Member
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Join Date: Feb 2009
Posts: 312
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Member
Join Date: Feb 2009
Posts: 312
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inadequate to say the least!
We are well aware (and so are clinical researchers) that our current batteries of "measurements" are woefully inadequate. Efforts to refine these scales over the years (including the newest UPDRS) reflect greater appreciation for a broader array of symptoms at play in PD. And we are certainly acknowledging that this "movement disorder" is much more than a movement disorder.
We are utterly convinced that we need more informative markers (of diagnosis, of progression, of therapeutic reach)...all things that MJFF has been funding since 2002. The biology is really hard; the heterogeneity of the disease is confounding. But, we do have some good leads ($30 million later!) and this has led to the new biomarker initiative, PPMI, a $40 million initiative of it's own, to see if we can validate these possible markers.
I think many of you will appreciate what is going into the study (of 400 de novo patients and 200 matched controls). The study will document extensive clinical (everything from UPDRS, to depression, to cognition, to sense of smell) and biologic (DNA, blood, urine and spinal fluid) and neuroimaging. Some of this data will be pre-meds and some will be after meds. This will produce the most comprehensive observations of "early PD" and provide a rich data set in which to (hopefully) identify correlations and verify much more exact "measures" of PD---essential for clinical trials.
These "measures" become the "endpoints" that are evaluated in clinical trials--without improved tools, it's likely that trials may just continue on with mixed results and ultimately bring hope for progress to its knees. Can you imagine how transformative it would be if a brain scan, lumbar puncture and blood test could, with confidence, tell you what "stage" of disease you are in and how fast/slow you are progressing? It would not only improve information for patients/docs in disease management, but it would reinvigorate clinical trial investment on PD.
We are excited.
Debi
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