Team FD/MAS 2023 Research Grants Announced
FD/MAS Alliance is thrilled to share that three fibrous dysplasia, McCune-Albright syndrome (FD/MAS) research grants have been awarded from Team FD/MAS’s 2023 Million Dollar Bike Ride efforts!
Last spring, Team FD/MAS raised $161,620 for FD/MAS-focused research through the UPenn Orphan Disease Center’s Million Dollar Bike Ride. After careful review of nine applications by the FD/MAS Alliance’s Scientific Advisory Council and the UPenn Orphan Disease Center’s staff, three grants have been awarded:
- Jaymin Upadhyay of Boston Children’s Hospital will receive $40,321 for his project “Probing Central and Peripheral Mechanisms of Pain and Dysfunction in Fibrous Dysplasia/McCune-Albright”
This study team will aim to obtain a deeper understanding of biopsychosocial factors that contribute to pain in FD/MAS. In a small pilot study, they will also determine the effects of intensive interdisciplinary pain treatment in patients with FD/MAS.’
- Julia Charles of Brigham and Women’s Hospital will receive $40,321 for her project “Spatial transcriptomics to elucidate the mechanism of anti-RANKL inhibition of fibrous dysplasia bone lesions”
Recent groundbreaking work from Dr. Alison Boyce has demonstrated the benefit of denosumab, an antibody that blocks the cytokine RANKL, for treating bone lesions in fibrous dysplasia, but is the molecular mechanism of this effect is not known. Dr. Charles’s team plans to use a mouse model of FD to investigate this. They will use spatial transcriptomics, which profiles the genes expressed by individual cells within a section of tissue, to understand how both mutant and bystander wild-type cells within mouse FD bone lesions respond in the hours and days after RANKL is blocked. This is important because blocking RANKL has side effects that limit use and understanding how it improves bone lesions could lead to developing alternative therapies.
- Fernando Fierro of the University of California Davis and Dr. Charles Hoffman of Boston College will receive $80,642 for their project “Testing the specificity and efficacy of compounds that inhibit cAMP signaling, for the development of potential therapeutics for FD/MAS”.
The award to Dr. Fernando Fierro (UC Davis) and Dr. Charles Hoffman (Boston College) is to test small molecules that could block the hyperactive cAMP signaling observed in FD/MAS lesions. Specifically, Dr. Fierro’s laboratory developed a system to model FD in a culture dish, using genetically modified human bone marrow-derived mesenchymal stromal cells (BM-MSCs) and will test if candidate compounds can restore cAMP levels. Dr. Hoffman will elucidate how exactly these compounds work and to what specific proteins they bind. The candidate compounds were previously identified based on either a large screening done in yeast, or based on computer modeling of Gsa (wild type and mutant) done by our collaborators at UCSF (Drs. Hsiao and Wentworth).
These research projects have been made possible by the FD/MAS community and the over 205 donors who gave to support these grants. Special thanks to Cindi Brandt Levin, who acted as captain for Team FD/MAS as well as the Cohen family, Clay C., the Healy family, and Aurelie Lagneau of Association MAS/FD for their significant contributions. We are so grateful.
Team FD/MAS has participated in the Million Dollar Bike Ride for 9 years and raised over a million dollars.
This past year was an especially exciting one for us, as previous Team FD/MAS Awardees used the preliminary data they generated with Team FD/MAS funding to compete for and receive over $6.5 million in federal research funding through the Peer-Reviewed Medical Research Fund and NIH research grants. “Team FD/MAS grants cover a critical funding gap by making early research initiatives possible,” explained Dr. Mike Collins, FD/MAS Alliance Scientific Advisory Council Chair. “By funding those initial investigations, researchers can build the evidence they need to compete for larger funding opportunities that would otherwise be out of reach. Each year we see a competitive pool of applicants for these pilot grants and it’s gratifying to see that the research Team FD/MAS has funded continues to make progress.”
Team FD/MAS awardees had a reinvigorated appreciation for the FD/MAS community after this year’s FD/MAS Community Conference. “I really felt the warmth of the community at the recent international consortium meeting and made great connections, including starting a side project with Biagio Palmisano [a 2021 Team FD/MAS awardee]. That kind of fun interaction leading to new ideas is the best part of science.” said Dr. Julia Charles, “I want to particularly thank Cindi for her tireless fundraising and community-building efforts that have made this possible.”
Our next big milestone will be seeing some of the science we’ve funded impact the lives of actual people living with FD/MAS. Team Captain Cindi Brandt Levin remains hopeful: “Between our dedication to continuing Team FD/MAS grants and the larger federal funding that these grants have led to, I know that together, we can achieve that goal!”
As you make plans for the upcoming year, mark your calendars for June 8th and the 2024 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!