ACT (Accelerating Cures and Treatments) for FOP

2018 Grant Awardees

The 2018 ACT for FOP Grant Program included lead gifts from: Joshua’s Future of Promises, FOP Australia, FOP Friends®, FOP Italia, friends and family of Sona Brinkman, and the Phoebe Snow Foundation.

X_Yu.jpgActivin A/ACVR1 Modulation of Nociceptive Dysfunction in FOP

Principal Investigator: Xiaobing Yu, MD
Institution: University of California, San Francisco
Country: United States
Award Amount: $49,610

Project Description: Debilitating pain is a major problem for people with FOP. Despite the prevalence and impact on quality of life, pain in FOP has not been extensively studied. Dr. Yu from the University of California, San Francisco will be using quantitative sensory testing (QST) to better understand the underlying pathway of pain in FOP. By further researching nociceptor (pain receptor) dysfunction in FOP, more targeted pain therapies may be identified to improve patient care and comfort.


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Apigenin-like Compounds for the Inhibition of HO in a R206H FOP Mouse Model

Principal Investigators: Robert Pignolo, MD, PhD and Haitao Wang, PhD
Institution: Mayo Clinic
Country: United States
Award Amount: $50,000

Project Description: While the mutated ACVR1 receptor is the direct cause of heterotopic bone formation in FOP, there are many other processes that impact disease intensity and duration. For example, FOP flare-ups are associated with inflammation and hypoxia (a lack of oxygen in tissues), and elevated levels of a protein called HIF-1a. Research by Drs. Pignolo and Wang will look at whether naturally occurring compounds, called bioflavonoids, could reduce the amount of new bone growth in FOP mice by inhibiting HIF-1a. Since bioflavonoids are generally well tolerated, and available without a prescription, this research has the potential to impact near term treatment options.


Elucidation of the Activin A Mechanism for FOP Using Primary Cells From FOP Patients

Principal Investigator: Teun de Vries, PhD
Institution: VU University Medical Center ACTA
Country: The Netherlands

Award Amount: $23,700

Project Description: Dr. Tuen de Vries is looking to assess what genes get activated in FOP cells versus “healthy” control cells. By looking at gene expression, this research will provide novel and important clues on which pathways are solely switched on and off in FOP patients. By researching these pathways, our understanding of FOP will increase which may lead to new potential treatment targets.


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Single-cell Transactional Profiling of Skeletogenic Progenitors in FOP Mice

Principal Investigator: David Goldhamer, PhD
Institution: University of Connecticut
Country: United States
Award Amount: $84,000

Project Description: Dr. Goldhamer has developed a technique to separate FOP and healthy cells from a FOP mouse model.  By isolating the cells that are directly involved in FOP, we can begin to characterize the processes that drives FOP cells to become bone instead of muscle. Furthermore, having this knowledge may lead to: 1) a better understanding of the different mechanisms for FOP bone formation vs. muscle regeneration, 2) insights into new potential biomarkers for FOP, and 3) more information about immune cell involvement in FOP. 

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