MJFF Strategy for<br />Accelerating Development<br />of Parkinson’s Therapies<br />Research Roundtable<br />Chicago, Illin...
Today’s Agenda<br />MJFF Overview<br />Deborah W. Brooks<br />The Michael J. Fox Foundation for Parkinson’s Research<br />...
3<br />MJFF Overview<br />Deborah W. Brooks<br />Co-Founder/Executive Vice Chairman<br />The Michael J. Fox Foundation for...
Parkinson’s disease:  Defining the need<br />Parkinson’s disease (PD) is a progressive neurodegenerative disease that affe...
MJFF was founded with clear objectives<br />Drive the Best Parkinson’s Research<br />Deliver Improved Therapies and a Cure...
Since founded in 2000, MJFF has funded more than $240M in research<br /><ul><li>In 2010, we received nearly 65,000 contrib...
Core values are efficiency and accountability: nearly 90 cents of every $1 spent goes straight to research program efforts...
In 2010, we reviewed over 800 PD-specific grants and currently have roughly 250 active grants in our portfolio.  </li></li...
Our approach has evolved as we’ve grown and learned<br /><ul><li>Focus on research is required to move the needle
The science is ahead of the money
We are not a bank, assets must be deployed quickly to the best science
Our goal is to go out of business  10-year horizon </li></ul>____________________________________________________________...
Strategy must focus on tackling roadblocks to progress in a systemic fashion, i.e. target validation, research tools, clin...
More proactive outreach and engagement with industry partners is required
Turning our knowledge out can ensure others benefit from the field knowledge we are developing
MJFF is in a unique position to bring all constituents together in the name of faster progress</li></ul>2005-11<br />8<br ...
How does MJFF work to accelerate progress today? <br />Craft aninformed agenda<br /><ul><li>Understand needs of patients
View global field and identify most promising targets
Convene experts in non-competitive environment </li></ul>Prioritize critical research<br /><ul><li>Infuse capital at under...
Develop and share essential tools
Pragmatically push research in a goal-directed, milestone-driven fashion</li></ul>Problem-solve: <br />lead and innovate<b...
Showcase top ideas to industry
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  1. 1. MJFF Strategy for<br />Accelerating Development<br />of Parkinson’s Therapies<br />Research Roundtable<br />Chicago, Illinois<br />June 27, 2011<br />
  2. 2. Today’s Agenda<br />MJFF Overview<br />Deborah W. Brooks<br />The Michael J. Fox Foundation for Parkinson’s Research<br />MJFF Program Highlights<br />Sonal S. Das, PhD<br />The Michael J. Fox Foundation for Parkinson’s Research<br />Panelists<br />Christopher G. Goetz, MD<br />Rush University Medical Center<br />Jeffrey H. Kordower, PhD<br />Rush University Medical Center<br />Tanya Simuni, MD<br />Northwestern University<br />Glenn T. Stebbins, PhD<br />Rush University Medical Center<br />Questions & Answers Session<br />2<br />
  3. 3. 3<br />MJFF Overview<br />Deborah W. Brooks<br />Co-Founder/Executive Vice Chairman<br />The Michael J. Fox Foundation for Parkinson’s Research<br />
  4. 4. Parkinson’s disease: Defining the need<br />Parkinson’s disease (PD) is a progressive neurodegenerative disease that affects nearly one million Americans; related Parkinsonisms affect an additional half million Americans.<br />PD prevalence is expected to grow sizably over the next 25 years as the proportion of older Americans continues to increase.<br />Significant therapeutic needs exist:<br />Current therapies to treat motor and non-motor symptoms are inadequate leaving substantial unmet needs for those living with PD.<br />There is currently no therapy proven to modify the progression of the disease.<br />4<br />
  5. 5. MJFF was founded with clear objectives<br />Drive the Best Parkinson’s Research<br />Deliver Improved Therapies and a Cure<br />5<br />
  6. 6. Since founded in 2000, MJFF has funded more than $240M in research<br /><ul><li>In 2010, we received nearly 65,000 contributions and raised $57M.
