Christopher Moertel, MD is the Kenneth and Betty Jayne Dahlberg Professor of Pediatrics in the University of Minnesota School of Medicine. He is the medical director of the Katie Hageboeck Children's Cancer Research Fund Clinic and leads the Pediatric Brain Tumor Program at the Comprehensive Neurofibromatosis Clinic.

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"It’s up to us practitioners to provide good, anticipatory guidance regarding symptom management…and to realize what those AEs are and how to treat them effectively. If we can do our best to ameliorate these toxicities and enable patients to stay on [treatment] for a long time, we’ve done a good job.

Christopher L. Moertel, MD, a pediatric neuro-oncologist, professor, and director the Pediatric Neuro-Oncology Fellowship Program in the Division of Pediatric Hematology/Oncology, medical director of the Pediatric Neuro-Oncology and Neurofibromatosis Programs, co-medical director of the Katie Hageboeck Children’s Cancer Research Fund Clinic, clinical neuro-oncology leader of the Brain Tumor Program, and The Kenneth and Betty Jayne Dahlberg Profssor at the University of Minnesota School of Medicine, discussed the importance of informed, careful, and effective management of MEK inhibitor–associated toxicities for patients with neurofibromatosis type 1 (NF1)–associated plexiform neurofibromas (PN).

The introduction of MEK inhibitors as a key therapeutic option for NF1-associated PN has necessitated a greater focus on managing associated toxicities to ensure long-term treatment adherence, Moertel began. Patients receiving MEK inhibitors are susceptible to a range of adverse effects (AEs), including ocular, cardiac, dermatologic, and gastrointestinal (GI) toxicities, which can significantly affect quality of life and treatment continuation, Moertel stated. He noted that these agents require extended administration—often for a minimum of 18 months—making effective toxicity management critical.

Therefore, oncologists should adopt anticipatory guidance strategies to educate patients on potential AEs and provide proactive toxicity management when they receive a MEK inhibitor, Moertel recommended. For example, dermatologic toxicities, such as rashes and skin irritation, can often be mitigated with antibiotics, bleach baths, and emollient lotions, he shared. GI toxicity, which may include nausea and diarrhea, can be managed through dietary adjustments and supportive care measures, Moertel added. Recognizing these toxicities early and implementing targeted interventions allows patients to tolerate treatment more effectively, reducing the likelihood of treatment discontinuation, Moertel emphasized.

Despite the efficacy of MEK inhibitors in slowing tumor growth, these agents do not eliminate NF1-associated PN, Moertel stated. There remains significant opportunity for research and innovation in this area, particularly in developing strategies to improve treatment tolerability and enhance therapeutic outcomes, he said. By refining toxicity management protocols and expanding treatment options, oncologists can better support patients in maximizing the benefits of MEK inhibitors and minimizing the effect of these agents on daily life, Moertel concluded.

Videos

Christopher L. Moertel, MD, discusses the management of MEK inhibitor–associated toxicities in NF1-associated plexiform neurofibromas.


Dr Moertel on the Importance of Managing MEK Inhibitor–Associated Toxicites in NF1-Associated PN

, which provides oncology professionals with the resources and information they need to provide the best patient care. In both digital and print formats, 

 covers every angle of oncology practice, from new technology to treatment advances to important regulatory decisions.

In today’s episode, we had the pleasure of speaking with Christopher L. Moertel, MD, about the 

(Gomekli) for the treatment of adult and pediatric patients at least 2 years of age with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PNs) that are not amenable to complete resection. Dr Moertel is a pediatric neuro-oncologist, professor, program director of the Pediatric Neuro-Oncology Fellowship Program, a faculty member in the Department of Pediatrics, medical director of the Pediatric Neuro-Oncology and Neurofibromatosis Programs, co-medical director of the Katie Hageboeck Children’s Cancer Research Fund Clinic, the clinical neuro-oncology leader of the Brain Tumor Program, and the Kenneth and Betty Jayne Dahlberg Professor at the University of Minnesota School of Medicine in Minneapolis.

In our exclusive interview, Dr Moertel discussed the significance of this approval, key efficacy and safety data from the phase 2 ReNeu trial (NCT03962543), and considerations for integrating mirdametinib into clinical practice for patients with NF1-associated PN.

That’s all we have for today! Thank you for listening to this episode of 

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Podcasts

Christopher L. Moertel, MD, discusses the significance of the FDA approval of mirdametinib, based on efficacy and safety data from the phase 2 ReNeu trial.

FDA Approval Insights: Mirdametinib for NF1-Associated Plexiform Neurofibromas: With Christopher L. Moertel, MD

“The approval of mirdametinib is significant because this a highly selective MEK inhibitor that will provide benefit to both adults and children with neurofibromatosis type 1 who have plexiform neurofibromas that are not amenable to surgery and are causing significant quality-of-life or pain issues.”

Christopher L. Moertel, MD, pediatric neuro-oncologist; professor; director, Pediatric Neuro-Oncology Fellowship Program, Division of Pediatric Hematology/Oncology; faculty, Department of Pediatrics; medical director, Pediatric Neuro-Oncology and Neurofibromatosis Programs; co-medical director, Katie Hageboeck Children's Cancer Research Fund Clinic; clinical neuro-oncology leader, Brain Tumor Program; and The Kenneth and Betty Jayne Dahlberg Professor, University of Minnesota School of Medicine, discusses the FDA approval of mirdametinib (Gomekli) for the treatment of adult and pediatric patients with neurofibromatosis type 1 (NF1)–associated plexiform neurofibromas (PN).

 for adult and pediatric patients 2 years of age and older with NF1 who have symptomatic PN not amenable to complete resection.

The regulatory decision was supported by data from the phase 2b ReNeu trial (NCT03962543), which demonstrated that treatment with mirdametinib led to significant reductions in tumor volume, pain relief, and improvements in quality of life for patients with NF1-PN that was not amenable to surgical resection. The trial enrolled both adult and pediatric patients and evaluated mirdametinib’s efficacy and safety in this patient population.

Findings that supported the approval showed that adult patients (n = 58) achieved a confirmed overall response rate (ORR) of 41% (95% CI, 29%-55%); pediatric patients (n = 56) experienced a confirmed ORR of 52% (95% CI, 38%-65%).

Beyond tumor reduction, the trial demonstrated significant clinical benefits in symptom management. Patients reported marked reductions in pain severity, which translated into improved daily functioning and quality of life.

Moertel emphasizes that this approval is particularly meaningful for adult patients with NF1-PN, who have historically lacked FDA-approved treatment options.

The approval of mirdametinib represents a significant advancement in the treatment landscape for NF1-PN, Moertel concludes. Future research may focus on optimizing long-term disease management strategies and evaluating combination approaches to further enhance clinical outcomes.

Christopher L. Moertel, MD, discusses the FDA approval of mirdametinib for patients with neurofibromatosis type 1–associated plexiform neurofibromas.

Christopher L. Moertel, MD

Articles

Dr Moertel on the Importance of Managing MEK Inhibitor–Associated Toxicites in NF1-Associated PN

FDA Approval Insights: Mirdametinib for NF1-Associated Plexiform Neurofibromas: With Christopher L. Moertel, MD

Dr Moertel on the FDA Approval of Mirdametinib for NF1-Associated Plexiform Neurofibromas