Thanks to scientists at the Fred Hutchinson Cancer Research Center in Seattle, there is new hope for patients with active, advanced leukemia and myelodysplastic syndrome (a pre-leukemic condition), for which standard transplant therapy rarely works. All fifty-eight patients who came from around the world to participate in this Phase 1 clinical trial saw their blood cancers go into remission using a novel combination of low-intensity chemotherapy, targeted radiation delivery by an antibody and a stem-cell transplant. Forty percent of the patients were alive a year after treatment and approximately 35 percent had survived three years, about the same rates as patients who received similar treatment but whose disease was already in remission and who had much more favorable risk for relapse when therapy began. The purpose of the study was to find the maximum dose of radiation that patients could tolerate with acceptable toxic side effects—the more radiation that can be applied, the more cancer cells will be killed in preparation for donor stem cells to take over the diseased immune system and kill off the remaining cancer cells. Standard and low-dose therapies (a process sometimes known as a "mini transplant" and pioneered at the Hutchinson Center) used to kill leukemia cells in the bloodstream in preparation for a transplant usually require that patients be in remission. The patients in this study were in large part those with active relapsed disease that, in many cases, had failed to respond to standard therapies. "These were people who had extremely advanced high-risk disease. They were typically older—most of them were in their 60s and some were in their 70s—and had few or no other options for a potential cure. In fact most, if not all, would not been offered a stem cell transplant here or elsewhere. It is fair to say that these patients would likely have died without a transplant being performed if they had not been given the opportunity to participate in this study,” explained John Pagel, MD, PhD, a transplant oncologist and assistant member of the Hutchinson Center’s Clinical Research Division. Pagel said further research is needed to test more patients at the highest radiation dose both at the Hutchinson Center and at other transplant centers around the country.
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