Neurotrope, Inc., a clinical-stage company developing a disruptive therapy for the treatment of severe Alzheimer's disease (AD), has entered into a research collaboration with the International Rett Syndrome Foundation, DBA: Rettsyndrome.org, to explore the potential of Bryostatin for the treatment of Rett syndrome.
Between 85% and 95% of Rett Syndrome cases are reported to be caused by a mutation of the MECP2 gene. This mutation causes a conserved pathology that comprises impaired neurite development and impaired synaptic connectivity, along with a corresponding impairment in social and cognitive functions. Such synaptic dysfunctions result from genetically altered functions of postsynaptic density proteins. Normal neurite growth and postsynaptic development may be regulated and augmented by growth factors, such as brain derived neurotrophic factor (BDNF). Drugs that promote BDNF function may therefore be of use in the treatment of progressive developmental disorders such as Rett. Thus, there is a need for the development of therapies that activate key synaptic growth factors such as BDNF, insulin like growth factor (IGF) and nerve growth factor (NGF), which regulate and augment normal neurite growth and postsynaptic development in patients suffering from Rett syndrome. Bryostatin, by targeting PKC epsilon, activates these growth factors.
As part of the collaboration, the National Cancer Institute supplied the drug to conduct the study under the Rettsyndrome.org Scout Program. The purpose of the study is to run a behavioural battery of tests to screen and assess the potential for Bryostatin for the therapeutic treatment of Rett syndrome in a mouse model. Assessments will include body weight, grip strength, gait analysis, and neurologic/behavioural parameters. All raw data, samples and specimens arising from the performance of this study will remain the property of Neurotrope. The study will be funded and managed by Rettsyndrome.org.
"We are most excited to be chosen to participate in The Scout Program and are hopeful that Bryostatin PKCe activation properties, based on the scientific work of Dr. Dan Alkon, the company's chief scientific officer, may bring some benefit to the thousands of individuals afflicted with Rett syndrome," stated Charles S. Ramat, president and chief executive of Neurotrope, Inc. "In addition to our focus on Alzheimer's disease and our present phase 2b clinical trial, this is an important step in Neurotrope's commitment to orphan diseases in addition to our ongoing efforts in Fragile X syndrome and Niemann Pick Type C disease."
"Neurotrope Bioscience has a very exciting drug in regard to Rett syndrome and I am very anxious to see its actions in our Scout Program. This is the exact type of compound we are looking for and every indication is that it is pointed at a very important pathway with regard to Rett syndrome biology," stated Dr. Steven Kaminsky, chief science officer at the International Rett Syndrome Foundation.
Rett syndrome is a rare non-inherited genetic postnatal neurological disorder that occurs almost exclusively in between 1:10,000 to 1:15,000 live female births and leads to lifelong impairments, affecting nearly every aspect of a child's life: their ability to speak, walk, and eat is effected, seizures develop and scoliosis occurs, and many develop irregular breathing patterns. Those diagnosed with Rett syndrome require maximum assistance with basic daily activities. The hallmark sign of Rett syndrome is near constant repetitive hand movements. Cognitive assessment in children with Rett syndrome is complicated, but they understand far more than they can communicate to us, evidenced by their bright and attentive eyes, and their ability to express a wide spectrum of moods and emotions.
Rettsyndrome.org aims to accelerate more discoveries to the clinic and committed over $1 million towards the Scout Program, a Drug Discovery Screen in a mouse model of Rett syndrome. The Scout Program will aggressively accelerate the testing of compounds, whether new or repurposed, through standardized preclinical studies in the Rett mouse model. This program will test many novel compounds and small molecules.
Rettsyndrome.org is the most comprehensive nonprofit organization dedicated to accelerating research of treatments and a cure for Rett syndrome and related disorders while providing information and family empowerment. As the world's leading private funder of Rett syndrome research, Rettsyndrome.org has funded over $38M in high-quality, peer-reviewed research grants and programs to date.
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.
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.