Matrix Metalloproteinase-2/-9 Inhibitor For The Treatment Of ALS

用于治疗 ALS 的基质金属蛋白酶-2/-9 抑制剂

• 批准号: 9763774
• 负责人: IRVING SUCHOLEIKI
• 金额: $ 45.76万
• 依托单位: AQUILUS PHARMACEUTICALS, INC.
• 依托单位国家: 美国
• 财政年份: 2019
• 资助国家: 美国
• 起止时间: 2019-09-15 至 2021-01-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9763774
• 关键词: Address; Amyotrophic Lateral Sclerosis; Animal Model; Applications Grants; Award; Behavioral; Biochemical; Biological; Biological Availability; Biological Markers; Body Weight decreased; CASP1 gene; Chemicals; Clinic; Deterioration; Devices; Diagnosis; Diagnostic; Disease; Disease Progression; Disease model; Dose; Drug Kinetics; FDA approved; Female; Gelatinase A; Gelatinase B; Goals; Investigational New Drug Application; Label; Life; Life Expectancy; Measures; Methodology; Morphology; Motor Neurons; Mus; National Institute of Neurological Disorders and Stroke; Nervous system structure; Neurodevelopmental Disorder; Onset of illness; Oral; Penetration; Pharmaceutical Preparations; Pharmacologic Substance; Phase; Proteins; Riluzole; Rodent Model; Rotarod Performance Test; Running; Small Business Innovation Research Grant; Spinal; Technology; Testing; Therapeutic; Tissues; Work; animal efficacy; diagnostic assay; dosage; efficacy study; efficacy testing; inhibitor/antagonist; male; mouse model; nervous system disorder; novel; painful neuropathy; pre-clinical; research clinical testing; response; superoxide dismutase 1

Project Summary/Abstract. Aquilus Pharmaceuticals, Inc. proposes to evaluate the effects of a selective, dual active, matrix metalloproteinase-2 (MMP-2) and MMP-9 inhibitor, AQU-118, in the superoxide dismutase-1 (SOD-1) mouse model of amyotrophic lateral sclerosis (ALS). The goals for Phase I are to chemically synthesize AQU-118, determine its oral pharmacokinetics (PK) in SOD-1 mice and then determine its efficacy in the SOD-1 mouse model of ALS with dose response. Lastly, a tissue morphology & immunohistochemical study will then be performed to determine whether or not oral dosing with AQU-118 results in any biochemical changes within spinal tissues as compared to controls. The ultimate goal of the proposed work is to evaluate whether a dual active MMP-2/-9 inhibitor can be used to treat ALS.

项目总结/抽象。Aquilus制药公司提议评估a