A Phase 2 study found that the drug Velcade had limited efficacy in myelodysplastic syndromes patients.

However, the study authors suggested that Velcade’s efficacy in myelodysplastic syndromes (MDS) may be improved in the combination with other drugs.

Few treatment options exist for myelodysplastic syndromes (MDS) patients besides stem cell transplantation. Transplantation is often not an option because a donor match cannot be found or the patient is not healthy enough.

Velcade (bortezomib), which is manufactured by the pharmaceutical company Millenium Pharmaceuticals, is a drug that kills cancer cells. It is currently approved for the treatment of multiple myeloma, a cancer that affects the plasma cells.

Velcade is capable of blocking a protein that is activated in MDS, particularly higher risk populations, and that may be a cause of the disease.  The study authors therefore hypothesized that Velcade may be a potential treatment for MDS.

In this study, researchers investigated the efficacy and safety of Velcade in 16 lower-risk and three higher-risk MDS patients.  For various reasons including vascular disease, disease progression, and intestinal hemorrhage, only six patients received all eight planned cycles of the drug.

The researchers observed a red blood cell response in four patients, all of whom were lower risk.

None of the six patients who received all eight planned treatment cycles achieved a white blood cell or platelet response.

Of the nineteen patients, nine remained in stable disease.

All patients experienced blood-related side effects as a result of the treatment, with four patients experiencing severely low white blood cell levels and six experiencing severely low platelet levels.

Other side effects such as diarrhea, fever, rash, and pneumonia were observed in seven patients.

For further information, please see the study in Leukemia Research.