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, 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.
About The Michael J. Fox Foundation for
Parkinson's Research
As the world's largest
nonprofit funder of Parkinson's research, The Michael J. Fox Foundation is
dedicated to accelerating a cure for Parkinson's disease and improved therapies
for those living with the condition today. The Foundation pursues its goals
through an aggressively funded, highly targeted research program coupled with
active global engagement of scientists, Parkinson's patients, business leaders,
clinical trial participants, donors and volunteers. In addition to funding
more than $600 million in research to date, the Foundation has fundamentally
altered the trajectory of progress toward a cure. Operating at the hub of worldwide
Parkinson's research, the Foundation forges groundbreaking collaborations with
industry leaders, academic scientists and government research funders;
increases the flow of participants into Parkinson's disease clinical trials
with its online tool, Fox Trial Finder; promotes Parkinson's awareness through
high-profile advocacy, events and outreach; and coordinates the grassroots
involvement of thousands of Team Fox members around the world.
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