Highlights from the 2026 VCP International Scientific and Community Summit
- Cure VCP Disease, Inc.
- 2 hours ago
- 4 min read
This July, VCP researchers, clinicians, patients, and caregivers from around the world sat in the same room together for the first time, not a scientific meeting on one weekend and a patient conference on another, but one Summit with eleven unique sessions packed across three days in Atlanta.
We're currently putting together the full details on the learnings and insights as a scientific publication. For now, here's a quick look at what happened, day by day.
Day One: The Biology
The VCP Summit opened by digging into the basic science of the VCP protein and relating it to how it causes disease in humans. Researchers walked through the structure of the VCP protein itself and how it burns through ATP to do its job. The expert presenters also delved into the dozens of partner proteins VCP works with, and how those partnerships fall apart when the VCP gene is mutated. One international study alone has pulled together data on 234 people living with VCP-multisystem proteinopathy from 52 centers across 24 countries.
Dr. Michael Benatar closed the day with a keynote on VCP's overlap with ALS, sharing early data suggesting motor neuron involvement may be far more common in VCP multisystem proteinopathy than anyone realized. Dr. Benatar also announced the new multi-site effort to study symptomatic VCP patients over two years as part of the CReATe Consortium. The sites are UCI, Miami, UPenn, UT San Antonio, WashU in St. Louis and Newcastle in the UK.
The day wrapped with a poster session, seventeen presenters in all, covering everything from protein structure to clinical translation. Two posters took home awards. In the Structure and Function category, Daniela Tamayo Jaramillo, PhD, from the University of Utah (Shen lab) won for her work developing a covalent VCP/p97 inhibitor called UTE-156. In Clinical and Translational, Jocelyn Wood, PhD, from Louisiana State University (Johnson lab) won for showing how a protein called SVIP taps into tubular lysosomes to help fix proteostasis problems linked to MSP-1 throughout the body.
Day Two: From the Lab Toward a Treatment
Day two was the day that the Summit's key stakeholders converged: 61 patients and family members with 55 researchers and industry leaders. Talks covered how VCP connects to TDP-43 and tau, the same proteins behind ALS and FTD, and what that overlap could mean for treatment down the road. Researchers shared early data on small molecules built to speed VCP up rather than shut it down, and on antisense oligonucleotides designed to knock down the mutant copy of the gene while leaving the healthy copy alone. Some potential therapies are being tested in cell and animal models, an important step before it is safe to move into clinical trials in humans. A genetic counseling panel walked through what testing and family planning actually look like for people living with VCP-multisystem proteinopathy. The day closed with a candid conversation about funding, open science, and where the community should focus next. One of the highlights of the discussion was the news that the new multi-site natural history study has its first site up and running at the University of Miami with others soon to follow.
Day Three: Breaking It Down with Patients and Researchers
The final day focused on the people living with this disease every day. A "Real Talk with Patients" panel dug into how differently VCP can affect people, even within the same family. Interestingly, having so many diseases caused by the same gene is the reason why there is a shift toward calling VCP disease "multisystem proteinopathy." A live poll of the room ranked “disease awareness” as a surprisingly high priority, a close second to “finding a cure”. Dr. Han Phan, a Cure VCP Disease Board Member, walked people through what it actually takes to get a drug from an idea to a clinical trial: roughly $2.6 billion and three to six years just to clear the animal testing phase. Dr. Phan highlighted that a strong patient registry and community make VCP a more attractive bet for drug companies willing to invest in developing a therapy for our patients. The day closed with a session on everyday healthy choices, like nutrition, sleep, and stress, that patients and families can make right now to improve their quality of life.
At the same time, in a different room, more than 25 researchers joined our invitation-only scientific roundtable to reflect on their key takeaways and brainstorm on ideas to bring effective treatments to patients. Several senior researchers shared that they brought their trainees to the Summit because it was the best place to meet all the key stakeholders while learning highly scientific content. Many researchers reflected on how the interactions with the patient community at the Summit motivated them to continue their VCP research with urgency. An overall feeling was that the field of VCP science was converging with many collaborative opportunities to move the science forward.
What's Next
None of this came together overnight. The Summit was the product of eight years of building a patient registry, investing in key research, collecting biosamples, and cultivating relationships with scientists and families. We look forward to sharing the session videos soon and publishing the full details in a scientific journal. For now, thank you to everyone who traveled to Atlanta, presented, asked hard questions, or just showed up for each other. As one attendee put it that week, “we're the family and friends nobody asked for, and we're awfully glad to have each other.”
