• By mayonewsreleases

Three-Drug Combo Shows Promise Treating Rare Blood Disease

February 16, 2012

SCOTTSDALE, Ariz. — A powerful combination of three chemotherapy drugs used to treat the blood cancer multiple myeloma also shows promise for patients with the rare and deadly blood disease AL amyloidosis, a study led by Mayo Clinic shows. The results appear in the American Society of Hematology journal Blood.

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VIDEO ALERT: Click here to watch Dr. Mikhael explain the study.

AL amyloidosis is an incurable disease in which abnormal proteins produced in the bone marrow travel to major organs of the body, leaving deposits that typically damage the heart and kidneys. The median overall survival for those with this disease is approximately three years. For those where the disease causes heart damage, the overall survival is less than a year.

Researchers tried a three-drug regimen known as CyBorD, a combination of
cyclophosphamide, bortezomib and dexamethasone. They found it rapidly reduced abnormal proteins, was easier for patients to tolerate than chemotherapy previously used, was effective as a first-line treatment and in relapses, and could be given to patients whether they were eligible or ineligible for transplants.

"This novel drug combination was able to effectively shut off the production of the abnormal proteins," says lead researcher Joseph Mikhael, M.D., a hematologist at Mayo Clinic in Arizona.

Prior to the CyBorD regimen, those with AL amyloidosis had few effective treatment options to keep the disease in check, explains Dr. Mikhael. "What we found remarkable was how fast the combination worked — the average time to response was less than two months," he says. "Furthermore, over 90 percent of patients had at least a 50 percent drop in the protein, with over 70 percent having the protein disappear entirely."

Typically, treatment includes a lengthy regimen of chemotherapy and possibly a stem cell transplant for those few patients who meet the medical criteria to receive a transplant.

Media Contact: Jim McVeigh, Public Affairs, 480-301-4222


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