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BloodBrainBarrierSafelyBreached
FranLowry

November16,2015

DoctorsatSunnybrookHealthSciencesCentreinToronto,Ontario,Canada,havenoninvasivelypenetratedtheblood
brainbarriertodeliverachemotherapeuticagentdirectlyintoapatientsmalignantbraintumor.
Thisisthefirsttimethatthebloodbrainbarrierhasbeensafelybreachedinahuman,theresearcherssay.
Thebloodbrainbarrierhaslongthwartedthedeliveryofchemotherapeuticandotheragentsintothebrain.
ButtheTorontodoctorshopethatthetechniquetheyused,focusedultrasound,willcontinuetobesuccessfulinsafely
penetratingwhathasbeenapersistentobstacletotreatingnotonlybraintumorsbutalsootherdiseases,suchas
Alzheimer'sdiseaseandParkinson'sdisease.
"Iwanttostressthatthishasonlybeendoneinonepatient,a56year
oldwomanwithglioblastoma,andtheresult,asexcitingasitis,isas
preliminaryasitgets,"neurosurgeonToddMainprize,MD,fromthe
UniversityofToronto,toldMedscapeMedicalNews.
"Thisisaproofofconceptphase1clinicalstudytomakesurethat
whenweopenupthebloodbrainbarrierwedon'tcauseany
hemorrhagesorinfection,andthatwecansafelyandeffectivelydeliver
adrugwhereinthebrainwewantto,"DrMainprizesaid.
DrMainprizeconfessedhewasabitsurprisedbytheintensemedia
interestthatthiscasegenerated.

CourtesyofSunnybrookHospital

"Ican'tstressenoughthatthisisaphase1trial.Wedonothavea
publishedpaper,wehaveonlydonethisinonepatientsofar.Thatbeingsaid,wethinkthisisaveryexcitingtreatment
andhasalotofpotentialapplicationsinthefuture.Butitisessentiallyadrugdeliverymethod,"hesaid.
TheSunnybrookteamplannedtotestfocusedultrasoundin10patientsaspartoftheirphase1trial.However,
becausethefirstcasewentsowell,theywillprobablyonlydo4beforetheygointoaphase2trial,DrMainprizesaid.
"Wewillsee,thisisonlythefirstpatient,buttheresultshavebeensoconclusive,itappearstobequitesafe.We'llsee
howtheothersgoandwemayjuststopbefore10patients,"hesaid.
Theresearchersinfusedthechemotherapyagentdoxorubicin,along
withtinygasfilledbubbles,intothebloodstreamofthepatient.They
thenappliedfocusedultrasoundtoareasinthetumorandsurrounding
brain,causingthebubblestooscillateandbypassthetightjunctionsof
thecellsofthebloodbrainbarrier.
"Wethinkthattheoscillationinsizewherethecellsswellandshrink
pokesholesinthebloodbrainbarrierandallowsthevariousdrugsor
whatevertocrossthebloodbrainbarrierintothesubstanceofthe
brain,"DrMainprizeexplained.

CourtesyofSunnybrookHospital

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"Inourfirstpatientwecouldclearlyseeenhancementintheareathat
weopened.Wecouldseethatthegadoliniumwascrossingtheblood
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brainbarrierintothebrain,makingthatareaturnwhitewhereweopenedit,asshownontheMRIsequenceswe
obtained.Itclearlyworkedinthispatient,thereisnodoubtthatwewereabletoopenthebloodbrainbarriersafely,
withoutanyproblems,basedonthegadoliniumuptake,"hesaid.
"Wereveryexcitedaboutitandwethinktherearemanypotential
[uses]forthisdrugdeliverysystem,butIhavetostressthatthisisvery
earlyandtherewillnotbewidespreadapplicationsforawhile,"Dr
Mainprizesaid.
FocusedUltrasoundforAlzheimer'sandParkinson's
ElisaKonofagou,PhD,professorintheDepartmentofBiomedical
EngineeringatColumbiaUniversity,NewYork,NewYork,andher
grouparealsostudyingfocusedultrasoundfortheopeningofthe
bloodbrainbarrierfordrugdeliveryinbothAlzheimer'sdiseaseand
Parkinson'sdisease.

CourtesyofSunnybrookHospital

"Wehavedonesomeworkinmiceandmonkeysandwehaveshown
bothneuroprotectionandneurorestorationinmousemodelsofParkinson'sdiseasebyopeningthebloodbrainbarrier
anddeliveringaneurotrophicfactor.Wehavealsoshownthatdrugdeliverythroughtheultrasoundinducedbloodbrain
barrieralsoworksinnonhumanprimates,"DrKonofagoutoldMedscapeMedicalNews.
SheaddedthathergrouphassecuredfundingbytheNationalInstitutesofHealthtobegin
clinicaltrialsonusingfocusedultrasoundforbloodbrainbarrieropeninginpatientswith
Alzheimer's,afterhavingshownpreliminaryresultsonfeasibilityinmousemodels.
TheworkwassupportedbytheFocusedUltrasoundFoundation,theCanadaFoundationfor
InnovationandOntarioMinistryofResearchandInnovation,theBombardierFoundation,the
CanadianInstitutesofHealthResearch,andtheNationalInstitutesofHealth.

DrElisa
Konofagou
MedscapeMedicalNews2015WebMD,LLC
Sendcommentsandnewstipstonews@medscape.net.
Citethisarticle:BloodBrainBarrierSafelyBreached.Medscape.Nov16,2015.

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