Research press release


Nature Medicine

Bringing Galatea to life?



ヒトの場合と同じ変異をもったFOPマウスモデルはまだ作られていないが、P Yuたちは、原因となる変異型に類似した変異受容体を発現させることによって、一般的症状のマウスモデルを開発した。この病気を引き起こすには、単にこのタンパク質受容体の変異型を発現するだけでは不十分で、ほかに炎症刺激も必要なことが判明した。また、炎症をグルココルチコイドで抑えるという臨床でよく用いられる治療法が、このモデルの異所性骨化の発生を減少させることもわかった。


Inflammation is a key step in the progression of heterotopic ossification ? where soft tissue turns into bone ? according to research in Nature Medicine this week. The study shows that an inhibitor of the disease gene’s protein product is partially therapeutic, and therefore offers hope for this devastating condition.

In a reverse of the ancient myth of Pygmalion, where a statue comes to life, sufferers of heterotopic ossification have their fibrous tissue ‘ossified’ ? in effect, turning the patients into statues. A major form of heterotopic ossification is fibrodysplasia ossificans progressiva (FOP), which in about 98% of cases results from a mutation in a specific bone morphogenetic protein receptor.

A mouse model of FOP involving the same mutation found in people has yet to be made, but Paul Yu and colleagues have now developed a mouse model of the general phenomenon by expressing a related version of the mutated receptor. They found that just expressing the mutant version of the protein receptor was not sufficient to cause the disease ? an inflammatory stimulus was also needed. Yu’s team also show that inhibiting inflammation with glucocorticoids?a treatment commonly used in the clinic?helps reduce the incidence of heterotopic ossification in their model.

Importantly, the authors also show that a small molecule inhibitor of the protein receptor likewise reduced the incidence of disease progression. This form of treatment represents a potential breakthrough, as long-term use of glucocorticoids causes severe side-effects. The authors caution, however, that much more research is needed before the drug could be considered for human trials.

doi: 10.1038/nm.1888


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