Drug in Clinical Trials for Breast Cancer Shows Promise for Treating Blood Cancers

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Prime Highlights:

Washington University research identifies a potential new use for an existing drug.

RSK1 inhibitor shows promise in treating myeloproliferative neoplasms (MPNs) and acute myeloid leukemia (AML).

The drug, PMD-026, is already in clinical trials for breast cancer and may soon be tested for blood cancers.

Key Background:

Two new studies led by researchers at Washington University School of Medicine in St. Louis have uncovered a potential therapeutic strategy for treating certain blood cancers, using a drug already undergoing clinical trials for breast cancer. The studies, published in Nature Communications and Blood Cancer Journal, explore the role of the RSK1 protein in the progression of myeloproliferative neoplasms (MPNs) and acute myeloid leukemia (AML). By inhibiting RSK1, the drug PMD-026 shows promise in halting cancer progression and could offer new treatment options for these aggressive blood cancers.

MPNs are slow-growing blood cancers that can lead to secondary AML, a highly aggressive and difficult-to-treat form of leukemia. Current treatments for MPNs are limited to symptom management, and there are no effective therapies to prevent the disease from progressing to AML. Senior author Dr. Stephen T. Oh, a hematology expert at Washington University, emphasizes the hope that the RSK1 inhibitor may not only halt MPN progression but potentially lead to remission for patients, ultimately improving long-term survival outcomes.

In mouse models, PMD-026 was shown to significantly reduce fibrosis and cancerous growth in bone marrow, with up to 96% of cancer cells eliminated within four weeks. The drug also prevented MPN from transforming into secondary AML. Additionally, in the case of FLT3-ITD AML, which commonly develops resistance to existing therapies, the RSK1 inhibitor could offer an alternative approach by targeting a different cancer pathway.

Currently in clinical trials for metastatic breast cancer, PMD-026 has shown promising safety and tolerability profiles. Researchers are now exploring the potential of moving forward with trials for blood cancers, with the goal of providing a treatment for patients who are not eligible for stem cell transplants.