NANOFIBER SOLUTIONS LLC — Department of Health and Human Services STTR Phase II: 300

Description

PROJECT ABSTRACT Short bowel syndromeSBSis a major clinical problem affecting newbornschildren and adultsfor which there is currently no known cureThe disorder results from the functional or anatomic loss of extensive segments of small intestine rendering the intestinal absorptive capacity severely compromisedSBS is clinically defined by malabsorptiondiarrheasteatorrheafluid and electrolyte disturbancesas well as malnutritionSBS has an overallyear survival ofand in newborn infants with less thanof expected intestinal lengthyear survival is onlyCurrent treatment options are inadequate and associated with severe complications and deathPatients with SBS require total parenteral nutritionTPNto surviveCurrentlyoverSBS patients per year are on home TPN in the US aloneAnnual costs of home TPN exceed $per year per patient$billion yearIn addition to the expense of TPNits use is associated with numerous central venous catheterassociated infectious and thrombotic complicationsresulting in additional high costsThusSBS represents an extremely costlyand deadlyburden to societyAt presentthe treatment for SBS is mainly supportiveAlthough small bowel transplantation is an optionthe results are suboptimalwithyear andyear survival rates ofandrespectivelyIn additionsmall bowel transplantation requires life long immunosuppression that causes numerous substantial secondary complicationsNovel approaches for the treatment of patients with SBS are critically neededOur approach to this unsolved medical problem is the production of tissue engineered intestineTEIusing the patient s own intestinal cells seeded onto a biodegradable scaffoldresulting in a novel solution to this unmet clinical needOur Phase I funding allowed us to develop successful standard operating procedures for the production of TEI in rodentsIn this Phase II project we will we will expand upon our Phase I findings in a large animal model and will accomplish the following specific aims necessary to move towards commercializationAimTo successfully produce tissue engineered intestine in pigs using freshly isolated ISCcontaining organoidsAimTo optimize production of tissue engineered intestine in pigs using in vitro cultured and expanded enteroidsAimTo successfully anastomose porcine tissue engineered intestine in line with host intestineThis Phase II STTR project will allow us to continue the path to developing a fully functional human tissue engineered intestineWe have assembled a world class team that includes a leading pediatric surgeona leading pediatric institute where we can do our first in man studyand a pioneering small business with expertise in scaffold design that will allow us to build the strategic partnerships with large medical device companies needed to successfully commercialize this urgently neededlife saving technology