Stem Cell Procedure Successfully Treats Amyloidosis Patients

AL amyloidosis occurs when clonal plasma cells in bone marrow produce proteins that misfold and deposit in tissues, leading to organ failure and death. Between 1,200 and 3,200 new cases are reported each year in the United States, although researchers believe the disease is highly underdiagnosed.

To determine whether a second course of this treatment could be beneficial, the researchers enrolled 62 patients on this trial, of whom 17 patients received a second course of high-dose chemotherapy and blood stem cell transplantation who had not received a complete remission from their initial treatment. Of the 17, five (31 percent) were successfully treated and achieved a complete hematologic remission of their amyloidosis after receiving a second course of treatment.

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

“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 study was supported by grants from the National Institutes of Health, Food and Drug Administration, the Gerry Foundation, and the Amyloid Research Fund at Boston University.