The Kendall Morgan FAST-TRAC (Targeted Research to Advance a Cure) Award

“Effectiveness of established therapeutics for the treatment of Angelman Syndrome”

Awarded to: Edwin Weeber, Ph.D., Department of Molecular Pharmacology and Physiology, University of South Florida.

Dates: December 1st, 2010 – November 30th, 2011 (extended through 2012)

Summary: There is mounting evidence to suggest that a treatment for Angelman syndrome is not just possible, but probable. The lack of known molecular targets associated with AS has hampered the development of specific therapeutics. However, a recent surge of potential therapeutics for other disorders associated with cognitive disruption has begun to be used in human clinical trials. The molecular modes of action for many of these new therapeutic agents have correlates to counter the molecular defects observed in AS. Thus, this proposal seeks to determine the effectiveness of compounds that are FDA approved and currently being used in clinical trials on the well-established AS mouse model. We propose to look at 4 of these compounds at the level of: 1) Degree of cognitive enhancement. 2) Rectification of a biological and genetic abnormality. 3) Increase in synaptic function and/or plasticity. It is our hope that these compounds will have a positive effect on one or more of these aspects. Furthermore, any positive results will prompt a full preclinical evaluation of the compound(s) and may lead to the development of an effective AS therapeutic.

FAST-TRAC (Targeted Research to Advance a Cure) Award

“Minocycline Pre-Clinical Animal Studies”

Awarded to: Edwin Weeber, Ph.D., Department of Molecular Pharmacology and Physiology, University of South Florida.
Dates: June 30th, 2011 – June 30th, 2012

Summary: The previous FAST-TRAC award to Dr. Weeber identified minocycline as a potential therapeutic for Angelman Syndrome; reversing deficits of learning, memory, and motor skills in the AS mouse model.  This award provides additional funds to repeat these studies, increase the number of mice analyzed, and add additional methods for testing the efficacy of minocycline in this model.  These studies will form the basis of the application for IRB approval for a potential clinical trial of minocycline in patients with AS.

FAST-TRAC (Targeted Research to Advance a Cure) Award

“Efficacy of Minocycline for the Treatment of Angelman Syndrome”

Clinical Trial Completed in March 2013 – Results being prepared for publication

Awarded to: Edwin Weeber, Ph.D., Department of Molecular Pharmacology and Physiology, University of South Florida.Dates: March 1st, 2012 – March 1st, 2013

Summary: Angelman syndrome is a rare genetic disorder characterized by phenotypic traits such as global developmental delay, ataxia and seizure. Children diagnosed with AS display a behavioral profile consisting of a happy demeanor with easily provoked laughter and hyperactivity. Building on our previous FAST-TRAC studies, the objective of this study is to examine the efficacy of minocycline to improve the cognitive and behavioral deficits of the Angelman Syndrome participant. Currently, the only treatment available for AS patients consists of supportive care including seizure control and behavioral therapy. Our laboratory has collected data indicating improved motor function and cognition after the administration of MC as well as enhanced synaptic function. Further, previously published data suggests the administration of MC to children with other genetically based neurologic disorders (e.g.Fragile X syndrome) dramatically improved dendritic spine morphology and behavioral performance. Taken together, we posit, children treated with MC will experience the same beneficial effects observed in previous studies and the AS mouse model.

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