Intravesical Liposome Treatment for Interstitial Cystitis

描述（由申请人提供）：项目总结/摘要：间质性膀胱炎（IC）是一种慢性、严重的膀胱衰弱性疾病。它可以被描述为膀胱壁的慢性炎性病症，其特征在于尿频和尿急，以及严重的耻骨上和/或骨盆疼痛。目前，IC的药物治疗有限，新的有效治疗是一个主要的未满足的医疗需求。Lipella制药公司打算成为专门使用膀胱内脂质体技术治疗IC的领先公司。我们已经开发了一种配制用于膀胱内安装的高度特异性脂质体的方法。这项技术是由匹兹堡大学的Leaf Huang博士和Michael Chancellor博士发明和研究的。根据他们的研究，脂质体本身可以提供一个舒缓的作用，刺激膀胱，Lipella制药公司预计，膀胱内脂质体制剂可以提出一个改善的活动，在患者与IC。Lipella Pharmaceuticals目前具有许多脂质体组合物，其在大鼠模型中显示出治疗诱导的膀胱刺激的功效。最有前途的化合物LPA-08是这项资助提案的主题。LPA-08的特定组成和在大鼠中的初始功效结果表明，LPA-08显示出作为膀胱刺激的安全有效疗法的前景。本文提出的努力将部分有助于在提交FDA研究性新药（IND）申请之前收集临床前数据，这是在人体施用LPA-08之前的监管要求。这些努力包括探索LPA-08的药理学，以及其安装后的分布。项目叙述：我们提出在动物模型中进行关键的临床前实验，以验证对膀胱疼痛综合征的新型治疗的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，涉及疼痛，尿急，睡眠不足，抑郁和焦虑，国内约有50万女性。

DESCRIPTION (provided by applicant): Project Summary/Abstract: 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 installation. 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 provide a soothing effect to an irritated bladder, Lipella Pharmaceuticals expects that intravesical liposome formulations can present an improved activity in patients with IC. Lipella Pharmaceuticals currently has a number of liposome compositions that demonstrate efficacy in treating induced bladder irritation in a rat model. The most promising compound, LPA-08, is the subject of this grant proposal. The specific composition of LPA-08, and initial efficacy results in rats, indicate that LPA-08 shows promise as a safe and effective therapy for bladder irritation. 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 man. Such efforts include exploring the pharmacology of LPA-08, as well as its distribution post-installation. Project Narrative: We propose key preclinical experiments in an animal model to validate follow-on inves ment for clinical development of a novel treatment for painful bladder syndrome, an in-curable chronic illness involving pain, urinary urgency, loss of sleep, depression and anxiety in approximately 500,000 women domestically.