An Improved Method of Delivering a Sclerosing Agent for the Treatment of a Malignant Pleural Effusion.

项目摘要/摘要。 我们正在开发一种改进的临床方法来治疗恶性胸腔积液(MPE)，一种 美国每年有150，00-175,000名患者发生严重疾病，影响到7%至15%的 所有癌症患者。MPE导致胸腔内积液过多，经常离开胸腔 患者在剩下的几个月里会出现呼吸急促和总体生活质量不佳的情况。 MPE的治疗是姑息性的，目前胸膜融合术等技术的主要问题是 由于硬化剂的低效和分布不均匀而导致的频繁临床失败率。 目前，医学界对最佳分娩方式还没有标准化和共识 硬化剂技术，因为患者满意度、有效性、成本 和复杂性。这导致进展甚微，目前许多临床黄金标准技术 已经有80多年没有进化过了。我们的重点是开发一种改进的临床方法来 使用硬化剂，这将克服目前使用的方法存在的问题。 在第一阶段可行性研究中，我们开发和评估了几种泡沫塑料配方，并选择了一种 领先候选人。我们对铅进行了充分的表征，展示了其所需的粘附性、凝胶性和 逆转热敏特性及其在动物模型中的功效。这些结果使我们能够 在完成我们专有交付系统设计的同时，着手优化销售线索 这一拟议的第二期项目。这项研究的目标将是生成一个功能齐全的原型， 完整的制造规范和用户指南，并评估安全和有效性 在GLP条件下进行的生物相容性研究期间的铅配方，以准备数据 用于向食品和药物管理局提交IND申请的包。一旦成功完成，这些 第二阶段的结果将为我们启动首个人类临床试验做准备，我们将在这些试验中测试 我们与几个享有盛誉的临床合作伙伴合作的联合药物设备的原型，这些合作伙伴是 这是这个第二阶段团队的重要组成部分。我们的多学科团队由一位经验丰富的心胸外科医生领导 外科医生，拥有从化学和机械方面的创新所需的专业知识 工程，驾驭FDA对组合产品的监管途径，成功商业化 医药和医疗器械产品，成功退出公司。通过以下途径寻求的资金 SBIR第二阶段计划将对我们实现重要的研发里程碑至关重要，我们将 进一步利用杠杆从投资者那里吸引外部资金来源，许多投资者已经表示 利息。我们的新方法预计将对当前的临床护理标准和结果产生重大影响 通过一种简单、微创的程序显著改善临床患者的预后。

Project Summary / Abstract. We are developing an improved clinical method to treat patients with malignant pleural effusions (MPE), a serious condition that occurs in 150,00-175,000 patients in the US each year, affecting between 7 to 15% of all cancer patients. MPE results in an excessive accumulation of liquid in the pleural cavity, frequently leaving the patient to suffer from shortness of breath and overall poor quality of life in their remaining months. Management of MPE is palliative and the main problem with the current techniques such as pleurodesis is its frequent clinical failure rate due to the inefficient and non-uniform distribution of the sclerosing agent. Currently, there is no standardization and consensus within the medical community on the optimal delivery technique for a sclerosing agent because of the tradeoffs and variability in patient satisfaction, efficacy, costs, and complexities. This has led to little progress and many of the current clinical gold standard techniques have not evolved in over 80 years. Our focus is on the development of an improved clinical approach to administering a sclerosing agent, which will overcome the problems with those currently practiced methods. In Phase I feasibility studies, we developed and evaluated several foam formulations and have selected a lead candidate. We fully characterized the lead, demonstrating its desired adhesiveness, gelation and reverse thermo-sensitive properties as well as its efficacy in an animal model. These results enable us to proceed with optimization of the lead in parallel with finalizing the design of our proprietary delivery system in this proposed Phase II project. The objectives of the study will be to generate a fully functional prototype with complete manufacturing specifications and user guides, and to also evaluate the safety and efficacy of the lead formulation during biocompatibility studies conducted under GLP conditions, in order to prepare a data package for an IND application to the Food and Drug Administration. Once successfully completed, these Phase II results will prepare us to initiate first-in-human clinical trials where we will test the functional prototype of our combination drug device in collaboration with several prestigious clinical partners who are an important part of this Phase II team. Our multidisciplinary team lead by an experienced cardiothoracic surgeon, has the demonstrated requisite expertise ranging from innovating in chemical and mechanical engineering, navigating the FDA regulatory pathway for combination products, successfully commercializing pharmaceutical and medical device products, to successfully exiting companies. The funding sought through the SBIR Phase II program will be critical to enable us to achieve important R&D milestones that we will further leverage for attracting outside sources of capital from investors, many who are already expressing interest. Our novel approach is expected to significantly impact the current clinical standard of care and result in markedly improved clinical patient outcomes with a simple, minimally invasive procedure.