2016 Competitive Research Grant Program Winners Announced

The IFOPA is pleased to announce the three recipients of nearly $108,000 in research grants through its Competitive Research Grants (CRG) Program

“It’s a great opportunity to be able to provide the Competitive Research Grants which focus on the discovery and advancement of new therapeutic approaches to FOP, in addition to the IFOPA’s funding of the Center for Research in FOP and Related Disorders at The University of Pennsylvania,” shared Paul Brinkman, board chair of the IFOPA. This is the second year research grants were made available through the CRG program. In a special partnership initiated this year, FOP Friends® UK and The Canadian FOP Network partnered with the IFOPA to help fund grants that were awarded to researchers based in their countries. “We are pleased to see three FOP organizations come together to directly fund FOP research grants,” offered Michelle Davis, executive director of the IFOPA. “Together, we can achieve more for the FOP community!”

Applications are reviewed by a voluntary Scientific Advisory Board (SAB) that has relevant and appropriate expertise related to FOP to evaluate proposals, and is free of conflict of interest. Members include: SAB Chair Vicki Rosen, PhD, Harvard School of Dental Medicine; Karen Lyons, PhD, UCLA/Orthopaedic Hospital Department of Orthopaedic Surgery; Ernestina Schipani, MD, PhD, University of Michigan; Michael Whyte, MD, Washington University School of Medicine; and Michael Zasloff, MD, PhD, Georgetown University Medical Center.

Congratulations to this year’s winners who continue to accelerate the development of safe and transformative therapies for FOP!

Allosteric inhibitors of ALK2 for FOP therapy

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Principal Investigator: Alex Bullock, PhD
Co-investigators: Paul Brennan, PhD and Frank von Delft, PhD
Institution: Oxford University, United Kingdom
Award Amount: $26,421
This grant is funded in partnership with FOP Friends® UK.

Project Description: The University of Oxford FOP Research Team plans to develop a second generation ALK2/ACVR1 inhibitor for FOP therapy by targeting novel allosteric sites that allow for exquisite target selectivity and further improved drug safety. The team will screen a drug fragment library using X-ray crystallography and solve hundreds of 3D structures of the ALK2/ACVR1 protein to identify drug fragments that bind outside the ATP pocket and lock the kinase domain in an inactive state. Further chemistry will then be used to optimize these fragments into potent inhibitors that can block heterotopic ossification (HO) in FOP.

Development of antisense oligonucleotide therapy for FOP

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Principal Investigator: Oana Caluseriu, MD and Toshifumi Yokota, PhD
Co-applicant: Rika Maruyama, PhD
Institution: University of Alberta, Canada
Award Amount: $35,070
This grant is funded in partnership with the FOP Canadian Network.

Project Description: The University of Alberta team will develop a new therapy for FOP using small DNA-like molecules. These molecules can reduce the gene product that induces abnormal bone formation in FOP patients. The team will evaluate the efficacy and safety of these molecules in an FOP mouse model. This research will identify new drug candidates for FOP.

Experimental therapy to prevent secondary heterotopic ossification following surgical intervention in FOP

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Principal Investigators: Paul B. Yu, MD, PhD and Dong-Dong Xia, MD
Institution: Brigham and Women’s Hospital and Harvard Medical School, United States
Award Amount: $46,446

Project Description: In advanced stages of FOP, progressive disease affects nearly all joints, with very limited mobility and high risk for traumatic injury. Surgical excision of heterotopic bone from muscle and soft tissues has been attempted, but almost always leads to rapid recurrence of heterotopic bone and recurrent loss of function. Current options for elective surgery in FOP are extremely limited, due to the lack of effective measures to prevent secondary bone formation following surgical trauma. We hypothesize that dysregulated ACVR1 ALK2 activity, in addition to driving primary heterotopic ossification (HO) in FOP, is responsible for the formation of secondary HO following surgical injury. We propose that pharmacologic inhibition of ALK2 kinase activity for a limited period following surgical intervention will prevent recurrent bone formation while permitting normal wound healing. We will test the efficacy and tolerability of this approach in an animal model of FOP expressing the ACVR1R206H mutant allele. It is hoped these experiments will provide a rationale for initiating clinical trials investigating the efficacy of this strategy in patients with advanced FOP.

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