Early Stage Drug Cancer, Bone Marrow Shows Promise

Ongoing clinical trial testing guadecitabine in Myelodysplastic syndromes and acute myelogenous leukemia.

While still in early testing, an experimental drug that treats cancer and bone marrow disorders offered hope in early testing.

Researchers from Temple University Hospital and Fox Chase Cancer Center conducted an international phase 1, randomized clinical trial evaluating guadecitabine (SGI-110) in the treatment of Myelodysplastic syndromes (MDS), a disorder where bone marrow fails to produce enough healthy blood cells, and acute myelogenous leukemia (AML).

The next generation small molecule treatment limits DNA methylation, a mechanism utilized by cells to control gene expression. Abnormal DNA methylation is linked with a greater malignancy rate.

SGI-110 seeks to reverse aberrant DNA methylation.

Published recently in Lancet Oncology, researchers noted the study was the first time the drug was administered to human patients. Investigators sought to evaluate the safety and efficacy of SGI-110 in patients diagnosed with MDS or AML.

The researchers found SGI-110 is both easy to administer and well-tolerated. Additionally, the drug was found to be biologically and clinically active in treating MDS and AML.

The researchers also found that reversing DNA demethylation as a result of treatment with SGI-110 was associated with clinical response in patients receiving the drug. Patients who responded to treatment showed significantly more demethylation than did those who did not respond.

"This means that the drug is safe and those patients who had more changes of their epigenome responded more to the drug," said lead author Jean-Pierre Issa, MD.