  7. 7. Core values are efficiency and accountability: nearly 90 cents of every $1 spent goes straight to research program efforts. We work to redeploy all donations quickly. We have no endowment or excess reserves.</li></ul>6<br />(est.)<br /><ul><li>Our in-house staff of 7 PhDs and 6 business strategists serve as portfolio managers, incorporating the advice and input of experts from academia and industry into their decision-making.
  8. 8. In 2010, we reviewed over 800 PD-specific grants and currently have roughly 250 active grants in our portfolio. </li></li></ul><li> Progress requires translation of discoveries through the pipeline <br />7<br />Preclinical<br />Convert biology into therapies Partly done in academic and biotech laboratories<br />Clinical<br />Determine safety and efficacy in patients<br />Mostly done by large pharma<br />$680 million/year<br />Basic Discovery<br />Understanding disease mechanisms<br />Mostly done by academics<br />$156 million/year<br />Unique opportunity for MJFF’s strategic funding/leadership to bridge the gap<br />
  9. 9. Our approach has evolved as we’ve grown and learned<br /><ul><li>Focus on research is required to move the needle
  10. 10. The science is ahead of the money
  11. 11. We are not a bank, assets must be deployed quickly to the best science
  12. 12. Our goal is to go out of business  10-year horizon </li></ul>________________________________________________________________________________________________________________________<br />2000-05<br /><ul><li>MJFF grant-making cannot be done in a vacuum – a comprehensive view of the landscape is key
  13. 13. Strategy must focus on tackling roadblocks to progress in a systemic fashion, i.e. target validation, research tools, clinical trial design, recruitment
  14. 14. More proactive outreach and engagement with industry partners is required
  15. 15. Turning our knowledge out can ensure others benefit from the field knowledge we are developing
  16. 16. MJFF is in a unique position to bring all constituents together in the name of faster progress</li></ul>2005-11<br />8<br />8<br />
  17. 17. How does MJFF work to accelerate progress today? <br />Craft aninformed agenda<br /><ul><li>Understand needs of patients
  18. 18. View global field and identify most promising targets
  19. 19. Convene experts in non-competitive environment </li></ul>Prioritize critical research<br /><ul><li>Infuse capital at underfunded, high-risk stages
  20. 20. Develop and share essential tools
  21. 21. Pragmatically push research in a goal-directed, milestone-driven fashion</li></ul>Problem-solve: <br />lead and innovate<br /><ul><li>Facilitate handoffs and orchestrate connections
  22. 22. Showcase top ideas to industry
  23. 23. Tirelessly pursue smart, creative solutions to challenges</li></ul>DE-RISK PD<br />9<br />
  24. 24. 10<br />2011 MJFF Program Highlights<br />Need for Disease-Modifying Therapies<br />Biomarkers and The Parkinson’s Progression Markers Initiative<br />Improving Symptomatic Treatments<br />Sonal S. Das, PhD<br />Associate Director, Research Programs<br />The Michael J. Fox Foundation for Parkinson’s Research<br />
  25. 25. MJFF priority area efforts drive progress in key research topics<br />11<br />
  26. 26. Altering the course of PD: Need for Disease-Modifying Therapies<br />12<br />MJFF has committed over $100M to advance disease modifying therapies and seeks a path forward to patients. <br />
  27. 27. Expert Insight, Jeffrey H. Kordower, PhD<br />13<br />Disease-Modifying Therapies<br />Jeffrey H. Kordower, PhD<br />Director, Research Center for Brain Repair<br />The Jean-SchweppeArmour Professor of Neurological Sciences<br />Rush University Medical Center<br />
  28. 28. 14<br />Obstacles include:<br /><ul><li>Current trial paradigm requires large number of subjects
  29. 29. Efficacy testing is a long process, diminishing patent value
  30. 30. Reliance on clinical measures alone confound trial interpretation
  31. 31. There are no biological indications of underlying disease that complement clinical changes</li></ul>Markers of progression are essential to achieve near-term hopes for needed therapies<br />Challenges remain in testing disease-modifying therapies<br />
  32. 32. MJFF has supported almost $31M in biomarker discovery<br />Biomarkers<br />Therapeutic Markers<br />Progression Markers<br />Diagnostic Markers<br /><ul><li>Identify PD patients and, perhaps, even determine who is at risk for developing PD
  33. 33. Assist with patient selection for clinical studies…Are we studying the right population of people?
