Genetic Predictors of Prognosis: GBA and SNCA Meta-Analysis Project
Research Grant, 2014
The research from this grant has continued with the supplementary grant:
- GBA Meta-analysis Initiative: Charting a Path to Success for Clinical Trials in Patients with GBA-PD
Study Rationale: † † † † † † † † ††
Predicting the prognosis of Parkinsonís disease (PD) for a patient is currently not possible. Genetic predictors of prognosis would help inform clinical trial design and, possibly, patient care.
We hypothesize that genetic variants in the glucocerebrosidase (GBA) and the alpha-synuclein (SNCA) genes are clinically relevant, powerful predictors of prognosis in sporadic PD.
Sequence variants in the GBA and SNCA genes will be analyzed using highly multiplexed, next-generation sequencing of 785 patients with PD enrolled in three longitudinal studies. Analysis will be performed for each study separately and across the studies in this international consortium.
Impact on Diagnosis/Treatment of Parkinsonís Disease: † † † † † ††
Prognosis predictors will improve trial design and precision care for patients with PD. They can enable recruiting of trial populations with a more homogeneous disease course thereby increasing power, lowering sample sizes, saving costs and facilitating mechanism-directed therapies.
Next Steps for Development:
Confirmed genetic prognosis predictors will be used to shape future clinical trials for mechanism-directed therapies.
Head of the Neurogenomics Lab and the Parkinson Personalized Medicine Initiative at Harvard and Brigham and Womenís Hospital
Associate Professor of Neurology at Harvard Medical School
Location: Boston, Massachusetts, United States