2020 ACT for FOP Grant Awardees

The IFOPA offers the ACT (Accelerating Cures and Treatments) for FOP grant program to accelerate the development of new drugs for FOP. The research grant program provides, through a competitive application process, funding to scientists conducting research on fibrodysplasia ossificans progressiva (FOP).

2020 Funders

The ACT for FOP Grant Program was launched in 2015 by friends and family of Sona Brinkman. Today others who are passionate about curing FOP have joined them in funding the ACT for FOP Grant Program with lead gifts from: FOP Australia, FOP Friends®, FOP Italia, friends and family of Sona Brinkman, Joshua’s Future of Promises and Terri Hendley.

2020 Grant Awardees

Combined Effects of an IL-1β Inhibitor and Microbiota Ablation on Heterotopic Ossification in FOP Mice

Principal Investigators: Daniel Perrien, PhD and Ed Hsiao, MD, PhD
Institutions: Emory University and University of California San Francisco
Country: United States
Award Amount: $50,000

Project Description: FOP flares are highly inflammatory and published evidence suggests immune cells from FOP patients are more sensitive to inflammatory activity. We and others have proposed there may be anti-inflammatory approaches to reduce heterotopic ossification (HO) and flare intensity and/or frequency. Microbiota, the bacteria and viruses that live on or in our bodies, have recently been recognized as a major regulator of systemic inflammation, including levels of the inflammatory protein IL-1β. Our preliminary data show that the removal of normal gut bacteria reduced injury-induced HO in FOP mice by about 50%. A published case report also suggests the use of an IL-1β inhibitory drug reduced ongoing and future flares in multiple FOP patients. Therefore, this project will test the combined effects of blocking IL-1β and removing gut bacteria on flare progression and HO formation in FOP mice. The goals are to generate key initial data needed to determine if manipulation of gut microbiota combined with anti-IL-1β therapy holds the potential to reduce inflammation and HO in FOP. If our hypotheses are proven correct, the data may support a future interventional clinical trial in FOP patients and collaborative applications for National Institutes of Health (NIH) funding.

Efficacy and Safety of anti-IL-1 Treatment in Children, Adolescents and Young Adults with FOP

Principal Investigator: Ruby Haviv, MD
Institution: Meir Medical Center
Country: Israel
Award Amount: $69,144

Project Description: FOP is a result of ongoing intra-cellular signaling through the bone morphogenic protein (BMP) pathway. Interleukin-1 (IL-1) has been linked to the mineralization of human and mice bone marrow mesenchymal cells. Investigators hypothesized that treating FOP patients with anti-IL-1 agents will help improve life with this disease, including minimizing FOP flares. Dr. Haviv currently treats three FOP patients with canakinumab, an anti-IL-1 agent. To date, encouraging responses are being observed with flares rates and heterotopic ossification progression. This grant is to support a small pilot study to expand our understanding of IL-1 inhibition in FOP and its role as a potential treatment against flares.

In Vivo Gene-editing for Fibrodysplasia Ossificans Progressiva Based on Transgene Reconstitution

Principal Investigators: Shailesh Agarwal, MD; Vicki Rosen, PhD and Yuji Mishina, PhD
Institutions: Brigham and Women's Hospital, Harvard Dental School and University of Michigan
Country: United States

Award Amount: $87,000

Project Description: In this study, the investigators will develop a gene therapy that empowers the patients’ cells to express an inhibitor of Activin A. As a result, upon injury, we expect that patients' own cells will automatically protect against the development of FOP lesions. The project draws on the principal investigators' previous experience with studying FOP and will make use of the ACVR1 R206H mouse model. This approach will include in vitro studies to validate the gene therapy and in vivo studies to quantify ectopic bone volume using microCT imaging. Our goal is to develop a gene therapy that has the potential for clinical evaluation.

In Vivo Proof-of-Concept Analysis of Antibodies to Mutant ALK2 and a Next-Generation Bispecific anti-ALK2-Inflammatory Pathway Inhibitor

Principal Investigator: Nicholas Nicolaides, PhD
Institution: Navrogen
Country: United States
Award Amount: $37,180

Project Description: Navrogen’s research team 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 inflammatory flare-ups associated with the underlying cause of heterotopic ossification. One of the benefits of this approach is the preferential targeting of the mutant FOP receptor and local inflammatory suppression in FOP lesions. They have formatted these agents to be suitable for future human testing. Grant funds will enable the further development and preclinical testing of these agents in vivo for efficacy and safety as the next step towards possible human testing.

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