News Bryostatin-1, Molecule to Protect Synapses, May Move Into MS Trials Bryostatin-1, Molecule to Protect Synapses, May Move Into MS Trials by Marta Figueiredo, PhD | February 24, 2022 Share this article: Share article via email Copy article link SynaptogenixĀ announced plans to advance bryostatin-1, its lead candidate, into clinical trials forĀ multiple sclerosisĀ (MS), marking the third neurological disease the small molecule therapy is being developed to potentially treat. āMultiple sclerosis joins Alzheimer’s disease (“AD”) and Fragile X syndrome as our third indication with potential clinical benefit from Bryostatin-1,ā Daniel Alkon, MD, Synaptogenixās president and chief scientific officer, said in a press release. āElimination of synapses in MS patients, like those lost in AD, has not been addressed by currently available drug strategies,ā Alkon added. Synapses are the points of near contact between nerve cells where cells transmit chemical and electrical signals to one another. āThrough its [synaptic formation-promoting], restorative mechanisms of action, we believe that Bryostatin-1 is uniquely positioned to target synaptic loss and cognitive dysfunction in MS, and potentially other aspects of the disease such as inflammation and [myelin loss],ā he said. Recommended Reading January 26, 2022 Columns by Beth Ullah The Trials and Tribulations of MS Medications SynaptogenixĀ expects to file an investigational new drug application to the U.S. Food and Drug Administration in the coming months, asking for clearance to launch a first trial of bryostatin-1 in people with MS. Should it be cleared, the study ā aiming to assess the therapyās safety and effectiveness in this patient population ā will be conducted in collaboration with Cleveland Clinic through a new consulting agreement. āWe will work with the Cleveland Clinic to finalize a protocol as soon as possible with the goal of moving towards a clinical trial soon thereafter,ā Alkon said. Alan Tuchman, MD, Synaptogenixās CEO, noted this collaboration would be similar to one the company forged with Nemours A.I. DuPont Hospital for Children to bring bryostatin-1 into trials for fragile X. āAdvancing our clinical development plans through partnership has been a strategic focus over the past year and will continue to be a key focus going forward,ā Tuchman said. Bryostatin-1 is a small molecule that works by activating protein kinase C, an enzyme required for maintaining synapse health, and involved in learning and memory. The therapy can also influence the immune system toward an anti-inflammatory environment. Notably, bryostatin-1 can cross the blood-brain barrier, a highly selective membrane that prevents microbes and large molecules in circulating blood from reaching the central nervous system (CNS; the brain and spinal cord). This barrier is often an obstacle for CNS-targeting therapies. As such, the therapy is expected to improve synaptic health, cognitive function, and reduce inflammation and its damaging effects in people with neurodegenerative diseases such as MS and Alzheimerās, and neurodevelopmental conditions that include fragile X syndrome. In preclinical studies, bryostatin-1 restored synapses and triggered the formation of new ones, while preventing nerve cell death. It also was reported toĀ improve learning and memory in animal models of fragile X syndrome, Alzheimerās, and toĀ reverse neurological deficits in a mouse model of MS. When administered directly into the bloodstream, the therapy showed a favorable safety profile in previous clinical trials in cancer and Alzheimerās. Bryostatin-1 is currently being evaluated in Alzheimerās patients in a Phase 2 trial (NCT04538066) and a trial in people with fragile X syndrome is expected. Synaptogenix was formed as a spinoff from Neurotrope ā bryostatin-1ās original developer ā in late 2020, as part of Neurotropeās merger with Metuchen Pharmaceuticals, forming Petros Pharmaceuticals. Print This Page About the Author Marta Figueiredo, PhD Marta holds a biology degree, a masterās in evolutionary and developmental biology, and a PhD in biomedical sciences from the University of Lisbon, Portugal. She was awarded a research scholarship and a PhD scholarship, and her research focused on the role of several signaling pathways in thymus and parathyroid glands embryonic development. She also previously worked as an assistant professor of an annual one-week embryology course at the University of Lisbonās Faculty of Medicine. Tags Alzheimer's disease, Cleveland Clinic, cognitive function, neuroinflammation
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