UCSF and Juliana’s Journey Foundation Seeking Funding for Clinical Trial

1.24.2014 press release final.pdf-page-0011.24.2014 press release final.pdf-page-002


UCSFandJuliana’s JourneyFoundation Seeking FundingforClinical Trial



SOUTHSANFRANCISCO, Calif.—January 23,2014 9:00pmPST:  Juliana’s JourneyFoundation isactivelyseeking funding tosupporta clinical trial focusingon arare PediatricCancer calledNeuroblastoma.

UCSF scientists,Dr. WilliamWeissand Dr.W. Clay Gustafson,areutilizing cuttingedge robotics andhigh-through-put liquid handling techniques tocombine large numbersof clinically available targetedinhibitors in different combinations tofind the mosteffective therapeuticcocktail. Once thistrial isfullyfunded and effectivecombination therapies are found, they will berapidly translated intoanimalmodelsof neuroblastomadevelopedat UCSF and then, in collaboration withDr.KatherineMatthayandher New Approaches toNeuroblastomaTherapy (NANT) consortium,these newtherapeuticcombinationscan berapidlymoved into clinical trialsin children with relapsedorresistantneuroblastoma for safetyand effectiveness againstthis deadly pediatric cancer.JJF needs to raise

$25,000.00 by March 31stto fully fund thistrial andare depending on local and national leaders aswell as theircommunitytohelp raise awareness andreach this goal.


Juliana’s Journey Foundation is a 501(c)3 Non-Profit Organization formed bythe parentsof JulianaPeña,who at thetenderageof2½ , losther courageous battleagainstrelapsed Neuroblastoma in Februaryof2012.The purposeof the foundation istohelp identify andgenerate fundingforresearchers tocreateless invasive, non-damaging treatment andmore successful therapies for Neuroblastoma. Along with their Boardmembers and manysupportersfromallover the country,they’ve committedthemselvesto raising awarenessabout Childhood Cancerand eventually, acure forthis deadly disease.


Neuroblastomahasthehighestmortality rateof all pediatric cancers.Although it is a common childhood cancer it is often difficult to treat. Neuroblastomais themost commonextracranial solid tumorcancer in children.The standardprotocolforneuroblastoma uses30-yearold chemotherapy agents, and the short and long-term effectsare justas destructive asthe treatment itself.In the UnitedStates, about650 children are diagnosedwith neuroblastoma eachyear,and it isthemostcommon tumor found in children youngerthan1 yearof age.Every16 hoursa child with neuroblastomadies and there is noknowncure forrelapsed neuroblastoma.Nearly70%of those children firstdiagnosed with neuroblastoma have diseasethat hasalreadymetastasized orspread to other partsofthe body. Whendisease has spread atdiagnosisand achild is overthe age of 2,thereis lessthana 30%

chanceof survival. Currentclinical trialsperformedby UCSF and NANT aretrying todevelopmoreeffective,less damaging therapies for NB, butwithsolittle funding from the government(lessthan 3%),these studies can take10years tocomplete.









Dr. KatherineMatthay,M.D., ChiefofPediatric Hematology andOncologyandLeaderof thePediatric

MalignanciesProgram,isone oftheworld’s leading doctors andresearchers intreatmentsforchildhood

cancer. She directsa national studyof the Children’sOncology Group to test newtherapies for

neuroblastoma (NANT). She and her colleagues demonstrated that theoutcomeof neuroblastoma can

be significantlyimproved by theuseof innovativetreatment thatincludes chemotherapy, bonemarrow

transplantationandthevitamin A derivative,13-cis-retinoic acid.

Matthayearned an undergraduatedegree in chemistry at Bryn MawrCollege inPennsylvania and a medical degreeat theUniversityofPennsylvania inPhiladelphia. She completed aresidency in pediatrics at theUniversityofColoradoinDenver and afellowshipinpediatric hematology andoncologyat UCSF MedicalCenter.Dr.Matthay’smanyaccolades include The American Cancer Society Scholar Award (1992),BourseHenri deRothschildfor Neuroblastoma Research Award (2000, 2001), Hadassah DoctorofDistinctionAward(2007)andwasawarded a VFoundation designated grantin2008 to support herworkon neuroblastoma.

Dr. WilliamA.Weiss,M.D.,is a professorof Neurology, Pediatrics, andNeurosurgery at UCSF, directorofClinical Child Neurology atSan Francisco GeneralHospital, directorof Child Neurology atUCSF’s BenioffChildren’s Hospital,co-directorofthePediatricsMalignancies Programand chairof theweekly seminarseries inUCSF’s Helen Diller FamilyComprehensive CancerCenter. He’s hadmultiple grantsawarded tohim for his ground-breaking researchin multiple cancer studies. Dr.WeisseditsBreaking Advances forCancer Research,is an associateeditorofNeuro-Oncology,servesonthe neuroblastoma biologycommittee for the Children’s Oncology Group, andis an external advisorto BrainTumorPrograms at theMayoClinic,Saint Jude Children’s Research Hospital,Universityof Calgary,Children’s HospitalofLosAngeles, and Southern California BrainTumorProgram.Hewasan external reviewerfortheDepartmentsof Geneticsand of Tumor CellBiologyatSt. Jude’sChildren’s Research Hospital(2009) andserved the NIH formany years andis currentlyapermanent memberof the NINDS NSDA studysection,and a permanent reviewerfor basicscienceswithin the CancerPreventionandResearch InstituteofTexas(underPhil Sharp, Albert Gilman, andChuckSherr).

Dr. ClayGustafson,M.D.isa Pediatric Hematologist and Oncologistandworks alongsideDr. Matthayand Dr. Weisswith allclinical trials attheHelenDillerCancer Care Center in SanFrancisco,Ca. Dr.

Gustafsonreceivedhis degreefromUniversityof Texas at Galveston.He completed a residency inpediatrics hematology/oncology atthe University ofTexas SouthwesternMedical School and afellowship in pediatric hematology andoncology atUCSFMedicalCenter.Hisspecializes inneuroblastomas, glioblastomas (braintumors), Dosimetry and genomicanalysis.


Please visithttp://JulianasJourney.orgtolearn moreabout Juliana and what JJFis doing tomake adifference inthe lives of our children orhttp://www.gofundme.com/JulianasJourneyUCSFifyou’dliketo help Juliana’sJourney Foundationfund this important clinicaltrial.

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