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Targeting protein tyrosine phosphatase non-receptor type 2 with a novel inhibitor for AML therapy

Kuang, W.; Jiang, J.; Wang, X.; Wang, D.; Ji, M.; Zhu, Y.; Yuan, K.; Ding, J.; Wang, W.; Song, M.; Min, W.; Huang, F.; Wang, L.; Gu, W.; Hao, H.; Xiao, Y.; Yang, P.

2024-05-10 cancer biology
10.1101/2024.05.07.592940 bioRxiv
Show abstract

Acute myeloid leukemia (AML) is a fatal disease characterized by a bleak prognosis. For over four decades, treatment options for AML have been constrained to administering high-dose cytotoxic chemotherapy. However, the emergence of drug resistance and the resultant toxic side effects have created an urgent necessity for identifying novel therapeutic targets. In this study, non-receptor protein tyrosine phosphatase type 2 (PTPN2) is highly expressed in AML. Remarkably, knock down of PTPN2 expression markedly mitigated AML burden both in vitro and in vivo. Additionally, we unveiled the direct interaction between PTPN2 and C-MYC, establishing C-MYC as a direct acting substrate of PTPN2. Then, we performed a small-molecule compound screening to identify K73, a selective inhibitor of PTPN2. At the same time, K73 showed a good safety profile and exhibited strong activity against AML in vivo. In conclusion, we have pinpointed a considerable therapeutic potential in targeting PTPN2 for AML treatment and discovered a novel class of selective PTPN2 inhibitors suitable for AML therapy.

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