Myelodysplastic syndrome currently is untreatable and often leads to acute leukemia. Researchers involved with the study, which is being conducted in 15 medical centers across the nation, hope that decitabine, the proposed drug, will prevent this progression.
The physicians at the Lineberger Comprehensive Cancer Center said they are very excited about the study and hope it will indicate the benefits of decitabine that previous trials have.
"I hope it will be a home run," said Dr. Tom Shea, head of the Bone Marrow Transplant Center at UNC Hospitals. "Hopefully it will show some benefits, and if the drug cures anyone it will be outstanding."
Decitabine works by permitting bone marrow stem cells to mature at a normal rate instead of being hidden by carbon methyl groups that bind to the surface of DNA in patients with MDS.
Melissa Hall, the nurse coordinating the study, said there has already been a 30 to 50 percent response rate in phase one and phase two, trials that tested both the toxicities of the drug as well as its effectiveness. This new study -- phase three -- will compare the standard treatment, either chemotherapy or supportive care, to decitabine.
While there is no cure for MDS, patients usually are given treatments which include infusions of growth factor compounds to stimulate red and white blood cell production and also transfusions of platelets.
"What's so encouraging is that there is not a current treatment for MDS and to have any kind is exciting," Hall said. "We expect to receive patients from all over North Carolina and not just the Triangle area."
Without a cure, Shea said most individuals with the disease either develop acute leukemia or their disease worsens and they require more transfusions.
The study is headed by Beverly Mitchell, a professor at the School of Medicine and division chief of Hematology-Oncology.