Functional Neural Transplantation IV Translation to Clinical Application, Part A PDF
Preview Functional Neural Transplantation IV Translation to Clinical Application, Part A
Serial Editor Vincent Walsh InstituteofCognitiveNeuroscience UniversityCollegeLondon 17QueenSquare LondonWC1N3ARUK Editorial Board MarkBear, Cambridge, USA. Medicine& TranslationalNeuroscience Hamed Ekhtiari, Tehran, Iran. Addiction Hajime Hirase, Wako, Japan. NeuronalMicrocircuitry Freda Miller, Toronto,Canada. DevelopmentalNeurobiology ShaneO’Mara, Dublin, Ireland. Systems Neuroscience SusanRossell, Swinburne, Australia. Clinical Psychology&Neuropsychiatry Nathalie Rouach, Paris, France. Neuroglia Barbara Sahakian,Cambridge, UK. Cognition &Neuroethics Bettina Studer,Dusseldorf,Germany. Neurorehabilitation Xiao-Jing Wang, New York, USA. ComputationalNeuroscience AcademicPressisanimprintofElsevier 50HampshireStreet,5thFloor,Cambridge,MA02139,UnitedStates 525BStreet,Suite1800,SanDiego,CA92101–4495,UnitedStates TheBoulevard,LangfordLane,Kidlington,OxfordOX51GB,UnitedKingdom 125LondonWall,London,EC2Y5AS,UnitedKingdom Firstedition2017 Copyright#2017ElsevierB.V.Allrightsreserved. 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Inusingsuchinformationormethodstheyshouldbemindfuloftheirownsafetyandthesafety ofothers,includingpartiesforwhomtheyhaveaprofessionalresponsibility. Tothefullestextentofthelaw,neitherthePublishernortheauthors,contributors,oreditors, assumeanyliabilityforanyinjuryand/ordamagetopersonsorpropertyasamatterofproducts liability,negligenceorotherwise,orfromanyuseoroperationofanymethods,products, instructions,orideascontainedinthematerialherein. ISBN:978-0-12-811738-5 ISSN:0079-6123 ForinformationonallAcademicPresspublications visitourwebsiteathttps://www.elsevier.com/books-and-journals Publisher:ZoeKruze AcquisitionEditor:KirstenShankland EditorialProjectManager:AnaClaudiaGarcia ProductionProjectManager:MageshKumarMahalingam CoverDesigner:MarkRogers TypesetbySPiGlobal,India Contributors Nozie D.Aghaizu UCL Instituteof Ophthalmology, London,United Kingdom Robin R.Ali UCL Instituteof Ophthalmology, London,United Kingdom ArturoAlvarez-Buylla The Eli and EdytheBroad Center ofRegeneration MedicineandStem Cell Research, University ofCalifornia, San Francisco, San Francisco, CA, United States (cid:1) Anne-Catherine Bachoud-Levi Assistance Publique-H^opitauxde Paris, Centrede R(cid:1)ef(cid:1)erence Maladie de Huntington, Servicede Neurologie, H^opital HenriMondor-Albert Chenevier; INSERMU955,Equipe 01 Neuropsychologie Interventionnelle; Universit(cid:1)e Paris Est, Facult(cid:1)e de M(cid:1)edecine,Cr(cid:1)eteil; D(cid:1)epartement d’Etudes Cognitives, Ecole NormaleSup(cid:1)erieure,PSL* Research University, Paris, France Roger A.Barker Wallenberg Neuroscience Center, Lund University, Lund,Sweden;Wellcome Trust-MRC Cambridge Stem Cell Institute and John van Geest Centrefor Brain Repair,University ofCambridge, Cambridge, United Kingdom Allan I. Basbaum University ofCalifornia—San Francisco, San Francisco, CA, United States € Anders Bjorklund Wallenberg Neuroscience Center, Lund University, Lund,Sweden Joao M.Braz University ofCalifornia—San Francisco, San Francisco, CA, United States Vania Broccoli San Raffaele Scientific Institute;CNR-Institute ofNeuroscience, Milan, Italy Mary B.Bunge TheMiamiProjecttoCureParalysis;DepartmentofCellBiology;Departmentof NeurologicalSurgery,UniversityofMiamiLeonardM.MillerSchoolofMedicine, Miami,FL,UnitedStates Monica Busse Centre for Trials Research, College ofBiomedical &Life Sciences, Cardiff