WrittenTestimonyofDr.JeffreyW.Chell

beforetheHouseCommitteeonEnergyandCommerce

Hearingon“ExaminingPublicHealthLegislation:

H.R.2820,H.R.1344,andH.R.1462”

June25,2015

  

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SummaryofKeyPoints

• The NMDP/Be The Match appreciates the continuing bipartisan support to reauthorize the C.W. Bill Young Cell Transplantation Program and the National Cord Blood Inventory. ItiscriticallyimportanttoreauthorizetheseprogramsbeforetheirauthorizationsexpireattheendofSeptember2015.

• SincethelateCongressmanBillYoungwasfirstinspiredby11‐year‐oldBrandyBlywhodidnotsurviveherbattlewithleukemiaandhetestifiedbeforetheCommerceCommitteein1987,significantprogresshasbeenmadetoprovideaccesstotransplantforallAmericansinneedofalife‐savingbonemarroworcordbloodtransplant.

• Today,theNMDP/BeTheMatchisthegloballeaderinhelpingpatientsseekingacureforoneofthemorethan70differentdiseasesorconditions,includingbloodcancersandsicklecelldisease.Cellulartherapyistheirbesthopeforacure.

• TheBeTheMatchRegistryhasgrowntoincludenearly12.5milliondonorsandmorethan209,000cordbloodunitsandthroughinternationalrelationships,wehaveaccesstomorethan24.5millionpotentialdonorsand622,000cordbloodunitsworldwide.

• Between1988and2005,theone‐yearsurvivalrateforthesepatientshasincreasedfromjustover40percentto70percent.Accesstomultiplecellsourcesallowsustofindthebestmatchpossibleforpatients.

• In2014,wefacilitatedmorethan1,200unrelatedtransplantsforpatients18oryoungerusingbonemarroworcordblood.Currentpediatricresearchfocusesnotonlyonmalignancies,butalsooncuringnon‐malignantdiseasessuchcertainmetabolicdiseasesandhemaglobanopathieslikesicklecelldiseaseandthalassemia.

• Thecalculatedneedforunrelatedtransplanthasincreasedby25percentsince2005.Muchofthereasonisduetotheexpansioninthenumberofindicationsfortransplant.

• Thenumberoftransplantsforminoritypatientshasincreasedfrom253in2000to990in2014.

• Thenon‐matchbarrierstoaccessandcarehaveaprofoundaffectonourabilitytomaketransplanttherapiesaccessibletoall.

  

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GoodmorningMr.Chairman,andotherdistinguishedmembersofthe

Subcommittee.MynameisDr.JeffreyChellandIamtheChiefExecutiveOfficeof

theNationalMarrowDonorProgram(NMDP)/BeTheMatch.WeoperatetheC.W.

BillYoungCellTransplantationProgram(Program)throughfourcompetitivelybid

contractswiththeHealthResourcesandServicesAdministration(HRSA).These

contractsincludetheSinglePointofAccessandOfficeofPatientAdvocacy,theBone

MarrowCoordinatingCenter,andtheCordBloodCoordinatingCenter.Collectively,

thesecontractsallowNMDP/BeTheMatchtooperatethenationalBeTheMatch

Registryandprovidelife‐savingbloodandmarrowtransplantsusingindividual

adultdonorsandumbilicalcordbloodunits.AndwiththeMedicalCollegeof

Wisconsin,NMDP/BeTheMatchalsoholdsthecontractfortheStemCell

TherapeuticOutcomesDatabase,knownastheCenterforInternationalBloodand

MarrowTransplantResearch(CIBMTR)ofwhichIamtheexecutivedirector.

Tobegin,IwouldliketothankChairmanPitts,RankingMemberGreen,andallofthe

MembersoftheHealthSubcommitteeforinvitingmetospeakwithyoutoday.On

behalfof565NetworkpartnerorganizationsandeveryoneattheNMDP/BeThe

Match,wealsowanttothankyouformaintainingtheCongressionalcommitmentto

patientsfightingbloodcancersandotherdisorderswhoseonlyhopeforacureisa

bonemarroworcordbloodtransplant.

