Today, the Penn Medicine Orphan Disease Center announced the winning research proposals for two grants to investigate FD/MAS. Each of the $58,500 grants will go to qualified and esteemed researchers, with exciting and important ideas that bring the FD/MAS community closer to effective treatments.
The grants were funded through fundraising by FD/MAS community members, including the Levin family, the Fibrous Dysplasia Foundation, the Ruotolo family, and the Yarlagadda family. More information on how these grants were funded is available in our recent news post.
In order to fund more research next year, we need new volunteers to captain teams in the 2016 Million Dollar Bike Ride. Every dollar raised up to $50,000 is matched by the Penn Medicine Orphan Disease Center. Please contact us if you may be interested in raising funds for another round of research.
The winning proposals are:
Mouse Models of Fibrous Dysplasia as a Tool for Developing Rational and Effective Therapies,
led by primary investigator Dr. Mara Riminucci of Sapienza University of Rome,
Development of Next-Gen Animal Models for Fibrous Dysplasia(FD)/McCune-Albright Syndrome(MAS),
led by primary investigator Dr. J. Silvio Gutkind of Moores Cancer Center, UC San Diego.
Both projects will test potential therapies on mice with FD/MAS.
Mouse models are essential tools for developing treatments for diseases. Researchers engineer mice to have the disease that they want to treat or cure, and then use those mice to understand the disease, and test hypotheses about how to treat or cure the disease. The mice they create are called “mouse models.” The experiments researchers conduct with mouse models are either ethically impossible or financially impractical to run on human beings. When the researcher discover treatments that work on mice, work can begin on how to bring those treatments to human beings.
Dr. Riminucci’s lab has already developed a mouse model. Dr. Riminucci will use this grant to study those mice, and design and test potential therapies.
Dr. Gutkind’s project will develop a new mouse model. The new mouse model will develop symptoms sooner than other mouse models, so research can be conducted more efficiently. Dr. Gutkind will use the new, “next-gen,” mouse models to study FD/MAS progression, and test potential therapies.
The winning grants emerged from a competitive funding cycle. Dr. Fred Singer, a Board Member of the Fibrous Dysplasia Foundation who contributed to the application evaluation process, said that of the proposals submitted, “all applications were worth funding.” Unfortunately, there was not enough funding to award additional researchers.
Research in fibrous dysplasia and McCune-Albright syndrome has reached an exciting and crucial time. There are researchers who have ideas for treatments that could slow down or reverse FD/MAS. Those researchers rely on the FD/MAS community to allow them to conduct that work.
Your donation makes a difference. The two projects described here were funded through small donations from FD/MAS patients, and their friends and family.