Preventing and treating RSV pneumonia in immunocompromised cancer patients

DESCRIPTION provided by applicant Pneumonia resulting from respiratory syncytial virus infections RSV is well known as a cause of morbidity and mortality in patients undergoing immune suppressive therapies such as hematopoietic stem cell transplant recipients and leukemia patients At some sites these patients may be treated with an extensive course of ribavirin aerosol or oral forms to alleviate the risk of pneumonia The effectiveness of ribavirin s not well established and the treatment is expensive and burdensome to patients and clinical staff alike While adult treatment is not currently an approved indication immunocompromised patients with RSV have no other treatment options Pulmotectandapos s Solution Pulmotect has identified and is developing a novel technology to prevent respiratory infections The lead drug PUL is a combination of two TLR ligands that stimulates the lungandapos s own innate defense mechanisms to create a hostile environment for pathogens and prevent or attenuate respiratory infections Both in vitro and in vivo experiments have been completed to validate this technology and the drug is progressing through the regulatory process for a treatment to benefit cancer patients during periods of immunocompromise The focus of this project is to accomplish key milestones that will further transition this technology for commercialization against RSV viral infections as an addition to ribavirin treatment The project is organized into three measurable Specific Aims This SBIR application is focused on addressing important preclinical issues The three Aims included are designed to validate and expand upon the proof of concept data already obtained to establish the safety of the drug in combination with ribavirin in IND enabling toxicology studies and to submit the results of the described studies to the FDA as part of an IND in order to initiate clinical trials The aims are focused for transitioning this technology ito the clinic Achieving these milestones would provide significant data for an IND application for a product that is safe and efficacious Positive results from this phase II proposal would lead to a clinical trial to test the proposed treatment PUL in immunocompromised cancer patients with respiratory syncytial virus infections Additionally by demonstrating subsequent discoveries in Pulmotectandapos s pipeline additional outside funds from investors can better be pursued to transition this technology into the clinic and market place Overall the outlined milestones of ths proposal would build upon previous work add to the foundation of this platform technology and help bring this technology to the clinic PUBLIC HEALTH RELEVANCE Pulmotect Inc is developing novel therapeutics that stimulate the innate immune system to protect against infectious diseases even in cases of severely compromised immunity Proof of concept data has shown that this technology effectively protects against a broad range of inhaled pathogens including viral pathogens that often cause significant morbidity and mortality in immunocompromised patients with cancer This project aims to provide essential data to help transition this technology from the lab to the clinic to better assist patients at serious risk of viral pneumonia