Merritt Engel published 2019 ACT for FOP Grant Awardees in ACT for FOP Grants 2019-10-16 17:34:59 -0400
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).
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: Joshua’s Future of Promises, Canadian FOP Network, FOP Australia, FOP Friends®, FOP Italia and friends and family of Sona Brinkman. The IFOPA's 2019 #GivingTuesday Campaign also raised funds for the ACT for FOP Grant Program.
2019 Grant Awardees
Development of Humanized Antibodies to Mutant ACVR1 and Next-Generation Bispecific Anti-ACVR1-inflamatory Pathway Inhibitors
Principal Investigator: Nicholas C Nicolaides, PhD
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
Award Amount: $65,000
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
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.
Merritt Engel published Cars2CureFOP Turns a Vehicle Into Support for FOP Families in IFOPA News 2019-10-15 10:49:33 -0400
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Merritt Engel published Million Dollar Bike Ride Grant Program in For Researchers 2019-09-16 15:02:43 -0400
September 23 is the Deadline to Apply for Two FOP Research Grants
The IFOPA is pleased to partner again this year with the Orphan Disease Center (ODC) at the University of Pennsylvania to expand FOP research grants through the 2019 Million Dollar Bike Ride Pilot Grant Program.
Two FOP research grants valued at $40,208 each are now seeking applicants. The Orphan Disease Center’s application process is two steps – a Letter of Interest (LOI) due at 8 pm EST on September 23, 2019, followed by an invitation to complete a full application. This Request for Applications (RFA) is open to the international community. All individuals holding a faculty‐level appointment at an academic institution or a senior scientific position at a nonprofit institution or foundation are eligible to respond to this RFA. The two areas of FOP research focus for grant consideration are:
Awardees of the research funding may have access to the IFOPA’s FOP Mouse Model (IFOPA will support the cost of animal models with the exception of shipping) or available samples from the IFOPA's FOP Biobank, if needed. Please contact the IFOPA at firstname.lastname@example.org for further details on these resources.
- Research that seeks to identify biomarkers, including novel imaging techniques, capable of measuring and predicting early FOP disease progression and/or treatment response.
- Research that investigates and further elucidates the immunologic mechanism in FOP.
These grants are made possible by Team #cureFOP riders who participated in the Million Dollar Bike Ride and the IFOPA.
Review all program details and application instructions
Official Program Announcement From the Orphan Disease Center
The Orphan Disease Center (ODC) at the University of Pennsylvania is pleased to announce the 2019 Million Dollar Bike Ride Pilot Grant Program. The program is now open and offering up to 38 different research grant opportunities focusing on 30 different rare diseases. This program provides a one‐year grant to support research related to a rare disease represented in the 2019 Million Dollar Bike Ride. Number of awards and dollar amounts vary per disease based on fundraising totals by each disease team. This Request for Applications (RFA) is open to the international community. All individuals holding a faculty‐level appointment at an academic institution or a senior scientific position at a nonprofit institution or foundation are eligible to respond to this RFA. We ask that you please share this funding opportunity with your department or other relevant researchers.
For more details about this grant program, rare disease focus areas, and how to apply, please visit the Orphan Disease Center. The RFA Guidelines are available. Letters of Interest (LOIs) are due no later than Monday, September 23, 2019 by 8 pm EST. Please refer to our website for instructions on LOI submission.Questions?Please contact Samantha Charleston by email, or call +1 215-573-6822 with any questions.
Merritt Engel published Battle of the Biotech Bands Check Presentation in IFOPA News 2019-09-16 14:51:51 -0400
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Merritt Engel published Surprise birthday party raises nearly $2,000 for the IFOPA! in IFOPA News 2019-09-16 14:51:16 -0400
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Merritt Engel published Million Dollar Bike Ride Grant Program in IFOPA News 2019-09-16 14:50:39 -0400
September 23 is the Deadline to Apply for Two FOP Research GrantsRead more
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Merritt Engel published Meet FOP Connect Mentor Cassie Eckart in IFOPA News 2019-08-30 15:44:53 -0400
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Merritt Engel published IFOPA Active in Supporting International Meetings in Germany and Russia in IFOPA News 2019-08-28 16:39:47 -0400
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Merritt Engel published Dr. Pacifici’s research on RAR gamma agonists inspired two palovarotene clinical trials in FOP News 2019-08-28 14:49:21 -0400
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Merritt Engel published FOP Fun Day in the Pacific Northwest in IFOPA News 2019-08-28 10:50:16 -0400
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Merritt Engel published What's a Biosample and How Can It Help FOP Research? in IFOPA News 2019-08-20 15:37:00 -0400
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Merritt Engel published Million Dollar Bike Ride Fundraising Totals Are In in IFOPA News 2019-08-12 14:23:52 -0400
Team#cureFOP raises over $79,000 for New FOP Research Grants in the 2019 Million Dollar Bike RideRead more
Merritt Engel published Natural History Study Data Shared With The FOP Registry in IFOPA News 2019-08-12 14:23:14 -0400
Data sharing benefits the FOP communityRead more