Unituxin (dinutuximab) / United Therapeutics Corp, Ohara Pharma - LARVOL DELTA

Pulmonary Toxicity Associated With Concurrent Lorlatinib and Anti-GD2 Monoclonal Antibody Therapy in Patients With Neuroblastoma. (PubMed, Pediatr Blood Cancer) - "Concurrent use of lorlatinib with anti-GD2 mAbs may be associated with severe pulmonary toxicities. Temporarily withholding lorlatinib during immunotherapy appeared to mitigate this risk."

Adverse events • Journal • Cough • Neuroblastoma • Oncology • Respiratory Diseases • Solid Tumor • ALK

Phase I Study of 131I-Metaiodobenzylguanidine With Dinutuximab ± Vorinostat for Patients With Relapsed or Refractory Neuroblastoma: A New Approaches to Neuroblastoma Therapy Trial. (PubMed, J Clin Oncol) - "MIBG combined with dinutuximab was well tolerated with encouraging antitumor activity. Vorinostat added to this combination may augment responses in this heavily pretreated patient population."

Journal • P1 data • Fatigue • Neuroblastoma • Oncology • Pneumonia • Solid Tumor

A phase 2 randomized study of chemoimmunotherapy with or without eflornithine (DFMO) in relapsed/refractory neuroblastoma: A Children's Oncology Group (COG) report. (ASCO 2025) - P2 | "National Institutes of Health Clinical Trial Registration Number: NCT03794349 Background: Dinutuximab, irinotecan, temozolomide and GM-CSF (DIT) is widely used in first relapsed/ refractory high-risk neuroblastoma (r/r HRNB), however <50% of patients (pts) respond. The addition of DFMO to DIT did not improve response rates in pts with first r/r HRNB, though the response rates in both arms confirmed DIT activity in this population. The ORR to DIT is higher than previously reported, likely due to use of the 2017 INRC that includes MR in calculation of ORR. Rates of CR+PR in this trial were similar to those previously reported."

Clinical • P2 data • Neuroblastoma • Oncology • Otorhinolaryngology • Pain • Solid Tumor • CSF2 • MYCN

Phase I Trial of rhIL-15 Plus Dinutuximab Plus Irinotecan/Temozolomide for Children and Young Adults With Relapsed/Refractory Neuroblastoma (clinicaltrials.gov) - P1 | N=34 | Not yet recruiting | Sponsor: National Cancer Institute (NCI)

New P1 trial • Neuroblastoma • Oncology • Solid Tumor

Advancements in neuroblastoma treatment: FDA-approved drugs and role of phytochemicals. (PubMed, Mol Biol Rep) - "FDA-approved drugs like Entrectinib, Dinutuximab, Unitaxin, Doxorubicin, Hydrochloride, Lorlatinib, and Crizotinib are pivotal in treating neuroblastoma. Additional plant extracts have exhibited activity against neuroblastoma. This review highlights the synergistic potential of FDA-approved drugs and plant-derived phytoconstituents in improving neuroblastoma treatment outcomes."

FDA event • Journal • Review • Neuroblastoma • Oncology • Pediatrics • Solid Tumor

NANT 2021-01 Phase II STING (Sequential Temozolomide, Irinotecan, NK Cells and GD2 mAb) Trial (clinicaltrials.gov) - P2 | N=62 | Active, not recruiting | Sponsor: New Approaches to Neuroblastoma Therapy Consortium | Recruiting ➔ Active, not recruiting

Enrollment closed • Neuroblastoma • Oncology • Solid Tumor

Pleuroparenchymal Fibroelastosis Following Hematopoietic Stem Cell Transplant for Neuroblastoma: Report of 3 Pediatric Cases (ATS 2025) - "All patients received chemotherapy (cisplatin, cyclophosphamide, doxorubicin, etoposide, topotecan and vincristine), 13-cis retinoic acid with immunotherapy (dinutuximab and alternating GM-CSF and IL-2), radiation (2160 cGy), and autologous stem cell transplant. Ann Am Thorac Soc. 2019; 16(11):1351-9."

