2015 Competitive Research Grant Awardees

Assessment of small agents in FOP primary fibroblast cultures to explore new therapeutic targets

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Principal Investigators:  Gerard Pals, PhD and Marelise Eekhoff, MD, PhD 
Institution:  VU University Medical Center, The Netherlands
Award Date: October 22, 2015
Award Amount:  $55,000
Project Start/Stop Date: December 2015 to November 2016
Project Description: The Dutch Amsterdam FOP dedicated research team (VUMC/ACTA) of the Netherlands developed an innovative in vitro model to study ectopic bone formation from very small skin biopsies that can be obtained safely. In this model small molecular compounds will be investigated on their treatment potential. The result will generate new treatment options for FOP and support personalized treatment in the future.

The role of exercise in the progression of fibrodysplasia ossificans progressiva

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Principal Investigators:  Paul B. Yu, MD, PhD and Yue Shen, MBBS 
Institution:  Brigham and Women’s Hospital and Harvard Medical School, United States
Award Date: October 22, 2015
Award Amount:  $28,000
Project Start/Stop Date: December 2015 to November 2016
Project Description: The impact of physical activity on the progression of FOP is not currently known, and thus clinicians are unable to provide evidence-based recommendations to patients on whether or not to pursue organized athletic activities or regular exercise as part of their routine. Using a knock-in mouse model which expresses the classic FOP-causing mutation ACVR1R206H under endogenous promoter control, they have observed spontaneous ligamentous and joint ossification that occur slowly but progressively with age. They will test the impact of voluntary exercise versus limb immobilization, versus normal cage activity on the progression of heterotopic ossification in these mice, hypothesizing that physiologic levels of activity have a beneficial impact on the disease.

Validation of novel diagnostic and targeted prophylaxis for FOP related heterotopic ossification

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Principal Investigators:  Yuji Mishina, PhD and Benjamin Levi, MD
Institution:  University of Michigan, United States 
Award Date: October 22, 2015
Award Amount:  $41,800
Project Start/Stop Date: December 2015 to November 2016

Project Description: The University of Michigan team plans to define the early chondrogenic environment that precedes heterotopic bone formation. They then plan to use a repurposed HIF-1-alpha inhibitor, both alone and in combination with an ACVR1-specific kinase inhibitor, to alter this environment and subsequently inhibit the formation of heterotopic bone in vivo.

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