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Findings from a study at the Dana-Farber Cancer Institute in Boston show that a new oral chemotherapy drug called PKC 412 may reduce the number of blasts in patients with acute myelogenous leukemia (AML), a cancer that attacks the cells in the blood, bone marrow, and lymph nodes.
This phase II clinical trial involved 14 patients with advanced AML who received PKC 412 in pill form three times a day.
The study results showed:
- In 12 of 14 patients, the number of blasts circulating in their blood decreased by more than 50%. In two of these 12 patients, blasts completely disappeared.
- Five patients experienced a reduction of more than 50% of the number of blasts in their bone marrow. One out of the five patients had their blood counts return to normal three months after starting PKC 412 treatment.
"We're very encouraged by the patients' initial responses to PKC 412," said the study's lead investigator, Richard Stone, MD, Dana-Farber Cancer Institute. "This could be the start of truly targeted therapy for AML."
All of the patients involved in the study were known to have a genetic abnormality of the FLT3 gene. In patients with an abnormal FLT3 gene, a protein that normally switches on and then off during blood cell maturation, never turns off. This abnormality has been directly linked to AML in one-third of all adult AML patients. The drug PKC 412 is designed to block the FLT3 gene.
Based on the promising results of the current study, Dr. Stone and his colleagues have expanded the trial and have long-term plans to use the drug in combination with chemotherapy (the standard treatment) in patients with AML, as well as in patients who do not have an abnormality in the FLT3 gene.