Clinical • Bone Marrow Transplantation • Fibrosis • Immunology • Infectious Disease • Interstitial Lung Disease • Neuroblastoma • Oncology • Pediatrics • Pneumonia • Respiratory Diseases • Solid Tumor • Transplantation • CSF2 • IL2

Successful Targeting of a FGFR1 Mutation With Futibatinib in a Patient With Refractory Neuroblastoma (ASPHO 2025) - "Initially treated with two cycles of high-risk therapy per Children's Oncology Group (COG) trial ANBL 1531, significant complications required transition to chemo-immunotherapy with Dinutuximab per the COG ANBL 1221 protocol...End of therapy imaging showed persistent paraspinal MIBG avid disease not amenable to surgery; thus, proton beam radiation followed by Isotretinoin was initiated... This case showcases the successful use of a FGFR1 inhibitor in the case of a pediatric patient with refractory neuroblastoma who had limitations in therapeutic options due to toxicity. This emphasizes the importance of tumor sequencing, and the need for further clinical trials exploring the use of these medications in childhood cancers with this mutation."

Clinical • IO biomarker • Brain Cancer • Breast Cancer • CNS Tumor • Lung Cancer • Neuroblastoma • Non Small Cell Lung Cancer • Oncology • Pediatrics • Solid Tumor • FGFR1

Children’s Hospital Los Angeles Experts to Present at 2025 Advances in Neuroblastoma Research Meeting (Newswise) - "Several Children’s Hospital Los Angeles experts will present their latest research at the 2025 Advances in Neuroblastoma Research (ANR) Meeting May 25-28 in Washington, DC....At ANR 2025, the team will share several new studies—including a promising cell therapy in development, a novel stem cell model of the disease, new insights into cancer-associated fibroblasts, and more."

Clinical data • Neuroblastoma

STING: NK Cells Infusions With Irinotecan, Temozolomide, and Dinutuximab (clinicaltrials.gov) - P1/2 | N=31 | Recruiting | Sponsor: Nationwide Children's Hospital | Trial completion date: Dec 2025 ➔ Dec 2027 | Trial primary completion date: Dec 2024 ➔ Dec 2026

Trial completion date • Trial primary completion date • Neuroblastoma • Oncology • Solid Tumor

Investigating the immunological effect of MYCN depletion in MYCN-amplified neuroblastoma (IMMUNOLOGY 2025) - "This upregulation appeared specific to MYCN depletion, as it was absent in cells treated with doxorubicin or etoposide. MYCi975 also enhanced NK-cell mediated killing of MYCN-amplified cells in the presence of dinutuximab, an FDA-approved anti-GD2 monoclonal antibody for high-risk neuroblastoma, and reduced secretion of the immunosuppressive cytokine IGFBP-2. These findings reveal a mechanism by which MYCN influences anti-tumor immunity and suggest a broader role in TIME modulation, providing a foundation for exploring the therapeutic potential of HLA-G and DR5 co-upregulation with MYCN targeting.Keywords: Cells Natural Killer Cells; Molecules Cell Surface Molecules MHC Transcription Factors; Processes Cytotoxicity"

IO biomarker • Late-breaking abstract • Tumor mutational burden • Neuroblastoma • Oncology • Pediatrics • Solid Tumor • IGFBP2 • MYCN • TMB • TNFRSF10B

Testing the Combination of Two Immunotherapy Drugs (Magrolimab and Dinutuximab) in Patients With Relapsed or Refractory Neuroblastoma or Relapsed Osteosarcoma (clinicaltrials.gov) - P1 | N=12 | Completed | Sponsor: National Cancer Institute (NCI) | Terminated ➔ Completed

