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Alzheimer’s disease and related dementias are a series of complex brain disorders that affect millions of Americans and many more worldwide. They have an enormous impact on individuals and their families, long-term care facilities, health care providers, health care systems and infrastructure, and the communities in which we all live. As the economic, social, and personal costs of these diseases climb, the research community is working to discover solutions that will improve the lives of those with dementia, their caregivers, and their communities.
The federal government’s Alzheimer’s and related dementias research strategy focuses on engaging a cross-disciplinary team of geneticists, epidemiologists, gerontologists, behavioral scientists, disease and structural biologists, pharmacologists, clinical researchers, and others to bring the greatest and most diverse expertise to the field. This includes training new generations of researchers and clinician-scientists and engaging in innovative partnerships with private industry, nonprofit groups, and more to foster collaboration and broaden access to research resources and data.
Critically, the government’s research strategy includes the search to find treatment and prevention strategies, as well as interventions, services, and supports to improve quality of life for those already living with these diseases and their families.
The National Institutes of Health (NIH) is made up of Institutes, Centers, and Offices that conduct and fund research into all aspects of human health. The National Institute on Aging (NIA) leads NIH’s efforts in clinical, behavioral, and social research in Alzheimer’s and related dementias through efforts aimed at finding ways to treat and ultimately prevent the disorder. NIA collaborates closely with the National Institute of Neurological Disorders and Stroke (NINDS), which manages a research portfolio targeting Alzheimer’s disease-related dementias. While some of this research takes place in NIH laboratories, the vast majority of NIH support is provided through a competitive grants process to institutions and small businesses across the country. Other federal agencies support a range of activities focused on public health and community programs.
Advances in Alzheimer’s and Related Dementias Research
As the nation’s biomedical research agency, the National Institutes of Health (NIH) supports research spanning from basic biology to drug development to clinical studies to evaluating public health outcomes. Within the past several decades, researchers have made great strides toward better understanding what causes Alzheimer’s and related dementias and discovering approaches that may prevent, diagnose, and treat them. Some highlights of these efforts include:
- Early detection and diagnosis. Researchers have made significant progress in developing, testing, and validating biomarkers that detect signs of the disease process. For example, NIH-supported scientists have made advances in blood-based tests that can be used to screen volunteers for research studies. It is likely that in the future, physicians will be able to use such tests to screen their patients for Alzheimer’s and related dementias before symptoms appear. Researchers are also studying behavioral and social indicators, including problems with paying bills and a combined decline in memory and walking speed, that may be early signs of these diseases. Other early markers are also under study. Early and clear diagnoses of Alzheimer’s and related dementias will enable researchers to develop appropriate treatment and prevention strategies based on an individual’s disease profile.
- Population studies and precision medicine. By studying large, diverse groups of people, researchers are identifying which genes, behaviors, and lifestyle choices are linked with dementia. Population studies have shown that sedentary behavior, low socioeconomic status, low level of education, and living in a poor neighborhood may increase the risk of developing dementia. These observational discoveries, along with knowledge of genetic and other factors, can be used to advance the development of methods for diagnosis, prevention, and treatment at an individualized level.
- Lifestyle interventions. Researchers are investigating interventions around exercise, healthy eating, cognitive training, preventive health care, and management of chronic conditions that — if made early in life — may be able to prevent or delay disease symptoms. NIA currently supports more than 100 trials testing behavioral and lifestyle interventions.
- Disease pathways. NIH’s research investments to identify the biological mechanisms that lead to Alzheimer’s and related dementias are fundamental for the discovery of potential drugs that target them. There are many biological pathways that scientists can target with investigational drugs. For example, several recent studies have further revealed how components of the immune system, brain inflammation, and possibly viruses and bacteria — including the many tiny organisms that live in the digestive system, known as the gut microbiome — contribute to the development of these diseases. Scientists are also exploring genetic variations that may contribute to or prevent disease.
