AstudyattheUniversityofSouthFloridahasshownthattransplantsofmononuclearhumanumbilicalcordblood（MNChUCB）cellsmayhelppatientssufferingfromAmyotrophicLateralSclerosis（ALS）,alsoknownasLouGehrig'sdisease.Adiseaseinwhichthemotorneuronsinthespinalcordandbraindegenerate,ALSleavesitsvictimswithprogressivemuscleweakness,paralysisand,finally,respiratoryfailurethreetofiveyearsafterdiagnosis.
Inthisstudy,USFresearcherstransplantedhumanumbilicalcordblood（HUCB）cellsintomousemodelswithALS.Cellsweretransplantedatthreedifferentdosestrengthlevels--low,moderateandhigh--todeterminethedegreetowhichdoselevelsoftransplantedcellsmightdelaydiseasesymptomprogressionandincreaselifespan.InresultsrecentlypublishedonlineatPloSOne（PublicLibraryofScience）,researchersdeterminedthatthemoderate-strengthdoseofHUCBcellswasmosteffectiveinincreasinglifespanandreducingdiseaseprogression.
"OurresultsdemonstratethattreatmentforALSwithanappropriatedoseofMNChUBCcellsmayprovideaneuroprotectiveeffectformotorneuronsthroughactiveinvolvementofthesecellsinmodulatingthehostimmuneinflammatorysystemresponse,"saidthestudy'sleadauthorSvitlanaGarbuzova-Davis,PhD,DSc,oftheCenterofExcellenceforAgingandBrainRepairatUSF.
Accordingtotheresearchteam,modulatingimmuneandinflammatoryeffectorswithHUCBcellscouldhaveaprotectiveeffectondyingmotorneurons.TheteamhadpreviouslyshownthathUBCcelltransplantsreducedinflammationandprovidedneuroprotectioninmodelsofstrokeandAlzheimer'sdisease.
"ThispreclinicalstudyindicatesthatMNChUBCcellsmayprotectmotorneuronsbyinhibitinganimmuneinflammatoryresponsebydecreasingpro-inflammatorycytokines,signalingproteinsinthebrainandspinalcordthatplayaroleinimmuneresponse,"Garbuzova-Davisandcolleagueswrote."Proinflammatorycytokinesmaybeindirectmediatorsforglialcells'contributiontomotoneurondeathandthedecreaseinthesecytokinesmightbeduetoareductionofactivatedmicroglia,thecellsthatformactiveimmunedefenseinthecentralnervoussystem."
Theresearchteamnoted,however,thatthemechanismunderlyingthebeneficialeffectofhUBCcellsforrepairingdiseasedmotorneuronsinALSstillneedsmoreclarification.
Suggestingthat'moreisnotbetter,'itwasthemoderate,notthehigh,doseofhUBCcellsthatprovedmosteffective.Researchersspeculatedthatthehighdosemayhavebeenlesseffectivebecauseitinducedanimmunologicalconflictwithinthemousemodel.
"Futurestudiesshouldlookatmultipleinjectionsofsmallerdosesovertime,inordertohelptranslatethisresearchtoclinicaltrials,"accordingtoco-authorPaulR.Sanberg,PhD,DSc,directoroftheCenter.
"DevelopinganeffectivetreatmentforALSiscomplicatedbythediffusenatureofmotorneurondeath,"concludedGarbuzova-Davis."However,celltherapymayofferapromisingnewtreatment."
Theotherco-authorsofthestudywereCyndyDavisSanbergandNicoleKuzmin-NicholsofSaneronCCELLTherapeutics,Inc.,andAlisonE.Willing,CarmelinaGemma,PaulaC.Bickford,ChristinaMiller,andRobertRossifromUSF.
