Powering Collaboration and Connections at Cure VCP Disease
- Cure VCP Disease, Inc.
- May 9
- 4 min read
Zollie Yavarow, PhD, MA, is the Director of Scientific Engagement and Collaborations at Cure VCP Disease. Because of your support, she is building relationships and making connections that push research forward.
My I CAN challenge is "I CAN accelerate VCP Science." I've learned that it takes a team … a very big team. At Cure VCP Disease, my role is a bit like being a specialized search engine, but for people, ideas, research tools, and ongoing projects. I serve as a liaison between key stakeholders across the VCP research landscape, helping connect the dots that turn isolated efforts into powerful collaborations.
In seven years, our team has built the infrastructure to enable progress on a global scale. We have made tremendous strides and accomplished great milestones:
Launched a patient registry, enrolling 120+ participants from 10 countries.
Hosted three patient conferences and two international scientific conferences, connecting over 70 patient families and 200 researchers.
Co-designed, funded, and published a two year clinical study with Nationwide Children’s Hospital to study disease progression and enable future clinical trials in our disease space.
Led a global consortium of 50 clinicians with 10 working groups to establish clinical care guidelines, aimed at reducing time to diagnosis and improving clinical care.
Created 12 scientific cell models that have powered a drug-screening project and gene therapy projects for VCP disease, while owning the IP.
Founded a patient biobank, including over 600 samples from 40 patients and family member controls
As the Director of Scientific Engagement and Collaborations, it is exciting to sit at the intersection of science, strategy, and stewardship for VCP disease. I help guide our research priorities and ensure that every scientific project we support—every donation, every cell line, every vial of blood, every hour of patient time—moves us meaningfully closer to improving patients’ lives. Here are a few of the lessons that I have learned about the importance of relationships in accelerating science:
Sharing resources can accelerate science. In the world of rare disease research, resources are precious, especially those donated by our patient community. So, when I know one lab is running a protein screen on muscle tissue and hear another is looking for samples for a similar study, I connect them. That one connection means we conserve samples, expand the scope of the experiment, stretch research budgets further, and most importantly, bring together brilliant minds to answer more questions.
Connections don’t happen by accident. They come from knowing the people and building trust with them. They come from intentionally cultivating the relationship— asking how their kids are doing, checking in on their favorite hobby, and asking if their mother is doing better home from the hospital (all real examples!). It's more than how many patients they see in the clinic and the next publication they're working on. That’s why I attend conferences, why I will drive an hour to share a meal in person, and why I facilitate regular check-ins with our research network. These sorts of efforts mean that when I reach out to the very busy head of a medical department about a new connection, they reply.
The VCP Research Network is growing because it has become a community. We have been hosting a monthly VCP Scientific Focus Group for nearly five years. It started with just 15 scientists presenting their latest research. Now, our research network includes over 30 institutes from seven countries. We have found that not only does the VCP Scientific Focus Group give emerging scientists a platform to elevate their work, it brings together scientists, clinicians, and advocates for open discussion. These meetings promote collaboration, generate feedback, and have led to new efforts, including a multi-PI grant working group and expanded use of Cure VCP Disease’s new cell lines.
Connections and collaborations can produce very meaningful scientific progress. Our 2024 International Scientific Conference in California brought the entire VCP community into the same room for the first time. By bringing together the various stakeholders and encouraging dialogue, we created a paradigm shift that changed the field’s focus toward VCP’s cellular localization and protein interactions. Young scientists were able to sit next to seasoned experts from different institutes and learn from each other. More importantly, scientists were able to sit with patients and learn from their lived experience—adding an urgency and passion to their work.
Designing with intention allows breakthroughs. Having a biobank is essential to enable research, but we must also intentionally design it so it can be utilized to answer our top priority research questions. That means ensuring researchers can easily access samples and link them to essential phenotypic data. It also means focusing on collecting samples from the same patients over time, allowing researchers to assess longitudinal changes in potential biomarkers. And for strong experimental rigor, we must have control samples collected in the same method, meaning donations from friends and family without VCP disease are also essential for moving our research forward. I am building our biobank with purpose, meaning every decision is shaped by what researchers will need to test therapies, track progression, and drive discovery forward.
Together, we have made great progress, and moving forward together with partners like you, WE CAN enhance our programs and provide resources that are vital to improving the lives of individuals and families. Will you consider joining the I CAN MOVEment too?
Zollie's work could not be done without support from our community.
Thank you for supporting our I CAN MOVEment!
