Treatment of acute leukemia with methylglyoxai-bis-guanylhydraxone (methyl GAG)

Methyl GAG was administered to 83 patients with acute leukemia. Significant antileukemic activity was identified in patients with acute myeloblastic leukemia who received the drug in an average daily dose of 126 to 160 mg. per square meter (45 per cent complete remissions). The remission rate is higher than that reported for other agents and is reflected by the median increase in survival from 2.5 to 6.5 months when compared to patients with myeloblastic leukemia treated with 6-mercaptopurine. Adverse effects of methyl GAG are severe, and include mucosal ulcerations, diarrhea, phlebitis, desquamation of feet, furunculitis, and marrow hypoplasia. The effective dose range is quite narrow; minor alterations in dose result in either greater toxicity or decreased antileukemic effect. Leukemic cells of patients with the highest response rate to methyl GAG (72 per cent) were characterized morphologically by Auer rods and/or significant granulation. Remissions lasted 4 months (median) and 4 patients had reinduction by a subsequent course. Methyl GAG has antileukemic activity which, although associated with substantial toxicity, is unique in the treatment of acute myeloblastic leukemia.