Michael J. Fox Foundation Continues to Lead Search for Biomarkers of Parkinson's Disease
“Discovering a definitive biomarker for Parkinson’s disease is critical,” said Sarah Orsay, MJFF’s chief executive officer. “By adding this tool to the Parkinson’s research ‘toolbox,’ we would gain the ability to objectively diagnose PD and to more accurately measure its progression. And we would remove a significant hurdle to effective clinical testing of new therapies, particularly treatments with potential to slow or stop the disease rather than just mask its symptoms.”
The development of neuroprotective therapies is greatly hindered by the lack of markers capable of serving as objective endpoints for clinical trials testing these treatments. In recognition of this issue, Biomarkers 2007 will exclusively accept proposals with a focus on biomarkers that can have significant impact on neuroprotective trials. Among the most significant challenges currently facing Parkinson’s clinicians:
- To measure whether a treatment alters the course or progression of the disease, researchers currently can only measure changes in patients’ clinical features, or the time it takes to reach the onset of specific disease-associated disabilities. Unfortunately, these endpoints leave a great deal to be desired. They may vary drastically between patients or clinical raters; require long trial durations before significant effects are seen; and not be accurate measures of disease progression.
- Clinical measures, though the most important means for determining the ability of a treatment to improve overall patient well-being, generally do not allow researchers to draw a clear distinction between symptom-masking versus disease-modifying (neuroprotective) effects of therapies being tested.
- Current trials must select patients largely based on clinical criteria that may not adequately reflect the highly variant nature of the underlying etiology and pathogenesis of PD. Participation of inappropriate patient subtypes in a trial could result in a seeming lack of effect and ultimately halt further development of an otherwise promising treatment.
- Only limited measures exist in many trials to determine whether a treatment is reaching its hoped-for target site in the brain and exerting its desired biological action. This can hinder ability to determine appropriate therapeutic dosing and lead to trial results (especially if results are negative) that are difficult to interpret.
“Identification of markers that can address the significant limitations of neuroprotective trials, or act as surrogate endpoints for clinical outcomes, would greatly improve our ability to develop and test new disease-modifying therapeutics for PD,” said Gene Johnson, PhD, MJFF’s chief scientific advisor. “For this reason, the Foundation has deliberately focused the current RFA on markers with potential to improve the way neuroprotective and disease-modifying clinical trials are carried out.”
The Michael J. Fox Foundation has been a field leader in spearheading the search for a PD biomarker, with approximately $5 million in biomarker research funded to date.
Pre-proposals under Biomarkers 2007 are required and must be submitted online by Eastern Daylight Time on