  

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IwouldalsoliketothankCongressmanChrisSmith,CongresswomanDorisMatsui,

CongressmanDavidJolly,andCongressmanChakaFattahfortheirleadershipin

introducingH.R.2820toreauthorizetheProgram,aswellastheNationalCord

BloodInventory(NCBI)grantprogram.Theseprogramsareexamplesofhowthe

CongresscaninspireinnovationtobringcurestopatientsacrossAmerica.

WhoWeAre

AsItestifybeforeyoutoday,Icannothelpbutberemindedofanotherhearingthat

tookplacein1987.Onthatday,thelateCongressmanBillYoungcalledonthe

Congresstoestablishanationalregistrywheremen,women,andchildrenwith

leukemiaandotherfatalblooddisorderscouldfindanunrelateddonortosavetheir

lives.HewasjoinedbyDr.RobertGraves,whosedaughterreceivedthefirst

unrelatedbonemarrowtransplantforleukemia,andNavyAdmiralBudZumwalt.

TheCongressheardthatcallandestablishedthenationalregistry.Firsthousedin

theDepartmentoftheNavy,itfounditspermanenthomeasoneofthenation’s

premierpublichealthprogramsatHRSA.

CongressmanYoung’squestwasinspiredbyachild,11‐year‐oldBrandyBly,and

herfamilyracingtosaveherlife.Unfortunately,nooneinherfamilywasasuitable

matchandshedidnotsurvive.Itwasthesimplestatementfromherphysician–

“Wouldn’titbegreatiftherewereawaythatdoctorscouldsearchforadultswilling

todonatetheirbonemarrow?”–thatledtothenationalregistry.

  

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Sincethatfirsthearingin1987,wehavemadegreatprogress.Today,theNMDP/Be

TheMatchisthegloballeaderinprovidingacuretopatientswithlife‐threatening

bloodandmarrowcancerssuchasleukemiaandlymphoma,aswellasother

diseases.ThroughthecontractwithHRSA,wemanagetheworld’slargest

registry—theBeTheMatchRegistry—ofpotentialmarrowdonorsandumbilical

cordbloodunits,connectpatientstotheirdonormatchforalife‐savingmarrowor

umbilicalcordbloodtransplant,educatehealthcareprofessionals,andconduct

researchsomorelivescanbesaved.Today,childrenlikeBrandyhaveamuch

betterchancetofindthatlife‐savingmatchandultimatelyacure.

But,theC.W.BillYoungProgramasithascometobeknown,ismorethanthe

nationalregistry.ThroughtheProgram,theNMDP/BeTheMatchalsooperatesthe

OfficeofPatientAdvocacy.TheOfficeofPatientAdvocacyassistspatientsandtheir

familiesinnavigatingthecomplexitiesofhealthinsuranceandhelpsthemovercome

logistical,psychosocialandinformationalbarriersthroughoutthetransplant

continuum.Wealsoworkcloselywithdonorandcollectioncentersthroughthe

BoneMarrowCoordinatingCentercontracttorecruitandretainvolunteerpotential

donors,produceacomprehensiveplanfordonorretention,andplanforincreasing

operationalefficiencies.Wesimilarlyprovidefinancialandeducationalsupportto

publiccordbloodbanksasthemanageroftheCordBloodCoordinatingCenterand

provideguidancetoHRSAintheadministrationoftheNationalCordBlood

Inventoryprogramtodetermineoptimalcompositionofcordbloodinventory.And,

wepartnerwiththeMedicalCollegeofWisconsintooperatetheStemCell

  

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TherapeuticOutcomesDatabase,whichfacilitatesresearchtoimprovepatient

outcomesandfindnewandexcitingwaysbonemarrowandumbilicalcordblood

canbeusedtosavelives.

