Amyloidosis Patients Treated Successfully with Stem Cells

Boston University, July 3, 2007

Patients with immunoglobulin-light chain (AL) Amyloidosis who did not respond to initial treatment with high-dose chemotherapy and blood stem cell transplantation can be helped by utilizing tandem cycles of the treatment. The finding, which was published in the June 25th issue of Bone Marrow Transplantation on-line, was made by researchers from the Stem Cell Transplant Program and the Amyloid Treatment and Research Program at Boston University Medical Center (BUMC).

Leading to organ failure and death, when clonal plasma cells in bone marrow produce proteins that misfold and deposit in tissues, the condition is called AL amyloidosis. Researchers believe the disease is highly under-diagnosed, despite the fact that in the United States between 1,200 and 3,200 new cases are reported each year.

17 patients who had not achieved a complete remission from their initial treatment out of the initial 62 enrolled in the trial received a second course of high-dose chemotherapy and blood stem cell transplantation. This was the process used to determine whether or not a second course could be beneficial. After receiving a second course of treatment, a complete hematologic remission of their amyloidosis was achieved by 5 of the 17 patients, equating to a 31 percent success rate.

This approach appears to be associated with an improvement in overall survival as it increases the proportion of patients who ultimately achieve a complete response stated lead researcher, Vaishali Sanchorawala, MD, who is the clinical director of the Stem Cell Transplant Program, section of hematology/oncology at BUMC and associate professor of medicine at Boston University School of Medicine.

“For patients who can tolerate this treatment approach, such treatment appears to result in the highest complete hematologic response rate of any treatment for AL amyloidosis to date,” said Sanchorawala. “In addition, patients showed improvements in both organ function and quality of life,” she added.

The Amyloid Research Fund at Boston University, Food and Drug Administration, Gerry Foundation, and National Institutes of Health, all supported the research through grants.