We all want to find a cure for rare disease, but where will it come from? Unfortunately, a single scientist or patient can't go into the wilderness with a magnifying glass and find a cure that's fallen from the sky. There are brilliant scientists with good ideas and many dedicated individuals who want to help. But, how do we connect people with great ideas and connect them quickly?
That’s why we are so excited about the research agenda developed by Dr. Armelle Pindon, the Chief Scientific Officer for Cure VCP Disease. This plan helps us connect the dots and identify the building blocks to put in place to develop a treatment for VCP disease. With your help, we can use this research agenda to build a strong foundation for drug discovery and a better future.
Pre-Clinical Toolbox
By being a research partner, Cure VCP Disease can stimulate advancement and innovation by making research tools readily available.
The pre-clinical toolbox is a major component of our research agenda. It's made up of scientific tools to discover, select and evaluate the most effective potential treatments for VCP disease. Last year with your donations, Cure VCP Disease funded two patient-derived cellular models (iPSCs) to test new medications, investigate gene editing, and validate breakthrough therapies.
The benefit of having research tools like this readily available is that Cure VCP Disease can quickly provide for different projects in the scientific community to test for the most effective and safe treatments before testing on humans. Our iPSC models enabled our first research collaboration, a gene-editing project with Genethon. We have pledged to contribute $83,000 to this program.
We have plans to expand the pre-clinical toolbox to create more iPSCs, establish a biobank with patient samples, and develop organoids. Each new model costs $10,000-$50,000, and with additional funding for additional models, we can build an even stronger foundation.
The path to new treatments is just starting, and we are not stopping. Cure VCP Disease plans to expand its funding mechanism so that more funds are available for new collaborations such as the Genethon gene editing project. No longer do patients and their families and friends have to sit around and wait for someone else to make something happen. You can take a leadership role today to help find treatments faster.
Every dollar you contribute drives us closer to finding a cure by accelerating drug discovery and treatment development.
Your gift to Cure VCP Disease not only helps VCP disease patients but also benefits other patient groups. The cutting-edge science that we invest in can also benefit the broader community. Join us by making a gift before the year-end.