ACT (Accelerating Cures and Treatments) for FOP

2019 Grant Awardees

The 2019 ACT for FOP Grant Program included lead gifts from: Joshua’s Future of Promises, Canadian FOP Network, FOP Australia, FOP Friends®, FOP Italia and friends and family of Sona Brinkman.

Development of Humanized Antibodies to Mutant ACVR1 and Next-Generation Bispecific Anti-ACVR1-inflamatory Pathway Inhibitors

Principal Investigator: Nicholas C Nicolaides, PhD
Institution: Navrogen
Country: United States
Award Amount: $90,000

Project Description: The research team at Navrogen has developed a novel approach to treating FOP by developing immune-based agents that target the FOP receptor and an immune component believed to contribute to the underlying cause of FOP flare ups.  One of the benefits of using this approach in FOP is the preferential targeting of the mutant FOP receptor, thus enabling the normal receptor to perform its natural functions.   

Recently, the Navrogen team has shown that this approach was able to significantly impact heterotopic ossification (HO) in a FOP mouse model.  Funds from the ACT for FOP grant will enable the further development and testing of these agents for advancement towards human testing.


In Vivo Evaluation of Antisense Oligonucleotide Therapy for FOP in Humanized Model Mice

Principal Investigator: Toshifumi Yokota, PhD
Institution: University of Alberta
Country: Canada

Award Amount: $60,000

Project Description: 

The University of Alberta team led by Dr. Yokota is looking to continue their research in FOP using antisense oligonucleotides (AONs) as way to stop FOP.  This approach is unique in that the AONs target the mutated gene products (i.e. mutated ACVR1/ALK2 receptor) in FOP. As a result of prior funding from the IFOPA and the Canadian FOP Network, the University of Alberta has developed compounds (called a “gapmers”) that target the mutated part of the ACVR1/ALK2 gene.  These gapmers were able to efficiently reduce most of the mutated gene products, while allowing normal (healthy) receptors to remain unaffected. This approach has been proven effective in other rare diseases, including familial hypercholesterolemia and hereditary transthyretin amyloidosis. 

New funding from the ACT for FOP grant program would allow Dr. Yokota’s team to optimize these gapmers to better differentiate between normal and mutant gene expression and to better understand the dosing and delivery of these investigational drugs.


Neutrophil Based Translational Therapies for Fibrodysplasia Ossificans Progressiva

Principal Investigators: Amanda Huber, PhD & Benjamin Levi, MD
Institution: University of Michigan
Country: United States
Award Amount: $65,000

Drs. Amanda Huber and Benjamin Levi at the University of Michigan are investigating the role of the innate immune system in FOP.  This research team leverages the expertise of Dr. Huber who is an immunologist by training and Dr. Levi who is a stem cell biologist with expertise in the response to injury. They will build on their exciting preliminary data identifying the central role of neutrophils and their function in FOP. Drs. Levi and Huber have identified the pathway through which this alteration in neutrophil function occurs which they propose to target with two new pharmacotherapies. 

Funding of this research will allow these investigators to further our understanding about the inflammatory response in FOP and would potentially identify two new inhibitors (one which is currently regulatory approved) that could help mitigate HO formation for people living with FOP. 


PI3K inhibitors as a New Therapy for Fibrodysplasia Ossificans Progressiva

Principal Investigator: Francesc Ventura Pujol, PhD
Institution: University of Barcelona
Country: Spain
Award Amount: $60,000

Project Description: Investigators at the University of Barcelona, let by Dr. Francesc Ventura, are looking to continue their research reported in Cell Signaling and Biology. Dr. Ventura described the ability of an inhibitor, BYL719, to block the formation of heterotopic bone in FOP mice.  BYL719 works by inhibiting phosphoinositide 3-kinase (PI3Kα), an enzyme that is involved in many cellular functions including cell growth, proliferation, and differentiation.  

Research conducted under the ACT for FOP grant program will analyze the mechanisms of BYL719 inhibition of heterotopic ossification (HO) in FOP mice; including which cell types are involved, which steps of HO are inhibited and optimize the timing of BYL719 administration. This research could lead to a new way of treating of people with FOP.

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