Phase I trials of the lysine-specific demethylase 1 inhibitor, GSK2879552, as mono- and combination-therapy in relapsed/refractory acute myeloid leukemia or high-risk myelodysplastic syndromes.

Two patients achieved a morphologic leukemia-free state at the 12 mg dose level and with GSK2879552 + ATRA combination therapy, and improvement in leukemia cutis was observed in one patient following treatment with GSK2879552 12 mg (Supplementary Figure 2). Among the 10 patients who received GSK2879552 + ATRA combination therapy, treatment-related Grade 2 pruritus led to treatment discontinuation due to investigator discretion in one patient. Outcomes remain poor for patients with advanced myelodysplastic syndrome (MDS) or relapsed/refractory acute myeloid leukemia (AML) [[1]] due to a lack of effective treatment regimens [[3]], highlighting an unmet clinical need for novel treatments for these patients. [Extracted from the article]