NeuroAnalytic Services Accelerating Brain Research & Drug - PowerPoint PPT Presentation

NeuroAnalytic Services Accelerating Brain Research & Drug Development Grace Medical Institute August 2016

Crucial Gap in Biomedical Research Research advances in animal models Often lost in translation… Why? 2 Problem of CNS variability Grace Medical Institute

Risks & Solutions • Funding follows human relevance!!! • Best projects estimate translational success. • Moving targets… 3 Grace Medical Institute

Risks & Solutions • Moving targets? CNS solutions rarely involve a single variable • Extraordinary multi-variate problem • Brains contain organs within an organ! – Neuroanatomy • Brain organs vary in cell composition – Cell types • Brains are optimized by species – Species • Brains change over time – Development, Environment, Age • Brains change all the time – Individual, Physiological • Brains are built differently – Connections, Genes, Proteins, etc. • Brain research has severe resource constraints • Expertise, data, time, etc. • Simplifying assumptions will reduce the constraints, but increase the translational risk. • Now possible to check assumptions to lower the risk 4 Grace Medical Institute

“Did You Check?” Testing assumptions earlier improves risk estimate, improves success. Examples Mouse Non-human Human Risk Profile primate (NHP) Interesting developmental Similar to mouse Similar to mouse Additional line profile of Gene X and NHP of evidence! (neurodevelopmental disease target) Strong expression of Gene Somewhat similar Less specific, Any efficacy Y (target for schizophrenia) to mouse lower levels issue? in basal forebrain Gene Z (target for seizure) Strong expression Variable or Any safety highly localized in in Cerebellum indeterminate concern? 5 Hippocampus besides data Hippocampus Grace Medical Institute

Research Directions at GMI Drug Development • Help validate/select drug targets in early stage of drug development, ensuring safer and more efficient investment. Discovery • Test hypothesis and provide preliminary data or guidance to better tackle major CNS diseases. Preferred provider / Sanity Check • Proxy for reproducibility, translational relevance, corroboration for many applications via NIH, FDA, EPA, Journals, etc. 6 Grace Medical Institute

“Get Ahead” • Validation of research aims or results • Use “Big science” to reinforce research efforts. • Stand out grant applications – multiply lines of evidence. Discovery • Stronger manuscripts with original and corroborating evidence. • Strategic guidance of research directions • Screening genes and mechanisms (GWAS, CNV, etc.) o Who’s the winner? Where to start? What’s next? • “Dry Lab” allows more experiments, better predictions. • Expertise and experiences in large scale brain mapping projects. “Let us help you to maximize the benefits of Big Science.” 7 Grace Medical Institute

Biomedical “Valley of Death” “The major barriers to clinical success have been the limited Drug Development predictive value of pre- clinical models… Most clinical trials of CNS compounds have been a black box exercise…” - William Potter, Neuropsychopharmacology Reviews 2012 Resource Wasted Time Delayed Cost Increase And More Patients At Risk 8 Grace Medical Institute

How To Cross “Valley of Death” • This gap in drug development could be alleviated if addressed Drug Development earlier with recent advances in basic and clinical neurosciences, especially in the early target validation process. A little Nudge, Big Savings 9 Grace Medical Institute

NeuroAnalytic Services • We help predict CNS specific issues Drug Development • Target identification, selection, vetting, and ranking • Animal model translation, potential efficacy/side effects • Reinforce assumptions and de-risk investment 10 Grace Medical Institute

Summary “Fueling Discovery” Science Business • Translational relevance • Fill translation gap • Big Data – standardized, • Interpretation by trusted comprehensive, CNS- experts who produced focused (e.g. Allen brain brain maps maps) • Dry-lab / light resource • Multiple data modalities • Scalable – API, streamlined • Vetted to literature • Accessible, collaborative 11 Grace Medical Institute