EVENT DETAILS

**Postponed Until Further Notice Due to COVID-19**

The VCP Scientific Conference is an international meeting that will take place on September 18-19, 2020 at California Institute of Technology, Pasadena, California. This conference will be the first and only VCP focused meeting that exists in the U.S.

Given the rapid progress in the field, there is an urgent need for researchers to meet, exchange knowledge, share unpublished data and expertise and set the agenda for future research and translational research. The conference will bring lab scientists, clinician scientists and patient representatives together.

One key focus is to attract and retain trainees in the VCP field. The multidisciplinary nature of the conference will significantly impact the innovation and coordination of the research and accelerate translational discoveries in the field to bring about therapeutics in the near future.

A unique feature of this meeting is that we will partner with a patient advocacy organization, Cure VCP Disease, Inc, which is a 501(c)3 non-profit patient advocacy organization. Cure VCP Disease aims to drive efforts to find cures for IBMPFD and ALS. Specifically, the organization will provide much needed patients’ perspectives to researchers in the meeting, and facilitate an ongoing dialog between researchers and patients and their families.

Objectives of the conference:

To provide a forum that facilitates sharing of published and unpublished information on physiological roles of VCP/p97, and on how mutations of VCP lead to diseases;
To bolster understanding of mechanisms involved in VCP/p97-relevant diseases and to enable identification of therapeutics to treat these conditions;
To identify gaps and barriers of further discoveries and translational research in the VCP/p97 field.
To set a concrete basic and translational research agenda for the future studies. This will include crucial discussions on biomarker discoveries and patient longitudinal studies to facilitate near term clinical trials.
To accelerate cross-disciplinary research collaborations amongst VCP/p97 researchers;
To enable attendees to learn about new tools and reagents with potential to facilitate VCP/p97 research;
To assist trainees in propelling their research and to foster mentorship from leaders in the field;
To promote diversity and inclusion of under-represented minorities in VCP/p97 research. Diversity is critically important for strong scientific research.

Location Information

Location Name
Broad Center for the Biological Science, Room 200
Address
360 S Wilson Ave, Pasdena, CA, 91225 US
Directions
Google, Bing, MapQuest

Organizers

TSUI-FEN CHOU, PhD Research Professor in Biology at California Institute of Technology

As a chemist and biochemist, Dr. Chou has developed a library of 200 small molecule inhibitors of VCP/p97 ATPase. Using a key cell-based assays for the activity of ATPase, she identified anti-VCP drugs to treat cancer.

Ming Guo, PhD, MD Professor in Neurology & Pharmacology at UCLA

Dr. Guo's lab was the first to find the role of PINK1. They report that PINK1 and parkin (mutations of them lead to inherited Parkinson’s disease) function in a common pathway to regulate mitochondrial quality and integrity. Her lab also generated VCP disease models and reported that the disease mechanisms of IBMPFD-mutations are hyperactive in nature. Furthermore, 2 VCP-inhibitors suppress pathology in both animal models and patients’ cells. Dr. Guo is also a member of the Cure VCP Disease Medical Advisory board.

Virginia Kimonis, MD, MRCP Professor in the Division of Genetics and Genomic Medicine in the Department of Pediatrics at UC Irvine

Dr. Kimonis identified VCP as the causal gene for inclusion-body myopathy in combination with Paget disease of bone, and frontotemporal dementia. Her lab is currently focused on translational studies in cell and animal models to develop treatments for patients with this devastating disease. Dr. Kimonis is also a member of the Cure VCP Disease Medical Advisory Board.

Conference Schedule

Friday, September 18

9:00 AMWelcome

Remarks from organizers

9:30 AMSession 1 - Introduction to VCP disease

Discussion of clinical symptoms, diagnostic tools and pathological features

10:30 AMSession 2 - New molecular and cellular mechanistic insight of IBMPFD

This session will provide mechanistic insight that we gained over the past few years, including the role of VCP in protein quality control, proteasome degradation, ERAD, endolysomal function, mitochondrial function, autophagy regulation among others.

