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Drug Candidates

As scientific knowledge of Alzheimer’s and related dementias has grown, drug development projects have evolved to reflect this complexity. Like cancer and its many subtypes, the many types of dementias will likely require targeted treatments aimed at an individual’s unique disease characteristics.

While FDA’s June 2021 accelerated approval of aducanumab is a milestone in Alzheimer’s research, an array of treatments will be necessary to successfully halt or reverse the effects of these diseases for everyone. A precision medicine approach would deliver specific treatments based on a person’s type of dementia as diagnosed through lab and imaging tests.

As knowledge advances, scientists are developing more precise treatments and preventions aimed at specific types of Alzheimer’s and related dementias. Today, many kinds of compounds are chosen for testing and development as drug candidates.

In addition to drugs aimed at a specific disease pathway, NIH-funded researchers are also exploring many variations of targets simultaneously, considering combinations of treatments, and working to repurpose existing drugs to treat these diseases.

New drug development projects in the lab

NIA recently funded several new projects (see table) through its Alzheimer’s Drug Development Program. Each project is focused on developing a new drug that targets a different biological process, such as brain inflammation, known to go awry during the development of Alzheimer’s and related dementias. If successful, these NIH-supported preclinical drug development studies will result in new candidate drugs that could then be tested in people.

Different types of drug candidates. See full PDF for details
New drugs tested in 2020, see full PDF for details

Drugs in clinical trials

As a result of the substantial research progress achieved over the last several years in understanding how Alzheimer’s and related dementias develop and worsen, the drug development pipeline has never been more diverse.

Drug candidates now in clinical studies target multiple aspects of the disease process. Thanks to increased investments in Alzheimer’s and related dementias research, NIH-funded clinical studies extend beyond drug candidates that target amyloid plaques in the brain. The continuously updated list of NIA-Funded Active Alzheimer’s and Related Dementias Clinical Trials and Studies catalogs the many different disease pathways.

NIA is supporting many late-stage drug trials and early-stage drug trials for Alzheimer’s and related dementias. In addition, several studies are evaluating drug candidates for treating psychological or neurological symptoms of dementia, including apathy, agitation, and restless legs.

Drug candidates that are shown to be safe and effective in late-stage trials can then be presented to FDA. Before drugs are approved for use in people, FDA ensures that drugs work correctly and that their health benefits outweigh their known risks.

Bridge showing steps to clinical drug discovery. See full PDF for details