Our Research Strategy
The vision of Cure VCP Disease is to make treatments available to patients affected by a pathogenic variant in the VCP/p97 gene.
Goal: An Ultra Rare Disease Strategy for Treatment Discovery & Clinical Trial Readiness
Our goal is to develop and apply a strategy to understand the gaps, remove the roadblocks, and fulfill the requirements needed for drug discovery and treatment development.
With limited financial resources and a small team, a four-pillar strategy is essential to building a pathway for treatments.
Collected to accelerate drug discovery and research for academia and industry
Why a Toolbox?
To enable drug discovery, our team has assembled a pre-clinical toolbox to provide a standardized workflow and easy access to test molecules in relevant disease models at the in vitro and in vivo levels. Our pre-clinical toolbox may be used for research purposes and commercial purpose entities.
If the tools listed do not meet your current research needs, any researcher may contact Cure VCP Disease directly and our organization may be able to facilitate collaborations.
Learn more about our collection of tools by selecting below:
PATIENT-DERIVED CELLULAR MODELS
Our approach to in vitro models is to make standardized models available to all researchers and host biobanks with patient samples.
Our approach to in vivo models is to evaluate existing VCP models and explore funding and grant opportunities for new models.
The primary assay for VCP is the binding and enzymatic activity of VCP hexamers. The secondary assays are VCP functions at the cellular level.
LIBRARY OF RESEARCH RESOURCES
Reference chemical compounds ready to store in 96 well-plates in-house
Clinical Trial Readiness
We collaborate with partners to power the understanding of the disease's progression, capture the patient's perspective, and establish clinical endpoints.
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Master Protocol in Development:
Design a master protocol for clinical trials with the input of patients and clinicians.
Develop a clinical network in several locations
Establish a relationship with FDA advisors
Longitudinal Natural History Study
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Nationwide Children’s Hospital Longitudinal Study:
40 participants, two years of data collected
Functional measures, including strength & movement, cognitive.tests, and patient reported data
Remote and in-clinic assessments
Progression measured is sufficiently sufficient for endpoints
Emmes Video Scorecard:
17 participants, 3 timepoints
Creating custom VCP Video Scorecard
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Current Biomarker Collaboration:
Analyze patient blood samples (from 0, 12, and 24 months) collected during the Longitudinal Study
Blood assays in UPLC/MS for VCP levels, FGF21, Neurofilament light chain, TDP43P
Potential Biomarker Collaboration:
Gene Editing Therapy
Stress granules assay
GENE EDITING THERAPY
Collaborative research agreement:
A joint effort between Cure VCP Disease and Genethon
PI: Isabelle Richard at Genethon
Our Collaborations Leverage Small Molecules & DNA/RNA Modulations.
Regulatory & Industry
We offer programs to drive awareness and support education for FDA, clinicians, and patients.