Alzheimer's Disease - Research

11/20/19 ·National Institute on Aging

Research supported by the National Institutes of Health (NIH) and other organizations has expanded knowledge of brain function in healthy older people, identified ways that may lessen age-related cognitive decline, and deepened our understanding of Alzheimer’s. 

Many scientists and physicians are working together to untangle the genetic, biological, and environmental factors that might cause Alzheimer’s disease. This effort is bringing us closer to better managing and, ultimately, better treating and preventing this devastating disease.

Types of Research

Different types of research—basic, translational, and clinical research—are conducted to better understand Alzheimer’s and find ways to treat, delay, or prevent the disease.

  • Basic research helps scientists gain new knowledge about a disease process, including how and why it starts and progresses. 
  • Translational research grows out of basic research. It creates new medicines, devices, or behavioral interventions aimed at preventing, diagnosing, or treating a disease.
  • Clinical research is medical research involving people. It includes clinical studies, which observe and gather information about large groups of people. It also includes clinical trials, which test a medicine, therapy, medical device, or other intervention in people to see if it is safe and effective.

Basic Research

Basic research seeks to identify the cellular, molecular, and genetic processes that lead to Alzheimer’s disease. Basic research has focused on two of the main signs of Alzheimer’s disease in the brain: plaques and tangles. Plaques are made of a protein called beta-amyloid and form abnormal clumps outside nerve cells in the brain. Tangles are made from a protein called tau and form twisted bundles of fibers within nerve cells in the brain. 

Scientists are studying how plaques and tangles damage nerve cells in the brain. They can see beta-amyloid plaques and tau tangles by making images of the brains of living people. Such imaging has led to clinical trials that are looking at ways to remove beta-amyloid from the human brain or halt its production before more brain damage occurs. 

Scientists are also exploring the very earliest brain changes in the disease process. Findings will help them better understand the causes of Alzheimer’s. As they learn more, they are likely to come up with better targets for further research. Over time, this might lead to more effective therapies to delay or prevent the disease. 

Genetics is another important area of basic research. Discovering more about the role of genes that increase or decrease the risk of developing Alzheimer’s will help researchers answers questions such as “What makes the disease process begin?” and “Why do some people with memory and other thinking problems develop Alzheimer’s disease while others do not?”

Genetics research helps scientists learn how risk-factor genes interact with other genes and lifestyle or environmental factors to affect Alzheimer’s risk. This research also helps identify people who are at high risk for developing Alzheimer’s and can participate in clinical research on new prevention and treatment approaches.

Translational Research 

Translational research allows new knowledge from basic research to be applied to a clinical research setting. An important goal of Alzheimer’s translational research is to increase the number and variety of potential new medicines and other interventions that are approved for testing in humans. Scientists also examine medicines approved to treat other diseases to see they might be effective in people with Alzheimer’s.

The most promising interventions are tested in test-tube and animal studies to make sure they are safe and effective. Currently, a number of different substances are under development that may one day be used to treat or prevent the symptoms of Alzheimer’s disease and mild cognitive impairment.

Clinical Research 

Clinical research is medical research involving people. It includes clinical studies, which observe and gather information about large groups of people. It also includes clinical trials, which test medicines, therapies, medical devices, or other interventions in people to see if they are safe and effective. 

Clinical trials are the best way to find out whether a particular intervention actually slows, delays, or prevents Alzheimer’s disease. Trials may compare a potential new treatment with a standard treatment or placebo (mock treatment). Or, they may study whether a certain behavior or condition affects the progress of Alzheimer’s or the chances of developing it. 

NIH, drug companies, and other research organizations are conducting many clinical trials to test possible new treatments that may

  • improve memory, thinking, and reasoning skills in people with Alzheimer’s or mild cognitive impairment
  • relieve the behavior problems of Alzheimer’s, such as aggression and agitation
  • delay the progression from mild cognitive impairment (MCI) to Alzheimer’s 
  • prevent Alzheimer’s disease.
A wide variety of interventions are being tested in clinical trials. They include experimental drugs as well as non-drug approaches.