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Multi-path DCS as a novel non-invasive treatment for amyotrophic lateral sclerosis

多路径 DCS 作为肌萎缩侧索硬化症的新型非侵入性治疗方法

基本信息

批准号：
10384856
负责人：
Zaghloul Ahmed
金额：
$ 37.16万
依托单位：
PATHMAKER NEUROSYSTEMS, INC.
依托单位国家：
美国
项目类别：
财政年份：
2021
资助国家：
美国
起止时间：
2021-09-23 至 2023-08-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/10384856
关键词：
ALS pathology ALS patients Affect Amyotrophic Lateral Sclerosis Animal Model Animals Anodes Bilateral Blood flow Brain Brain-Derived Neurotrophic Factor Cathodes Cessation of life Characteristics Chronic Clinical Clinical Data Clinical Trials Cohort Studies Data Development Devices Diagnosis Disease Disease Progression Effectiveness Electrodes Electrophysiology (science)FDA approved Feasibility Studies Funding Goals Growth Factor Heat shock proteins Heat-Shock Proteins 70 Human Impairment Lead Methods Modality Modeling Molecular Motor Motor Neuron Disease Motor Neurons Mus Muscle Muscle Weakness Na(+)-K(+)-Exchanging ATPase Neural Pathways Neurodegenerative Disorders Neurons Paralysed Pathologic Patients Pharmaceutical Preparations Pharmacology Phase Physiological Placebos Potassium Chloride Preparation Prophylactic treatment Pump Reporting Research Research Personnel Rodent Small Business Technology Transfer Research Sodium Source Spinal Spinal Cord Techniques Technology Testing Therapeutic Therapeutic Uses Time Tissues Transgenic Mice Transgenic Organisms Translating United States United States National Institutes of Health Validation Vascular Endothelial Growth Factors Work active method amyotrophic lateral sclerosis therapy base clinical development clinical practice cost density design efficacy evaluation improved motor disorder mouse model nervous system disorder neuronal excitability neuroregulation novel novel strategies pre-clinical programs protective effect spasticity spinal pathway success superoxide dismutase 1 symporter translational study

项目摘要

PROJECT SUMMARY / ABSTRACT Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects motor neurons in the spinal cord and brain. ALS causes muscle weakness and paralysis and eventually death. According to the ALS Association, every 90 minutes there is a new diagnosis of ALS, as well as an associated death. Nearly 5,000 people are diagnosed with ALS each year and at least 16,000 are living with the disease at any given time in USA alone. There are 3 FDA-approved drugs for ALS, however, the effectiveness of these drugs is limited. In this Phase 1 STTR project, we propose translational studies to evaluate the effects of a recently developed neuronal hyperexcitability suppression technique in ALS models. If successful, the implications of the current project are far-reaching changes in clinical practice for treating ALS using non-invasive hyperexcitability suppression. PathMaker Neurosystems Inc. is a clinical-stage neuromodulation company developing first-in- class devices for the treatment of serious neurological disorders based upon our pioneering multi-path DCS technology, which enables the non-invasive modulation of spinal circuits and motor neuron activity. Our objective for this Phase 1 STTR project is to conduct key translational studies that will be essential for advancing this hyperexcitability suppression technology towards human clinical trials as a safe and efficacious new treatment for ALS. The project consists of 3 Specific Aims: In Specific Aim 1, we will develop and deliver research-use multi-path DCS devices facilitating concurrent animal stimulations. In Specific Aim 2, we will expand on initial transgenic ALS mouse studies to further assess the effects of multi-path DCS on survival, motor function and excitability. In Specific Aim 3, we will conduct motor neuron counts to assess the effects of multi-path DCS on motor neuron survival. These Specific Aims will help generate a package of pre-clinical data that will provide a basis to move the technology into a human clinical feasibility study. As our company works to translate the groundbreaking academic studies that have identified a novel approach to the treatment of ALS based on neuronal hyperexcitability suppression, Phase 1 STTR support will advance this revolutionary treatment option to the brink of human clinical trials, and will pave the way for application of this technology to potentially other motor neuron diseases.

项目摘要/摘要