For the FOP community, every discovery in research brings us closer to a future with better treatments—and ultimately, a cure. During the In Pursuit of a Cure campaign, we’re highlighting the tangible impact of your support and the many ways your generosity helps accelerate FOP research.
Progress depends on more than groundbreaking ideas. Researchers need access to the tools and resources that allow them to turn those ideas into action.
Thanks to the generosity of IFOPA donors, the IFOPA FOP mouse model remains one of those tools. Here’s how one shared resource is helping advance studies around the world and move FOP research forward.
A living model for understanding FOP
Mouse models play an important role in biomedical research because mice share immense genetic and physiological similarities with humans. Nearly every human gene has a comparable mouse counterpart. This provides researchers with complex biological systems to study how diseases develop and progress, and allows them to explore potential treatments before they are tested in people.
For FOP research, this model is especially important. Thanks to a generous sponsorship from La Jolla Pharmaceutical Company in 2018, the IFOPA created a conditional ALK2R206H FOP mouse model, with the expressed purpose of sharing it and making it freely available to other researchers to support their work in FOP without restrictions. The FOP mouse model recreates key features of FOP, including heterotopic bone formation and flare-ups, allowing scientists to better understand how the disease develops and investigate potential treatments.
Donor support makes it possible for researchers from around the world to access a FOP mouse model without the barriers that can slow scientific progress.
Some pharmaceutical companies and academic institutions have their own animal models, but obtaining them can involve complex agreements, fees, and other requirements. Sometimes, this actually stopped companies from testing their products for treating FOP. The IFOPA model takes a different approach: our FOP mice are available to any researcher with a valid research plan, and the researcher pays only the shipping costs. The result is a shared, open-access resource designed to put research first.
One mouse model. Many paths to a cure.
Daniel Perrien, PhD, Associate Professor in the Division of Endocrinology at Emory University, manages the IFOPA’s mouse model. He shares that over the past eight years, the IFOPA’s FOP Mouse Model has been used to test potential therapies from at least 7 companies and answer numerous questions about FOP in:
- 13 labs
- 4 countries
- 2 continents
- 11 publications
- 2 funded R01 and one funded R21 grants from the National Institutes of Health
The model has become a shared resource that accelerates research by freeing up the time and resources that scientists would need to spend to develop their own FOP mouse model, or negotiate with other research institutions or organizations to access their FOP mouse model.
One particularly significant area of research has been gene therapy.
Jae Shim, PhD, of the University of Massachusetts Chan Medical School, and his team have used the IFOPA mouse model to advance novel genetic medicines targeting both the ACVR1 R206H mutation and the inflammatory flare-ups associated with FOP.
“Animal models recapitulating FOP phenotypes … have been instrumental for elucidating disease mechanisms and developing therapeutic strategies,” Dr. Shim says.
The ultimate goal is to translate promising approaches into clinical trials. The IFOPA’s mouse model is one piece of that journey—helping researchers understand FOP biology and test potential therapeutic approaches.
Your support amplifies research. This mouse model has already contributed to many important research projects and helped secure millions of dollars in additional NIH research funding, multiplying the impact of donations from supporters of FOP research.
Your support makes research move faster
The IFOPA mouse model is a tangible example of what a donation to the In Pursuit of a Cure campaign makes possible.
A gift doesn’t simply support an abstract idea of “research.” It helps keep critical research resources, such as the FOP mouse model, open access for researchers.
Over the past eight years, dozens of research studies have used the IFOPA’s mouse model. Each of those studies asked a new question, tested a new possibility, and brought us closer to the cure.
Your gift helps remove red tape, making research move faster by giving scientists free access to a critical research tool that advances their work, including bringing potentially transformative strategies like gene therapy to life.
Thank you for making this kind of progress possible.
As we Journey to the Future, your support will help us continue to provide this mouse model and other critical resources to the next set of researchers, who may discover the next breakthrough in pursuit of the FOP cure.
Give to In Pursuit of a Cure today.
