New Clinical Trial Treatment Causes Myelofibrosis Cell Death

What is navtemadlin, and how does this treatment in clinical trials help stop myelofibrosis cells and improve cancer-related symptoms? Learn answers to these questions below! 

How Navtemadlin Works Against Myelofibrosis 

Navtemadlin is a pill taken by mouth. It is part of a new class of drugs called an MDM2 inhibitor. 

Myelofibrosis cells have too much of the MDM2 protein. Blocking MDM2 with navtemadlin activates the p53 protein. This supports the cell death cycle and prevents cancer cells from multiplying uncontrollably, reducing cancer progression and improving cancer-related symptoms. 

One thing to note: Patients must have wild-type TP53 for navtemadlin to be effective, meaning the TP53 gene must be normal and not mutated, ensuring proper p53 creation and activation.

John O. Mascarenhas, MD, from the Icahn School of Medicine at Mount Sinai, presented the phase 3 results of the BOREAS study testing navtemadlin for myelofibrosis at the 2024 ASH conference. 

Read a summary of the findings below, and learn how you may be eligible to receive navtemadlin.  

Navtemadlin in the BOREAS Trial

The BOREAS study evaluated navtemadlin compared to the best available therapy, which included hydroxyurea, chemotherapy, immunomodulatory drugs (such as lenalidomide), and supportive care. 

The trial enrolled people with myelofibrosis who had previously taken or became intolerant to JAK inhibitors. Patients in the best available therapy group had the option to switch to navtemadlin. 

Key Benefits of Navtemadlin for Myelofibrosis

Cancer-Modifying Effects

At week 24 of treatment, navtemadlin reduced the number of myelofibrosis cells by an average of 82% for patients. This was measured by testing CD34+ bone marrow stem cells and progenitor cell levels, the cells commonly affected in myelofibrosis.

Navtemadlin also decreased cancer-driving mutations and reduced inflammatory markers, suggesting that navtemadlin could slow or alter the cancer course rather than simply managing symptoms.

Reduction in Spleen Size

One of the primary goals of myelofibrosis treatment is reducing spleen volume, as an enlarged spleen can cause pain, discomfort, and other complications. 

By week 24 of treatment, navtemadlin helped 15% of patients reduce their spleen volume by ≥35%, compared to 5% in the control group. 

Improvement in Bone Marrow Scarring

Nearly half of the patients treated with navtemadlin showed a one-grade improvement in bone marrow scarring, indicating the therapy reduced myelofibrosis’ effects. 

Improved Symptom Control

Navtemadlin significantly improved symptoms like fatigue, night sweats, and bone pain, enhancing patients' overall well-being.

How You Can Receive Navtemadlin for Myelofibrosis Treatment 

If you have relapsed/refractory myelofibrosis and would like to review your eligibility for receiving navtemadlin in the BOREAS study, click here. 

If you have not previously taken a JAK inhibitor and would like to review your eligibility for receiving navtemadlin in combination with ruxolitinib in the POIESIS study, click here. 

To learn more about how clinical trials work, read: What are Clinical Trials, and How May They Help Me?

Summary

Navtemadlin represents a promising new treatment in clinical trials for people with myelofibrosis who have limited options after JAK inhibitors stop working. Restoring p53 function and reducing cancer burden offers a new approach that may improve survival and quality of life. 

While more research is needed, current clinical trial results suggest that navtemadlin could significantly advance myelofibrosis treatment. 

Read More News

Learn about additional myelofibrosis treatments by clicking the button below to explore HealthTree’s news page. 

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Sources: 

• Disease-Modifying Activity of Navtemadlin Correlates with Clinical Responses in a Randomized, Multicenter, Global Phase 3 Study (BOREAS) in JAK-Inhibitor Relapsed/Refractory Myelofibrosis
• Results from the Randomized, Multicenter, Global Phase 3 BOREAS Study: Navtemadlin Versus Best Available Therapy in JAK Inhibitor Relapsed/Refractory Myelofibrosis