Methods of treating myelodysplastic syndrome

A method of treating myelodysplastic syndrome (MDS) in a subject, the method comprising:

• classifying the subject as having a reduction in human telomerase reverse transcriptase (hTERT) expression level in a biological sample of target cells after administration of the telomerase inhibitor as compared to a baseline expression level prior to administration of the telomerase inhibitor, wherein a 50% or greater reduction in hTERT expression level in the biological sample after administration of the telomerase inhibitor identifies the subject as having an increased likelihood of benefiting from treatment with the telomerase inhibitor;
• and
• administering to the subject an effective amount of a telomerase inhibitor, wherein the administering is effective for treating MDS in the subject, and
• wherein the telomerase inhibitor is imetelstat or a pharmaceutically acceptable salt thereof.

A method of monitoring therapeutic efficacy in a subject with myelodysplastic syndrome (MDS), the method comprising:

• measuring human telomerase reverse transcriptase (hTERT) expression level in a biological sample of target cells obtained from the subject after administration of a telomerase inhibitor;
• and
• comparing the hTERT expression level in the biological sample to a baseline hTERT expression level prior to administration of the telomerase inhibitor;
• wherein a 50% or greater reduction in hTERT expression level in the biological sample after administration of the telomerase inhibitor identifies a subject who has an increased likelihood of benefiting from treatment the telomerase inhibitor, and
• wherein the telomerase inhibitor is imetelstat or a pharmaceutically acceptable salt thereof.

A method of identifying and treating a subject with myelodysplastic syndrome (MDS) with a telomerase inhibitor, the method comprising:

• diagnosing the subject with trisomy 8, wherein the diagnosis of trisomy 8 in the subject identifies the subject having an increased likelihood of benefitting from treatment with the telomerase inhibitor;
• and
• administering to the subject with trisomy 8 an effective amount of the telomerase inhibitor;
• wherein the administering is effective for treating MDS in the subject, and
• wherein the telomerase inhibitor is imetelstat or a pharmaceutically acceptable salt thereof.