TheSuccessoftheProgram

Duringthepast28years,theNMDP/BeTheMatchhasbeenhonoredtoserveasthe

stewardofthiscriticalnationalresource.Today,theBeTheMatchRegistryserves

asthesinglepointofaccessforbothumbilicalcordbloodunitsandadultvolunteer

donors.Thissinglepointofaccessassuresthatthesephysicianswillhaveaccessto

anypotentialdonororumbilicalcordbloodunitregardlessofwherelocatedacross

theglobeinordertoperformadultstemcelltransplantsthatcancuremorethan70

differentdiseasesorconditions,includingbloodcancersandsicklecelldisease.The

BeTheMatchRegistryhasgrowntoincludenearly12.5milliondonorsandmore

than209,000cordbloodunits.Throughinternationalrelationships,theNMDP/Be

TheMatchhasaccesstomorethan24.5millionpotentialdonorsand622,000cord

bloodunitsworldwide.

Forpatientsbattlingthesefatalcancersandotherblooddisorders,theNMDP/Be

TheMatchofferssupportandeducationtohelpthemlivehealthylivesafter

transplant.Weprovidepatientservices,caregiversupport,andfinancialsupport

throughtheBeTheMatchFoundation.Wealsoworkcloselywithtransplant

physiciansthroughoutthecountrybydevelopingandimprovinguponpost‐

transplantguidelinestoimprovesurvivalrates.

  

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Ourfocusisonpatientsforwhomcellulartherapyisthebesthopeforcureoftheir

diseasesandisoftentheonlytherapyavailablewithanintenttocure.Today,we

areabletotreatpatientswithcancersandpre‐cancers,suchasleukemia,

Myelodysplasia,andlymphomas;bonemarrowfailuredisorders,suchasaplastic

anemiaandimmunodeficiencysyndromes;andgeneticdiseases,suchassicklecell

disease.Totreatthesediseases,weinfusebonemarrow,peripheralbloodstem

cells,orcordbloodcellsintoapatientafterhavingeliminatedhis/hercurrentbone

marrow.Thesenewcellsrestorethepatient’sabilitytomakebloodcellsorprovide

anewimmunesystemtoattackcancercells.Findingthebestmatchpossibleis

importantbecauseifdonorstemcellsarenotthesameHLAtypeastherecipient

theywillrecognizetherecipientasbeingdifferentandattack,leadingtorejection.

Becausewecollectdataonalltransplants,wehavebeenabletoimprovepatient

outcomesandreducerejection.Between1988and2005,theone‐yearsurvivalrate

forthesepatientshasincreasedfromjustover40percentto70percent.Accessto

multiplecellsourcesallowsustofindthebestmatchpossibleforpatients.Initially

focusedonlyonbonemarrow,theProgramtodayalsoallowsphysicianstoselect

peripheralbloodstemcellsandcordblood,aswellasbonemarrow,asthesourceof

theadultstemcellsusedintransplant.

Weareespeciallyproudoftheworkwehavedonetohelpchildreninneedofa

transplant.In2014,theNMDP/BeTheMatchfacilitatedmorethan1,200unrelated

  

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transplantsforpatients18oryoungerusingbonemarroworcordblood.Our

currentresearchfocusesnotonlyonmalignancies,butalsooncuringnon‐malignant

diseasessuchcertainmetabolicdiseasesandhemaglobinopathieslikesicklecell

diseaseandthalassemia.Theseblooddisorderscanbefatalifleftuntreated.Prior

totransplantationtherapy,childrenwiththesediseaseswouldoftendie

prematurely.

Wearealsolearningmoreabouthowtoimproveoutcomesforchildrenfighting

bloodcancers.Byhavingaccesstoallthreesourcesofadultstemcells,physicians

canselectthebestsourcetomeettheiryoungpatients’needs.Forexample,most

physicianspreferafullymatchedbonemarrowgraftifavailableforallpatients,

includingchildren.Incertaininstances,umbilicalcordbloodisused,especiallyif

thereisnofullymatchedadultdonor.Inthisway,umbilicalcordbloodhas

significantlyextendedtheopportunityforallpatientswhootherwisewouldnot

havefoundanacceptableadultmatch.ThroughtheCIBMTR,researchers

throughouttheworldarefindingnewandexcitingwaysthatbonemarrowandcord

bloodtransplantscanhelpchildrenfightlife‐threateningdiseases.