University, Cardiff,United Kingdom MelissaK. Carpenter CarpenterGroup Consulting,Seattle,WA, United States Susanne Clinch Centrefor Trials Research,College ofBiomedical &LifeSciences; BrainRepair Group,School ofBiosciences, Cardiff University, Cardiff,United Kingdom v vi Contributors Peter Coffey Institute ofOphthalmology; NIHR BiomedicalResearchCentre,Moorfields Eye Hospital NHS FoundationTrust,London, United Kingdom;Centerfor Stem Cell Biology and Engineering, Neuroscience Research Institute, University of California, Santa Barbara,Santa Barbara,CA, United States Lyndon da Cruz Institute ofOphthalmology; NIHR BiomedicalResearchCentre;Moorfields Eye Hospital NHS Foundation Trust,London,United Kingdom € € MateD. Dobrossy University Freiburg—MedicalCentre,Freiburg, Germany Elsa Diguet CEA,DSV,MolecularImagingResearchCenter(MIRCen),Fontenay-aux-Roses; InstitutdeRecherchesServier,NeuropsychiatryUnit,CroissysurSeine,France Stephen B.Dunnett BrainRepair Group, SchoolofBiosciences, CardiffUniversity, Cardiff, UnitedKingdom Alex Etlin University ofCalifornia—San Francisco, San Francisco, CA, United States RobinJ.M. Franklin Wellcome Trust-Medical Research Council Cambridge Stem Cell Institute; University ofCambridge, Cambridge, United Kingdom KateFynes Institute ofOphthalmology, London,United Kingdom Steven A.Goldman Center for Neuroscience, University ofCopenhagen Faculty ofHealth and MedicalSciences, Copenhagen,Denmark;Center for Translational Neuromedicine,University ofRochester Medical Center,Rochester, NY, UnitedStates AnaiGonzalez-Cordero UCL Institute ofOphthalmology, London,UnitedKingdom Genevieve Gowing BoardofGovernorsRegenerativeMedicineInstitute, Cedars-SinaiMedical Center;Cedars-Sinai MedicalCenter,Los Angeles,CA,UnitedStates € Magdalena Gotz Physiological Genomics, BiomedicalCenter;SYNERGY, Excellence Cluster of SystemsNeurology,BiomedicalCenter,Ludwig-MaximiliansUniversityMunich, Planegg;Instituteof Stem Cell Research,Helmholtz Center Munich, Munich, Germany Contributors vii Philippe Hantraye CEA,DSV,MolecularImagingResearchCenter(MIRCen);CNRS,CEA,Paris-Sud University, University of Paris-Saclay, Neurodegenerative DiseasesLaboratory (UMR9199), Fontenay-aux-Roses, France Dina Juarez-Salinas University ofCalifornia—San Francisco, San Francisco, CA, United States Aisha Khan The Interdisciplinary Stem CellInstitute,University ofMiami LeonardM. Miller SchoolofMedicine, Miami, FL,United States Agnete Kirkeby WallenbergNeuroscienceCenter;LundStemCellCenter,LundUniversity,Lund, Sweden Zaal Kokaia Laboratory ofStem CellsandRestorative Neurology, Lund Stem CellCenter, Lund,Sweden Kamil Kruczek UCL Instituteof Ophthalmology, London,United Kingdom Tilo Kunath MRCCentreforRegenerativeMedicine,InstituteforStemCellResearch,School ofBiologicalSciences,TheUniversityofEdinburgh,Edinburgh,UnitedKingdom Meng Li CardiffUniversityNeuroscienceandMentalHealthResearchInstitute,Schoolof Medicine; Cardiff University SchoolofBiosciences, Cardiff,United Kingdom Olle Lindvall Laboratory ofStem CellsandRestorative Neurology, Lund Stem CellCenter, Lund,Sweden IdaJ. Llewellyn-Smith Cardiovascular Medicine,Human Physiology and Centre for Neuroscience, Flinders University, Bedford Park, SA, Australia Paul Lu VeteransAdministration San Diego HealthcareSystem; University ofCalifornia, San Diego, CA,United States Walter R. Mancia Leon The Eli and EdytheBroad Center ofRegeneration MedicineandStem Cell Research, University ofCalifornia, San Francisco, San Francisco, CA, United States Paula V.Monje The Miami Project to CureParalysis; Department of NeurologicalSurgery, University