Cured blood cancer: a drug originally developed for another condition shows promise in battling this dangerous form of oncology.

Researchers from the Washington University School of Medicine in St. Louis have discovered an effective method to halt the progression of certain types of blood cancer using a drug originally developed for breast cancer treatment. Two recent studies indicate that blocking the RSK1 protein, which is involved in inflammation, may be key to stopping the growth of myeloproliferative neoplasms (MPN) and an aggressive form of acute myeloid leukemia (AML), reports Washington University School of Medicine.

The findings from the scientists, based on trials in mice and patient samples, suggest that PMD-026, a drug currently undergoing testing for breast cancer, may also be effective against several types of blood cancer.

MPN refers to slowly growing blood tumors that can worsen over time, increasing the risk of developing secondary AML, a more aggressive and fatal disease. Patients with chronic MPN often endure debilitating symptoms like severe fatigue and weight loss, but they lack reliable treatment options to prevent disease progression or reduce the likelihood of transformation into AML.

Dr. Stephen T. Oh, the senior author of the study and co-director of the hematology division at Washington University, explained: "We hope that this new drug will help fill this gap in clinical care. At the very least, we hope it can halt the progression of chronic disease to AML."

The studies provided compelling evidence supporting the authors' claims. In the first, published in the journal Nature Communications, researchers found that blocking RSK1 reverses the progression of MPN in mice, reducing scar tissue in the bone marrow and destroying up to 96% of cancer cells within just four weeks.

The second study, published in the journal Blood Cancer Journal, demonstrated that blocking RSK1 also treats a specific type of AML known as FLT3-ITD AML. While existing treatments for this form of AML often fail as the cancer becomes resistant, the RSK1 inhibitor offers an alternative approach by targeting a different pathway.

The PMD-026 drug, a tablet that blocks all four forms of the RSK protein, was developed by Phoenix Molecular Designs for breast cancer treatment. Initial clinical trials showed that breast cancer patients tolerate it well, with side effects being manageable and controllable. Encouraged by these results, Dr. Oh's team collaborated with the drug developers to explore its potential in combating blood cancer.

Researchers believe that the drug may also prepare some MPN patients for stem cell transplantation—the only known cure for many types of blood cancer, but not always accessible due to age or health complications. Identifying RSK1 as a key factor in the progression of MPN and AML builds on earlier work by Oh's group, which identified another protein, DUSP6, as a critical element in these diseases. By understanding how DUSP6 signals subsequent effects, they pinpointed RSK1 as a target protein.

If PMD-026 is approved for breast cancer treatment, it will be the first drug on the market that inhibits the RSK protein family, potentially opening new avenues for treating various types of cancer, according to the authors. Their research has already received support from several organizations, including the National Institutes of Health (NIH), the Leukemia and Lymphoma Society, and the MPN Research Foundation. The next steps for the scientists involve developing clinical trials to test the drug's efficacy on blood cancer patients who have exhausted other treatment options.

Important! This article is based on the latest scientific and medical research and does not contradict them. The text is for informational purposes only and does not contain medical advice. For diagnosis, please consult a physician.