Intravesical Liposome Treatment for Interstitial Cystitis

描述(申请人提供)：项目摘要/摘要：间质性膀胱炎(IC)是一种慢性、严重衰弱的膀胱疾病。它可以被描述为膀胱壁的慢性炎症状态，特征是尿频和尿急，以及严重的耻骨上和/或盆腔疼痛。目前，IC的医学治疗有限，新的有效治疗是一个主要的未得到满足的医学需求。Lipella制药公司打算成为专业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用于膀胱内安装的高度特异性脂质体的制备方法。这项技术的授权来自匹兹堡大学的黄叶博士和迈克尔·查赫尔博士的发明和研究。根据他们的研究，脂质体本身可以为受刺激的膀胱提供缓解作用，Lipella制药公司预计，膀胱内脂质体制剂可以改善IC患者的活性。Lipella制药公司目前有许多脂质体组合物，这些组合物在治疗大鼠模型中诱导的膀胱刺激方面具有疗效。最有希望的化合物LPA-08是这项赠款提案的主题。LPA-08的特殊成分和对大鼠的初步疗效表明，LPA-08有望成为治疗膀胱刺激的安全有效的药物。这里提出的努力将在一定程度上有助于在提交FDA研究新药(IND)申请之前收集临床前数据，这是在MAN中使用LPA-08之前的监管要求。这些努力包括探索LPA-08的药理学，以及它在安装后的分布。项目简介：我们建议在动物模型中进行关键的临床前实验，以验证对痛性膀胱综合征临床开发的后续投资。痛性膀胱综合征是一种涉及疼痛、尿急、失眠、抑郁和焦虑的不可治愈的慢性疾病，在国内约500,000名女性中进行。

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.