HNB Therapeutics LLC — Department of Health and Human Services STTR Phase I: NIA

Description

Summary Glucocorticoid induced osteoporosis is the most common cause of secondary osteoporosisand the primary cause before ageIt affects overof the general populationwhereinare womenIn postmenopausal women and men agedand overtreatment would be recommended in at least half based on theAmerican College of Rheumatology guidelinesAdult stem cells are a promising class of regenerative cellssome lineages of which can be induced to differentiate into osteoblasts and potentially cure osteoporosisWe have shown thatfold nanofiber expanded hematopoieticCDstem cells can reverse glucocorticoid induced osteoporosis in an aged murine modelNow we have discovered a way to expand CDstem cells over a million fold while preserving their stemnesswhich promises to make this therapy cost effectiveThese cells can be obtained noninvasively from umbilical cord bloodUCBUCB is readily available and is considered safe for human application for treating a variety of diseases such as leukemia and other blood disordersIf these million fold expanded CDstem cells can be shown to be safe and efficacious in reverting glucocorticoid induced osteoporosisthis cell based therapy may extend to other types of osteoporosisand would represent a major paradigm shiftPatients with osteoporosis must undergo lifetime therapy with traditional therapeuticswhereas stem cells promise to be atotime treatmentFurthermorepatients will not have extended hospitalization nor surgery costsas stem cell therapy in many cases is an outpatient IV infusionMillion fold expansion provides the opportunity to reduce costs furtheras one cord blood unit will be able to treat overpatientsThe aims of this Phase I project are therefore toadetermine the potential of million fold expanded CDstem cells to differentiate into osteoblastsandbtest the efficacy of million fold expanded CDstem cells for reverting glucocorticoid induced osteoporosis in aged miceIf successfulthis research will be extended to more severe forms of osteoporosisand potentially provide a pathway towards a cost effective clinical regenerative treatment for this debilitating diseaseIt may also have extensions for the lower cost treatment of leukemias and a variety of other blood disorderswhich will be the subject of future investigations Public Health Relevance Glucocorticoid induced osteoporosis is the most common cause of secondary osteoporosisand the primary cause before ageIt affects overof the general populationwhereinare womenThis research focuses on investigating a promising cell based therapy using million fold expanded CDstem cells derived from human umbilical cord blood for reverting glucocorticoid induced osteoporosis in aged miceIf successfulthis research will provide a pathway towards a cost effective clinical regenerative treatment for this debilitating diseasewith potential extension to other forms of osteoporosis as well as a variety of blood related disorders