ACT (Accelerating Cures and Treatments) for FOP

2017 Grant Awardees

H-SAADDs: Hypoxia-Selective ALK2 Allosteric Destabilizers & Degraders for FOP Prophylaxis

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Principal Investigator: Jay Groppe, Ph.D
Institution: Texas A&M University College of Dentistry, United States
Award Amount: $69,832
Funded by Joshua's Future of Promises

Project Description: Dr. Jay Groppe has developed a very new approach to targeting the faulty receptor in FOP. Instead of turning the receptor off, Dr. Groppe has designed a compound that will degrade and eliminate the mutant receptor. One very unique characteristic of this compound is that it is more active during periods of hypoxia (i.e. lack of tissue oxygen), which is found in FOP lesions. Consequently, this drug could be taken chronically, but it would only “turn on” when there is a flare or during bone formation. Funding this research would allow this investigator to optimize this compound and prove the drug is effective at stopping HO in FOP cells. This could be a “next generation” treatment option for people with FOP.

Interrogating New Therapeutic Targets for FOP

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Principal Investigator: Ed Hsiao, M.D. and Pam Yelick, Ph.D.
Institution: University of California, San Francisco and Tufts University, United States
Award Amount:  $75,431
Funded in partnership with FOP Australia

Project Description: Dr. Hsiao has identified genetic variations in 4 FOP patients exhibiting unusually “mild” FOP disease progression. This research will validate if the same genetic variations are also observed in other less severe FOP patients. The identified human genetic variations will then be “placed” in Dr. Yelick’s FOP zebrafish to assess how these genetic changes impact FOP progression. Ultimately, this research would help the research community better understand why some patients have less severe FOP, and could potentially identify new and effective targets to treat people with FOP.

Modes of Treating FOP with Clinically Relevant ALK2 Inhibitory Compound

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Principal Investigator: Daniel S. Perrien, PhD
Institution: Vanderbilt University Medical Center, United States
Award Amount: $50,000
Funded by Joshua's Future of Promises

Project Description: The mutated ACVR1/ALK2 receptor is the direct cause of heterotopic bone formation in FOP. This research uses a highly specific ALK2 inhibitor that has been developed at Vanderbilt University and licensed to a pharmaceutical company for further drug development. This aim of this research study is to define the stages of heterotopic ossification (HO) formation at which the ALK2 inhibitors is effective at stopping bone formation. By knowing this information, we will know the point at which a potent therapeutic could reverse the early stages of FOP, before a lesion has progressed to mature bone.

Validation of Novel Muscle Regenerating Prophylaxis for FOP-related Heterotopic Ossification

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Principal Investigator: Yuji Mishina, Ph.D. and Benjamin Levi, M.D.
Institution: University of Michigan School of Dentistry and University of Michigan, United States
Award Amount:  $52,208
Funded in partnership with FOP Friends®

Project Description: Drs. Mishina and Levi will be investigating the therapeutic approach of inhibiting a signaling enzyme called TAK1 (Transforming growth factor beta-Activating Kinase 1) and the ability of this inhibitor to prevent spontaneous heterotopic ossification (HO) in a mouse model. The role of TAK1 in early cartilage formation makes it a compelling target for HO prevention. The research will further look at whether TAK1 could also play a role in stimulating muscle regeneration following surgery. The results from this research will inform us whether TAK1 has a role as a future therapeutic that could be used in FOP to both prevent HO and to stimulate muscle regeneration following surgery.

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