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.
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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 antiretroviral medication for adults, including the elderly. Anti-retroviral drugs are medications for the treatment of infection by retroviruses, a virus whose genes are encoded in RNA instead of DNA that infect and then make copies of cells to spread.
“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, 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, 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.
Source: Case Western Reserve University