Intravesical Liposome Treatment for Interstitial Cystitis

膀胱内脂质体治疗间质性膀胱炎

基本信息

批准号：
7536874
负责人：
JONATHAN H KAUFMAN
金额：
$ 37.5万
依托单位：
LIPELLA PHARMACEUTICALS, INC.
依托单位国家：
美国
项目类别：
财政年份：
2006
资助国家：
美国
起止时间：
2006-09-15 至 2010-06-30
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/7536874
关键词：
Achievement Address Affect Agreement American Animal Experiments Animal Model Animals Antineoplastic Agents Anxiety Applications Grants Award Biochemistry Biodistribution Biological Biological Assay Biological Availability Biology Biophysics Biotechnology Bladder Book Chapters Book Reviews Budgets Businesses C Fiber Calculi Canada Catheters Chairperson Chronic Chronic Disease Clinical Clinical Data Clinical Research Clinical Trials Conceptions Condition Congresses Consult Contracts Data Collection Development Disease Doctor of Philosophy Dose Drug Delivery Systems Drug Formulations Educational workshop Engineering Expenditure Faculty Felis catus Film Freeze Drying Frequencies Funding Future Goals Grant Growth Hand Health Hepatic Human Hydration status Hyperactive behavior Increased frequency of micturition Inflammatory Institutes Intellectual Property International Interstitial Cystitis Intravesical Administration Intravesical Instillation Investigational Drugs Investigational New Drug Application Investments Laboratories Left Legal patent Licensing Life Lipids Liposomes Literature Marketing Master&apos s Degree Measures Medical Medical center Membrane Lipids Mental Depression Methods Modeling Molecular Muscle satellite cell Neck New Drug Approvals North Carolina Numbers Organ Outcome Pain Paper Patients Peer Review Pelvic Pain Pennsylvania Pharmacists Pharmacologic Substance Pharmacology Pharmacy Schools Phase Phase I Clinical Trials Plant Leaves Plasma Potassium Chloride Procedures Production Program Research Project Grants Protamine Sulfate Public Health Publishing Quality Control Rate Rattus Reaction Research Rights Room, Clean Route Safety Sales Schools Scientist Senior Scientist Site Sleep Small Business Funding Mechanisms Small Business Innovation Research Grant Societies Solutions Spinal cord injury Stream Students Talents Taxes Technology Testing Therapeutic Thinking Time Toxic effect Training Translational Research Treatment Protocols United States United States Food and Drug Administration United States National Institutes of Health Universities Urinary Incontinence Urology Validation Variant Viscosity Woman Work base commercialization controlled release cooking cost design dosage experience gene therapy improved intravesical irritation manufacturing process medical schools member micturition urgency nociceptive response non-viral gene therapy novel painful bladder syndrome pre-clinical programs reconstitution research and development research clinical testing research facility research study residence response scale up size stem cell technology success uptake

项目摘要

DESCRIPTION (provided by applicant): Interstitial cystitis (IC) is a chronic, severely debilitating disease of the urinary bladder. It can be described as a chronic inflammatory condition of the bladder wall, characterized by urinary frequency and urgency, and severe suprapubic and/or pelvic pain. Presently, there is limited medical therapy for IC and new and effective treatment is a major unmet medical need. Lipella Pharmaceuticals Inc. intends to be the leading company specializing in the use of intravesical liposome technology to treat IC. We have developed a method of formulating highly specific liposomes for intravesical instillation. The technology is licensed from inventions and research by Drs. Leaf Huang and Michael Chancellor at the University of Pittsburgh. Based on their research that a liposome itself can provides a soothing effect to an irritated bladder (RO1 DK068556; Fraser et al. 2003), Lipella Pharmaceuticals expects that intravesical liposome formulations can present an improved activity in patients with IC. Prior to submitting this phase-II proposal, phase-I funding was used primarily to explore preclinical safety considerations of LP-08, a novel intravesical liposome therapy for IC, in a rat model. This phase-II proposal includes further safety studies, specifically measuring consequences of expected repeat dosing, as well as developing manufacturing scale-up procedures necessary for subsequent human testing. The effort proposed here would, in part, contribute to pre-clinical data collection prior to filing an FDA Investigational New Drug (IND) application, a regulatory requirement prior to administering LPA-08 in humans. PUBLIC HEALTH RELEVANCE We propose key preclinical experiments in an animal model to validate follow-on investment for clinical development of a novel treatment for painful bladder syndrome, an incurable chronic illness involving pain, urinary urgency, loss of sleep, depression and anxiety in approximately 500,000 women domestically.

描述（由申请人提供）：间质性膀胱炎（IC）是一种慢性、严重使人衰弱的膀胱疾病。它可以被描述为膀胱壁的慢性炎症，其特征是尿频和尿急，以及严重的耻骨上和/或骨盆疼痛。目前，IC 的药物治疗有限，新的有效治疗方法是未满足的主要医疗需求。 Lipella Pharmaceuticals Inc. 旨在成为专门利用膀胱内脂质体技术治疗 IC 的领先公司。我们开发了一种配制用于膀胱内滴注的高度特异性脂质体的方法。该技术已获得 Drs 的发明和研究许可。 Leaf Huang 和匹兹堡大学迈克尔·校长。基于他们的研究，脂质体本身可以为受刺激的膀胱提供舒缓作用（RO1 DK068556；Fraser et al. 2003），Lipella Pharmaceuticals 预计膀胱内脂质体制剂可以改善 IC 患者的活性。在提交此 II 期提案之前，I 期资金主要用于探索 LP-08（一种新型膀胱内脂质体治疗 IC）在大鼠模型中的临床前安全性考虑。该二期提案包括进一步的安全性研究，特别是测量预期重复给药的后果，以及开发后续人体测试所需的生产放大程序。这里提出的努力将在一定程度上有助于在提交 FDA 新药研究 (IND) 申请之前收集临床前数据，这是在人体中施用 LPA-08 之前的监管要求。公共卫生相关性我们建议在动物模型中进行关键的临床前实验，以验证膀胱疼痛综合征新疗法临床开发的后续投资，膀胱疼痛综合征是一种无法治愈的慢性疾病，涉及国内约 500,000 名女性的疼痛、尿急、睡眠不足、抑郁和焦虑。