Alzheimers Archives • Fragile X Research

Crossroads of Fragile X and Alzheimers Research

Katie Clapp Research Updates July 20, 2015May 16, 2018Alzheimers, amyloid precursor protein

Researchers investigate treatment targets which may address both Fragile X Syndrome and Alzheimer’s disease Last week researchers at VIB Leuven in Belgium published evidence that a brain pathway involving the protein APP (Amyloid Precursor Protein) plays a vital role in development of Fragile X syndrome, one of the most common causes of autism. Scientists led by Dr. Emanuela Pasciuto in the laboratory of Prof Claudia Bagni published findings of their study in the journal Neuron. The team identified the molecular mechanisms behind the elevated levels and metabolism of APP protein in a mouse model. This dysregulation affects brain development and behavior, at a stage where the infant’s neuronal connections i.e. synapses are being formed and remodeled. Using a newly developed agent the team was able to reduce the cellular dysfunction and behavioral alterations paving way for potential new therapies for autism in the future. Dysregulated ADAM10-Mediated Processing of APP during a Critical Time Window Leads

Bryostatin Restores Learning and Memory in Adult Fragile X Mice

FRAXA Research Foundation Research Updates March 11, 2015May 8, 2018Alzheimers

Just as the Amazon rainforest may hold a cure for cancer if only scientists can find it, a bizarre marine critter found off the California coast — Bugula neritina— is the only known source of a potential new Fragile X treatment, Bryostatin. Last month, FRAXA sat down with scientists from Neurotrope BioScience, a specialty biopharmaceutical company developing medicines for rare diseases and Alzheimer’s based on Bryostatin. Their Fragile X program is based on research by a West Virginia team led by Daniel Alkon, MD, which showed that Bryostatin-1 restores hippocampal synapses and spatial learning and memory in adult fragile X mice. “Our results show that synaptic and cognitive function of adult FXS mice can be normalized through pharmacologic treatment and that bryostatin-1-like agents may represent a novel class of drugs to treat fragile X mental retardation even after postpartum brain development has largely completed,” remarked Dr. Alkon. Bugula neritina, the source of bryostatin. Photo source http://seanet.stanford.edu/Bryozoa/ Bugula and Bryostatins Often mistakenRead more

Ab-Mediated Translation in Fragile X Syndrome

FRAXA Research Foundation 2011 Grants, 2012 Grants, Biomarkers, Cara Westmark, PhD, Completed Research Grant, Treatment Targets, University of Wisconsin September 13, 2012April 25, 2018Alzheimers

With a $120,000 grant from FRAXA Research Foundation during 2011-2012, Dr. Cara Westmark at the University of Wisconsin explored the role of AbPP as a potential treatment option for fragile X. AbPP produces b-amyloid which is over-expressed in Alzheimer’s disease (AD) and Down syndrome.

Using Fenobam to Reduce APP and Abeta in Fragile X Mice

FRAXA Research Foundation 2006-2010 Grants, Cara Westmark, PhD, Completed Research Grant, James Malter, PhD, Treatment Targets, University of Wisconsin February 14, 2008April 28, 2018Alzheimers, Amyloid beta, amyloid precursor protein, fenobam

With a $130,000 grant from FRAXA Research Foundation over 2008-2008, Drs. James Malter and Cara Westmark at the University of Wisconsin studied the relationship between the fragile X protein FMRP and APP, a protein important to the pathology of Alzheimer’s Disease. APP may also contribute to the pathology of fragile X, and its major metabolite, Aß, may contribute to abnormal protein synthesis via a positive feedback loop. This project sought to restore normal dendritic protein synthesis in fragile X mice by breaking into this loop.