In June, former vice presidential candidate Geraldine Ferraro revealed that she had been taking thalidomide for a rare blood cancer.
Myelodysplastic syndrome represents a group of disorders in which the bone marrow does not function normally and not enough red blood cells, white blood cells and platelets are present in the blood. These cells are the body's natural defense system against many diseases, including cancer.
Dr. Azra Raza, director of the Myelodysplastic Syndrome Center at Rush-Presbyterian-St. Luke's Medical Center administered thalidomide to 83 patients with myelodysplastic syndrome, which can lead to acute myeloid leukemia. In the study, 20 percent of patients responded favorably to treatment with thalidomide.
Raza believes that thalidomide works because it suppresses the production of cytokines - proteins that play a role in causing important bone marrow cells to "commit suicide" or aptosis - and blocks the formation of the blood vessels needed to make tumors grow. The drug also appears to have a positive impact on the body's immune system.
In the study, 83 patients received 100 mg of thalidomide daily, and the dose was gradually increased to a maximum of 400 mg per day. Before 12 weeks was up, 32 patients dropped out of the study due to unrelated medical conditions or adverse reactions to the medicine. Of the remaining 51 patients, 16 showed hematologic improvement as measured by increased platelets, decreased dependence on transfusions and improved erythroid series. Significantly, 10 previously transfusion-dependent patients become transfusion independent.
"These patients are now able to be free of regular blood transfusions that sustained them, thus improving their quality of life," Dr. Raza said.
For some people, the drug thalidomide is a painful reminder of one of medicine's darkest periods. In the 1950s and 1960s, thousands of babies were born with severe deformities, such as missing or misshapen limbs, as a result of pregnant women taking the morning sickness drug thalidomide. West German physicians estimated that 40,000 patients suffered from peripheral neuritis and 12,000 infants were deformed by thalidomide, 5,000 of whom survived past childhood, making it one of Medicine's worst nightmares.
It took only one tablet at the wrong time to cause the horrendous birth defects. Although it was pulled from the market in 1962 (it was never available in the United States), the drug has long been a source of great interest in the research community. And in recent years, it has been evaluated in the treatment of a variety of illnesses - from leprosy to AIDS to cancer. "The thought behind the research has been that, if thalidomide is powerful enough to stop limbs from growing, perhaps it can prevent cancer or other diseases from progressing," said Raza. Raza has had even more encouraging results in patients given thalidomide in combination with other treatments such as low-dose chemotherapy and other anticytokine agents, with a 40 to 50 percent response rate.
"Right now, the standard treatment for MDS is blood transfusion, which leads to accumulation of iron in the body and serious organ damage," Raza says. "Our hope is to find gentler, more effective treatments. Thalidomide may be part of that."
Raza is now working with an analog of thalidomide which is several thousand times more effective than the paneul compound and has none of its side effects.
"It will be an important day when we can consign thalidomide to the annals of history forever by replacing it with the more powerful and less toxic analogs," Raza said.
As for the risk of patients having children with birth defects, Raza has ensured that women in her studies are all postmenopausal. And when the time comes for younger women to be evaluated, pregnancy tests will be part of the screening process. "The drug company and researchers are taking every precaution to ensure safety," she says. "Our patients' safety is always our highest priority."
Additional Contact: John Pontarelli at 312-942-7820
Rush-Presbyterian-St. Luke's Medical Center includes the 824-bed Presbyterian-St. Luke's Hospital; 110-bed Johnston R. Bowman Health Center for the Elderly; Rush University (Rush Medical College, College of Nursing, College of Health Sciences and Graduate College); and seven Rush Institutes providing diagnosis, treatment and research into leading health problems. The medical center is the tertiary hub of the Rush System for Health, a comprehensive healthcare system capable of serving about two million people through its outpatient facilities and five memb
Journal
Blood