IFOPA and Canadian FOP Network Partnering to Fund FOP Research 

The Canadian FOP Network and the IFOPA are joining forces to fund research conducted by rare disease experts at the University of Alberta through the IFOPA’s Competitive Research Grant (CRG) Program. The study will test DNA-like molecules known as “antisense oligonucleotides” in a mouse model of FOP, with the hope that successful completion may one day lead to clinical trials for patients with FOP. 

“We are using short DNA-like molecules that target mutated products from DNA in FOP patients,” said Toshifumi Yokota, PhD, Assistant Professor at the University of Alberta, who is partnering with fellow faculty members at the University of Alberta to conduct the study. 

This method has several unique advantages, including the possibility of designing a molecule that targets only the mutated DNA copy. Another advantage is that DNA-like molecules do not need a carrier such as a virus for delivery to tissues throughout the body. 

Yokota went on to say, “We are hopeful these DNA-like molecules will also prove effective in the treatment of FOP, as they have been for Duchenne muscular dystrophy and spinal muscular atrophy.”

Carrie Connell, President of the Canadian FOP Network (CFOPN), said, “The Canadian FOP Network is thrilled for the opportunity to co-fund the research grant with our expert partners, the IFOPA. We are very hopeful about the University of Alberta's research and the results it may produce.” 

The idea for the study initiated when Oana Caluseriu, Medical Geneticist and Assistant Professor at the University of Alberta, began working with four FOP patients. 

“That was certainly a unique opportunity to learn not one, but four very different realities of the same disorder from four young adults I’ve seen in clinic,” she related. 

Caluseriu verified that the culprit mutation in each of these patient’s DNA was the same recurrent mutation, R206H, that affects 97% of FOP patients around the world.

She said, “This raised in my mind the theoretical possibility of a unifying therapy for my patients.” 

After reviewing FOP literature, she found an attempt to use antisense therapy in mouse tissues. Caluseriu then proposed to her colleague, Toshifumi Yokota, a study aimed at figuring out how the therapy would work in human tissues.

They secured initial funding from the University Hospital Foundation, which yielded encouraging preliminary results on antisense therapy. 

“This new funding will help us take our initial research to a whole new level, and allow us to test in an FOP mouse model engineered in the United States,” Caluseriu said.

Caluseriu and Yokota stressed their thanks and appreciation for the support they’ve received from CFOPN and the IFOPA.

“I was deeply impressed by the passion of presenters I saw at the FOP Drug Development Forum in Boston,” Yokota said. “We were very excited when we learned of the funding from CFOPN and the IFOPA to continue our project.”