After a Tragic Past, Thalidomide's Promising Future in Fighting Cancer Explored in Mayo Clinic Proceedings

Thursday, July 29, 2004

Media Contact:
Lynn Closway
Mayo Clinic
480-301-4222
closway.lynn@mayo.edu

For Immediate Release - July 29, 2004

SCOTTSDALE, Ariz. - From the late 1950s to the end of 1961, thalidomide was a popular sedative and treatment for morning sickness until it was discovered to cause fetal malformations, which proved fatal within the first year of life in 40 percent of affected infants.

The drug was never marketed in the United States or approved by the U.S. Food and Drug Administration. But researchers recognized the drug's properties might have cancer-fighting potential. This possibility has driven promising studies into thalidomide's role in fighting blood disorders, such as multiple myeloma, a deadly cancer for which there is no cure. An article in the July 2004 issue of Mayo Clinic Proceedings offers four studies that have probed thalidomide's promising future after its tragic past.

Multiple myeloma researcher Rafael Fonseca, M.D., site director for Hematology Malignancies and vice chair for Research for Hematology and Oncology at Mayo Clinic in Arizona, was a participant in these studies.

"We have been impressed by the newly-found ability of a drug otherwise forgotten as an effective therapeutic agent for myeloma," said Dr. Fonseca. "My colleagues and I have been working with new ways to use thalidomide for the treatment of patients, both at the time of diagnosis and when the disease recurs. We are happy to have an extra tool in our bag for the treatment of this serious disorder."

Multiple myeloma is a cancer of plasma cells, which are found in bone marrow, the blood producing tissue that fills in spaces within bone. Plasma cells produce antibodies (substances that help the bone fight infection). Typically plasma cells make up one to two percent of all cells in the bone marrow, but in a person with multiple myeloma, abnormal plasma cells (or myeloma cells) multiply in a cancerous manner. The growth of myeloma cells results in reduced production of blood, which produces anemia. Patients may also have associated destruction of bones.

The article in Mayo Clinic Proceedings includes studies and editorials regarding thalidomide and myeloma:

• Mayo Clinic researchers present findings in a long-term follow-up of patients with monoclonal gammopathy of undetermined significance (241 patients were examined at Mayo Clinic from 1956 to 1970) and found that the risk of progression to multiple myeloma or related cancer is indefinite and persists even after more than 30 years of follow-up. In an editorial, Joan Blade, M.D., a hematologist at Hospital Clinic IDIBAPS in Barcelona, Spain, writes that Robert Kyle, M.D., a Mayo Clinic hematologist who led the study, first recognized the possibility of malignant transformation of monoclonal gammopathy. Because of Dr. Kyle's detailed studies, physicians have learned about the actual outcome of persons with monoclonal gammopathy of undetermined significance, Blade writes.
• Mayo Clinic researchers report their findings in patients who have had relapses in multiple myeloma, noting that the response duration of the follow-up treatment or salvage therapy decreased. The researchers said this must be remembered when making treatment decisions for these patients and must be factored in when assessing the efficacy of new therapies.
• Researchers from Dana-Farber Cancer Institute in Boston; Celgene Corporation in Warren, N.J.; St. Vincents Catholic Medical Center in New York; and H. Lee Moffitt Cancer Center in Tampa studied the dosage of thalidomide for patients with relapsed multiple myeloma after high-dose chemotherapy and stem cell transplantation. They report that the optimal thalidomide dose varies and should be based on individual patients to ensure that it is well tolerated and a response is achieved.
• When thalidomide is used alone or combined with prednisone, it can also be an effective frontline treatment for symptomatic anemia or low platelets in patients with myelofibrosis with myeloid metaplasia, a chronic form of leukemia. The Mayo Clinic College of Medicine researchers outline their findings regarding 36 patients. In an editorial about this study, Richard Silver, M.D., of New York-Presbyterian Hospital-Weill Cornell Medical Center in New York, writes that the results provide an important therapeutic contribution in terms of quality of life. A peer-review journal, Mayo Clinic Proceedings publishes original articles and reviews dealing with clinical and laboratory medicine, clinical research, basic science research and clinical epidemiology. Copies of the articles are available online at www.mayo.edu/proceedings

Dr. Rafael Fonseca has received a joint appointment with Mayo Clinic in Scottsdale and TGen to lead a collaborative research project investigating biology, genetics and targeted treatment of multiple myeloma. This program is part of a research collaboration agreement signed by Mayo Clinic and TGen in Sept. 2003 to broaden areas of joint research endeavors.

Mayo Clinic is a private group practice of medicine dedicated to providing diagnosis and treatment of patient illnesses through a systematic focus on individual patient needs. As a leading academic medical center in the Southwest, Mayo Clinic focuses on providing specialty and surgical care in more than 65 disciplines at its outpatient facility in north Scottsdale and at Mayo Clinic Hospital. The 202-licensed bed hospital is located at 56th Street and Mayo Boulevard (north of Bell Road) in northeast Phoenix, and provides inpatient care to support the medical and surgical specialties of the clinic, which is located at 134th Street and Shea Boulevard in Scottsdale.

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