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AMLAL-101 is a novel small molecule activator of the gamma-aminobutyric acid type A receptor (GABAAR) being developed by Amlal Pharmaceuticals. It is designed to enhance GABAAR-mediated chloride transport in melanoma cells, which triggers mitochondrial dysregulation and subsequent tumor regression. In preclinical studies, AMLAL-101 has demonstrated the ability to sensitize melanoma brain metastases to stereotactic radiosurgery (SRS), potentially allowing for reduced radiation doses while maintaining therapeutic efficacy. Beyond its direct anti-tumor effects, AMLAL-101 appears to modulate the tumor microenvironment by increasing the infiltration of polyfunctional CD8+ T cells and M1-phenotype tumor-associated macrophages (TAMs), thereby promoting a potent abscopal effect in non-irradiated tumors.
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