KAER BIOTHERAPEUTICS — Department of Health and Human Services SBIR Phase I: NHLBI

Description

The causal factors in neonatal Respiratory Distress SyndromeRDSare insufficient production and presence of surfactant in the developing lungs of babies born prematurelyThis deficit results in markedly impaired respiratory function that not only causes a high mortality but leads to respiratory complications throughout childhood and into adult lifeOur goal is to change the paradigm of surfactant therapy in babies born prematurely and in so doing improve their survival and reduce the associated chronic lung problemsOf themillion premature babies born around the worldone million will die annually within the first month of lifeand many of themillion survivors are faced with a lifetime of serious health complicationsRespiratory distress syndromeRDSis the single most important cause of mortality and morbidity in preterm infantsBoth surfactant instillation into the lungs and continuous positive airway pressureCPAPhave been shown to improve outcomes in premature babies with RDSHoweverthe complication ratecomorbidities and mortality rate due encountered due to the need for intubation to instill surfactant remain high in spontaneously breathing premature babies remains highTo mitigate these adverse sequelaethis proposal focuses on the development of non invasive aerosol surfactant replacement therapyNamelythe delivery ofm MMAD pure surfactant aerosol that deposits in the peripheral regions of the neonatal lungsThe major issue inhibiting the realization of a successful therapy has been the inability to reliably deliver a therapeutically relevant mass of surfactant to the peripheral lung in an acceptably short treatment timeCompared to an aqueous aerosol generated from amg ml surfactant suspensiona similar sized pure solidphase fine particle surfactant aerosol deliverstimes the doseWhen pure surfactant aerosols are produced from higher concentrations of surfactant suspensions together with higher atomization rates and delivered as pure solid phase aerosolseven higher delivery rates are achievableIn additionwhen the pure solid phase aerosol is less thanm MMAD losses related to the delivery of the aerosol are minimized together with greater peripheral deposition improved efficacy can be obtained within a shorter treatment timeA game changing system for aerosol surfactant replacement therapy that overcomes present limitations of mesh nebulizers and jet atomizers is proposedSUPRAER MiniAn aerosol is generated from an aqueous surfactant suspensionthe water is rapidly evaporated from the suspended droplets and the resulting surfactant aerosol concentrated using a virtual impactorThis proposal is targeted at demonstrating that using such an aerosol generation and processing system and an integrated with an aerosol friendly interfacehigh concentrations ofm MMAD surfactant aerosols with narrow size distributions can be delivered atl min tol minThe aerosols will be delivered at under conditions of CPAP at an absolute humidity ofmg lThe performance of the aerosol processing systemthe device interfaceand the effectiveness of this aerosol generation and processing system will be evaluated on the benchThe physiological responses of surfactant aerosol delivery will be demonstrated in a spontaneously breathing lavage induced rabbit model of respiratory distress Of themillion premature babies born around the worldone million will die annually within the first month of lifeand many of themillion survivors have respiratory complications throughout childhood and into adult lifeRespiratory distress syndromeRDScaused by respiratory surfactant deficiencyis the single most important cause of mortality and morbidity in preterm infantsOur goal is to develop SUPRAER Minia noninvasive aerosol delivery system that will rapidly deliver of fine particles of surfactant aerosols to the peripheral regions of the neonatal lungsThis game changing therapeutic treatment is anticipated to result in a substantial improvement in the survival of premature babies and reduce long term respiratory disabilities resulting from their inherent surfactant deficiency