A drug being tested for breast cancer might help to block the progression of myeloproliferative neoplasms, and possibly become a treatment for acute myeloid leukaemia, researchers have reported.
Scientists studied the impact of inhibiting the protein RSK1 and report that it reduced inflammation as well as stopping the progression of myeloproliferative neoplasms (MPNs). The studies, conducted on patient samples and animal models, also suggest RSK1 inhibitors might treat an aggressive form of acute myeloid leukaemia (AML).
An inhibitor of the RSK family of proteins, PMD-026, is already undergoing clinical trials for breast cancer, they say.
The work at the Washington University School of Medicine, St Louis, USA, has been reported in Nature Communications and Blood Cancer Journal.
The study in Nature Communications reports that the inhibitor helped reverse the progression of myeloproliferative neoplasms in mice and reduced fibrosis in the bone marrow.
In Blood Cancer Journal, the researchers report testing the inhibitor on FLT3-ITD acute myeloid leukaemia, suggesting it may help overcome disease resistance to existing treatments.
Researcher Dr Stephen Oh said: “These patients [with myeloproliferative neoplasms] have no effective medical therapies, so we hope this new drug will help fill that gap in clinical care. At minimum, we’re hopeful this drug can stop the chronic disease from progressing to acute myeloid leukaemia. But the goal is to eliminate the disease and get patients into remission.”
He added: “We are excited about these studies because they highlight RSK1 as a novel therapeutic target for MPNs and AML with a viable strategy for moving an investigational drug into clinical trials in the near future."
Source:
Kong T, Laranjeira ABA, Letson CT, Yu L, Lin S, Fowles JS, Fisher DAC, Ng S, Yang W, He F, Youn M, Mark K, Jose AS, Liu J, Kim AB, Cox MJ, Fulbright MC, Jayanthan A, Los G, Rentschler SL, Ding L, Sakamoto KM, Dunn SE, Challen GA, Oh ST (2025) “RSK1 is an exploitable dependency in myeloproliferative neoplasms and secondary acute myeloid leukemia.” Nature Communications, 16 January 2025, doi: 10.1038/s41467-024-55643-7
Link: https://www.nature.com/articles/s41467-024-55643-7
Kong T, Laranjeira ABA, Letson CT, Yu L, He F, Jayanthan A, Los G, Dunn SE, Challen GA, Oh ST. (2024) “RSK1 dependency in FLT3-ITD acute myeloid leukemia.” Blood Cancer Journal, 25 November 2024, doi: 10.1038/s41408-024-01187-4.
Link: https://www.nature.com/articles/s41408-024-01187-4
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