In Early Tests, Blood, Bone Marrow Rx Sparked Hope

Guadecitabine (SGI-110) showed promise for myelodysplastic syndromes, acute myelogenous leukemia in phase I trial

/ Author:  / Reviewed by: Joseph V. Madia, MD Beth Bolt, RPh

(RxWiki News) Early testing of a medication for blood cancers and bone marrow disorders yielded promising results. Now, researchers are looking to the future.

In a phase I trial, researchers found that guadecitabine (SGI-110) had an effect that could one day help patients with myelodysplastic syndromes (MDS) and acute myelogenous leukemia (AML). According to these researchers, a currently unpublished phase II trial (which reportedly produced more promising results) is complete, and a phase III trial is already underway.

In a phase I trial, a small group of patients receives a new treatment. Researchers study the treatment's safety, effectiveness and side effects. Phase II trials are similar, except they involve a larger group of test patients. Phase III trials involve an even larger patient group, and researchers compare the new treatment to commonly used treatments while gaining more insight into the treatment's safety.

"The most exciting outcome of these studies is that this drug also started a new phase III clinical trial, and if successful, it can lead to US Food and Drug Administration approval to treat leukemia patients," said lead study author Jean-Pierre Issa, MD, director of the Fels Institute for Cancer Research and Molecular Biology at Temple University School of Medicine, in a press release.

In the recently published phase I trial, Dr. Issa and team gave SGI-110 to 93 patients — 74 of whom had AML and 19 of whom had MDS — from 13 cancer centers in the US and Canada. AML is a type of cancer that can affect patients' blood and bone marrow. MDS are bone marrow disorders that prevent patients' bone marrow from producing enough blood cells.

"The goal was to measure the safety in patients diagnosed with MDS or AML and determine whether the drug changed the patients' epigenetic bookmarks," Dr. Issa said.

By changing the patients' "epigenetic bookmarks," Dr. Issa was referring to the biological activity of the drug.

In addition to appearing effective, SGI-110 was easy to administer and not hard on patients' bodies, these researchers said.

"This means that the drug is safe and those patients who had more changes of their epigenome responded more to the drug," Dr. Issa said.

While these results were promising, testing is still in its very early stages. If proven safe and effective in further study, SGI-110 still may not be available to patients for months or years.

This study was published Aug. 19 in The Lancet Oncology.

Astex Pharmaceuticals, which manufactures SGI-110, funded this research. Study authors disclosed ties to pharmaceutical companies like GlaxoSmithKline, Janssen, Astex, Roche and Novartis, among others.

Review Date: 
August 21, 2015
Last Updated:
August 30, 2015