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Clinical TrialsExplained and ExploredPresented By:Jerry CallMarch 11, 2013

1What is a Clinical Trial?Health-related research studyRequires ethics review (IRB)

Follows a pre-defined protocolRequires informed consent

Only 2-4% of cancer patients participate in clinical trials

No animation on this slide2Why do Patients Participate in Trials*?89%-Obtaining possible benefit very important17%-Helping future cancer patients/treatmentsOther factors cited as very important66%-Trust in doctor66%-Being treated by the latest treatment available61%-Better standard of care and closer follow-up71% stated that surviving for as long as possible was the most important thing for them

*Survey of 38 patients participating in phase I and phase II trials British Journal of Cancer (2005) 92, 1001-1005

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3May not be effectiveSide effectsAdditional testing, time and travel (travel expenses)Some costs may not be covered by insurance

Option to access treatment after standard treatments failReceive treatment at a major GIST trial centerHelp future patientsBenefitsRisks

Last bullet point is Help future patients4Clinical Trial PhasesEffectRegulatoryApprovalFurther StudySafety1234Pre-Clinical Evidence of Effect in GISTEffective in GISTCompareAfter Approval?GIST TargetsDrug CandidatesSometimes CombinedLong TermEffectsApprox 10% of drugs that start get approvedPhase IIFurther defines the safety and begins to evaluate effectivenessPhase IIICompare a new agent with the current standard treatmentRandomized to groups

Phase IVUsually take place after the treatment is approvedFurther evaluate long-term safety and effectivenessPhase IFirst step in humansIncreasing doses (cohorts) determine safe doseEvaluate route of administrationSide effects

5The overall picture:In general cancer drugs have taken 7-9 years to travel this pathGleevec did it in two years in GIST (but it had the benefit of CML preceding)It can be done when efficacy is high (70 % benefit versus