Biopharmaceutical company, Cell Therapeutics, Inc. (CTI) has announced that the University of Washington (UW) has begun enrolling patients in a randomized phase II study testing the combination of tosedostat with either cytarabine or decitabine for elderly patients with newly-diagnosed acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS).

Drs John Pagel and Elihu Estey, from the University of Washington School of Medicine and Fred Hutchinson Cancer Research Centre, are leading the study.

“This is the first study to examine the effects of tosedostat in combination with either cytarabine or decitabine as a first-line therapy. The study will evaluate how well patients tolerate these combinations, and their effectiveness. Given that there have been no major advances in treatment of elderly patients with AML, and the results of a previous study of tosedostat by itself in relapsed or refractory patients with AML or MDS showed promising anti-leukaemic effects and acceptable tolerability, we are hopeful that this study will demonstrate that tosedostat increases the limited efficacy of these commonly used anti-leukaemic agents,” Dr Estey said.

The study's primary objectives are to determine the four-month survival and complete response (CR) rates of tosedostat in combination with either cytarabine or decitabine in untreated AML or high-risk MDS. The study's secondary objectives are to assess the safety and tolerability of tosedostat in combination with either cytarabine or decitabine, to determine the treatment-related mortality rate (defined as death within the first 30 days of beginning treatment), and to estimate the rates of disease-free survival, one-year survival, and overall survival.

Each arm of the study will enroll up to 20 patients, with the possibility of enrolling an additional five patients each (for a maximum of 25 per arm), if an arm is not closed. Those eligible for the trial are adults with newly-diagnosed AML (including those with high-risk MDS). Patients with certain favourable AML features would be excluded from the study.

Patients interested in participating in this study or wanting more information can call Carol Dean (FHCRC) at (206) 667-1926.

AML is a cancer characterized by the rapid growth of abnormal white blood cells that accumulate in the bone marrow and interfere with the production of normal blood cells. AML is the most common acute leukemia affecting adults, and its incidence increases with age.

The symptoms of AML are caused by replacement of normal bone marrow with leukemic cells, which causes a drop in red blood cells, platelets, and normal white blood cells leading to infections and bleeding. AML progresses rapidly and is typically fatal within weeks or months if left untreated. Although a substantial proportion of younger individuals who develop AML can be cured, AML in the elderly typically responds poorly to standard therapy with few complete remissions.

Tosedostat is an oral, aminopeptidase inhibitor that has demonstrated significant anti-tumor responses in blood-related cancers and solid tumors in phase I-II clinical trials. CTI has exclusive marketing and co-development rights to Chroma Therapeutics Ltd.'s drug candidate tosedostat in North, Central and South America.

CTI is a biopharmaceutical company committed to developing an integrated portfolio of oncology products aimed at making cancer more treatable.