VCP Research Programs
The research tools and programs of Cure VCP Disease are centered around the four pillars of our research strategy and our vision of making treatments available to patients affected by a pathogenic variant in the VCP/p97 gene.
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.
Master Protocol
- Learn More -
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
- Learn More -
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
-
Assessments complete
-
Creating custom VCP Video Scorecard
Biomarkers
- Learn More -
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:
-
Proteomics
-
Transcriptomics
Gene Editing Therapy
Small Molecules
SMALL MOLECULES
​
NCATS Collaboration on phenotypic screening using muscle organoids
​
Industry Interactions
-
VCP modulation
-
TDP43 aggregation
-
Stress granules assay
GENE EDITING THERAPY
Prime gene editing with Genethon:
-
A joint effort between Cure VCP Disease and Genethon
-
PI: Isabelle Richard at Genethon
Point gene editing with Nationwide:​
-
Young Investigator Eleonora D’Ambrosio
Treatment Discovery
Our collaborations leverage small molecules & DNA/RNA modulations.
Regulatory & Industry
We offer programs to drive awareness and support education for FDA, clinicians, and patients.
Engage the Scientific Community
2024 International Scientific Conference (Feb 22-25)
•Cross-disease integrated approach for VCP application, including Oncology, Virology, and Neuromuscular Disorders
•Presentations, roundtables and posters
Scientific Focus Group
70+ Scientists and Clinicians
•Monthly virtual meeting
•Active presentations, brainstorming
•Fosters new collaborations in basic and clinical science
​
Engage the Patient Community
2020 FDA Listening Session
Monthly virtual convening for patient peer-to-peer support and networking
Monthly scientific office hours
with the Chief Scientific Officer to increase patient and caregiver understanding
Annual in-person patient and caregiver conference
The third Connections Conference was held July 21-22, 2023
Align Clinical Applications & Endpoints
Patient registry:
100+ participants from Australia, Brazil, Germany, New Zealand, Taiwan, Thailand, United Kingdom, USA
Patient preference survey :
To understand patient priorities and perspectives
Three unique patient panel/workshops:
Focused on patient-focused drug discovery
​
​
