Consortium, led by
Parkinson's UK and the Critical Path Institute, aggregates and analyzes study
data to develop a model for quantitative characterization of Parkinson's
progression This asset would enrich
the design of trials for novel therapies
Consortium will work with international regulators for acceptance of a quantitative model to streamline review processes
NEW YORK and LONDON and TUCSON,
Ariz.,
May 19, 2016
The Michael J. Fox
Foundation for Parkinson's Research (MJFF), with Parkinson's UK and the
Critical Path Institute (C-Path) in Tucson, Arizona, announce that MJFF has
joined the Critical Path for Parkinson's Consortium (CPP). The consortium aims
to create a quantitative model of Parkinson's progression from its earliest
stages that will allow researchers to optimize clinical trial design for faster
and more effective testing of novel therapies.
Parkinson's UK and C-Path launched the CPP in
October 2015, and seven major pharmaceutical companies, the Parkinson's Disease
Foundation, the Davis Phinney Foundation and Cure Parkinson's Trust have
subsequently signed on to the initiative.
"More therapies with potential to slow or
stop Parkinson's progression in its early stages are moving through clinical
trials," said MJFF Senior Vice President of Research Programs Mark
Frasier, PhD. "A quantitative characterization of that early progression
would help evaluate the impact of these treatments and usher effective
therapies to the people who need them."
Currently clinical trials for potential new
therapies are time-consuming and costly. Due to the variability in Parkinson's
symptoms and progression and the lack of objective biomarkers, trials must
enroll hundreds of patients and follow them for many months if not years.
Development of a quantitative model of Parkinson's progression, potentially
combining both biological and clinical factors, may allow for efficient trials
that aim to treat the right patient with the right drug at the right time.
To create the model, C-Path will standardize
and aggregate data from several large-scale Parkinson's studies — including the
MJFF-led Parkinson's Progression Markers Initiative (PPMI) comprising nearly
1,000 participants, which has made its data available in real time since it
launched in 2010. C-Path will apply modeling and computational tools using the
pooled database to produce the quantitative progression model — which will be submitted to the U.S. Food and Drug
Administration and the European Medicines Agency for feedback — with the ultimate goals of saving time and money and providing a
platform for the best chance of a positive outcome.
In future stages, the consortium plans to
develop a clinical trial simulation platform. Drug developers make educated
guesses on the number of volunteers to enroll, how long to follow them and how
to evaluate therapeutic impact. The simulation platform will inform trial
sponsors of the required parameters when planning a trial and allow them to
enter data on individual participants to determine the number of subjects and
the length of the trial required. For example, with a study population of
"fast progressers" as defined by the CPP progression model, sponsors
may be able to plan a shorter trial. Such a tool would help drug developers
plan efficient studies and avoid false negative results.
"We are very pleased to welcome The
Michael J. Fox Foundation to this important collaboration," said Martha A.
Brumfield, president and CEO of C-Path. "The Foundation's unwavering
commitment to helping patients with PD, their years of experience in
articulating the patient perspective, and their research priorities will enrich
the work of the Critical Path for Parkinson's Consortium."
Arthur Roach, PhD, director of research at
Parkinson's UK, the principal funder of the consortium, says: "Parkinson's
UK is determined to get better treatments to people living with this
devastating condition in years, not decades. A key part of this is joining
forces with regulators, researchers, pharma and medical research charities such
as MJFF to maximize the benefit of data-sharing and learnings. Working with CPP
partners will play a crucial role in helping develop and evaluate the
effectiveness of potential new therapies, which could one day lead us to a cure
for Parkinson's."
The PD data platform will serve as the
resource for advancing the regulatory science goals of CPP, including
qualification of drug development tools, and eventually will be made available
to qualified researchers. Through CPP analyses or independent investigation,
the standardized, aggregated data may allow researchers to identify events that
underlie trajectories of decline and biomarkers or other determinants of early
Parkinson's disease.
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.
About Parkinson's UK
Every hour, someone in the UK is told they
have Parkinson's. It affects 127,000 people in the UK, which is around one in
500 of the population. Parkinson's is a degenerative neurological condition,
for which there currently is no cure. The main symptoms of the condition are
tremor, slowness of movement and rigidity. Parkinson's UK is the UK's leading
charity supporting those with the condition. Its mission is to find a cure and
improve life for everyone affected by Parkinson's through cutting edge
research, information, support and campaigning. For advice, information and
support, visit www.parkinsons.org.uk.
About Critical Path Institute (C-Path)
Established in 2005 as a nonprofit
organization, C-Path was formed to deliver on the vision of FDA's Critical Path
Initiative. C-Path is committed to improving human health and well-being by
developing new technologies and methods to accelerate the development and
review of medical products. An international leader in forming collaborations
around this mission, C-Path has established twelve global, public-private
partnerships that include more than 1,400 scientists from regulatory and other
government agencies, academia, patient advocacy organizations, and 41 major
pharmaceutical companies. C-Path has headquarters in Tucson, AZ, and has been
funded by public and private philanthropic support from the University of
Arizona, Science Foundation Arizona (SFAz), the US Food and Drug Administration
(FDA), and the Tucson community. For more information, visit www.c-path.org.
SOURCE The Michael J. Fox
Foundation; Critical Path Institute; Parkinson's UK
http://www.prnewswire.com/news-releases/the-michael-j-fox-foundation-for-parkinsons-research-joins-multinational-critical-path-for-parkinsons-consortium-300271614.html
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