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Activin A receptor, type I (ACVR1)
- Target
- ACVR1
- Molecular classification
- Receptor, Serine/threonine kinase receptor, Bone morphogenetic protein (BMP) type I receptor, Transforming growth factor-beta (TGF-β) superfamily receptor
01
Overview
Activin A receptor, type I (ACVR1, also known as ALK-2), is a transmembrane serine/threonine kinase receptor that mediates signaling by members of the bone morphogenetic protein (BMP) and transforming growth factor-beta (TGF-β) superfamilies. ACVR1 forms receptor complexes with type II BMP receptors for ligand binding and activation, leading to phosphorylation of SMAD1/5/8 proteins and regulation of diverse biological processes, most notably bone and tissue morphogenesis, cell differentiation, and cardiac development. Pathogenic gain-of-function mutations in ACVR1 are causative for fibrodysplasia ossificans progressiva (FOP), a rare disorder characterized by heterotopic bone formation, and also contribute to certain pediatric gliomas. ACVR1 is a validated therapeutic target for inhibition in FOP and is under study for several drug programs focused on rare diseases and oncology.
- Other names
- ALK-2Activin receptor-like kinase-2ACVR1Bone morphogenetic protein receptor type IA
02
Mechanism of action
Inhibition of ACVR1/ALK2 kinase activity (main mechanism for anti-FOP drugs and cancer research); Modulation of BMP/SMAD signaling, leading to alteration of transcriptional programs
03
Biological functions
Signal transductionCell differentiationBone morphogenesisOsteoblast differentiationHeart and cartilage developmentNervous system and reproductive system developmentRegulation of transcription (via SMADs)Branching morphogenesis in blood vesselsCell proliferation
04
Disease associations
Fibrodysplasia ossificans progressiva (FOP)Cancer (notably diffuse intrinsic pontine glioma, DIPG)Skeletal dysplasiaPotential implications in cardiovascular and reproductive diseases
05
Safety considerations
On-target toxicity leading to impaired normal bone, reproductive, cardiac, and vascular developmentOff-target kinase inhibition causing cytopenia, cardiac dysfunction, or vascular abnormalitiesSuppression of normal ACVR1 function causing abnormal skeletal, cardiac, or vascular development
06
Interacting drugs
Dorsomorphin (experimental inhibitor)
4 more in the full profile.
07
Biomarkers
Mutant ACVR1 allele (especially R206H mutation for identifying susceptibility to FOP)Heterotopic ossification (imaging/clinical marker in FOP)SMAD1/5/8 phosphorylation status (for pathway activation)
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