Doctors have been reporting “unprecedented success” in using gene therapy to transform the blood cells of patients blood cells into soldiers that seek and destroy leukemia.
"It's really exciting," exclaimed Dr. Janis Abkowitz, blood diseases chief at the University of Washington in Seattle and president of the American Society of Hematology. "You can take a cell that belongs to a patient and engineer it to be an attack cell."
In one study, all five adults and 19 of 22 children including 8-year-old Emily Whitehead of Philipsburg, PA. who disease was so advanced that her major organs were expected to fail within days, had complete remission, “meaning no cancer could be found after treatment.”
Prior to the gene therapy, each of the patients in the study had undergone multiple bone marrow transplants as well as 10 different types of chemotherapy to no avail. Despite the fact that 6 of the trial patients have had relapses, doctors are considering a second gene therapy attempt, while others have performed similar treatments for people suffering from lymphoma and myeloma.
In fact, Dr. James Kochenderfer and others at the National Cancer Institute have treated 11 patients with lymphoma and four with chronic lymphocytic leukemia for the past two years, and currently report that “six had complete remissions, six had partial ones, and one has stable disease.” Results regarding the others. will be presented at the hematology group's conference that starts Saturday in New Orleans.
If all continues to go well, the new treatment is expected to become the first gene therapy approved in the United States and the first for cancer worldwide. To date only one other gene therapy has been approved for a rare metabolic disease in Europe.