Critical Path for Parkinson’s

Overview

The Problem

Parkinson’s affects over 10 million individuals worldwide, but there is currently no cure and only limited treatments to manage its progression, which impacts both motor and nonmotor functions. This wide range of manifestations makes it difficult to both fully capture the real-life experiences of individuals living with Parkinson’s and to measure the efficacy of drugs in clinical trials in this heterogeneous population.

Existing approaches and measures used to evaluate Parkinson’s fail to capture the aspects of the disease that hold genuine significance for people living with Parkinson’s and their care partners. Thus, there is an urgent need to redefine and expand our understanding of the early stages of Parkinson’s.

The Solution

The focus on the impact of early-stage Parkinson’s Disease (PD) will enable us to reach a greater number of individuals affected by the disease and effectively address their specific needs, ensuring improved outcomes and a better quality of life.

The goal of the CPP Consortium is to broadly advance the drug development landscape for Parkinson’s prevention and treatment by working collaboratively with the Parkinson’s Disease global community to share data, knowledge, and resources towards the development and regulatory endorsement of novel drug development tools.

We do this by prioritizing efforts focused on broadening populations for trial recruitment, collecting, understanding, and sharing real-world data, and by leveraging innovative technologies to capture what truly matters.

The Impact

With a focus on the “right drug, right patient, right time” approach, CPP streamlines regulatory processes to optimize drug development with greater efficiency in advancing data-driven solutions for clinical trials. CPP aims to align with regulatory strategies spanning from model-informed drug development, patient-focused drug development, digital heath technologies, biomarkers, and real-world data.

Regulatory Endorsement

Accelerating effective treatments for Parkinson’s by engaging with regulators to foster advancements in research for expanded treatment options.

Integrated Parkinson's Database

Empowering researchers with comprehensive patient-level data for enhanced Parkinson’s research and optimized clinical trial design.

Shaping the Parkinson's Landscape

Pioneering accurate diagnosis, personalized treatments, and patient-centric advancements for improved outcomes in Parkinson’s care.

Leading Digital Health Innovations

Optimizing device-agnostic Digital Health Technologies for Parkinson’s research, empowering drug development, gaining valuable insights.

Integrated Parkinson’s Database

The database includes integrated and standardized data from both PD observational studies and randomized clinical trials and contains thousands of participants’ data, anonymized and deidentified. The database covers over 600 variables, such as demographic information, clinical measures, medication usage data, dropouts, and more.

The CPP integrated database is rich in patient-level outcome measures and includes item level patient level data. It contains limited biomarker and genetics data and does not include data collected using digital health technologies. In accordance with the Data Contribution Agreements between CPP and data contributors, CPP is obligated to protect the identity of the individual datasets and provides access to the datasets in aggregate with masked study identifiers.

To read more about the Integrated Parkinson’s Database, including FAQs, click here.

We would like to thank our collaborators for their generous contributions to the CPP integrated Parkinson’s Database. View acknowledgements.

Early Motor Parkinson’s DAT Clinical Trial Enrichment Tool

This quantitative clinical trial enrichment tool helps optimize clinical trial design in the early-motor stages of Parkinson’s, using MDS-UPDRS part III as the primary endpoint. For scientists and clinicians of all backgrounds, a cloud-based graphical user interface (GUI) has been developed, which allows a user-friendly experience to perform simulations based on the model.

Regulatory Successes

Why is it important to achieve regulatory success?

Regulatory endorsement relieves trial sponsors of the burden of having to convince the regulators that methods are reliable and reproducible and could accelerate effective patient treatments. Tools endorsed by regulators save time and money, which incentivizes more trials, which results in increased chances of getting more Parkinson’s disease treatments approved.

Impact Videos

"Changing the face of clinical trials for Parkinson’s,” Florence Pite Memorial Lecture, London, November 15, 2017

CPP’s primary funder, Parkinson’s UK, outlines strategy to help us find better treatments for Parkinson’s in years not decades

This video from the Michael J. Fox Foundation explains the process of drug development

This C-Path video uses the analogy of NASA’s space program to explain the benefits of clinical trial simulation tools

Parkinson’s patient and longtime C-Path supporter Carl Ames describes Parkinson’s disease from the patient’s perspective

Collaborators

Founding Partners

• Critical Path Institute
• Parkinson’s UK
  • C-Path would like to thank and acknowledge Parkinson’s UK’s generous support in founding the consortium in 2015.

