Using Innovative Funding Mechanisms to Discover, Develop, and Test a Potentially Promising AD Drug

AIT-082 is a novel small molecule that crosses the blood-brain barrier to enhance nerve function. It does this by increasing the levels of nerve growth factors and encouraging nerve sprouting in the brain. The process that AIT-082 has gone through–from initial discovery to tests in humans–illustrates well the way in which NIA’s innovative funding mechanisms work together to foster the development of potentially promising drug treatments for AD.

A Small Business Innovation Research Grant (SBIR) was the first NIH step for AIT-082. This grant allowed scientists to develop the compound and conduct initial tests on its function. Based on the promising results from this work, NIA provided funds through the Investigational New Drug Toxicology for Drugs to Treat Alzheimer’s Disease contract mechanism to begin the formal drug testing process. The studies showed that AIT-082 did not have an adverse toxicologic effect in laboratory animals. The National Institute of Mental Health also contributed to this effort by providing support through a contract to synthesize sufficient quantities of AIT-082 for the toxicologic testing.

These results allowed researchers to take the step from animal tests to tests in humans, and NIA is currently supporting Phase I clinical trials of AIT-082. In Phase 1 studies, a small number of volunteers are given a drug to assess safety at various doses and establish some initial indication of effectiveness. The studies have been conducted through the Alzheimer’s Disease Cooperative Study at four Alzheimer’s Disease Centers sites (the University of California at San Diego, Indiana University, the University of Michigan, and Washington University in St. Louis). In a Phase IA study, eight healthy older volunteers were given single, weekly, increasing doses of AIT-082 or a placebo for 5 weeks. During that time they were tested for side effects and for whether they properly absorbed the drug. They also underwent a battery of neuropsychological memory tests, including word and number recall tests. The study period is complete and the research team is now analyzing the results. A Phase IB study is currently ongoing in which safety and initial effectiveness is being assessed in 36 people with AD.

If the results show that AIT-082 is both safe and has some indication of effectiveness, the next step will be to test the drug in a larger clinical trial for its effect on AD symptoms, development, or progression.


National Institutes of Health

National Institute on Aging


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