NEWARK, N.J., March 08, 2016 /PRNewswire/ -- Neurotrope, Inc. (OTCQB: NTRP) today announced that its lead Alzheimer's drug Bryostatin improved disease symptoms and restored normal synaptic networks in a young transgenic animal model of Fragile X Syndrome (FXS), a common genetic cause of mental retardation and autism. The study led by scientists at the Blanchette Rockefeller Neuroscience Institute (BRNI) demonstrated that chronic treatment with bryostatin-1 rescues young fragile X mice (corresponding to 15 – 16 year old children) from the disorder phenotypes, including reversal of most FXS abnormalities of 1) hippocampal brain-derived neurotrophic factor (BDNF) expression, 2) the synaptic anchoring protein, PSD-95, levels, 3) transformation of immature dendritic synapses to fully mature, developed synapses, 4) sub-cellular densities of the presynaptic and postsynaptic membranes, and 5) spatial learning and memory. These results were long lasting and confirmed by electron microscopy. While other attempts at Fragile X therapeutics have focused on reduced mGluR5 receptor levels, the Bryostatin efficacy for treating Fragile X did not require changes of mGluR5 and were even more dramatic than those of a late-onset treatment in adult Fragile X mice. As a result of the mutation of the FMR1 gene, the Fragile X mental retardation protein (FMRP) is not produced. It is known that this protein is abundant in the neurons of the hippocampus and cerebellum of non-affected individuals, but it is not yet fully understood why the absence of FMRP results in the phenotype of Fragile X. We have found that chronic activation of PKCε in the hippocampus by Bryostatin can rescue synapses and spatial cognition, as well as other FXS phenotypes. This rescue in Fragile X mice is remarkably similar to the rescue of synapses, spatial cognition, and other Alzheimer's disease (AD) phenotypes in AD transgenic mice. The new Fragile X study in late brain development is entitled "Rescue of Synaptic Phenotypes and Special Memory in Young Fragile X Mice," was authored by Miao-Kun Sun, Jarin Hongpaisan, and Dr. Daniel L. Alkon, and is published, in the Fast Forward edition of the Journal of Pharmacology and Experimental Therapeutics, dated March 3, 2016.
"What our studies consistently exhibit is that all of the synaptogenic growth factors such as BDNF, NGF, IGF, etc. are activated by Bryostatin/PKC epsilon. Bryostatin has the ability to potentially regenerate the brain wiring that has been lost and restore memory capacity due to many causes of neurodegeneration such as Alzheimer's, Fragile X, Traumatic Brain Injury, Frontal Dementia, Lewy Body disease, etc." Dr. Alkon, the Chief Scientific Officer of Neurotrope, went on to say, "We believe that this latest study provides new evidence that Neurotrope is developing a potential 'universal' therapeutic for neurodegeneration in the brain, however it arises, even due to disease genes as distinct as Fragile X, ApoE and Presenilin."
About Fragile X Syndrome
FXS is the most common cause of inherited intellectual disability and the most common known genetic cause of autism or autism spectrum disorders. There is currently no FDA approved treatment for FXS available on the market today. Symptoms of FXS include moderate to severe learning disabilities, behavioral disorders, seizures and cognitive impairment. FXS is caused by a partial or a full mutation of the FMR1 gene.
Neurotrope BioScience, Inc., a wholly owned subsidiary of Neurotrope, Inc., is at the forefront of biotechnology companies having a focus on developing a novel therapy for the treatment of moderately severe to severe Alzheimer's disease. The scientific basis of our treatment is activation of Protein Kinase C isozymes ε and α by bryostatin, a natural product, which can result in repair of damaged synapses as well as synaptogenesis, reduction of toxic amyloid generation, and enhancement of memory and learning, thus having the potential to improve cognition and behavior in Alzheimer's disease.
Neurotrope is also conducting preclinical studies of bryostatin as a treatment for Fragile X Syndrome and Niemann-Pick Type C disease, two rare genetic diseases for which only symptomatic treatments are currently available. The Food and Drug Administration has granted Orphan Drug Designation to Neurotrope for bryostatin as a treatment for Fragile X Syndrome. Bryostatin has undergone testing in over 1400 people establishing a large safety database.
NTRP has exclusively licensed technology from the Blanchette Rockefeller Neurosciences Institute for Alzheimer's disease and Fragile X Syndrome, has a world-wide, exclusive license with the Icahn School of Medicine at Mt. Sinai for Niemann-Pick Type C disease and is partnered with Stanford University to synthesize and find the next generation bryostatin – called bryologs.
About The Blanchette Rockefeller Neurosciences Institute
Located in Morgantown, WV, BRNI, at West Virginia University, is a unique, independent, non-profit institute dedicated to the study of memory and finding solutions to memory disorders. BRNI was founded in 1999 in memory of Blanchette Ferry Hooker Rockefeller, an Alzheimer's patient and mother of U. S. Senator John D. Rockefeller IV. BRNI is operated in alliance with West Virginia University as well as in collaboration with other academic institutions.
Any statements contained in this press release that do not describe historical facts may constitute forward-looking statements. These forward-looking statements include statements regarding the proposed study and timing of initiation, and continued development of use of bryostatin for Alzheimer's disease and other cognitive diseases, and the Company's ability to list its common shares on a major stock exchange. Such forward-looking statements are subject to risks and uncertainties and other influences, many of which the Company has no control over. Actual results and the timing of certain events and circumstances may differ materially from those described by the forward-looking statements as a result of these risks and uncertainties. Factors that may influence or cause actual results to differ materially from expected or desired results may include, without limitation, the Company's inability to obtain adequate financing, the significant length of time associated with drug development and related insufficient cash flows and resulting illiquidity, the Company's patent portfolio, the Company's inability to expand the Company's business, the Company's inability to meet listing requirements for major stock exchanges, significant government regulation of pharmaceuticals and the healthcare industry, lack of product diversification, availability of the Company's raw materials, existing or increased competition, stock volatility and illiquidity, and the Company's failure to implement the Company's business plans or strategies. These and other factors are identified and described in more detail in the Company's filings with the SEC, including the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2015. The Company does not undertake to update these forward-looking statements.
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