Drug for early Alzheimer's heads to clinical trial

NlH awards Johns Hopkins $7.5 million grant to test treatment to prevent or delay onset of dementia

Johns Hopkins University researchers have received an estimated $7.5 million National Institutes of Health grant to clinically test what would be the first treatment to prevent or delay the onset of Alzheimer's dementia.

Image caption: Michela Gallagher

The five-year grant will fund a global clinical trial to test a novel therapy for an existing drug that reverses a condition in elderly patients at high risk for dementia. The research that led to this therapeutic approach was led by Johns Hopkins neuroscientist Michela Gallagher, who is also co-primary investigator for the grant.

During the HOPE4MCI Phase 3 trial, which could start by early 2016, levetiracetam, which has been commonly prescribed for more than a decade to treat epilepsy, will be compared to a placebo. In prior studies, Gallagher and colleagues have shown that very low doses of the drug calms hyperactivity in the brain of patients with amnestic mild cognitive impairment (aMCI), a condition in which memory impairment is greater than expected for a person's age and which greatly increases risk for Alzheimer's dementia. More than 5.6 million Americans and 25 million people globally suffer from aMCI.

"The state of the science points to hippocampal overactivity in aMCI as a strong predictor of progression to Alzheimer's dementia and the earliest point at which this pre-dementia condition can be diagnosed before significant irreversible neurodegeneration kills brain cells," Gallagher said. "We expect our upcoming trial to demonstrate efficacy in preserving cognition and memory in aMCI patients while delaying progression to the clinical stage of Alzheimer's dementia."

Also see: Hopkins, AgeneBio to launch trial of Alzheimer's drug in partnership with NIH (The Baltimore Sun)

In the HOPE4MCI Phase 3 trial, a public-private partnership between the NIH's National Institute on Aging, Johns Hopkins and biopharmaceutical company AgeneBio, patients will receive the drug AGB101 in an extended release once-a-day tablet at approximately one-fifth to one-twelfth of the dose most commonly prescribed for epilepsy, or a placebo. The trial is expected to last 18 months to two years.

The Johns Hopkins research team includes co-primary investigator Marilyn S. Albert, director of the Division of Cognitive Neuroscience in the Department of Neurology and director of the Johns Hopkins Alzheimer's Disease Research Center; Professor of Neurology Gregory Krauss; Michael I. Miller, director of the Center for Imaging Science; Susumu Mori, professor in the department of radiology; and professors of biostatistics Scott Zeger and Brian Caffo.

Gallagher, the Krieger-Eisenhower Professor of Psychology and Neuroscience, is the founder of, and a member of the scientific board of, AgeneBio, a biotechnology company focused on developing treatments for diseases that affect brain function. The company is headquartered in Baltimore.

Gallagher will not participate in any clinical aspects of the study because she owns AgeneBio stock, which is subject to certain restrictions under Johns Hopkins policy. She is entitled to shares of any royalties received by the university on sales of products related to her inventorship of intellectual property. The terms of these arrangements are managed by the university in accordance with its conflict-of-interest policies.

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