One of the exciting aspects of the Million Dollar Bike Ride fundraiser is how quickly the gifts from patients, friends and family go directly towards actual FD/MAS science. In July, UPenn announced that $273,000 would be awarded to the best proposals for FD/MAS studies, and researchers from around the world have already turned in their proposals for the four grants available.
Grants awarded ten months ago in December 2017 are now in the final months of their project period. Dr. Mara Riminucci has continued her work, funded by previous Million Dollar Bike Ride grants, on RANKL inhibitors, a project that looks at FD bone and tries to prevent bone fragility and deformity. Previous Million Dollar Bike Ride grants have helped Dr. Riminucci progress this project forward and test a treatment that causes, in mice, the replacement of fibrotic tissue with bone, while halting the progression of FD, but “what is not properly defined today is the approach that will prevent or reduce the relapse of the disease following treatment withdrawal,” explains Dr. Riminucci. Right now, her lab continues to work with FD mouse models to focus on the exact dosage and course for her treatment so that, eventually, this same therapeutic strategy can help treat patients.
Our other 2017 awardee, Dr. Yingzi Yang is also working on therapeutic strategies that will improve the lives of FD/MAS patients further down the line. Like Dr. Riminucci, Dr. Yang is using mouse models to better understand how FD works on a molecular and cellular level. By learning more about the “underlying mechanisms of FD, we can then identify and test therapeutic targets for FD/MAS,” says Dr. Yang.
Mouse models are a common early step for new drug therapies because they allow researchers to make sure their findings are safe and effective before developing them for patients. This is an important step on the path towards a treatment patients can access in stores. “We hope that we can test some of these therapeutic targets using patient-derived cells,” Dr. Yang explains, highlighting other steps on the path to new treatments “we also hope that, eventually, we can recruit patients in possible clinical trials for these therapies.” Like Dr. Yang’s project, Dr. Riminucci’s project aims to create a treatment that will help patients down the line. Her aim is to convert FD tissue into fully mineralized bone in a lasting and meaningful way.
As Dr. Riminucci and Dr. Yang enter the final months of their award year, FDF’s Scientific Advisory Council will be looking over the applications for the 2018 awards. Along with UPenn scientists, FDF advisors will decide the four best projects to advance our understanding of FD/MAS and improving the lives of patients. Some awarded projects might, like the last five projects, continue the trend of studying the basic science of FD/MAS with preclinical models, but at least one award has been reserved for a promising clinical science study. This will help to promote diversity in the award winning projects, ensure that the FD/MAS community is advancing as many promising research opportunities as possible, and increase the speed it takes for funded research to directly touch the lives of the FD/MAS community.
Both Dr. Riminucci and Dr. Yang agree that the Million Dollar Bike Ride grants are an invaluable resource and support for the work that they do. These grants and this incredible research would not be possible without the dedication and generosity of the FD/MAS community and their friends and families. The 2018 awardees will be announced in December.