A new drug, called PF-04449913, which was found promising for treating blood cancer in its first human trial is now in phase II clinical trials, revealed a new study. PF-04449913 coaxes dormant cancer stem cells, residing in the bone marrow, to begin differentiating and exit into the blood stream where they can be destroyed by chemotherapy agents.
Study's senior author Catriona Jamieson, associate professor of medicine at University of California, San Diego School of Medicine in the US, said, "This drug gets that unwanted house guests to leave and never come back. It is a significant step forward in treating people with refractory or resistant myeloid leukemia, myelodysplastic syndrome and myelofibrosis. It is a bonus that the drug can be administered as easily as an aspirin, in a single, daily oral tablet."
For the first study conducted between 2010-2012, the drug was tested in 47 adults with blood and marrow cancer. The patients received escalating daily doses of the drug in 28-day cycles. The treatment cycles were repeated until a participant experienced unacceptable adverse effects without evidence of clinical improvement. The drug elicited clinical activity sufficient to establish proof-of-concept for the treatment in 23 individuals, or nearly half the study subjects.
Given the promising results in the phase I trial, the drug's efficacy as a treatment for different types of blood cancer is now being investigated in five phase II clinical trials. Jamieson said, "Our hope is that this drug will enable more effective treatment to begin earlier and that with earlier intervention, we can alter the course of disease and remove the need for, or improve the chances of success with, bone marrow transplantation."
The study appeared online in the The Lancet Hematology.