  34. 34. Facilitate measurement of modifications in the disease - could provide valid clinical trial endpoints
  35. 35. Help guide clinical trial design parameters like patient numbers, stratification, duration of treatment
  36. 36. Is the therapeutic reaching its target?
  37. 37. Is the therapeutic having its desired effect?</li></ul>Without markers of progression, clinical trials to test <br />new therapies in patients are at risk of yielding <br />inconclusive results <br />15<br />
  38. 38. The Parkinson’s Progression Markers Initiative (PPMI)<br />16<br /><ul><li>PPMI is a ground-breaking clinical study that aims to dramatically accelerate PD drug development by identifying biomarkers of the disease. PPMI aims to recruit 400 newly diagnosed people with Parkinson’s and 200 control participants.
  39. 39. A pre-competitive collaboration between government and industry.
  40. 40. A study of this size – $45M over a 5 year period – requires large scale funding: Abbott, Biogen Idec, Covance, GE Healthcare, Genentech, Merck, Pfizer and Roche are lead industry supporters.
  41. 41. As of Wednesday, June 22nd, 2011, 19 out of 21 PPMI sites in the US and Europe are currently recruiting volunteers. At these sites, 140 participants have been recruited (82 PD, 58 control) with an additional 43 consents.</li></li></ul><li>Expert Insight, Tanya Simuni, MD<br />17<br />Insights Into PPMI and What We Hope to Learn<br />Tanya Simuni, MD<br />A.C. Nielsen Research Professor of Parkinson's disease <br />Associate Professor of Neurology<br />Director, Parkinson’s Disease and Movement Disorders Center<br />Northwestern University<br />
  42. 42. Clear need for developing treatments for motor & non-motor symptoms<br />18<br />MJFF has funded nearly $17M in dyskinesia research and $18.5M in research towards improving treatments for non-motor symptoms. <br />Non-Motor Symptoms<br /><ul><li>Includes cognitive dysfunction, anxiety, memory loss and mood disorders
  43. 43. Relieving these symptoms leads to a better quality of life for those living with PD</li></ul>Dyskinesia<br /><ul><li>Uncontrolled body movements that result from dopamine-replacement therapy
  44. 44. Breakthroughs in treating dyskinesia would expand options for treating PD</li></ul>MJFF is funding two parallel tracks in dyskinesia research: <br />Developing new therapies <br />Determining how to best assess these therapies in the clinic (Dyskinesia Rating Scale Study)<br />
  45. 45. Expert Insight: Christopher G. Goetz, MD<br />19<br />Advancing Treatments for Dyskinesia<br />Christopher G. Goetz, MD<br />Professor of Neurological Sciences<br />Professor of Pharmacology<br />Rush University Medical Center<br />Co-Principal Investigator, Dyskinesia Rating Scale<br />Glenn T. Stebbins, PhD<br />Professor of Neurological Sciences<br />Rush University Medical Center<br />Co-Principal Investigator, Dyskinesia Rating Scale<br />
  46. 46. Connecting willing volunteers with clinical trials in their area<br /><ul><li>50% of clinical trial sites that enroll 1 or 0 subjects in their studies
  47. 47. 85% of clinical trials finish late due to recruitment troubles
  48. 48. Estimates hold that fewer than 1 in 10 people with Parkinson’s participate in clinical trials</li></ul>Empowered coordinators<br />Smart matches<br />Active alert system<br />Robust content<br />20<br />
  49. 49. Questions & Answers Session<br /><ul><li>Deborah W. Brooks, The Michael J. Fox Foundation for Parkinson’s Research
  50. 50. Sonal S. Das, PhD The Michael J. Fox Foundation for Parkinson’s Research
  51. 51. Christopher G. Goetz, MD Rush University Medical Center
  52. 52. Jeffrey H. Kordower, PhD Rush University Medical Center
  53. 53. Tanya Simuni, MD, Northwestern University
  54. 54. Glenn T. Stebbins,PhD, Rush University Medical Center</li></ul>21<br />
  55. 55. 22<br />Thank you for your participation!<br />For more information, please visit:<br />www.michaeljfox.org<br />Our 2011 Research Roundtable Series is generously <br />supported through an educational grant from Teva Neuroscience <br />

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