Dosing & Uses
Myelodysplastic Syndromes
Pending FDA approval for treatment of transfusion-dependent anemia in adults with low-to-intermediate risk myelodysplastic syndromes (MDS) who have failed to respond or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESAs)
Pharmacology
Mechanism of Action
Binds to telomerase and inhibits its activity, selectively killing malignant stem and progenitor cells in the bone marrow that are the source of disease in blood cancers
Telomeres are repetitions of a DNA sequence located at the end of chromosomes; normally, every time a cell divides, the telomeres shorten; eventually, they shrink to a critically short length, which prevents it from further cell division or leads to cell death (apoptosis)
Telomerase is a naturally occurring enzyme that maintains telomeres and prevents them from shortening during cell division; telomerase is upregulated in many malignant stem and progenitor cells, enabling the continued and uncontrolled proliferation of those malignant cells, driving tumor growth and progression
