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MaxWell Webinar with Dr. Makoto Tamura and Dr. Abduqodir Toychiev
Due to confidentiality reasons, this webinar will not be recorded, so make sure to join us live!
This webinar will cover:
iPSC-derived neurons with and without TARDBP mutations from ALS patients.
HD-MEA insights into functional changes, including hyperactivity and altered network dynamics in mutated neurons.
Foundational knowledge to advance targeted ALS treatment development.
Speaker
Dr. Makoto Tamura
Mitsubishi Tanabe Pharma Holdings America
USA
Speaker
Dr. Abduqodir Toychiev
Mitsubishi Tanabe Pharma Holdings America
USA
Host
Dr. Laura D’Ignazio
MaxWell Biosystems
Switzerland
Title
Functional Characterization of Induced Pluripotent Stem Cells in Patients with Amyotrophic Lateral Sclerosis with TARDBP Mutation
Abstract
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disorder characterized by motor neuron degeneration leading to muscle weakness and eventual death. Early signs of ALS include muscle twitching, cramps, stiffness, and weakness. As the disease advances, individuals may experience slurred speech, difficulties chewing and swallowing, and, in some cases, cognitive decline. The primary aim of current therapies is to slow disease progression rather than restore neuronal function. To help pave the way for future therapeutic strategies, this study focused on understanding fundamental alterations in ALS neurons with TARDBP mutations. Using single-cell electrophysiology and high-density microelectrode arrays, we identified significant changes in firing rates, action potential activity, neuronal hyperactivity, and network dynamics in neurons derived from induced pluripotent stem cells (iPSCs) of patients with ALS and the TARDBP mutation compared to patients with ALS absent this mutation. These changes were particularly pronounced during neuronal stimulation and modulation by synaptic blockers. Our findings offer crucial insights into the pathological mechanisms of ALS motor neurons, helping to provide the foundational understanding necessary for development of targeted treatments.
Speaker Bio – Dr. Makoto Tamura
Dr. Makoto Tamura is the Senior Director of the NeuroDiscovery Laboratory at Mitsubishi Tanabe Pharma America, leading pivotal efforts in drug development and foundational research for neuropsychiatric and neurological disorders. With a career in neuropharmacology, Dr. Makoto Tamura has expertise in molecular and cellular mechanisms, beginning at the University of Tokyo. As a postdoctoral fellow at Columbia University, he delved into neurophysiological deficits in animal models of psychiatric disorders, employing a systems neuroscience approach. As a senior research scientist at Mitsubishi Tanabe Pharma Corporation, Dr. Makoto Tamura established innovative animal models and physiological assays, bridging the gap from lab to clinical application. Now based in Cambridge, Massachusetts, he focuses on uncovering novel drug targets for neurodegenerative disorders, including amyotrophic lateral sclerosis and Alzheimer’s disease.
Speaker Bio – Dr. Abduqodir Toychiev
Dr. Abduqodir Toychiev is a Senior Research Associate for the NeuroDiscovery Lab at Mitsubishi Tanabe Pharma America. He received his PhD in cellular physiology in 2007 at the National Institute for Physiological Sciences, Okazaki, Japan. Before joining Mitsubishi Tanabe Pharma America in 2022, he worked as a visual neuroscience researcher for more than 10 years at Weill Cornell University and The State University of New York (SUNY) College of Optometry. His current work focuses on understanding the pathophysiological mechanisms of neurological diseases in the hope of developing functional assays for these conditions. He and his team utilize inducible pluripotent stem cells from human patients to study cellular behavior, neuronal activity, and synaptic connectivity. Their aim is to use these studies to better understand neuronal pathophysiology with the ultimate goal of developing successful treatment strategies. Dr. Toychiev is a recipient of the Japanese Government MEXT Scholarship as well as the Yang Investigator Grant from the Academy of Sciences of Uzbekistan.