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Busulfan Improves Outcomes for Stem Cell Transplant Patients

Conquest - Summer 2010

Early in his career, Borje S. Andersson, M.D., Ph.D., believed in the potential of busulfan as a preparative agent in stem cell transplantation. The problem was its potency. In solid form it worked to help patients’ bodies accept stem cells from outside donors. But swallowing the drug meant it had to pass through the digestive system where too much or too little could be absorbed by the liver.

Borje S. Andersson, M.D., Ph.D.

When two of his patients died of liver failure in 1989-1990 due to the damage caused by this agent, he knew he had a mission.

“The problem was that no one had discovered how to dissolve busulfan and keep its effectiveness stable so that it could safely and predictably enter the blood stream with the same potency as the oral (pill) form,” he says.

By 1993, Andersson, professor in MD Anderson’s Department of Stem Cell Transplantation and Cellular Therapy, had found a way through animal studies to keep busulfan stable in solution. By 1995, the drug moved forward into Phase I and Phase II studies. And on Feb. 4, 1999, the U.S. Food and Drug Administration approved it for allogeneic (unrelated donor) stem cell transplants for chronic myeloid leukemia patients.

Drug dramatically changed patient outcomes

Given to patients intravenously in combination with other drugs, commonly cyclophosphamide, or FludaraTM (fludarabine) or ClolarTM (clofarabine), busulfan has dramatically increased the safety of the transplant procedure. And it has reduced the risk of dying from complications in the first 100 days after transplant
from about 30% to 40% to about 3%.

This pre-transplant treatment has received regulatory approval for use in 45 countries, with the drug available on a limited basis in an additional three countries. In 2008, worldwide sales of busulfan were approximately $40 million with a royalty sharing benefit to MD Anderson, some of which is used to fund continued research.

For this remarkable achievement that has benefited thousands of patients, Andersson was honored in March with the 2010 Chancellor’s Innovation and Entrepreneurship Award. Established to promote a culture of entrepreneurship throughout The University of Texas System, the award recognizes researchers who exemplify ingenuity, creativity and innovation in translating research into useful products and services.

© 2015 The University of Texas MD Anderson Cancer Center