding boost targeted, for example, cutting-edge research into genetic risk and protective factors and development of new cellular models of Alzheimer’s to enable more rapid screening of potential therapies. The funding also made possible two new Alzheimer’s treatment and prevention clinical trials:
A $16 million prevention trial in people with early-onset Alzheimer’s, a rare, familial form that can occur in people in their 30s, 40s, and 50s. The trial, which is testing an experimental amyloid-clearing drug, is co-funded by the Banner Alzheimer’s Foundation and Genentech, a biotechnology company that is also providing crenezumab, the test drug.
In fiscal year 2013, NIA awarded approximately $45 million in new funding to test promising drugs aimed at preventing Alzheimer’s and to identify and validate biological targets for novel therapies. The studies are among the first to be developed with direction from the recommendations developed at the 2012 Alzheimer’s Disease Research Summit.
Four clinical trials will test amyloid-clearing or hormone treatments in symptom-free volunteers at risk for the disease, or those in the very earliest stages—where interventions may prove most effective.
Three studies will identify and validate novel therapeutic targets for Alzheimer’s disease. These efforts use cutting-edge scientific methods with the goal of speeding discovery. For example, one study will investigate the complex molecular networks and candidate genes that influence susceptibility to cognitive decline and Alzheimer’s disease, and will then test promising drugs that may normalize the dysfunctional networks.
Expand Genetic Epidemiologic Research to Identify Risk and Protective Factors for Alzheimer’s Disease
In 2012, NIH Director Dr. Francis Collins asked NIA and the National Human Genome Research Institute (NHGRI) to work together to analyze the genomes of a large number of older adults to identify Alzheimer’s risk and protective gene variants. The ultimate goal is to find new pathways for treatments and prevention. By December 2013, the Alzheimer’s Disease Sequencing Project, developed jointly by NIA and NHGRI, had sequenced the whole genomes of more than 580 volunteers and made the data freely accessible to researchers on the NIA Genetics of Alzheimer’s Disease Data Storage Site.
Increase Enrollment in Clinical Trials and Other Clinical Research Through Community, National, and International Outreach
An estimated 50,000 volunteers with Alzheimer’s, mild cognitive impairment, or normal cognition are needed for clinical trials and studies; researchers will need to screen at least half a million potential volunteers to reach this goal. To address this need, several HHS agencies collaborated on the Recruiting Older Adults into Research (ROAR) program in 2013. NIA, along with the Administration on Community Living and the Centers for Disease Control and Prevention, will promote research participation via outreach and messaging at the State and local levels. To reach eligible volunteers, ROAR will use existing networks and service programs, such as senior centers, nutrition sites, support groups, information and assistance hotlines, flu shot clinics, and community health worker programs.
The Alzheimer’s Prevention Registry was launched in October 2012 by the Banner Alzheimer’s Institute (one of 27 NIA-funded Alzheimer’s research centers). This online, shared recruitment resource enables people across the United States to learn about and consider participating in Alzheimer’s prevention studies. Currently, more than 22,000 people have signed on; the goal is to enroll 250,000 people by June 2015. Banner is a key partner in the Alzheimer’s Prevention Initiative, an international collaboration funded in part by NIA that will test treatments in people at high genetic risk for Alzheimer’s.
An NIA Request for Information sought ideas on how to increase participation in Alzheimer’s clinical trials, particularly from diverse communities. Responses from the research and advocacy communities suggested a wide array of actions, such as using volunteer registries, increasing access to trials, and standardizing consent forms.
In 2012 and 2013, NIA and the Administration for Community Living, also part of HHS, co-hosted Connecting to Combat Alzheimer’s webinars to improve coordination of Federal resources for people with the disorder and their caregivers, and to increase awareness of opportunities to participate in research. The free, award-winning HHS Innovates webinars and presentations for both the research and aging-services communities helped spur a 25 percent increase in prospective research participants. These efforts continue to inspire collaborations at the State, local, and grassroots levels. In 2013, a key focus of the series was addressing enrollment issues related to racial and ethnic minorities. Listen to the webinars and view related materials.
Conduct Clinical Trials on the Most Promising Pharmacologic Interventions
NIH is a primary funder of large, investigator-initiated clinical trials, currently supporting more than 35 pilot and large-scale trials of a wide range of interventions to prevent, slow, or treat Alzheimer’s and/or cognitive decline. More than 40 compounds are in preclinical development. These trials include testing newly designed drugs aimed at specific mechanisms to the possible broader effects of exercise in slowing down or preventing Alzheimer’s.
