US-based biopharmaceutical company Kura Oncology has dosed its first patient in a Phase II clinical trial of farnesyl transferase inhibitor, tipifarnib, to treat lower risk myelodysplastic syndromes (MDS).
The Phase II trial will include about 58 patients and has been designed to evaluate the efficacy of tipifarnib.
During the trial, the patient samples will be tested to determine the presence or absence of various biomarkers related to the activity of tipifarnib.
Kura Oncology chief medical officer Antonio Gualberto said: "We were encouraged by data from a previous Phase II trial of tipifarnib conducted by Johnson & Johnson that showed modest activity of this agent in the overall population of patients with intermediate to high-risk MDS.
"Using translational medicine methodologies, we identified potential predictive biomarkers of this activity, and one key goal of this study is to validate the ability of those biomarkers to identify the patients most likely to benefit from tipifarnib."
MDS represent a group of hematopoietic stem cell malignancies that may lead to mortality.
It is marked by ineffective blood cell production, or hematopoiesis, which leads to low blood cell counts, or cytopenias, and a high risk of progression to acute myeloid leukemia.
Columbia University Medical Center medicine professor and principal investigator Joseph Jurcic said: "Patients with MDS, a group of diverse bone marrow disorders, suffer from a dearth of treatment options and the development of new drugs is vital to combat the disease.
"The analyses from previous studies provide a strong rationale for evaluating tipifarnib in this indication."
Tipifarnib acts as an inhibitor of farnesylation, the cell which triggers the process to initiate cancer and its further development.
Image: CT scan displaying enlarged spleen due to myelodysplastic syndrome. Photo: courtesy of Tdvorak.