Scientists evaluate HIV/AIDS, diabetes and organ transplant drugs as possible therapies for Alzheimer's

More than ever before, Alzheimer's researchers understand that a variety of approaches will be needed - most likely used in combination - for effective treatment of the disease.

With the recent influx of new funding - including more than $2 billion annually at the National Institute on Aging - researchers are expanding the exploration of new treatment avenues. At the same time, scientists are more extensively testing the potential benefits of drugs approved for other diseases for the treatment of dementia.

As an example, through newly-awarded grants from the Alzheimer's Association Part the Cloud Translational Research initiative, scientists are evaluating the use of existing HIV/AIDS, diabetes and organ transplant drugs as possible therapies for Alzheimer's dementia. Other research funded by the recent grants will investigate novel drugs that might alleviate, delay or slow the brain changes associated with Alzheimer's.

To drive the field forward and create new therapies for people living with Alzheimer's and all dementia, the Alzheimer's Association believes it is important to fund innovative science that explores both new mechanisms and the repurposing of existing drugs. The Part the Cloud program is vital to advancing high-risk, high-reward research that might not otherwise be explored without this financial support."

Maria C. Carrillo, Ph.D., Alzheimer's Association chief science officer

Repurposing existing drugs for new uses can speed up the research process. Since scientists are building on previous research, much is already known about the drugs' potential side effects, it may take less time for the drugs to be tested, and the clinical trials may be less expensive. For many of the same reasons, repurposing can also speed review by the U.S. Food and Drug Administration.

The 2020 Part the Cloud: Translational Research Funding for Alzheimer's Disease grants provide essential support for early-phase clinical studies in people. Each researcher will receive up to $750,000 over two years. Part the Cloud awards are specifically designed to accelerate translation of findings from the laboratory, through trials, into possible therapies.

"No stone can be left unturned. We must advance all potential avenues of treatment, and explore methods for combining successful approaches," Carrillo said. "Alzheimer's and all dementia are complex, and their effective treatment and prevention will likely also be a complex but achievable task."

Research evaluating repurposing of drugs

Steven Arnold, M.D., Massachusetts General Hospital, Boston

Biomarker and Neural System Effects in Calcineurin Inhibition with Tacrolimus

A Phase Ib/IIa study to evaluate whether Tacrolimus - a Food and Drug Administration (FDA)-approved drug to prevent organ transplant rejection - can be repurposed as a potential therapy for Alzheimer's.

Paul Edison, M.B.B.S., F.R.C.P., Ph.D., Imperial College, London

Evaluating oral semaglutide as a treatment for Alzheimer's disease A Phase IIa clinical trial to evaluate a type 2 diabetes medication as a potential treatment for Alzheimer's disease. Type 2 diabetes is a risk factor for Alzheimer's disease. Researchers believe that semaglutide, a drug that helps the body to maintain appropriate levels of sugar, may also support brain function by improving thinking and memory.

John Sedivy, Ph.D., Brown University, Providence, Rhode Island

Stephen Salloway, M.D., Brown University and Butler Hospital, Providence, Rhode Island

Repurposing Nucleoside Reverse Transcriptase Inhibitors for Treatment of Alzheimer's Disease A Phase I clinical trial to evaluate whether an HIV/AIDS medication might be repurposed to reduce brain inflammation in individuals with Alzheimer's and other brain diseases.

Research evaluating new mechanisms

James Kirkland, M.D., Ph.D., Mayo Clinic, Rochester, Minnesota

ALSENLITE: An Open-Label Pilot Study of Senolytics for Alzheimer's Disease A Phase IIa clinical trial to determine if a novel drug targeting the removal of certain aging brain cells in regions impacting cognition, language and memory can benefit people with age-related brain disorders, including Alzheimer's disease.

Maurice Zauderer, Ph.D., Vaccinex, Inc., Rochester, New York

SEMA4D Blockade Safety and Brain Metabolic Activity in Alzheimer's Disease A Phase I clinical trial to evaluate a new experimental drug that may reduce inflammation in the brain.