Translational Medicine A new role for the non-profit medical research foundation Presented by Scott Johnson President and ...
Overview  <ul><li>Background of the MRF </li></ul><ul><li>The ARC™ Model: Principles and Practices </li></ul><ul><li>Facin...
Background <ul><li>What is the Myelin Repair Foundation? </li></ul><ul><li>Mission and goals </li></ul>
The Accelerated Research Collaboration ™ (ARC ™)  Model: Principles and Practice <ul><li>Assemble the best people  (experi...
Basic Science Consortium <ul><li>Who participates? </li></ul><ul><ul><li>Core academic labs </li></ul></ul><ul><ul><li>Spo...
Basic Science Consortium: Results <ul><li>Targets </li></ul><ul><li>Models and Assays </li></ul><ul><li>Published papers <...
The Valley of Death:  Broadly defining the problem <ul><li>Discoveries in academic labs lack industry rigor </li></ul><ul>...
The Valley of Death:  Special Challenges in the CNS <ul><li>Limited availability of human tissue </li></ul><ul><li>Blood-B...
Addressing the challenges:  A Novel Approach  <ul><li>De-risk development of drugs that enhance myelin repair thereby lowe...
Applying the principles and practices of the ARC ™  model to translational medicine <ul><li>Assemble the best people (Expe...
Applying the principles and practices of the ARC ™  model to translational medicine <ul><li>Create a non-competitive envir...
An ideal role for non-profits <ul><li>The best entity to coordinate and be involved in the entire continuum – from discove...
The ultimate benefit to pharma <ul><li>Access to assays, models and experts </li></ul><ul><li>Validated targets </li></ul>...
Summary <ul><li>The MRF is exploiting its position as a non-profit to benefit academics, pharma and  most important, patie...
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Carving a New Niche for Non-Profits: Filling the Translational Gap for Novel Treatments for Diseases with Unmet Needs - Scott Johnson, Myelin Repair Foundation

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Scott Johnson - Myelin Repair Foundation, Speaker at the marcus evans Discovery Summit Fall 2011 in Las Vegas, delivers his presentation on Carving a New Niche for Non-Profits: Filling the Translational Gap for Novel Treatments for Diseases with Unmet Needs

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Carving a New Niche for Non-Profits: Filling the Translational Gap for Novel Treatments for Diseases with Unmet Needs - Scott Johnson, Myelin Repair Foundation

  1. 1. Translational Medicine A new role for the non-profit medical research foundation Presented by Scott Johnson President and Founder Myelin Repair Foundation September 27, 2011
  2. 2. Overview <ul><li>Background of the MRF </li></ul><ul><li>The ARC™ Model: Principles and Practices </li></ul><ul><li>Facing the Valley of Death </li></ul><ul><li>Employing the ARC model in Translational Medicine </li></ul><ul><li>A unique / ideal role for non-profits </li></ul><ul><li>Benefit to pharma </li></ul><ul><li>Questions/Feedback </li></ul>
  3. 3. Background <ul><li>What is the Myelin Repair Foundation? </li></ul><ul><li>Mission and goals </li></ul>
  4. 4. The Accelerated Research Collaboration ™ (ARC ™) Model: Principles and Practice <ul><li>Assemble the best people (experience, expertise, diversity) </li></ul><ul><li>Provide them with the necessary resources </li></ul><ul><li>Be laser-focused on a single, complex problem </li></ul><ul><li>Develop a plan </li></ul><ul><li>Create a collaborative environment </li></ul><ul><li>Protect IP </li></ul><ul><li>Add value to all participants </li></ul><ul><li>Remove any and all impediments </li></ul><ul><li>Metrics and management </li></ul>
  5. 5. Basic Science Consortium <ul><li>Who participates? </li></ul><ul><ul><li>Core academic labs </li></ul></ul><ul><ul><li>Sponsored Researchers </li></ul></ul><ul><ul><li>Academic, Industry and Clinical Advisors </li></ul></ul><ul><ul><li>MRF staff scientists </li></ul></ul><ul><ul><li>MRF management </li></ul></ul>
  6. 6. Basic Science Consortium: Results <ul><li>Targets </li></ul><ul><li>Models and Assays </li></ul><ul><li>Published papers </li></ul><ul><li>Patents </li></ul><ul><li>Commercial interest </li></ul>
  7. 7. The Valley of Death: Broadly defining the problem <ul><li>Discoveries in academic labs lack industry rigor </li></ul><ul><li>Results in rodents may not translate to the human system </li></ul><ul><li>No biomarkers </li></ul><ul><li>Two cultures that are vastly different </li></ul>
  8. 8. The Valley of Death: Special Challenges in the CNS <ul><li>Limited availability of human tissue </li></ul><ul><li>Blood-Brain Barrier </li></ul><ul><li>No biomarkers </li></ul>High-risk situation for drug development
  9. 9. Addressing the challenges: A Novel Approach <ul><li>De-risk development of drugs that enhance myelin repair thereby lowering the bar for pharma engagement </li></ul><ul><ul><li>Standardize complex pathology-relevant models and assays for use in commercial drug discovery and development </li></ul></ul><ul><ul><li>Invest resources in building human in vitro assays </li></ul></ul><ul><ul><li>Invest in the identification of biomarkers </li></ul></ul>
  10. 10. Applying the principles and practices of the ARC ™ model to translational medicine <ul><li>Assemble the best people (Experience, expertise, diversity) </li></ul><ul><ul><li>Clear goals and metrics </li></ul></ul><ul><ul><li>Scientific Advisory Board </li></ul></ul><ul><ul><li>Drug Discovery Advisory Board </li></ul></ul><ul><ul><li>Clinical Advisory Board </li></ul></ul><ul><ul><li>Internal MRF Staff ( 103 years of pharma expertise) </li></ul></ul><ul><ul><li>Sponsored researchers </li></ul></ul><ul><ul><li>CROs </li></ul></ul><ul><ul><li>Pharma partners </li></ul></ul>
  11. 11. Applying the principles and practices of the ARC ™ model to translational medicine <ul><li>Create a non-competitive environment </li></ul><ul><li>Fill critical gaps </li></ul><ul><li>Create relevant incentives for each participant </li></ul><ul><li>Enforce a focus on patient need </li></ul><ul><li>Remove any and all impediments </li></ul><ul><li>Lead by example </li></ul>
  12. 12. An ideal role for non-profits <ul><li>The best entity to coordinate and be involved in the entire continuum – from discovery biology to FDA approval </li></ul><ul><li>Why? </li></ul><ul><ul><li>Not risk averse </li></ul></ul><ul><ul><li>The luxury of a laser focus on one problem </li></ul></ul><ul><ul><li>Bridge between academic and business cultures </li></ul></ul><ul><ul><li>The only incentive is patient need </li></ul></ul><ul><ul><ul><li>No quarterly earnings calls </li></ul></ul></ul>
  13. 13. The ultimate benefit to pharma <ul><li>Access to assays, models and experts </li></ul><ul><li>Validated targets </li></ul><ul><li>Freedom to operate </li></ul><ul><li>Precompetitive collaborations / consortia </li></ul>
  14. 14. Summary <ul><li>The MRF is exploiting its position as a non-profit to benefit academics, pharma and most important, patients. </li></ul>
  15. 15. Questions / Feedback

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