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Monday, February 29, 2016

World Rare Disease Day 2106

Today, February 29th, 
is 
World Rare Disease Day

It seems fitting that today the 40th and last participant has enrolled into the Clementia Phase 2 clinical trail that is investigating palovartene for the treatment of FOP.

When Hayden was diagnosed September 11, 2002, hope seemed to 
very, very far away.  However the following pretty much sums up the FOP Community.
"Alone we are rare. Together we are strong."

Nothing demonstrates this more that the determination of our families who fundraise, our board members who guide us along our mission and the researchers who are continually on a quest to discover a cure.

World Rare Disease Day is an annual observance held on the last day of February to raise awareness for rare diseases and improve access to treatments and medical representaiotn for individuals with rare diseases and their families. 


Hayden in Tahoe - 4th of July 2015

Monday, February 1, 2016

2016 Competitive Research Grant Program for FOP Research


In 2015 the IFOPA launched the Competitive Research Grant Program, to encourage collaborative research on many venues to further advance our search for a cure to FOP.  

The 2016, the IFOPA gave out three research grants.  Below are the winners.

Allosteric inhibitors of ALK2 for FOP therapy

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

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

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.