MoreNeedsToBeDone

However,moreneedstobedone.Theneedfortransplantsisincreasing,especially

amongolderAmericans.Thecalculatedneedforunrelatedtransplanthasincreased

by25percentsince2005.Muchofthereasonisduetotheexpansioninthenumber

ofindicationsfortransplant,aswellasadvancesthatallowolderAmericanstobe

  

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candidatesfortransplants.Transplantsforpatients51‐64yearsoldaregrowing

fasterthanotheragegroups.NMDP/BeTheMatchfacilitatedtransplantshave

grownby200percentoveralland250percentforminoritiessince2006.

Whilewehavemadesignificantimprovementsintransplantsforracialandethnic

minoritypatients,theretoomoreworkisneeded.Thenumberoftransplantsfor

minoritypatientshasincreasedfrom253in2000to990in2014.Wecontinueour

effortstoexpandthediversityofthenationaladultvolunteerdonorregistryand46

percentofcordbloodunitsontheregistryarefromaminoritydonor.Duringthe

last5yearscordbloodhasbeentheproductsourceforabout21percentofall

transplantsand37percentofminoritypatientswhoreceivedatransplantrelied

uponcordblood.

Federalfundingremainscriticaltocontinuingtoprovideaccesstotransplantation.

Weneedtocontinuetorecruitnewpotentialdonorsbothtoimproveaccessfor

minoritypatientsandtorenewthecurrentlistofdonorswithyoungerdonors.

Graftsfromyoungerdonorshaveshownimprovedclinicaloutcomes.Foreveryone

milliondollarsallocatedbytheCongress,theProgramcanadd10,000adult

volunteerdonorsor750cordbloodunitstothenationalregistry.Preservingthese

fundsthroughthereauthorizationoftheProgramallowsittocontinuetoimprove

thechancesofeveryAmericanneedingatransplanttofindamatchandprovidesthe

criticalinfrastructurethatallowsNCBIcordbloodunitstobeusedtosavelives.

  

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EventhoughtheNMDP/BeTheMatchhasimprovedtheabilityofthoseneedinga

transplanttofindamatch,thereareotherbarriersthatcontinuetomakeaccess

difficult.Thesenon‐matchbarrierstoaccessandcarehaveaprofoundaffectonour

abilitytomaketransplanttherapiesaccessibletoall.Language,literacy,finances,

insurance,geography,lackofknowledge,andpredispositionbygeneral

hematologistsandoncologiststowardsnon‐transplanttherapiesallhaveanimpact.

TheNMDP/BeTheMatchcontinuestoworkwithpatients,physicians,community

leaders,andotherstoaddresstheseproblemsaswell.

Conclusion

TheongoingCongressionalcommitmenthasmadetheseadvancespossibleand

turnedthetragiclossof11‐year‐oldBrandyBlyintohopefortensofthousandsof

Americans.OneofthoseAmericansisHadleyMercer.Whenshewasjustsix

monthsold,Hadleywasdiagnosedwithacutemyeloidleukemia(AML).Aftertwo

roundsofchemotherapy,herparentsbegantoconsiderabonemarrowtransplant

asanalternativetreatmentoptionforHadley.Theyconsultedvariousphysicians,

whoallagreedthathavingabonemarrowtransplantwouldbeherbestchanceof

survival.Thefamilyheldabonemarrowdriveandregisteredmorethan1,000

peopleinfivehours.Afewmonthslater,Hadleywasmatchedwithayoungmanin

histwenties.Nowalmosttwo,Hadleyisalivebecauseofher“donorangel”.Sheis

alsoalivebecauseofyourcontinuedsupportfortheC.W.BillYoungCell

TransplantationProgram.

  

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TheNMDP/BeTheMatchhasneverforgottentheimportanceofthephysician’s

simplestatementthatinspiredCongressmanYoungandeverydayweareinspired

bythepeoplewemeet,youngandold,whoareseekingtofindamatchandundergo

atransplant.

  

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Appendix:StatisticalChartsandGraphs

  

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