Rhabdomyosarcoma (RMS) is a highly aggressive form of cancer that arises from skeletal muscle progenitor cells, primarily affecting children. Despite advancements in treatment, RMS remains challenging to manage due to its propensity for metastasis and resistance to therapy. Consequently, there is a pressing need for innovative therapeutic approaches to combat this malignancy.

Methyl-β-cyclodextrin (MβCD), a derivative of cyclodextrin, has emerged as a promising candidate for RMS treatment. MβCD exerts its inhibitory effects on RMS cells through multiple mechanisms, including modulation of cellular cholesterol levels and disruption of lipid rafts.

Firstly, MβCD interferes with RMS cell proliferation by inducing cell cycle arrest and promoting apoptosis. By disrupting cholesterol-rich lipid rafts on the cell membrane, MβCD inhibits crucial signaling pathways involved in cell growth and survival, thereby suppressing RMS cell proliferation.

Secondly, MβCD hinders RMS cell migration, a critical step in cancer metastasis. Through its cholesterol-depleting activity, MβCD disrupts the formation of invadopodia, cellular structures essential for cancer cell migration and invasion. By inhibiting the activity of matrix metalloproteinases (MMPs), enzymes crucial for extracellular matrix degradation and tumor cell migration, MβCD impedes the migratory potential of RMS cells.

Lastly, MβCD attenuates RMS cell invasion, thereby inhibiting the spread of cancer to distant sites. By disrupting lipid raft integrity, MβCD interferes with the activation of signaling pathways implicated in tumor cell invasion. Additionally, MβCD suppresses the expression of genes associated with invasive phenotypes, further restraining RMS cell invasion.

Overall, Methyl-β-cyclodextrin demonstrates promising anti-cancer effects against RMS by targeting proliferation, migration, and invasion pathways. Further research is needed to elucidate its mechanisms of action and assess its therapeutic potential in preclinical and clinical settings.