A Buck Milestone-Our First Clinical Trial is Underway In Australia

The Buck Institute’s first clinical trial, which tests a potential treatment for amnestic mild cognitive impairment (aMCI), got underway recently when the first patient in Australia received an exploratory therapeutic. aMCI is often an early indicator of Alzheimer’s disease.

The drug being tested is called F03 - a therapeutic that binds three different receptors involved in cognitive function. The ongoing Phase 1b/2a trial is a randomized, double-blind, placebo-controlled study assessing the safety, tolerability, pharmacokinetics and preliminary efficacy of two dose levels of F03 in 36 aMCI patients.

Results could be in by the end of summer 2015. If they are positive, F03 will progress into more advanced clinical studies involving significant numbers of patients in the U.S. and Australia. F03 has been approved in Australia (and 48 other countries) for use in a different indication; it is very well tolerated and has a very strong safety profile and history.

Stelios Tzannis (left) is running the Buck’s first clinical trial based on research by Dale Bredesen (right).

“We are excited to commence the first clinical trial of F03 for the treatment of aMCI,” says Stelios Tzannis, PhD, the Buck’s Director of Clinical Sciences. “F03’s novel, multi-targeted mechanism of action gives us increased hope that it may be a highly valuable therapeutic candidate with potential disease-modifying capacity.”

Emanating from groundbreaking research by Dale Bredesen, MD and his lab at the Buck, F03 addresses a key process in the brain that is distorted early in the development of Alzheimer’s disease: the processing of amyloid precursor protein (APP). A number of supporting preclinical studies have demonstrated that F03 significantly improves cognition and reverses memory loss in mouse models of Alzheimer’s, providing early evidence for a potentially disease-modifying effect. The proof-of-principle for the research and the ensuing clinical trial were established in 2006 when Dr. Bredesen published a study involving genetically engineered mice that did not have memory problems despite the presence of amyloid plaques (a hallmark of Alzheimer’s) in their brains.

“It is extremely rewarding to have this work move into human trials—we are optimistic about this important first trial,” says Bredesen, whose Buck lab continues to work on Alzheimer’s therapeutics while he also takes the helm as the Director of the Mary S. Easton Center for Alzheimer’s Research at the University of California, Los Angeles.

The F03 clinical trial was enabled by federal grants as well as support from the Ellen and Douglas Rosenberg Foundation, Bechtel Foundation, Alzheimer’s Association, and philanthropic support from Hussam Abu Issa. “We are grateful for the varied sources of support that led to this groundbreaking work,” Tzannis says. “The Buck Institute continues to build and expand its position in the field of Alzheimer’s disease and other neurodegenerative disorders, and has multiple new funding opportunities. We are poised to move our groundbreaking science toward development of novel, safe and effective drugs to combat these devastating diseases.”

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