Thalidomide may be a potent anti-cancer treatment

Jun 20 2005

When people hear "thalidomide," many think "birth defects," however, evidence has come to light that this once-banned drug can be used as a potent anti-cancer treatment.

In a new study, researchers from the University of Bologna, Italy, demonstrate that Thal-Dex (thalidomide used in combination with dexamethasone) is more powerful than conventional chemotherapy for the treatment of multiple myeloma.

Their findings will be published in the July 1, 2005, issue of Blood, the official journal of the American Society of Hematology.

Each year, approximately 15,000 Americans are diagnosed with multiple myeloma, an incurable and painful disease of the bone marrow. Most patients who receive this diagnosis have less than five years to live; however, many can undergo autologous (self-donor) stem cell transplants to help prolong survival. Although thalidomide has been studied for the treatment of advanced stages of multiple myeloma since the late 1990s, this is the first large study to compare its effectiveness to standard drugs as part of front-line therapy with stem cell transplantation.

Because the odds for a successful transplant increase as the number of cancer cells decreases, patients receive chemotherapy a few months before the procedure. In this study, 100 multiple myeloma patients given Thal-Dex before transplant were compared to 100 patients given traditional chemotherapy with VAD (a combination of three drugs: vincristine, adriamycin, and dexamethasone).

Thal-Dex patients were more likely to have successful results; 76 percent received at least a partial remission. Of the VAD patients, only 52 percent had a positive response to treatment, meaning that nearly half had either no response, or a progression of their disease. Patients taking Thal-Dex also had a more profound reduction in the size of their tumors.

Not only was Thal-Dex a more effective treatment, but its two-pill regimen is also greatly preferred by patients over intravenous VAD treatments. A vital consideration for patients undergoing autologous stem cell transplant is that toxicity from chemotherapy not impair the collection of stem cells needed for the transplant. In that respect, patients in both the Thal-Dex and the VAD group were able to collect enough healthy stem cells for transplantation and there was no significant difference between the two.

main side effect of Thal-Dex was deep vein thrombosis, which 15 percent of patients on the drug experienced. Although this effect was treated successfully with anti-coagulants, researchers speculate that further research needs to be done to help predict which patients will experience this outcome in order to find ways to make treatment with Thal-Dex safer for them. The main side effect of VAD was granulocytopenia (low white blood cell levels), which was severe in 12 percent of the patients in that group.

"It's time to look at thalidomide in a new light," said Michele Cavo, M.D., professor at the University of Bologna and lead study author. "It's earned its place in modern medicine. Thalidomide has proven to be a highly effective, relatively safe, and more comfortable treatment for patients with multiple myeloma than traditional chemotherapy."

This work was supported by grants from the University of Bologna, the Ministero dell'Università e Ricerca Scientifica, and the Carisbo Foundation.