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UConn researchers publicize 'disappointing' results in disease study, hope for better options

Image of a mouse with bone malformation, used in research.
Courtesy
/
University of Connecticut
Image of a mouse with bone malformation, used in research.

University of Connecticut researchers have discovered that a potential treatment for a rare disease does not work — and they think it's important other scientists know.

The painful disease is known as Fibrodysplasia ossificans progressiva, or FOP. It causes muscles and ligaments to turn into bone after even the slightest injury.

A UConn professor of molecular and cell biology, David Goldhamer, worked on the study with the pharmaceutical company Alexion, which hoped a new antibody treatment it produced would stop that reaction.

But when researchers injected that antibody into mice, the bone formation was actually much worse, producing up to 50 times more bone in some cases.

"Of course, it was very disappointing," Goldhamer said. "And we still don't understand at a fine level what is going on."

UConn's paper is coming out in collaboration with another drug company, Regeneron, that independently found the same outcome. They are publishing the results so other scientists won't waste effort, Goldhamer said.

"I think the results are so dramatic and there's a great deal of interest in FOP because of how devastating this disease is," Goldhamer said. "I think this really serves as a cautionary tale for others who are interested in this approach. And so we agreed to co-submit our papers because the results are really mutually reinforcing."

He said ongoing studies do show that other antibody approaches may still be effective.

According to Goldhamer, about 800 to 900 people worldwide have been diagnosed with FOP, though up to about 4,000 people are likely to have it.

Aside from some new, limited approaches, Goldhamer said there is virtually no treatment other than pain relief.