Adding thalidomide to a standard regimen of high-dose chemotherapy in patients with the cancer multiple myeloma improved complete responses but did not give any advantage in five-year survival, according to a study published in the New England Journal of Medicine (March 9).
The study of 668 patients with newly-diagnosed multiple myeloma compared treatment with two courses of Celgene’s Alkeran (melphalan) to the combination of Alkeran and Celgene’s Thalomid brand of thalidomide alongside autologous stem cell transplantation.
The five-year study revealed that the complete response rate among the Thalomid group, defined as the disappearance of malignant cells in the blood, was 62%, significantly higher than the 43% seen in the control group. Thalomid was also associated with higher event-free survival at five years (56% versus 43%), but the overall survival rate was no different at 65% a piece.
The combination treatment was also associated with higher rates of blood clots and damage to the nerves. Last year, Celgene was forced to discontinue a trial of a thalidomide derivative – Revlimid (lenalidomide) – in multiple myeloma patients after patients developed blood clots. The latter drug was approved for another haematological disease, myelodysplastic syndromes – in December 2005, and has been fast-tracked as a multiple myeloma treatment by the US Food and Drug Administration (FDA).
Thalidomide, originally used as a treatment for morning sickness, was taken off the market in the 1960s because women who took it during pregnancy had a much higher rate of severe birth defects. However, studies suggesting it might help against cancer led to its reintroduction - with strict controls – for treating complications of leprosy in 1998.
In 2005, the FDA issued an approvable letter for Thalomid in the treatment of multiple myeloma, asking for additional information on the drug. Celgene said in January it had submitted the data and was now hoping for a verdict on its marketing application in the first half of this year. Sales of Thalomid were $388 million last year.