According to a story from GlobeNewswire, the biopharmaceutical company Onconova Therapeutics, Inc. has recently published results from a phase 1 clinical trial that was sponsored by the company. This trial tested a combination treatment of rigosertib and azacitidine as a treatment for acute myeloid leukemia and high-risk myelodysplastic syndromes. The results suggest that further investigation of this combination in myelodysplastic syndromes could be useful.
About Myelodysplastic Syndromes (MDS)
Myelodysplastic syndromes are a type of blood cancer in which developing blood cells remain immature and fail to transform into usable blood cells. Risk factors for this disease include exposure to radiation, chemotherapy, benzene, xylene, and Agent Orange. Family history is also a risk, as are certain genetic disorders such as Down syndrome. In an individual case, it is rare for the direct cause to be identified. Myelodysplastic syndromes rarely present with symptoms initially, but it can eventually present with anemia, neutropenia, thrombocytopenia, cell abnormalities, chromosome abnormalities, enlarged spleen and/or liver, easy bleeding and bruising, and infections. The disease also has the potential to evolve into acute myeloid leukemia. Treatment may include bone marrow transplant, stem cell transplant, blood products, and certain chemotherapy agents. Outcomes in this disease ranges widely and can depend on a number of factors. To learn more about myelodysplastic syndromes, click here.
About the Trial
The company also released some early data from an ongoing phase 2 study. In phase 1, which featured a dose-escalating design in order to determine the optimal dose for future testing, the combination was able to produce a 78 percent response rate in myelodysplastic syndromes (nine patients) compared to 29 percent in acute myeloid leukemia (seven patients). There were a total of three different dose cohorts in the trial and no dose-limiting adverse effects were reported.
About Rigosertib
Rigosertib is the company’s leading therapy candidate. The drug is orally available and is designed to target the RAS effector pathways. The drug is currently being developed for intravenous and oral administration. It’s availability by the oral route makes it an appealing candidate for combination treatments such as alongside azacitidine.
While the study was small in scale and was only an early phase assessment, these early findings are nevertheless encouraging, and rigosertib could one day become an important component of treatment for high risk myelodysplastic syndromes.