Critical Path for Parkinson’s Members

• AbbVie
• Acurex Biosciences
• Annovis Bio
• Biogen
• Biohaven
• CLARIO
• F. Hoffmann-LaRoche Ltd.
• IXICO
• Merck Sharp and Dohme (known as Merck & Co., Inc. in the United States and Canada)
• Neumora Therapeutics Inc.
• Neuron23
• Novartis
• Sanofi Genzyme
• Takeda Pharmaceuticals
• UCB
• Vanqua Bio

Academic Institutions

• Newcastle University
• Radboud University
• University of Cambridge
• University of Glasgow
• University of Oxford
• University of Plymouth

Nonprofit Organizations

• Cure Parkinson’s
• Davis Phinney Foundation
• Movement Disorders Society
• The Michael J. Fox Foundation for Parkinson’s Research
• Parkinson’s Canada
• Parkinson’s Foundation
• Parkinson & Movement Disorder Alliance
• Parkinson’s UK
• Parkinson’s Europe

Government and Regulatory Agencies

• European Medicines Agency (EMA)
• National Institute of Neurologic Diseases and Stroke (NINDS)
• U.S. Food and Drug Administration (FDA)

 

Collaborators

• Karim Azer, PhD, Critical Path Advisor, Quantitative Systems Pharmacology and Systems Biology
• Bastiaan Bloem, MD., Ph.D. Professor of Neurology, Center for Parkinsons, Radboud University
• Peter Bloomingdale, PhD – Critical Path Advisor, Modeling & Simulation and QSP
• Jesse Cedarbaum, MD. FAAN Neurology, Founder & Head, Coeruleus Clinical Sciences, LLC
• Ray Dorsey, MD., MBA. Professor, Department of Neurology, University of Rochester
• Sue Dubman, Director, IT & Informatics, UCSF
• Mike Edwards, Ph.D. Professor and Area Head, Quantitative Psychology, Arizona State University
• Derek Hill, Ph.D. President, Panoramic Digital Health
• Karl Kieburtz, MD, MPH. Professor, Department of Neurology, University of Rochester
• Sheng Luo, Ph.D. Associate Professor in Biostatistics, Duke University
• Eric Macklin, Ph.D. Assistant in Biostatistics, Massachusetts General Hospital
• Walter Maetzler, MD. Professor, Department of Neurology, University of Kiel
• Ken Marek, MD. Senior Scientist, Institute of Neurodegenerative Diseases
• Brit Mollenhauer, MD. Associate Professor, University of Gottingen Medical Center
• George Roussos, Ph.D. Professor of Pervasive Computing, Birkbeck, University of London
• David Russell, MD. Director, Clinical Research, Institute of Neurodegenerative Diseases
• Michael Schwarzschild, MD, Ph.D. Director, Molecular Neurobiology, Massachusetts General Hospital
• John Seibyl, MD. Executive Director & Senior Scientist, Institute of Neurodegenerative Diseases
• Ira Shoulson, MD. Professor of Neurology, University of Rochester
• Tanya Simuni, MD. Professor of Neurology (Movement Disorders), Northwestern University
• Glenn Stebbins, Ph.D. Professor, Department of Neurological Sciences, Rush University
• Charles Venuto, PharmD. Associate Professor, Department of Neurology, University of Rochester
• Daniel Weintraub, MD. Professor of Psychiatry, University of Pennsylvania

3DT Collaborators

• Jamie Adams, MD. Assistant Professor, Department of Neurology (Movement Disorders), University of Rochester
• Bastiaan Bloem, MD., Ph.D. Professor of Neurology, Center for Parkinsons, Radboud University
• Camille Carroll, Ph.D. Associate Professor of Neurology, University of Plymouth
• Jesse Cedarbaum, MD. FAAN Neurology, Founder & Head, Coeruleus Clinical Sciences, LLC.
• Derek Hill, MD. President, Panoramic Digital Health
• Melissa Kostrzebski, Senior Project Manager/Clinical Trials, University of Rochester
• Jennifer Mammen, Ph.D. APRN-CNP., Assistant Professor, College of Nursing, The University of Rhode Island
• Anat Mirelman, Ph.D. Director, Laboratory for Early Markers of Neurodegeneration, Tel Aviv Medical Center
• George Roussos, Ph.D. Professor of Pervasive Computing, Birkbeck, University of London
• Lisa Shipley, PhD, Shipley Pharma Insights
• Tanya Simuni, MD. Professor of Neurology (Movement Disorders), Northwestern University
• Glenn Stebbins, Ph.D. Professor, Department of Neurological Sciences, Rush University

Team

For more information on the Critical Path for Parkinson’s (CPP) Consortium, please contact CPP Executive Director Dr. Diane Stephenson at DStephenson@c-path.org.