A novel device and rescue solution for the improved post-thaw viability and function of human stem cells used in cell therapy.

There are overhuman stem cell trials currently underway worldwideMost of these trials use adipose derived mesenchymal stem cellsadMSCswhile others use differentiated human embryonic stem cellshESCsWhile human induced pluripotent stem cellsiPSCsare currently in limited trials in select countriesthe future promise of these cells for treating disease worldwide is indisputable and their increasing use in the drug discovery toxicity sciences is assuredThus it is imperative to improve the bioprocessing and delivery of stem cells for patient use through developing new stem cell dedicated instrumentsunique biomaterialsand improved methods of cell cryopreservationNearly half of stem cells are cryopreserved and thawed prior to infusion into the patientYet little attention has been paid to the post thaw recoveryfunction and homing of these cells in patients that can be compromised as a consequence of preservationCPSI Biotech personnel have worked in the stem cell therapy cryopreservation arena for nearlyyears and have reported that delayed onset cell death occurs up to several days post thaw in all cell types including stem cellsCurrentlymany stem cell therapy companies thaw their cryopreserved cells immediately prior to infusion using a cryopreservation solution that is a component of their FDA issued INDThe most common method of thawing these cells is a standardnon programmablewater bath and little to no emphasis is placed on delayed onset cell death that most assuredly occurs in the patient as a consequence of the thawing process that is exacerbated by the physical stress to the cells due to the infusion processCPSI Biotech will address this issue by developing two new devicesStemThaw and StemTransand one unique recovery reagentStemReviveIn Phase I CPSI s prototype dry thaw systemSmartThawwill be tested as a dedicated stem cell thawing deviceStemThawthat uses stem cell specific thaw profiles that yield improved post thaw outcome compared to standard methodsIn additiona post thaw dilution reagentStemRevivewill be developed that will reduce the delayed onset cell death known to occurtohours postthawThe combination of these two complementary technologies should improve the viabilityfunction and engraftment of stem cells while at the same time limiting the chance of posttherapy rejectionPhasewill be dedicated to developing a unique deviceStemTransthat will be designed for the improved shipping and storage of cryopreserved and hypothermically stored stem cellsThe PhaseSpecific Aims are the followingDefine and develop unique cell type matched thawing programs for StemThaw that improve the post thaw outcome of adMSCshESCs and iPSCsDefine the optimal formulation of StemRevive as an additive reagent to thawed adMSCshESCs and iPSCs to prevent delayed onset cell death resulting in optimal viabilityCombine StemThaw and StemRevive together to determine efficacyPhasespecific aims will includeDevelop and test unique thaw programs for StemThaw defined by PhaseDevelop StemTrans for the improved distribution of cryopreserved stem cellsDetermine if the optimal StemThaw and StemRevive combination defined in Phaseresults in improved homing and engraftment in a mouse SCID modelTest the combination of StemTransStemThaw and StemRevive as a candidate platform for the improved shippingstoringthawing and recovery system that will yield improved viabilityfunction and engraftment of stem cells for stem cell therapy thus limiting graft rejection and improving stem cell therapy efficacy Of thestem trails currently ongoing worldwidenearly half use cells that are cryopreserved in liquid nitrogen so that they can be shipped and stored prior to infusion into cell therapy patientsThis Phaseproject proposes to develop a unique instrumentStemThawand a companion solutionStemRevivethat when used either separately or together should yield improved viability and function of cryopreserved stem cells used for stem cell therapyThis will lead to improved health benefits and outcome to stem cell patients