ofMiami Leonard M. Miller School of Medicine,Miami,FL, United States viii Contributors Natalia A.Murphy Wellcome Trust-Medical Research Council Cambridge Stem Cell Institute; University ofCambridge, Cambridge, United Kingdom Masaya Nakamura KeioUniversity SchoolofMedicine, Tokyo, Japan AmmarNatalwala MRCCentreforRegenerativeMedicine,InstituteforStemCellResearch,School ofBiologicalSciences,The University ofEdinburgh; TranslationalNeurosurgery Group,Western General Hospital, Crewe RoadSouth, Edinburgh, UnitedKingdom Britta Nommiste Institute ofOphthalmology, London,United Kingdom SatoshiNori KeioUniversity SchoolofMedicine, Tokyo, Japan HideyukiOkano KeioUniversity SchoolofMedicine, Tokyo, Japan MalinParmar WallenbergNeuroscienceCenter;LundStemCellCenter,LundUniversity,Lund, Sweden Rachael A.Pearson UCL Institute ofOphthalmology, London,UnitedKingdom ConorRamsden Institute ofOphthalmology; NIHR BiomedicalResearchCentre,Moorfields Eye Hospital NHS Foundation Trust,London,United Kingdom AnneE. Rosser CardiffUniversityNeuroscienceandMentalHealthResearchInstitute,Schoolof Medicine; CardiffUniversity School of Biosciences,Cardiff, UnitedKingdom JulienSpatazza The Eli and Edythe Broad Center ofRegeneration Medicine and Stem Cell Research, University of California,San Francisco, San Francisco, CA, UnitedStates Lorenz Studer The Center for Stem Cell Biology, Developmental Biology Program, Memorial Sloan Kettering Cancer Center, New York, NY, UnitedStates CliveN. Svendsen BoardofGovernorsRegenerativeMedicineInstitute, Cedars-SinaiMedical Center;Cedars-Sinai MedicalCenter,Los Angeles,CA,UnitedStates SoshanaSvendsen BoardofGovernorsRegenerativeMedicineInstitute, Cedars-SinaiMedical Center;Cedars-Sinai MedicalCenter,Los Angeles,CA,UnitedStates Contributors ix Jun Takahashi Center for iPSCell Research and Application, Kyoto University, Kyoto, Japan Daniel Tornero Laboratory ofStem CellsandRestorative Neurology, Lund Stem CellCenter, Lund,Sweden Olof Torper Physiological Genomics, Biomedical Center;SYNERGY,Excellence Clusterof SystemsNeurology,BiomedicalCenter,Ludwig-MaximiliansUniversityMunich, Planegg;Institute ofStem Cell Research,Helmholtz Center Munich,Munich, Germany VictoriaE. Tovell Institute ofOphthalmology, London, UnitedKingdom Nadja Van Camp CEA,DSV,MolecularImagingResearchCenter(MIRCen);CNRS,CEA,Paris-Sud University, University of Paris-Saclay, Neurodegenerative DiseasesLaboratory (UMR9199), Fontenay-aux-Roses, France PatrickM. Wood The Miami Project to CureParalysis; Department of NeurologicalSurgery, University ofMiami Leonard M. Miller School of Medicine,Miami,FL, United States Preface Thepurposeofthisbookistoprovideaseriesofcriticalreviewsonrecentprogressin thefieldofneuraltransplantationwithafocusonfunctionalaspectsofcelltransplan- tationforbrainrepair.After35yearsofbasicresearchandinitialearlyattemptsatclin- icalapplicationthelast5–7yearshaswitnessedamajorstepchangeinboththerange ofclinical applicationsbeing considered and therefinementofalternative strategies andcellsourcesunderconsideration.Inparticular,recentadvancesinthesophistica- tionofstemcellbiologyanddifferentiationareprovidingrealisticprospectsofnew celltherapiesforneurodegenerativediseasesandinjuryreachingclinicalreality. This volume represents the fourth in a series of review volumes that we have editedat6-to10-yearintervalsfollowingtheemergenceofthefieldofneurotrans- plantationinthebrainoverfourdecades.Allbutthefirstvolume(FunctionalNeural Transplantation,RavenPress,1994)havebeenpublishedasvolumesinElsevier’s Progress in Brain Research