12:00 PMLunch

Complimentary with attendee registration

1:00 PMSession 3 - New tools and omics studies

This session will provide new tools to facilitate collaborations. Various genomic, proteomics and other omics studies have been performed. Cryo EM studies may yield new insight in structures of VCP. New vertebrate and invertebrate animal models have also been generated.

3:00 PMSession 4 - Trainee talks from the abstracts

We designate a specific session to trainees with a goal to attract and retain them in the VCP field.

5:00 PMPoster Session & Happy Hour

Attendees present posters.

6:30 PMDinner

Complimentary with attendee registration

Saturday, September 19

8:30 AMSession 5 - New inhibitors and small molecules of VCP/p97

With recent findings suggesting that VCP mutations lead to overactivation of the VCP protein, the identification of the new inhibitors and carrying out the mechanistic studies on these inhibitors are crucially important. We thus designate the whole Session addressing this.

9:45 AMSession 6 - Gaps in current research areas and clinical knowledge

This may take a form of discussion to engage all attendees to identify gaps in research and clinical knowledge and guide future research agenda.

11:00 AMBreak

11:15 AMSession 7 - Clinical Trial Readiness

One key outcome of basic research is to drive therapy development as well as critically assess the clinical trial readiness. Specifically, we’d like to address two areas: biomarker discovery and patient longitudinal studies.

12:30 PMWrap-up and final discussions

Create action items and any final discussions.

1:00 PMAdjournment

Speakers

Alyssa Johnson, PhD Assistant Professor, Louisiana State University

Dr. Johnson's lab uses Drosophila as a model system to study the pathogenesis of VCP-related diseases. Recently generated endogenous knock-in models of some of the most common and severe VCP disease-causing mutations and are studying how each mutation affects various cellular processes. We hope that these models will help accelerate new discoveries concerning the pathogenesis of VCP mutations.

Donna Huryn, PhD, ACSF Professor, University of Pittsburgh School of Pharmacy

Dr. Huryn's lab has a strong interest in developing small molecule modulators of Valosin Containing Protein (VCP; also known as p97) as potential therapeutics, but also as tool molecules to help understand VCP biology. Co-author on the first high resolution cryo-EM co-structure that shows small molecules bound to VCP, and have published a number of papers describing inhibitors of this fascinating protein.

Hemmo Meyer Professor, Department Molekularbiologie, Universität Duisburg-Essen

Dr. Meyer's group has made major contributions to understanding VCP cellar function and biochemical mechanism, and how both are affected by disease-associated mutations in VCP. They are now eager to help translate this knowledge into a cure for the patients.

Tahseen Mozaffar, MD, FAAN Director, UC Irvine Neuromuscular Program, University of California, Irvine

Dr. Mozaffar is currently working with a group of investigators at UC Irvine on putting together a UO1 grant aiming to examine the natural history in VCP-related Multisystem Proteinopathy and establish peripheral biomarkers that can be used to follow disease course and treatment related outcomes.

Gerald Pfeffer, MD, PhD Assistant Professor, University of Calgary

Dr. Pfeffer's research focuses on genetic neuromuscular diseases, and a major project of his lab focuses on the role of genetic modifiers as a possible explanation for the wide range of disease phenotypes which can be caused by VCP mutations. As a new investigator, his ability to build collaborations and develop new initiatives is greatly enhanced by the availability of subspecialty conferences, which bring together fellow researchers from around the world.

Malavika Raman, PhD Assistant Professor, Department of Developmental, Molecular and Chemical Biology, Tufts University School of Medicine

Dr. Raman's lab is dedicated to understanding the multiple roles of VCP in regulating protein quality control within the cell. They aim to develop a mechanistic understanding of how VCP regulates protein triage and uncover new and uncharacterized roles for this protein within the cell. The goal of their work is to determine how normal VCP function is perturbed by the disease mutation and identify key functions of VCP that are most impacted in VCP disease.

