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

Grace Medical Institute

Research advances in animal models Often lost in translation… Why? Problem of CNS variability

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Risks & Solutions

Grace Medical Institute

• Funding follows human relevance!!!
• Best projects estimate translational success.
• Moving targets…

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Risks & Solutions

Grace Medical Institute

• 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

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“Did You Check?”

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Mouse Non-human primate (NHP) Human Risk Profile

Interesting developmental profile of Gene X (neurodevelopmental disease target)

Similar to mouse Similar to mouse and NHP Additional line

• f evidence!

Strong expression of Gene Y (target for schizophrenia) in basal forebrain

Somewhat similar to mouse Less specific, lower levels Any efficacy issue?

Gene Z (target for seizure) highly localized in Hippocampus

Strong expression in Cerebellum besides Hippocampus Variable or indeterminate data Any safety concern?

Examples

Testing assumptions earlier improves risk estimate, improves success.

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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. Grace Medical Institute

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“Get Ahead”

Grace Medical Institute

• Validation of research aims or results
• Use “Big science” to reinforce research efforts.
• Stand out grant applications – multiply lines of evidence.
• Stronger manuscripts with original and corroborating evidence.
• Strategic guidance of research directions
• Screening genes and mechanisms (GWAS, CNV, etc.)
• 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.”

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Discovery

Biomedical “Valley of Death”

“The major barriers to clinical success have been the limited 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

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Drug Development

How To Cross “Valley of Death”

• This gap in drug development could be alleviated if addressed

earlier with recent advances in basic and clinical neurosciences, especially in the early target validation process.

A little Nudge, Big Savings

Grace Medical Institute

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Drug Development

NeuroAnalytic Services

• We help predict CNS specific issues
• Target identification, selection, vetting, and ranking
• Animal model translation, potential efficacy/side effects
• Reinforce assumptions and de-risk investment

Grace Medical Institute

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Drug Development

Summary

Science

• Translational relevance
• Big Data – standardized,

comprehensive, CNS- focused (e.g. Allen brain maps)

• Multiple data modalities
• Vetted to literature

Business

• Fill translation gap
• Interpretation by trusted

experts who produced brain maps

• Dry-lab / light resource
• Scalable – API, streamlined
• Accessible, collaborative

“Fueling Discovery”

Grace Medical Institute

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