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Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine Proceedings of a Workshop (2018) / Chapter Skim
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7 Patient Perspectives
Pages 82-86

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From page 82...
... T  his chapter summarizes the perspectives of representatives from patient advocacy organizations. PERSONALIZED MEDICINE COALITION Daryl Pritchard, senior vice president for science policy at the Personalized Medicine Coalition, invited workshop participants to change gears and focus on "the most important stakeholder in this process -- the patient." Pritchard started by drawing an analogy between precision medicine and modern aviation.
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From page 83...
... In 2005, there were 14 personalized medicines on the market, and today there are more than 160, with many more in development. Pritchard reported on a study commissioned by the Personalized Medicine Coalition with Tufts Medical Center that showed that 42% of all the drugs in the pharmaceutical pipeline in 2015 were personalized medicines, and a full 73% of oncology drugs in development were personalized (Tufts Center for the Study of Drug Development, 2015)
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From page 84...
... , said Brian Fiske, senior vice president for research programs, is dedicated to finding a cure for Parkinson's disease through an aggressively funded research agenda, and to ensuring the development of improved therapies for those living with Parkinson's today. MJFF has invested nearly $750 million in research programs to date, funding both domestic and international projects in both the non-profit and for-profit sectors.
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From page 85...
... Fiske noted that the description and classification of Parkinson's has not changed significantly since it was first identified 200 years ago. The disease is still largely diagnosed on the basis of motor symptoms, such as bradykinesia, rigidity, resting tremors, and postural instability, despite the fact that there are a number of non-motor symptoms associated with Parkinson's, such as cognitive impairment, mood disorders, autonomic dysfunction, and REM sleep behavior disorder.
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From page 86...
... A number of animal models have been developed to study Parkinson's, but to date none are considered predictive of the human condition, said Fiske. These models are divided into two categories: those that model environmental triggers, such as neurotoxins or inflammation, and those that model genetic triggers using strategies such as knockouts or transgenics.
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Key Terms

  • precision medicine
  • medicine coalition
  • personalized medicines
  • modern aviation
  • testing products

  • clinical differences
  • genetic testing
  • postural instability
  • disease tend
  • particularly germane

  • inform modeling
  • date none
  • studies feed
  • including mitochondrial
  • gis map

  • clinical description
  • environmental triggers
  • expanded dramatically
  • animal modeling
  • ideal target

  • model genetic
  • particularly stable
  • profound effect
  • tufts center
  • motor symptoms

  • major question
  • vitro research
  • debate remains
  • predictive validity
  • marker initiative


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