Opinion
Video
Lessons From Previously Used Therapies In NF1-PN
Panelists discuss key lessons learned from previous NF1-associated plexiform neurofibroma therapies, including the limitations of traditional treatments due to high toxicity and limited efficacy, and how these challenges are driving the development of more targeted therapies like MEK inhibitors, personalized medicine, and combination strategies to improve outcomes and reduce relapse.
Summary for Physicians
Lessons Learned From Previous NF1-PN Therapies
Previous therapies for neurofibromatosis type 1–associated plexiform neurofibromas (NF1-PN), including traditional chemotherapies and surgical approaches, have highlighted several challenges that are influencing the development of new treatments. One major lesson is the limited efficacy and high toxicity of conventional therapies, especially in terms of tumor shrinkage and symptom control. The lack of long-term effectiveness in many cases led to a push for more targeted therapies, such as MEK inhibitors, which offer a more focused mechanism of action and potentially fewer adverse effects.
Additionally, tumor heterogeneity and resistance mechanisms (eg, the activation of compensatory pathways) have underscored the need for combination therapies to improve response rates and minimize relapse. Patient selection, based on molecular profiling, is becoming more important, as it can guide the use of the most effective treatments for individual cases.
These challenges are helping shape the direction of future therapies, focusing on personalized medicine, combination strategies, and biomarker-driven approaches to optimize treatment outcomes and minimize adverse effects.
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