April marks two years from the start of our study with Nationwide Children’s Hospital. Thirty VCP patients have enrolled for in-clinic assessments. And these thirty patients continue to make an annual trip to Columbus, Ohio to be tested. I am happy to report that this study is breaking down barriers. Barriers to a clinical trial. Barriers to a cure. Two questions stand in our way to clinical trials: 1 - Can we measure change in a year? 2 - Is there a way to measure this change remotely? The results are astounding . . . Yes and Yes! Thanks to all the patients in the Nationwide study, we have data. Lots and lots of data. Our patients have volunteered their time and efforts so that we have statistical proof.
Shared with permission from Dr. Lindsay Alfano as an excerpt from a poster presentation at the World Muscle Society 2022 Congress illustrating performance on three different functional measures over time.
But, I share these charts with mixed feelings. These dots on a chart are not just cool statistics. These dots are our friends. This is our family. This is our loved ones. Declining. Everyday. The fact that significant change happens in a year is no surprise to VCP patients and their loved ones. We see it every day. What our loved ones could do last year is not the same as today. Abilities are declining. Strength is declining. Independence is declining. The proof is overwhelming. But, that does not change our resolve to keep fighting.
Even though the team at Nationwide is amazing. Even though we pay for travel. Even though this study is very, very important. This study is hard on patients and their families. Travel is hard with VCP disease. Scheduling time away from work is difficult. Scheduling a travel companion is challenging. Scheduling childcare can be complicated. But, our patients continue and persevere because they know this study is the way to possible clinical trials and a better future.
Our community needs each and every patient to continue to participate. And so many are.
I write to all of our patients and their families to say THANK YOU. Each and every one of you are true warriors. Together, we are fighting this. Together, we will find a cure. Allison Peck Co-founder, Care Partner and Friend Cure VCP Disease