Project Overview

Patients with neurological disease are understandably frustrated by the lack of meaningful pharmacological treatments for even comparatively common pathologies such as Parkinson’s Disease. One of the common complicating factors for these patients are broad disease labels, such as “Multiple Sclerosis”, which encompass several distinct biological entities under a common but imprecise diagnosis. Industry partners have responded to this reality by specifying increasingly complex inclusion criteria for new clinical trials when a patient’s specific profile (often both clinical and genetic) might affect the response to treatment. While this better allows researchers to understand if a therapy is having an effect in the intended population, this has the unintended and unwanted outcome of slowing down recruitment and data collection, and consequently delaying the development of desperately needed therapies.

Responding to these twin challenges, we propose the creation of a novel neuroscience-focussed ‘Virtual Integrated Patient Platform’ (NeuroVIPs), to dramatically expand a registry of genetically and clinically well-characterized patient partners to accelerate recruitment for precision medicine trials. Patient materials will be sent to our bioinformatics core to robustly genotype patient partners, and clinically relevant information shared with project leads in the Clinical Research Unit to improve patient care. While the specific journey of individual patient’s contribution may vary based on pathology, cells will be shared within this integrated platform to generate patient-derived cell lines to allow for in vitro testing of therapeutic and investigational compounds and will also be examined in the microscopy core using cutting edge tools and techniques. The platform has been entirely developed within the Neuro’s Open Science (OS) ecosystem, and all data, derived materials, and cell lines will be shared as openly using well-established procedures for tiered access. Importantly, a novel patient-facing data-portal will be established to provide patient partners insight into how their contributions are accelerating translational research.

Principal Investigator

Guy Rouleau , Montreal Neurological Hospital and Institute

Partners and Donors

Tanenbaum Open Science Institute

The Neuro Philanthropy

Neuro-CERVO Alliance for Drug Discovery in Brain Diseases (NCADD)