series (Functional Neural Transplantation II, PBR vol. 127, 2000, and Functional Neural Transplantation III parts 1 and 2, PBR vols.200 and 201, 2011). The field of functional neural transplantation has developed dramatically over thatperiod,fromtheearlyexperimentalforaysestablishingviabletechniquesforcell survivalintheadultrodentbraininthe1970s,thefirstclearexamplesoffunctional transplantationinrodentmodelsofneurodegenerationinthe1980s,earlyattemptsat applying fetal cell transplantation in patients in the 1990s, to the present mature phase of sophisticated translational and clinical trial research. This includes an explosion of new opportunities arising from different, more flexible and powerful cell sources, notably pluripotent stem cells. Theauthorsareamongthecurrentworldleadersinthisrapidlydevelopingfield. Consequently,theresultingvolumes,likethepreviousthreevolumesintheseries,pro- videsauthoritativereviewsofthecurrentstateofplayinthisfastemergingandhigh profile field of cell replacement therapies. In particular, harnessing the power and potential of stem cells holds promise to transform the range of clinical conditions that are now within realistic prospect of “brain repair.” As we are passing the 20th anniversaryofJamieThomson’sfirsthumanembryonicstemcellline,andthe10th anniversaryofYamanakaandTakahashi’sdeterminationofspecificfactorsforrepro- grammingofadulthumancellstoapluripotentstemcellstate,therehasbeenremark- ableprogressintheunderstandingoftheprinciplesfordifferentiationofprecursorand postmitoticcellsofdiverseneuronalandglialphenotypestypes,whichareviablefor transplantation, replacing our previous exclusive dependence on fetal cells for cell replacementtherapy.Advancesindevelopmentalbiologyarenow,forthefirsttime, allowingeffectiveandefficientcontrolofcellfatespecificationfrompluripotentcell sources, accompanied by dramatic technical advances instandardization, reliability, andqualitycontrolnecessarytounderpinclinicallyacceptable21stcenturymedicinal products.Indeed,therearenowmajoreffortsworldwidetoaddressthecriticalsafety issuesthatneedtoberesolvedpriortoanyclinicalapplication. xxv xxvi Preface The chapters have been commissioned to provide authoritative scholarly representationofthecurrentfrontlineoftranslationalresearchacrossabroadrange ofapplications ofemerging celltransplantationtechnologiesinregenerativemedi- cine,appliedtothebrainandspinalcord,withafocusonneurodegenerativediseases andinjury.Althoughthebookwasplannedasasinglevolume,thedetailedaccounts providedbytheauthorshaverequiredpublicationintwoparts,PartAandPartB,as consecutivevolumesintheProgressinBrainResearchSeries.Nevertheless,wepro- vide aunified contents list and index inorder to retain the integrityof the whole. In the first chapter ofpart A,aseditors, wediscuss the principles offunctional repairandrecoveryandoutlineourpresentunderstandingofthevariousmechanisms bywhichacellulartransplantmayimpactonthefunctionalstateofthehost,beitan experimentalanimalorapatient.Understandingalternativemechanismsofrecovery isanessentialfirststageindefiningthetaskrequirementsforanynewcelltherapy andsetsthedemandsofwhatisrequiredtoachievefunctionalefficacyineachspe- cific disease indication. This is followed by a series of chapters that focus on the currentandemergingstatesofthenewtechnologiesforexvivoandinvivorepro- grammingofneuronstodifferentcellfates,characterizationofalternativedelivery vectorsandstemcelllines,andthedemandsofregulatorycompliancefortakingany new therapy to the clinic. Inthesubsequentchaptersthemajorclinicalindicationsforwhichcelltherapies