The Alzheimer’s Disease Cooperative Study (ADCS) is an innovative research network for Alzheimer’s disease clinical studies. In January 2013, NIA renewed its support of the ADCS with an $11 million award, which could total as much as $55 million over the 5 years of the project. The consortium, coordinated by the University of California, San Diego, and led by Paul Aisen, M.D., is made up of more than 70 research sites in the United States and Canada, with a focus on advancing studies of interventions that might not otherwise be tested by industry. This award made possible four new trials. Read more about the ADCS and these trials at www.nia.nih.gov/newsroom/2013/01/nih-supported-alzheimers-studies-focus-innovative-treatments
Strategy 1.C: Accelerate Efforts to Identify Early and Presymptomatic Stages of Alzheimer’s Disease
Since 2004, NIA has led the Alzheimer’s Disease Neuroimaging Initiative (ADNI), a groundbreaking study that is defining subtle changes that may take place in the brains of older people many years before overt Alzheimer’s symptoms appear. ADNI is the largest public-private partnership to date in Alzheimer’s disease research. Now in its third phase, ADNI 2 is identifying and standardizing imaging and fluid biomarkers in brain, blood, and spinal fluids that make it possible to detect the onset of Alzheimer’s disease, track its progression, and evaluate the effectiveness of promising therapies. To learn more, go to www.nia.nih.gov/newsroom/features/conversation-dr-neil-buckholtz
Strategy 1.D: Coordinate Research with International Public and Private Entities
Inventory Alzheimer’s Disease Research Investments
The International Alzheimer’s Disease Research Portfolio (IADRP) is a publicly available database that captures the full spectrum of Alzheimer’s disease research investments and resources, both in the United States and internationally. Developed by NIA with the Alzheimer’s Association, IADRP enables public and private funders of Alzheimer’s research to coordinate research planning, leverage resources, avoid duplication of funding efforts, and identify new opportunities in promising areas of growth. Today, 13 major Alzheimer’s disease research funders—in the United States, United Kingdom, Canada, and Australia—have provided funding data, and many others use the database.
In April 2013, NIA convened the Enabling Partnerships for Alzheimer’s Disease Drug Development meeting to promote collaborations aimed at an integrated, seamless approach to Alzheimer’s research and drug development. More than 60 leaders from academia, industry, NIH, the U.S. Food and Drug Administration, foundations, and advocacy groups explored ways to adopt and adapt new models of data-sharing and translational science.
The New York Academy of Sciences and the Global CEO Initiative, in collaboration with NIA, held the Alzheimer’s Disease Summit: The Path to 2025 meeting in November 2013. Leading industry, academic, and government stakeholders discussed ways to coordinate efforts to build research resources, reengineer current drug development and evaluation systems, and identify innovative technologies and financing models.
The Alzheimer’s Association and NIA cohosted a funders meeting at the Alzheimer’s Association International Conference in July 2012 and July 2013. The Alzheimer’s Association and NIA also convene representatives of several funding organizations around the world for regular teleconferences. NIA is exploring collaboration with the European Union Joint Programming Initiative on Neurodegenerative Diseases Research, and the HHS Office of Global Affairs is exploring opportunities to collaborate on international initiatives focused on Alzheimer’s disease.
Strategy 1.E: Facilitate Translation of Findings into Medical Practice and Public Health Programs
NIA operates the Alzheimer’s Disease Education and Referral Center (ADEAR), the primary Federal Government resource for information on Alzheimer’s disease research and care. The Center educates the public about the latest research findings and provides evidence-based information at www.nia.nih.gov/alzheimers and in print. Information about Alzheimer’s and other dementias, participation in clinical trials, and caregiving, is available for people with the disease, their families and caregivers, health care and social service providers, and the public. Information specialists are available to answer calls via a toll-free number (1-800-438-4380) and email at firstname.lastname@example.org.
ADEAR tweets information about Alzheimer’s daily and covers such topics as caregiving tips and resources, the latest research findings, new clinical trials, and basic information about diagnosis and treatment. Follow ADEAR Twitter @Alzheimers_NIH.
Caregiving resources have been greatly have been greatly expanded to include easy-to-read tip sheets (now available as e-books for tablets, e-readers, and smartphones) as well as newly translated online Spanish topics.
The ADEAR Center distributes e-mail news alerts, including the online Connections newsletter and a monthly update of recruiting clinical trials and studies. Subscribers can sign up on the ADEAR Center home page at www.nia.nih.gov/alzheimers.