Evidence map›Paper›PMID 40037468›Full record

ReviewNeurobiology of disease2025

2024 VCP International Conference: Exploring multi-disciplinary approaches from basic science of valosin containing protein, an AAA+ ATPase protein, to the therapeutic advancement for VCP-associated multisystem proteinopathy.

A Peck, A Dadi, Z Yavarow, L N Alfano, D Anderson, M R Arkin, T F Chou, E S D'Ambrosio, J Diaz-Manera, J P Dudley and 31 more

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Neurobiology of disease, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

4 citing papers in PubMed.

  1. Article
  2. Review
  3. Review
  4. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

41 authors.

A PeckCure VCP Disease, Warner Robins, GA, USA.
A DadiCure VCP Disease, Warner Robins, GA, USA.
Z YavarowCure VCP Disease, Warner Robins, GA, USA.
L N AlfanoThe Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
D AndersonEikon Therapeutics, Hayward, CA, USA.
M R ArkinDepartment of Pharmaceutical Chemistry, University of California, San Francisco, San Francisco, CA, USA.
T F ChouDepartment of Biology, Caltech, Pasadena, CA, USA.
E S D'AmbrosioNationwide Children's Hospital, Columbus, OH, USA; Department of Genetic and Cellular Medicine and Department of Neurology, UMass Chan Medical School, Worcester, MA, USA.
J Diaz-ManeraThe John Walton Muscular Dystrophy Research Centre, Newcastle University Translational and Clinical Research Institute and Newcastle Hospitals NHS Foundation Trust, Center for Life, Central Parkway, Newcastle, Upon Tyne, United Kingdom.
J P DudleyLaMontagne Center for Infectious Disease, University of Texas, Austin, TX, USA.
A G ElderCure VCP Disease, Warner Robins, GA, USA.
N GhoshalDepartments of Neurology and Psychiatry, Washington University School of Medicine, St. Louis, MO, USA.
C E HartCreyon Bio, San Diego, CA, USA; Lilly, Indianapolis, IN, USA.
M M HartCure VCP Disease, Warner Robins, GA, USA.
D M HurynDepartment of Chemistry University of Pennsylvania, Philadelphia, PA, USA.
A E JohnsonDepartment of Biological Sciences, Louisiana State University, Baton Rouge, LA, USA.
K B JonesDepartment of Orthopaedics, Huntsman Cancer Institute, University of Utah School of Medicine, Salt Lake City, UT, USA.
V KimonisDepartment of Pediatrics, University of California, Irvine, CA, USA; Department of Neurology, University of California, Irvine, CA, USA.
E KiskinisThe Ken & Ruth Davee Department of Neurology, Department of Neuroscience, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.
E B LeeDepartment of Pathology and Laboratory Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.
T E LloydDepartment of Neurology Baylor College of Medicine, Houston, TX, USA.
M MapstoneDepartment of Neurology, University of California, Irvine, CA, USA.
A MartinHoward Hughes Medical Institute, University of California, Berkeley, CA, USA.
H MeyerCenter of Medical Biotechnology, Faculty of Biology, University of Duisburg-Essen, Essen, Germany.
T MozaffarDepartment of Neurology, University of California, Irvine, CA, USA.
C U OnyikeDivision of Geriatric Psychiatry and Neuropsychiatry, Johns Hopkins University School of Medicine, Baltimore, USA; Department of Psychiatry and Behavioral Sciences, Johns Hopkins University School of Medicine, Baltimore, USA.
G PfefferDepartment of Clinical Neurosciences, University of Calgary, Calgary, Canada; Department of Medical Genetics, Cumming School of Medicine, University of Calgary, Calgary, Canada.
A PindonCure VCP Disease, Warner Robins, GA, USA; Myhre Syndrome Foundation, Richardson, TX, USA.
M RamanDepartment of Developmental Molecular and Chemical Biology, Tufts University School of Medicine, Boston, MA, USA.
I RichardGénéthon, 91000 Evry, France; Université Paris-Saclay, Université Evry, Inserm, Généthon, Integrare research unit UMR_S951, 91000 Evry, France.
D C RubinszteinCambridge Institute for Medical Research, University of Cambridge, CB2 0XY Cambridge, UK; UK Dementia Research Institute, Cambridge Biomedical Campus, Cambridge, UK.
M SchiavaThe John Walton Muscular Dystrophy Research Centre, Newcastle University Translational and Clinical Research Institute and Newcastle Hospitals NHS Foundation Trust, Center for Life, Central Parkway, Newcastle, Upon Tyne, United Kingdom.
A K SchützDepartment of Chemistry and Pharmacy, Ludwig-Maximilians-Universität Munich, Munich, Germany.
P S ShenDepartment of Biochemistry, University of Utah, Salt Lake City, UT, USA.
D R SouthworthInstitute for Neurodegenerative Diseases, University of California, San Francisco, CA, USA.
A M StaffaroniUCSF Memory and Aging Center University of California San Francisco, CA, USA.
M Taralio-GravovacDepartment of Biochemistry & Molecular Biology, University of Calgary, Alberta, Calgary, Canada.
C C WeihlDepartment of Neurology, Washington University School of Medicine, St. Louis, MO, USA.
Q YaoDepartment of Medicine Stony Brook University, Renaissance School of Medicine, Stony Brook, NY, USA.
Y YeLaboratory of Molecular Biology, NIH, NIDDK, Bethesda, MD, USA.
N PeckCure VCP Disease, Warner Robins, GA, USA. Electronic address: nathan@curevcp.org.

