Parkinson's Disease affects 1-2% of the population over 65 years of age and increases to 3-5% in people 85 years and older. The FDA approval of Sinemet® (carbidopa/leovdopa) in 1988 revolutionized the treatment of Parkinson’s Disease. Over 20 years later, 15 new PD drugs (dopamine agonists, COMT inhibitors, MAO-B inhibitors) have been brought to market in the United States. With the exception of amantadine, all 15 medications treat Parkinson's in a similar way — adjusting for loss of dopamine in the brain by either increasing available dopamine or directly stimulating CNS dopamine receptors. The discovery of major breakthroughs in either neuroprotective or neurorestorative approaches to PD therapy remain elusive and are in part inhibited by the lack of progression biomarkers that can be successfully utilized in Phase II clinical trials. The Alzheimer’s Disease Neuroimaging Initiative (ADNI) has demonstrated the need to standardize clinical information and biospecimen collection for the successful identification and validation of biomarkers for Alzheimer’s disease. Certainly, the lack of standardization in biospecimen collection has impacted the ability to validate newly identified biomarkers for PD, as well as the availability and application of biomarkers for PD clinical trials. Strategic planning for PD biomarker development will enable NINDS in collaboration with the PD research community to: 1) develop guidelines for biospecimen collection and storage, that can be applied across studies; 2) identify current roadblocks to PD biomarker development; 3) identify metrics for moving from discovery to validation; and 4) identify key tools and resources needed for PD biomarker discovery and validation.
The format of the workshop included: 1) keynote sessions on the process of biomarker development – NCI, FDA and industry perspectives; 2) industry and clinical research perspectives concerning obstacles in the path to PD drug development; 3) analysis of clinical non-motor biomarkers for PD disease diagnosis and/or progression; 4) analysis of neuroimaging modalities for development of PD diagnosis and/or progression biomarkers; and 5) analysis of PD cohorts currently funded through NINDS or through Foundation support.
Margaret Sutherland, Ph.D.
National Institute of Neurological Disorders and Stroke
Phone: (301) 496-5680
Last updated December 23, 2013