Alicia Perzolli: Excited to share our latest publication on PROTACs in pediatric acute myeloid leukemia
Alicia Perzolli, PhD Student at Princess Maxima Center, shared a post on LinkedIn about a recent paper by her and colleagues published in Cancers 2025:
“I am very excited to share our latest publication on PROTACs in pediatric acute myeloid leukemia!
Proteolysis-targeting chimeras (PROTACs) are a groundbreaking technology that degrade harmful proteins driving cancer. While PROTACs have shown immense promise in various cancer types, their application in pediatric acute myeloid leukemia (AML) has been relatively unexplored until now.
In this study, we evaluated two PROTAC drugs, CC-90009 and GU3341, both of which target the degradation of the protein GSPT1. The treatment effectively inhibited tumor growth, suppressed cancer cell proliferation, and induced cell death.
Notably, it showed pronounced efficacy in two subtypes of pediatric AML driven by specific gene fusions: RUNX1::RUNX1T1 and FUS::ERG. We also observed that GSPT1 degradation reduced the levels of these leukemia-causing gene fusions, further underscoring GSPT1’s role in the disease and its potential as a therapeutic target.
This work highlights an exciting new approach for treating pediatric AML and demonstrates the potential of PROTACs to target proteins critical for leukemic cell survival.
A huge thanks to my co-authors and collaborators for their contributions and support throughout this journey.”
“PROTAC-Mediated GSPT1 Degradation Impairs the Expression of Fusion Genes in Acute Myeloid Leukemia”
Authors: Alicia Perzolli, Christian Steinebach, Jan Krönke, Michael Gütschow, C. Michel Zwaan, Farnaz Barneh, and Olaf Heidenreich
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