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 Grant Awardees

In vivo proof-of-concept analysis of antibodies to mutant ALK2 and a next-generation bispecific anti-ALK2-inflamatory pathway inhibitor

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

 

 

 


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

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

Award Amount: $87,000

Project Description: In this study, the investigators will develop a gene therapy which empowers the patients’ cells to express an inhibitor of Activin A. As a result, upon injury, we expect that patient’s own cells will automatically protect against the development of FOP lesions. The project draws on the PIs’ 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 which has the potential for clinical evaluation.


The gut microbiome regulates inflammation and EHO, making it a novel therapeutic target for FOP

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

 

 

 


Efficacy and safety of anti IL1 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: Fibrodysplasia Ossificans Progressiva (FOP) is a result of an 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. The investigators at Meir Medical Center in Israel hypothesized that treating FOP patients with anti-interleukin 1 agents will help ameliorate the progression of this disease (including minimizing FOP flares).

Dr. Haviv is currently treating 3 FOP patients with canakinumab, an anti-interleukin 1 agent. To date, encouraging responses are being observed with flares rates and HO 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 FOP flares.

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