2021 Team FD/MAS Research Grants Announced

FD/MAS Alliance is thrilled to share that three FD/MAS research grants have been awarded from the 2021 Million Dollar Bike Ride!

Last spring, Team FD/MAS raised $161,373 for fibrous dysplasia, McCune-Albright syndrome (FD/MAS)-focused research through the UPenn Orphan Disease Center’s Million Dollar Bike Ride. After careful review of several applications by the FD/MAS Alliance’s Scientific Advisory Council and the UPenn Orphan Disease Center’s staff, three grants of $53,791 each have been awarded:

• Dr. Biagio Palmisano, Sapienza University of Rome
  “Unraveling the role of Adiponectin-expressing bone marrow stromal cells in the cellular pathogenesis of Fibrous Dysplasia”
  
  

  Dr. Biagio Palmisano

  Fibrous dysplasia bone lesions contain a very heterogeneous population of cells. The cell responsible for the development of the disease is believed to be a progenitor of osteoblasts (the cells that produce bone), that due to Gsα mutations, accumulates in the fibrous tissue and recruits osteoclasts (the cells that destroy bone). However, its precise identity is still unknown. Our recent work points to a specific bone marrow cell type (Adiponectin-expressing stromal cell) as a potential candidate. In this project we will generate “new versions” of our FD transgenic mouse model to track Adiponectin-expressing stromal cells during FD lesion development and to assess their role in osteoclast recruitment. In addition, we will look for markers that are specific for Adiponectin cells with the aim to identify new druggable targets for FD.

• Dr. Fernando Fierro, University of California, Davis and Dr. Charles Hoffman of Boston College
  “Structure activity relationship studies of compounds to treat FD/MAS”
  
  This interdisciplinary project brings together a lab that has developed a novel compound detection method and a lab focused on bone biology and stem
  

  Dr. Charles Hoffman (far left) and his students at Boston College.

  cells. Compounds identified by their ability to counteract the effect of GNAS mutations on cyclic AMP levels have been shown to reduce osteogenesis in human bone marrow-derived multipotent mesenchymal stromal cells (MSCs). This project will further characterize compounds from a collection of 41 related structures to identify those most suitable for testing in animal models and human MSCs.

   

• Dr. Edward Hsiao and Dr. Kelly Wentworth of University of California, San Francisco
  “Identification and signaling characterization of GNASR201H/C selective inhibitors for FD/MAS”One of the major challenges for treating patients with FD/MAS is the lack of therapies directed against the causative GNAS mutations. This proposal characterizes a number of small molecules that may be able to bind directly to the GNAS R201H/C mutations in FD/MAS. We are very excited to pursue these studies as a stepping stone towards targeted therapies and to learn more about how we can target the GNAS pathway. We greatly appreciate the support of the FD/MAS community to make this possible.
  

“Dr. Michael Collins of the NIH, the acting chair of our Scientific Advisory Council, has described these proposals as exciting, creative, and-most important of all–promising,” said Team Captain, Cindi Brandt Levin, “We are  inspired by the continuing promise we see in these research proposals, and deeply grateful both for the scientists dedicating their careers to treating this disease and to the community members who come together year after year to ensure that research progress continues.”

Dr. Fernando Fierro and his lab team at UC Davis

This year’s research projects represent new collaborations as well as career long commitments to this disease. “Personally, I feel very inspired by working with the

FD/MAS community,” said 2021 awardee, Dr. Fierro, “it brings our scientific endeavor to a whole new level when thinking of the direct impact we hope our work can have in other people’s lives.” Each of the five renowned researchers named by these grants echoed this sentiment along with their gratitude for Team FD/MAS. Their work has been made possible by the record breaking 50 riders who registered to ride with Team FD/MAS and the over 200 donors and fundraisers whose generosity they inspired in their communities. We are so grateful. In the 7 years Team FD/MAS has participated in the Million Dollar Bike Ride, we have raised over $900,000 for FD/MAS research. “Every dollar raised helps our community move closer to our goal of better answers and better outcomes, and ultimately the hope of a cure for FD/MAS,” said Brandt Levin. “We are determined to make 2022 the year that we surpass the million dollar mark and we will need every rider, fundraiser, donor, and supporter in our corner to get there. With the help of the  incredible FD/MAS community, I have faith that we can do it!”

Dr. Ed Hsiao (far left) and Dr. Kelly Wentworth (center front) and their lab team of UCSF

As you make plans for the upcoming year, mark your calendars for June 11th and the 2022 Million Dollar Bike Ride with Team FD/MAS.

We’d love for you to join us in person in Philadelphia or virtually. Until then, be well, stay safe, and a happy healthy new year to you all!