Trial completion • Neuroblastoma • Oncology • Osteosarcoma • Sarcoma • Solid Tumor

Isotretinoin With or Without Dinutuximab, Aldesleukin, and Sargramostim Following Stem Cell Transplant in Treating Patients With Neuroblastoma (clinicaltrials.gov) - P3 | N=1449 | Active, not recruiting | Sponsor: National Cancer Institute (NCI) | Trial completion date: Mar 2025 ➔ Mar 2026

Trial completion date • Bone Marrow Transplantation • CNS Tumor • Neuroblastoma • Oncology • Solid Tumor • Transplantation

A first-in-human phase 1, multicenter, open-label study of M3554, a novel anti-GD2 antibody-drug conjugate (ADC), in patients with advanced solid tumors (AACR 2025) - P1 | "GD2 is a clinically validated target in pediatric neuroblastoma; however, currently approved anti-GD2 antibody treatments, such as dinutuximab and naxitamab, have been associated with severe adverse events (AEs) of pain, likely due to Fcγ receptor-mediated immune activation via antibody-dependent cellular cytotoxicity (ADCC) and complement-dependent cytotoxicity (CDC). The study is currently recruiting and aims to enroll 52 patients in the dose escalation phase and 110 patients in the dose expansion phase (STS, n=80; GBM, n=30), across different global sites including, Japan, Belgium, France, and the US. Clinical trial information: NCT06641908."

Clinical • Metastases • P1 data • Brain Cancer • CNS Tumor • Glioblastoma • Neuroblastoma • Oncology • Osteosarcoma • Sarcoma • Soft Tissue Sarcoma • Solid Tumor

Irinotecan Hydrochloride, Temozolomide, and Dinutuximab With or Without Eflornithine in Treating Patients With Relapsed or Refractory Neuroblastoma (clinicaltrials.gov) - P2 | N=94 | Active, not recruiting | Sponsor: Children's Oncology Group | Trial completion date: Mar 2025 ➔ Apr 2026

Trial completion date • CNS Tumor • Neuroblastoma • Oncology • Solid Tumor

NCCN has published updates to the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines), the NCCN Drugs & Biologics Compendium (NCCN Compendium), the NCCN Radiation Therapy Compendium, and the NCCN Imaging Appropriate Use Criteria (NCCN Imaging AUC) for Neuroblastoma, Version 1.2025. (NCCN)

NCCN guideline • Neuroblastoma

Ocular Toxicity in GD-2 Antibody Therapy: A Case Study. (PubMed, J Pediatr Hematol Oncol Nurs) - " A 6-year-old boy with high-risk neuroblastoma presented to the outpatient clinic 4 days after completion of dinutuximab therapy stating that he was "going blind" and "the light hurt his eyes." He was treated with 1% pilocarpine drops and sunglasses for comfort after evaluation by ophthalmology. Measuring a change in visual acuity and pupillary response is impossible without a baseline screening before commencing GD-2 antibody therapy. Although there is significant literature and reports on the identification and management of common side effects of GD-2 antibody therapy, less common ocular toxicities are possibly underreported with little guidance on management, thus making recognition and subsequent treatment challenging for nurses and providers."

Journal • CNS Tumor • Neuroblastoma • Oncology • Ophthalmology • Pediatrics • Solid Tumor

MIBG With Dinutuximab +/- Vorinostat (clinicaltrials.gov) - P1 | N=45 | Completed | Sponsor: New Approaches to Neuroblastoma Therapy Consortium | Active, not recruiting ➔ Completed

Trial completion • CNS Tumor • Neuroblastoma • Oncology • Solid Tumor • CD34

NANT: Lenalidomide and Dinutuximab With or Without Isotretinoin in Treating Younger Patients With Refractory or Recurrent Neuroblastoma (clinicaltrials.gov) - P1 | N=27 | Active, not recruiting | Sponsor: National Cancer Institute (NCI) | Trial completion date: Mar 2025 ➔ Mar 2026

