Cleveland Researchers Recruiting for Alzheimer’s Disease Clinical Trial

Case Western Reserve University School of Medicine and University Hospitals (UH) Cleveland Medical Center have enrolled their first participant in a new clinical research study evaluating the potential benefits of an investigational medicine for people with early onset Alzheimer’s disease.

The Phase 1 research study, called Efavirenz in Patients with Mild Cognitive Impairment, is evaluating small doses of the drug efavirenz (EFV) in people with mild cognitive impairment and mild Alzheimer’s disease. At a much larger dose, EFV is approved by the U.S. Food and Drug Administration as an anti-retroviral medication for adults, including the elderly.

“This clinical trial is based on our research findings suggesting that small doses of EFV activate a specific brain enzyme that eliminates excess cholesterol,” said Irina Pikuleva, PhD, the study’s co-principal investigator (PI), the Carl F. Asseff Professor of Ophthalmology, and professor of pharmacology at the School of Medicine. “We found that, in a mouse model of Alzheimer’s disease, activation of this enzyme leads to behavioral and other improvements.”

The goal is to find the EFV dose that activates this enzyme, called cytochrome CYP46A1, in the human brain. Pikuleva’s research is focused on eliminating cholesterol from the brain and retina to help develop new treatments for Alzheimer’s disease and age-related macular degeneration.

The clinical trial will evaluate the safety and tolerability of EFV in 36 people with mild cognitive impairment and early dementia from Alzheimer's disease. Half of the patients will be recruited by UH Cleveland Medical Center and half by Massachusetts General Hospital. The 18 people recruited at each hospital will be divided into three groups—two that will take specific doses of EFV and a placebo group—evaluated over 20 weeks.

“This study represents a new approach to the treatment of Alzheimer’s disease,” said Alan Lerner, MD, co-PI, director of the UH Brain Health and Memory Center, and Neurological Institute Chair for Memory and Cognition. “Cholesterol is involved with Alzheimer’s in several ways, and the levels of cholesterol in the brain may affect the progression of the disease.” Lerner is also a professor at the School of Medicine.

Clinicians at Case Western Reserve and UH are seeking eligible enrollees for the study. Participants must be between age 55 and 85, diagnosed with mild cognitive impairment or early dementia due to Alzheimer’s, and have no major health issues or diseases that could interfere with the study, such as use of a cholesterol-lowering drug. Criteria to participate can be found in more detail at Interested participants can also call (216) 368-3823 for more information. The identifier (NCT number) is NCT03706885.


About Alzheimer’s disease
An estimated 5.7 million people in the U.S. suffer from mild-to-moderate Alzheimer’s disease, with those numbers expected to triple by 2050 without prevention or cure. Alzheimer's disease is a progressive, fatal neurodegenerative dementia that accounts for 60 to 80 percent of dementia cases. Currently, at least 70,000 volunteers are needed to participate in more than 150 active clinical trials and studies that are testing ways to understand, diagnose, treat, and prevent Alzheimer’s disease.

About the Alzheimer’s Drug Discovery Foundation (ADDF):
Funds for the clinical trial were awarded to Case Western Reserve University School of Medicine by the ADDF.  The mission of the Alzheimer's Drug Discovery Foundation is to rapidly accelerate the discovery of drugs to prevent, treat and cure Alzheimer's disease. The ADDF follows a venture philanthropy model, funding breakthrough research in academia and the biotech industry. The ADDF has awarded more than $115 million to fund over 590 Alzheimer's drug discovery programs and clinical trials in 18 countries. Twenty percent of the drugs in clinical development for Alzheimer's disease received support from the ADDF.

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