arecurrentlyunderinvestigationarereviewed.ThisstartsinpartAwithtwowell- established basal ganglia disorders, Parkinson’s disease and Huntington’s disease, thathavebeenthefirsttoundergoclinicaltranslation.Here,theexperimentalstrat- egy isexplicitly reparative,i.e., toreplace lostneuronswith new cellsofthe same neuronalphenotypewiththecapacitytointegratefunctionallyintohostbrain,repair damagecircuits,andrestorelostfunction.Ineachcase,wereviewthestatusofon- goingclinical trials using existing cell sourcesas well asnew strategies for differ- entiating clinical grade cell therapy products from human stem cell sources. Cell therapytrialsinthesediseaseshavesetthebenchmarksforclinicaltrialdesign,in- cludingpatientassessmentstrategies,motorandcognitivetesting,andsophisticated imaging.Theyhavealsopavedthewayforthedevelopmentandimplementationof rehabilitationstrategies,notjusttopromotestrategiesforcompensationbutwiththe capacity toimpact graft survival, differentiation, and circuit repair. Next,inPartB,followsaseriesofchaptersaddressingtherecentandsignificant advancesinspinalcordinjuryandrepair.Thelong-distanceregenerationofprecisely targetedaxonpathwaysrepresentsafieldwheresustainedcarefulexperimentalanal- ysisovermanyyearsisnowgettingclosetoimplementationinclinicaltrials,build- ing, in particular, on the capacity of central and peripheral glial cells to support damagedaxons,promoteregeneration,andenhancecircuitrepair,aswellasneuro- protection and trophic support. New sources of neuroglia derived from pluripotent stem cells provide interesting alternatives to conventional oligodendrocyte and Schwann cells, but face the same biological, regulatory, and safety challenges as in applicationsinthe brain. Preface xxvii Amajorareaofadvanceforstemcell-basedtherapieshasemergedoverthelast decadeintheeye.Remarkableprogresshasbeenachievedinfunctionalcellreplace- mentinexperimentalanimalsusingretinalpigmentepithelium(RPE)andphotore- ceptorcells,andauthenticcellsofbothtypeshavebeeneffectively,generatedfrom pluripotentstemcells.Thereisarealisticprospectthatthefirstdemonstrablyeffec- tivestemcelltherapyinneurologicalmedicinewillemergefromongoingtrialsusing RPE cell replacement totreat blindnessduetoage-related maculardegeneration. Finally,toconcludePartB,weconsideranothermajorcauseofneurologicalill- nessworldwide,i.e.,theprospectsforcell-basedtreatmentofstrokeandrelatedneu- rovascular diseases.Thisisan area morethan any where therehave been spurious claims worldwideofnovelstem celltherapies.Thishasraisedfalse hopesforvul- nerablepatientsofmagiccuresintheabsenceofanycrediblescientificfoundation andpromotedaformof“stemcelltourism”thatthreatenstoovershadowtheimpor- tantprogressthatistakingplaceinacademicresearchcentersworldwide.Thefinal chapterprovidesacriticalreviewofwhatisnowpossibletoachieveandassessesthe realistic hopes oftrue progress inthis widespread disease. FunctionalNeural TransplantationIV, PartAand Part B,istargetedtoinform active basic and translational research scientists working in the field, although we anticipatethatthetopicwillattractamuchbroaderreadershipamongneuroscientists and clinicians interested in the current state of stem cell biology, cell therapy, and regenerative medicine. Readers can expect a balanced and realistic assessment of what is now possible, and the likely advances in the near future years, along with acriticalappraisalofthelimitsofcurrenttherapies,andadebunkingoftheunsub- stantiatedclaims. S. Dunnett, Cardiff, UK A.Bj€orklund, Lund, Sweden 13th February 2017
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