New York, N.Y., January 23, 1998 - Memorial Sloan-Kettering Cancer Center (MSKCC) researchers delving into the fundamental mechanisms underlying one form of leukemia have learned how to interfere with the genetic changes that lead to this potentially fatal type of cancer. In laboratory studies, the investigators identified a combination of drugs that turn on proteins called transcription factors, which may be turned off in leukemia. The drug duo induces leukemia cells to mature and behave like normal, healthy blood cells. This finding, the first evidence supporting a new approach called "transcription therapy," may have broad implications for improving the treatment of other types of cancer that arise as a result of faulty gene transcription. The study was published in the February 1998 issue of the scientific journal Nature Genetics.
"This is exciting because we are taking our knowledge of how genes are regulated and using it to design an effective, targeted approach to treating cancer," said Dr. Pier Paolo Pandolfi, the geneticist who led the study. "We were able to show that this treatment was effective in mice, and we are planning to evaluate it soon in patients."
Specifically, Dr. Pandolfi and his team studied the molecular genetics underlying acute promyelocytic leukemia (APL), a type of leukemia that afflicts 2,000 to 3,000 Americans each year and has an especially abrupt onset. Five years ago, MSKCC investigators led by Dr. Raymond P. Warrell, Jr. showed that a drug called retinoic acid (RA) could be used to treat many patients with APL by inducing their cancer cells to mature, or differentiate, into healthy white blood cells. Differentiation therapy is an especially intriguing treatment approach, since it doesn't kill cells, but rather prompts them to "grow up" and behave normally.