Therapeutic advances for blocking heterotopic ossification in fibrodysplasia ossificans progressiva.

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Wentworth KL, Masharani U, Hsiao EC

Therapeutic advances for blocking heterotopic ossification in fibrodysplasia ossificans progressiva.

Br J Clin Pharmacol. 2019 Jun;85(6):1180-1187. doi: 10.1111/bcp.13823. Epub 2019 Jan 6.

PubMed ID

30501012 [ View in PubMed

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Abstract

Fibrodysplasia ossificans progressiva (FOP) is a rare genetic disease in which heterotopic bone forms in muscle and soft tissue, leading to joint dysfunction and significant disability. FOP is progressive and many patients are wheelchair-bound by the 3rd decade of life. FOP is caused by an activating mutation in the ACVR1 gene, which encodes the activin A Type 1 receptor. Aberrant signalling through this receptor leads to abnormal activation of the pSMAD 1/5/8 pathway and triggers the formation of bone outside of the skeleton. There is no curative therapy for FOP; however, exciting advances in novel therapies have developed recently. Here, we review the clinical and translational pharmacology of three drugs that are currently in clinical trials (palovarotene, REGN 2477 and rapamycin) as well as other emerging treatment strategies for FOP.

DrugBank Data that Cites this Article

Drugs

Garetosmab

Drug Targets

Drug	Target	Kind	Organism	Pharmacological Action	Actions
Garetosmab	Inhibin beta A chain	Protein	Humans	Yes	Binder	Details