According to the company, these two disease conditions may represent a continuum in malignant progression of the abnormal production of blood cells in the bone marrow that results in a rapidly progressing form of leukemia. Patients that have myelodysplastic syndrome (MDS) which progresses to acute myeloid leukemia (AML) have been identified as an especially high-risk group for poor survival.
The clinical study, which will be sponsored by the US National Cancer Institute (NCI) under an agreement with Lorus, will be conducted by Dr Mark Kirschbaum, director of new drug development at the City of Hope National Cancer Center in Duarte, California.
“The extension of the GTI-2040 AML program to include MDS is consistent with the already announced priority focus by Lorus on GTI-2040 in AML,” said Dr Jim Wright, the CEO of Lorus.
GTI-2040 is an antisense oligonucleotide complementary to the R2 component of ribonucleotide reductase, an activity that is essential for DNA synthesis and tumor growth. R2 is frequently over-expressed in cancer cells, and has shown an ability to cooperate with a variety of oncogenes to increase the malignant potential of cancer cells.