ACVR1 (Activin A Receptor Type 1)

A biopsy was avoided due to the risk of disease exacerbation, and the diagnosis was made on clinical grounds.As no definitive cure exists, management was supportive with non-steroidal anti-inflammatory drugs for pain, nutritional supplementation and avoidance of surgery. Awareness and early recognition are crucial, while emerging therapies, such as palovarotene and gene-targeted approaches, hold promise for the future.

The potential target genes of activin A receptor type I (Acvr1), ribosomal protein S6 kinase B1 (Rps6kb1), and transforming growth factor beta receptor type 1 (Tgfbr1) were significantly lower in the kidneys of the LPS group. EVs-derived miR-128-3p in LPS induced periodontitis may cause kidney inflammation which may be mediated by, Rps6kb1, Tgfbr1, and Acvr1.

Taken together, experimental and computational evidence supports a dual-mechanistic model in which 4d promotes muscle proliferation and regeneration by (1) activating the MasR-PI3K/AKT/mTOR axis and (2) inhibiting the ACVR1/2A-SMAD pathway, counteracting the action of myostatin. These findings position compound 4d as a promising therapeutic candidate against muscle wasting disorders, including cancer-related cachexia, by inducing a robust and multifactorial anabolic response.

Furthermore, EGFR-mutant LUAD patients exhibited similar cTME characteristics as observed in mice, and high expression of ACVR1 correlated with worse patient prognosis. Overall, in addition to elucidating the role of pro-tumor macrophages in cTME formation, this study pinpoints targeting ACVR1 as a therapeutic strategy for treating EGFR-mutant lung cancers.

P2, N=113, Active, not recruiting, Clementia Pharmaceuticals Inc. | Trial primary completion date: Jul 2025 --> Mar 2029

Greater TAM exposure was significantly associated with lower odds of grade 3/4 anemia and higher odds of any-grade peripheral neuropathy, although the latter was infrequently observed in phase III trials. There was no significant relationship with grade ≥ 3 thrombocytopenia or any-grade diarrhea.

This comprehensive transcriptomic characterization of T-cell exclusion in PM reveals that targeting cilium-based Hedgehog signaling, in addition to multiple other actionable drug targets, could enhance the efficacy of ICB treatment in PM.

Survival analysis highlighted the prognostic relevance of pathway-specific disruptions. These insights emphasize the importance of precision medicine approaches that consider genetic heterogeneity and pathway-specific alterations to improve outcomes for H/L CRC patients.

The safety profile was similar to that reported in clinical trials, with most toxicities of grade I-II. In conclusion, our real-life results support the use of momelotinib as an effective and safe therapeutic option for heavily pre-treated, cytopenic MF patients in real-world clinical practice.

Nearly 50% of PMF patients experience anemia (hemoglobin (Hb) < 10 g/dL), often worsened by JAK inhibitors like ruxolitinib and fedratinib. However, heterogeneity in control groups limited direct efficacy comparisons. Larger studies are needed to confirm its effectiveness and safety.

ACVR1 is a promising therapeutic target that inhibits CAF proliferation. High BMP7 and ACVR1 expression is a significant prognostic factor in stage II CRC.