Funding

“Regulation of Protein Quality Control by the VCP AAA-ATPase”R01GM127557 · NIGMS · TUFTS UNIVERSITY BOSTON · PI Malavika Raman · 2018 to 2026
$3.5M
Visualizing the Mechanisms of Protein Quality ControlR35GM133772 · NIGMS · UTAH STATE HIGHER EDUCATION SYSTEM--UNIVERSITY OF UTAH · PI Peter Shen · 2019 to 2026
$3.4M
VCP in ASPSCR1::TFE3-driven oncogenic transcription reprogrammingR01CA293084 · NCI · UTAH STATE HIGHER EDUCATION SYSTEM--UNIVERSITY OF UTAH · PI Kevin Bruce Jones, Peter Shen · 2024 to 2026
$1.9M
Investigating the impact of p97 mutation in Amyotrophic Lateral SclerosisR21NS123631 · NINDS · TUFTS UNIVERSITY BOSTON · PI RAMAN, MALAVIKA · 2022 to 2023
$454k
Intramural NIH HHS Z99 DK999999NCI NIH HHS R01 CA293084NIGMS NIH HHS R01 GM127557NIGMS NIH HHS R35 GM133772NINDS NIH HHS R21 NS123631
6 · The paper itself

Abstract

Valosin-containing protein (VCP/p97) is a ubiquitously expressed AAA+ ATPase associated with numerous protein-protein interactions and critical cellular functions including protein degradation and clearance, mitochondrial homeostasis, DNA repair and replication, cell cycle regulation, endoplasmic reticulum-associated degradation, and lysosomal functions including autophagy and apoptosis. Autosomal-dominant missense mutations in the VCP gene may result in VCP-associated multisystem proteinopathy (VCP-MSP), a rare degenerative disorder linked to heterogeneous phenotypes including inclusion body myopathy (IBM) with Paget's disease of bone (PDB) and frontotemporal dementia (FTD) or IBMPFD, amyotrophic lateral sclerosis (ALS), Alzheimer's disease (AD), parkinsonism, Charcot-Marie Tooth disease (CMT), and spastic paraplegia. The complexity of VCP-MSP makes collaboration among stakeholders essential and necessitates a multi-disciplinary approach. The 2024 VCP International Conference was hosted at Caltech between February 22 and 25. Co-organized by Cure VCP Disease and Dr. Tsui-Fen Chou, the meeting aimed to center the patient as a research partner, harmonize diverse stakeholder engagement, and bridge the gap between basic and clinical neuroscience as it relates to VCP-MSP. Over 100 multi-disciplinary experts attended, ranging from basic scientists to clinicians to patient advocates. Attendees discussed genetics and clinical presentation, cellular and molecular mechanisms underlying disease, therapeutic approaches, and strategies for future VCP research. The conference included three roundtable discussions, 29 scientific presentations, 32 scientific posters, nine patient and caregiver posters, and a closing discussion forum. The following conference proceedings summarize these sessions, highlighting both the identified gaps in knowledge and the significant strides made towards understanding and treating VCP diseases.

Indexed as

Valosin Containing ProteinAnimalsCongresses as TopicFrontotemporal DementiaHumansMyositis, Inclusion BodyOsteitis DeformansValosin Containing ProteinVCP protein, humanATPaseInclusion body myopathy associated with Paget's disease of bone and frontotemporal dementia (IBMPFD)Motor neuron diseaseMultisystem proteinopathyValosin-containing protein (VCP)VCP-MSPVCP/p97

Identifiers

PMID40037468
PMCPMC11960434

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