HP-β-CD (HP-β-CD) is a widely used drug delivery vehicle to improve stability and bioavailability.

Cell treatment with (2-hydroxypropyl)-β-cyclodextrin can activate the activation of the transcription factor EB (the main regulator of lysosomal function and autophagy) and enhance the cell's autophagy clearance ability[1]. HP-β-CD treatment reduces intracellular cholesterol resulting in significant leukemic cell growth inhibition through G2/M cell-cycle arrest and apoptosis. The IC50 values for HP-β-CD after 72 hours exposure are in the range of 3.86–10.09 mM. HP-β-CD also shows anticancer effects against CML cells expressing a T315I BCR-ABL mutation (that confers resistance to most ABL tyrosine kinase inhibitors), and hypoxia-adapted CML cells that have characteristics of leukemic stem cells. In addition, colony forming ability of human primary AML and CML cells is inhibited by HP-β-CD[2].

Intraperitoneal injection of HP-β-CD significantly improves survival in leukemia mouse models. Systemic administration of HP-β-CD to mice has no significant adverse effects[2].