NEW YORK -- In an editorial recently published in Personalized Medicine, authors from The Michael J. Fox Foundation for Parkinson's Research and J. William Langston, MD, chief scientific officer and founder of the Parkinson's Institute and Clinical Center, outline a strategy to advance personalized medicine for Parkinson's disease (PD). By pivoting from traditional definitions of disease to profile pathology by genetic and molecular changes, neurodegeneration researchers can design and test more targeted therapies with higher likelihood of success.
With advances in the identification and characterization of key Parkinson's genetic targets — alpha-synuclein, LRRK2, glucocerebrosidase — the field is poised for acceleration in this area, similar to the transformation seen in oncology in recent years. However, hurdles to progress remain.
"Truly transforming PD treatment into a precision approach will require tackling key research and regulatory challenges, and the coordinated effort of the entire PD community," the authors write.
The editorial presents a roadmap for overcoming current challenges:
- Support a culture of access to data and biosamples, and build the infrastructure for standardization and cross-cohort analysis
- Create on-ramps to partner with study volunteers through technology, brain donation programs and study results reporting
- Pursue hypothesis-driven and novel discovery paths to generate and link deep molecular profiling and clinical data to build a clearer picture of Parkinson's disease
- Drive biomarker validation to include an array of imaging and biochemical markers to capture the potential biological diversity underlying PD
- Blur the lines between clinical diagnosis to test candidate therapies based on biological dysfunction rather than disease definition
The Michael J. Fox Foundation works in these directions through its support of independent studies and programs and its sponsorship of the landmark biomarker study the Parkinson's Progression Markers Initiative and the online clinical study Fox Insight. In addition, with its newly formed policy arm, the Foundation represents the patient voice to regulators and policymakers.
"With our growing understanding of the varied paths to Parkinson's disease, we see potential routes to personalized medicine for our field," said lead author Todd Sherer, PhD, CEO of The Michael J. Fox Foundation. "Increased attention and collaboration in this area will mean significant advancement in the identification and testing of new treatments to prevent, slow or stop disease."
The full text of the editorial is available online at no fee at http://www.futuremedicine.com/doi/pdf/10.2217/pme-2016-0052 and will publish in the September issue of the journal.