Intravesical Liposome Treatment for Interstitial Cystitis

描述(申请人提供)：间质性膀胱炎(IC)是一种慢性的，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滴注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。基于他们的研究，即脂质体本身可以为刺激的膀胱提供缓解作用(RO1DK068556；Fraser等人。2003年)，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。在提交这个第二阶段的提案之前，第一阶段的资金主要用于探索LP-08的临床前安全性考虑，LP-08是一种治疗IC的新型膀胱内脂质体疗法，在大鼠模型中。这一第二阶段的建议包括进一步的安全性研究，特别是测量预期重复剂量的后果，以及开发后续人体测试所需的制造扩大程序。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，这是在人类使用LPA-08之前的一项法规要求。 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试验，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不可治愈的慢性疾病，涉及疼痛、尿急、失眠、抑郁和焦虑，国内约有500,000名女性。

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.