A cure at last? New treatment options for an incurable paediatric leukemia

Researchers from the University Children’s Hospital in Zurich, in collaboration with the Bioinformatics Core Facility of the Swiss Institute of Bioinformatics, analysed the drug activity profiles for patient-derived leukemia xenografts (PDX) of a rare, but fatal pediatric acute lymphoblastic leukemia (ALL).

The drug panel consisted of one hundred drugs, including novel chemotherapeutics that are undergoing clinical development. Striking and specific responses were found for venetoclax (ABT-199), a drug that specifically targets the BCL2 protein, and which has been shown to work in other cancer strains. Follow-up work in mouse models have shown that the drug induces remission when combined with backbone chemotherapy for ALL.
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