Trial completion date • CNS Tumor • Neuroblastoma • Oncology • Solid Tumor

Therapy for Children With Advanced Stage Neuroblastoma (clinicaltrials.gov) - P2 | N=153 | Active, not recruiting | Sponsor: St. Jude Children's Research Hospital | Trial completion date: Dec 2024 ➔ Dec 2025

Trial completion date • CNS Tumor • Neuroblastoma • Oncology • Solid Tumor • MYCN

Post Transplant Outcomes of High-Risk Neuroblastoma From a Tertiary Care Unit in India. (PubMed, Indian Pediatr) - "All patients had febrile neutropenia; there was no ASCT-related mortality. Multidisciplinary therapy even with omission of dinutuximab in our cohort had acceptable outcomes."

Journal • Retrospective data • CNS Tumor • Febrile Neutropenia • Hematological Disorders • Neuroblastoma • Neutropenia • Oncology • Solid Tumor • Transplantation

Ketamine Infusion as an Adjunct to Opioid Analgesia in Pediatric Patients with High-Risk Neuroblastoma Undergoing Treatment with Dinutuximab: Adverse Effects and Safety in a Non-ICU Setting. (PubMed, J Pain Res) - "Demographics, pain scores, medication records, and total daily IV morphine milligram equivalents (IVMME) with and without a ketamine adjunct were collected. Findings suggest ketamine is safe in a non-ICU setting for treatment of complex pain during anti-GD2 immunotherapy. Additional prospective studies are needed to quantify the effect of reducing opioid side effects by including ketamine in pain management plans."

Adverse events • Journal • CNS Tumor • Neuralgia • Neuroblastoma • Oncology • Pain • Pediatrics • Solid Tumor

Targeting Ewing Sarcoma By IL1RAP CAR Modified Ex-Vivo Expanded TGFβ Imprinted NK Cells in Combination with IL-15 Agonist and Anti-GD2 Antibody (TCT-ASTCT-CIBMTR 2025) - "Conclusions Our data demonstrated enhanced anti-tumor efficacy of IL1RAP CAR NK/TGFβi-NK cells alone and combined with NKTR-255 and dinutuximab against ES in vitro and in vivo. These CAR engineered TGFβi NK cells in combination with NK function and persistence enhancing immune modulators constitute potential novel effective immunotherapies for patients with high-risk ES."

Combination therapy • IO biomarker • Preclinical • Ewing Sarcoma • Oncology • Sarcoma • Solid Tumor • IFNG • IL15 • IL1RAP • TGFB1 • TGFBI

Testing the Combination of Two Immunotherapy Drugs (Magrolimab and Dinutuximab) in Patients With Relapsed or Refractory Neuroblastoma or Relapsed Osteosarcoma (clinicaltrials.gov) - P1 | N=12 | Terminated | Sponsor: National Cancer Institute (NCI) | Trial completion date: Nov 2025 ➔ Sep 2024 | Active, not recruiting ➔ Terminated; Drug supply issues

Trial completion date • Trial termination • CNS Tumor • Neuroblastoma • Oncology • Osteosarcoma • Sarcoma • Solid Tumor

Prospects of anti-GD2 immunotherapy for retinoblastoma. (PubMed, Front Immunol) - "An anti-GD2 monoclonal antibody (Dinutuximab), which has been approved for the treatment of high-risk neuroblastomas, has shown promising efficacy in improving patients' prognosis. Additionally, chimeric antigen receptors (CAR)-T therapy, GD2 vaccines and nanoparticles are also potential therapeutics. Finally, we discuss the prospects and current limitations of these immunotherapeutic approaches for treating retinoblastoma, as well as how to address these problems."

Journal • Review • CNS Tumor • Eye Cancer • Neuroblastoma • Oncology • Ophthalmology • Retinal Disorders • Retinoblastoma • Solid Tumor