Specific treatments for (pediatric) acute myeloid leukemia (AML) are lacking.
Standard treatments still include intensive myelosuppressive chemotherapy, which is unsatisfactory in terms of general toxicity, with the persistent risk of relapse due to the escape of quiescent leukemic cells to chemotherapy. Importantly, only 11 to 23 % of pediatric patients diagnosed with the most aggressive leukemia subtype harboring a known genetic rearrangement are designated as event-free survivors at 5 years.
Our invention relates to novel analogs of thioridazine, that lack a characteristic CNS toxicity of the parent molecule, for a targeted treatment of the most aggressive pediatric subtype of acute myeloid leukemia (t(6;11)AML featuring disease free survival at 5 years of only 11-23%).

TRL

Lead Optimization/Preclinical

Inventors

Martina
Pigazzi

Claudia
Tregnago

Romeo
Romagnoli

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