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Predicting the Behavior of Amyloid Disease

Marina Ramirez-Alvarado, Ph.D.

Our bone marrow produces antibodies designed to fight off disease. Mayo Clinic Researcher Marina Ramirez-Alvarado, Ph.D. likens the proteins that form antibodies to strands of spaghetti. In order to function correctly, they must twist into a certain configuration. If the protein misfolds, the flawed antibodies accumulate in the bloodstream and eventually in the heart, kidneys, nerves, liver and spleen. Eventually, these organs stop functioning.

Dr. Ramirez-Alvarado and a team of researchers are studying protein kinetics, to understand how proteins find the proper shape or misfold. The work depends on samples donated by patients with light-chain amyloidosis, a disease without a cure.

"One of the reasons Mayo is a worldwide leader in treating this condition is that we see a large number of patients with amyloid disease," says Dr. Ramirez-Alvarado. "Basic scientists and clinical hematologists at Mayo meet each month; we get incredible feedback from our clinical colleagues in our effort to find a cure."

Two of those hematologists are Morie Gertz, M.D. and Angela Dispenzieri, M.D. Their goal is to determine whether high dose chemotherapy in combination with stem cell transplant is superior to conventional chemotherapy.

"We will enable our researchers to predict the behavior of amyloid disease and determine the best treatment options," says Dr. Gertz. "Working together as a team has become a major strength for us in researching this condition."

The trial, which started in 2005, is in the second year of a grant from the National Institutes of Health. From the lab to the bedside, Mayo researchers remain focused on the needs of the patient.