Chris Weihl, MD, PhD Professor of Neurology, Washington University School of Medicine in St. Louis

Dr. Weihl has studied the role of VCP disease mutations in the context of human disease for the past decade. His group has made sentinel observations that have helped define the pathogenesis. Dr. Weihl is also a member of the Cure VCP Disease Medical Advisory Board.

Lodging Details

Hilton Pasadena

168 S Los Robles Ave, Pasadena, CA 91101 Phone Number: 626-584-3204

Conference Rate: $181.00 per night

Booking link: https://www.hilton.com/en/hi/groups/personalized/P/PASPHHF-CURE6-20200917/index.jhtml?WT.mc_id=POG

Registration

Registration Options

Attendee - $100.00
Member of research or scientific community
Trainee - $50.00
Current student or post-doc

Registration includes lunch and dinner on Friday, September 19.

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2020 VCP SCIENTIFIC CONFERENCE

Postponed Until Further Notice Due to COVID-19
PASADENA, CA

ACCELERATING COLLABORATION AND PROGRESS IN VCP / p97 RESEARCH

Countdown:

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EVENT DETAILS

Location Information

Organizers

TSUI-FEN CHOU, PhD Research Professor in Biology at California Institute of Technology

Ming Guo, PhD, MD Professor in Neurology & Pharmacology at UCLA

Virginia Kimonis, MD, MRCP Professor in the Division of Genetics and Genomic Medicine in the Department of Pediatrics at UC Irvine

Conference Schedule

Friday, September 18

Saturday, September 19

Speakers

Alyssa Johnson, PhD Assistant Professor, Louisiana State University

Donna Huryn, PhD, ACSF Professor, University of Pittsburgh School of Pharmacy

Hemmo Meyer Professor, Department Molekularbiologie, Universität Duisburg-Essen

Tahseen Mozaffar, MD, FAAN Director, UC Irvine Neuromuscular Program, University of California, Irvine

Gerald Pfeffer, MD, PhD Assistant Professor, University of Calgary

Malavika Raman, PhD Assistant Professor, Department of Developmental, Molecular and Chemical Biology, Tufts University School of Medicine

Chris Weihl, MD, PhD Professor of Neurology, Washington University School of Medicine in St. Louis

Lodging Details

Hilton Pasadena

Registration

Billing Information

2020 VCP SCIENTIFIC CONFERENCE

**Postponed Until Further Notice Due to COVID-19**PASADENA, CA

ACCELERATING COLLABORATION AND PROGRESS IN VCP / p97 RESEARCH

Countdown:

NaN days, NaN hours, NaN minutes, NaN seconds

EVENT DETAILS

Location Information

Organizers

TSUI-FEN CHOU, PhD Research Professor in Biology at California Institute of Technology

Ming Guo, PhD, MD Professor in Neurology & Pharmacology at UCLA

Virginia Kimonis, MD, MRCP Professor in the Division of Genetics and Genomic Medicine in the Department of Pediatrics at UC Irvine

Conference Schedule

Friday, September 18

Saturday, September 19

Speakers

Alyssa Johnson, PhD Assistant Professor, Louisiana State University

Donna Huryn, PhD, ACSF Professor, University of Pittsburgh School of Pharmacy

Hemmo Meyer Professor, Department Molekularbiologie, Universität Duisburg-Essen

Tahseen Mozaffar, MD, FAAN Director, UC Irvine Neuromuscular Program, University of California, Irvine

Gerald Pfeffer, MD, PhD Assistant Professor, University of Calgary

Malavika Raman, PhD Assistant Professor, Department of Developmental, Molecular and Chemical Biology, Tufts University School of Medicine

Chris Weihl, MD, PhD Professor of Neurology, Washington University School of Medicine in St. Louis

Lodging Details

Hilton Pasadena

Registration

Billing Information

Postponed Until Further Notice Due to COVID-19
PASADENA, CA