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Jaume Mora, MD, PhD: Naxitamab Approval for High-Risk Neuroblastoma in the Bone or Bone Marrow

Jaume Mora, MD, PhD.

Recently, the FDA approved naxitamab in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF) for patients with relapsed/refractory high-risk neuroblastoma in the bone or bone marrow. In this interview, Jaume Mora, MD, PhD, Scientific Director of Oncology and Hematology at Sant Joan de Déu Barcelona Children's Hospital and lead investigator of one of the trials on which the approval was based, speaks with i3 Health about the benefits of naxitamab for patients with relapsed/refractory high-risk neuroblastoma and shares advice for the cancer care team regarding how to safely administer this medication to patients.

What are some of the most challenging aspects of treating patients with relapsed/refractory high-risk neuroblastoma in the bone or bone marrow?

Jaume Mora, MD, PhD: Newly diagnosed high-risk neuroblastoma is a very difficult disease to cure. Long-term survival rates hadn't reached 40% for decades, not until anti-GD2 antibodies came about, such as dinutuximab, which was approved in the US and Europe in 2015. However, survival rates remain low even for those who experience complete remission, which is achieved when patients receive first-line therapy, the tumor responds nicely, and the tests become negative. Those patients then receive dinutuximab, leading to survival rates around 50%. This is the standard of care nowadays in the US and Western Europe, although not for the rest of the world. For patients who do not respond completely to first-line therapy—those who have residual or refractory disease, mainly in the bone and bone marrow compartments—these patients have an even more dismal outcome with standard management. Long-term survival rates for this population are reported to be less than 20% with conventional treatment.

Can you comment on the approval of naxitamab plus GM-CSF for patients with relapsed/refractory high-risk neuroblastoma in the bone or bone marrow?

Dr. Mora: Naxitamab is an anti-GD2 antibody that was discovered at Memorial Sloan Kettering Cancer Center and then put forward into clinical trials, where it has shown complete remission rates of 60% in this patient population. You can see how potent these antibodies are, in that they can completely clear disease in the bone and bone marrow compartment that is refractory to first-line chemotherapy. The approval of naxitamab is a major breakthrough in the management of these patients, and it opens up the possibility of long-term cure for patients who had almost zero survival in the past.

Are there any particular adverse events that members of the cancer care team should monitor for in patients receiving naxitamab?

Dr. Mora: The safety profile of naxitamab is very similar to those of the other anti-GD2 antibodies. It can cause hypotension, changes in heart rate and respiratory rate, pain, and injection site reactions, which are common reaction patterns seen with other anti-GD2 antibodies. The difference between naxitamab and the prior antibodies—those in the dinutuximab family—is that naxitamab generates more hypertension, which should be monitored for during infusions. However, with regular antihypertensive drugs, this can usually be successfully managed with no major complications. The most significant side effect is posterior reversible encephalopathy syndrome (PRES), which is associated with hypertension and should be monitored for in patients receiving naxitamab.

Do you have any words of advice for members of the cancer care team treating patients with relapsed/refractory high-risk neuroblastoma in the bone or bone marrow?

Dr. Mora: Even though naxitamab is another anti-GD2 antibody, the way that it is administered and managed is nothing like that of dinutuximab. Cancer care teams that are experienced with dinutuximab infusions should go through specific training as they begin administering naxitamab. First of all, naxitamab has a very short infusion period, 30 to 45 minutes, as compared to 10 to 20 hours for dinutuximab. These very short infusions generate very intense pain, hypotension, and infusion-related reactions. The cancer care team, as well as the patients, should be prepared and ready to react when adverse events occur; the nursing team at the bedside should be ready to support the patients through these intense reactions. Basically, it's just a matter of getting experience using the drug and knowing how to manage the side effects. When all is said and done, both the patients and their families are really benefiting from receiving a short infusion that allows them to go home the same day.

About Dr. Mora

Jaume Mora, MD, PhD, is the Scientific Director of the Pediatric Cancer Center Barcelona of the Hospital Sant Joan de Déu and an associate member of the Oncology Program at the Institute for Research in Biomedicine in Barcelona. His research specializes in the origin of childhood solid tumors, mainly neuroblastoma, Ewing sarcoma, Wilms tumor and brain stem glioma. Dr. Mora has published more than 170 peer-reviewed publications in the field of developmental oncology. He has been distinguished with the American Society of Clinical Oncology (ASCO) Young Investigator Award (2000), the 16th Schweisguth Prize of the International Society of Pediatric Oncology (2001), the ASCO Career Development Award (2001), the First Research Grant for Pediatric Oncology of the Spanish Association Against Cancer (2006), the first Research Award of Translational Oncology of the FERO Society (2009), and the First Martí Via Award of Translational Oncology of the Fundació Vallformosa (2015).

For More Information

Mora J, Chan GC, Morgenstern DA, et al (2020). Naxitamab, a new generation anti-GD2 monoclonal antibody (mAb) for treatment of relapsed/refractory high-risk neuroblastoma (HR-NB). J Clin Oncol (ASCO Virtual Scientific Program Abstracts), 38(suppl_15). Abstract 10543. DOI:10.1200/JCO.2020.38.15_suppl.10543

Clinicaltrials.gov (2020). Combination therapy of antibody HU3F8 with granulocyte- macrophage colony stimulating factor (GM-CSF) in patients with relapsed/refractory high-risk neuroblastoma. NLM identifier: NCT01757626.

Transcript edited for clarity. Any views expressed above the speaker's own and do not necessarily represent those of i3 Health. 

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