- Drug discovery. Thanks to the substantial investment in Alzheimer’s and related dementias research over the past several years, NIH has increased drug discovery significantly. Of the many compounds in NIH-supported drug development programs for Alzheimer’s and related dementias, 10 drug candidates have now matured through the pipeline, from discovery in the lab all the way through preclinical development, to reach the stage of human testing. NIA currently supports more than 40 clinical trials testing drug candidates that target many different aspects of the disease.
- Infrastructure development. NIH is continually investing in research infrastructure to advance Alzheimer’s and related dementias research. Efforts in this area include launching a consortium for Alzheimer’s clinical trials, research efforts to validate cognitive tests in a primary care setting, and genetics and genomics sharing and collaboration initiatives.
Challenges for the Alzheimer’s Research Community
Even with the progress that we’ve made, there’s still a lot of work to do before we can find treatment and prevention strategies for the millions of people affected by Alzheimer’s and related dementias. These devastating diseases are highly complex conditions caused by an interplay of genetic, lifestyle, and environmental factors. They usually develop gradually — changes in the brain take place over years and even decades, long before the first symptoms appear. This complexity presents challenges to the discovery and development of new drugs and other prevention and treatment approaches. Alzheimer’s disease does not affect all communities at the same rate, and research has found that a person’s likelihood of getting Alzheimer’s can be affected by their sex, ethnicity, race, socioeconomic status, and other factors. These differences are called health disparities.
Researchers believe Alzheimer’s disease and related dementias will likely require multiple treatments customized to individuals. We also know that as the older adult population continues to grow — aging remains the most important risk factor for dementia — we will see increased numbers of people living with these diseases. That’s why thousands of researchers around the country are working on this issue.
Setting the Federal Research Agenda
NIH takes a collaborative, methodical approach to reviewing progress, identifying gaps, and setting the future agenda for research into Alzheimer’s disease and related dementias. NIH funding in this area is guided by gaps and opportunities identified in research summits, which alternate yearly to focus on Alzheimer’s disease, Alzheimer’s disease-related dementias, or dementia care and services. Smaller, focused workshops are held more frequently on specific aspects of this research.
NIH outlines its Alzheimer’s research efforts in the NIH AD/ADRD Research Implementation Milestones, a research framework detailing specific steps and success criteria toward achieving the goals of the National Plan to Address Alzheimer’s Disease. The milestones also showcase funding initiatives, accomplishments, and highlights of progress toward accomplishing the National Plan goals.
NIH’s research progress is highlighted in the annual Alzheimer’s and related dementias professional judgment budget, which is submitted to Congress each year.
Each year NIH submits a professional judgment budget that estimates the additional funding needed to advance NIH-supported research into the treatment and prevention of Alzheimer’s disease and related dementias. The report also summarizes progress and promising research opportunities. Only two other areas of biomedical research — cancer and HIV/AIDS — follow a similar process designed to accelerate research discovery. This approach is often referred to as a “bypass budget” because of its direct transmission to the President and then to Congress without modification through the traditional federal budget process.
Clinical Research Into Alzheimer’s and Related Dementias
No major advance in Alzheimer’s and related dementias treatment, prevention, or care will be possible without robust clinical research. Clinical research includes studies that involve people so scientists can learn more about disease progression, how behavior and lifestyle factors may affect health, and the safety and effectiveness of an intervention. Advances made through clinical research rely on the volunteers who participate in these types of studies. NIA is working on multiple initiatives to enhance recruitment and retention of diverse populations in clinical research. View some of those resources below.
NIA-funded clinical research includes both observational studies through which researchers gather important information, and clinical trials in which researchers test interventions to treat or prevent disease, improve care and caregiver support, and enhance quality of life for people living with dementia. NIA is currently funding more than 200 active clinical trials.
NIA also funds more than 30 Alzheimer’s Disease Research Centers across the country. Scientists at these centers conduct clinical research to improve diagnosis and care for people with dementia and their families, and to find a treatment or prevention.
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