Researchers Discover Novel Drug Treatment for Patients with Amyloidosis

in Health & Medicine, News Releases, School of Medicine
September 8th, 2006

Contact: Kristen Perfetuo, 617-638-8491 | kristen.perfetuo@bmc.org

(Boston) – Researchers in the Section of Hematology-Oncology and the Amyloidosis Treatment and Research Program at Boston University Medical Center (BUMC) recently discovered that lenalidomide, a relative of the controversial drug thalidomide, can help treat patients with primary amyloid light chain (AL) Amyloidosis.

The finding, which appears prepublished in the First Edition of Blood and can be viewed on Blood Online at www.bloodjournal.org/papbyrecent.shtml, could offer an alternative treatment for patients diagnosed with the rare blood disease.

AL amyloidosis occurs when 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. Until recently, the only known effective treatments for the disease were various forms of chemotherapy.

According to Vaishali Sanchorawala, MD, lead author of the study and associate professor of medicine at Boston University School of Medicine, treatment regimens used in multiple myeloma, a disease that causes plasma cells in bone marrow to grow out of control, can also be effective in treating patients with AL amyloidosis.

“Previous studies show thalidomide and lenalidomide have been effective in treating patients with multiple myeloma, a disease similar to AL amyloidosis,” said Sanchorawala. “However, thalidomide is poorly tolerated in patients with AL amyloidosis, causing severe fatigue, constipation, neuropathy and exacerbation of cardiac and renal dysfunction.”

To determine whether lenalidomide, also known as Revlimid, would be more effective and tolerable for AL amyloidosis patients, BUMC researchers analyzed 34 patients diagnosed with the disease who underwent the drug therapy.

“In contrast to thalidomide, we found lenalidomide to be much better tolerated by AL amyloidosis patients,” said Sanchorawala. “The hematologic responses produced by this regimen were associated with improvement in organ dysfunction caused by amyloid disease. Our study shows lenalidomide is a useful, new non-chemotherapy option for treatment of this life-threatening blood disorder.”

Lenalidomide is a relative of thalidomide, a drug that achieved notoriety in the 1950’s for causing birth defects.

“It is remarkable that lenalidomide is proving to be an effective treatment for a wide range of bone marrow and blood diseases,” added Sanchorawala.

Researchers note further follow-up and larger clinical trials will be required to determine if this treatment-related response is associated with improved survival.

The study was supported with funds from the National Institutes of Health, the Celgene Corporation, the Gerry Foundation and the Amyloid Research Fund at Boston University.

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