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

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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.