Leukine (sargramostim) / Partner Therap - LARVOL DELTA

Cytokine use in the Hematopoietic Subsyndrome of Acute Radiation Syndrome (H-ARS): Implications for the role of cytokines in a mass casualty radiologic/nuclear (R/N) emergency. (PubMed, J Radiol Prot) - "Ease of use favored pegfilgrastim (administered weekly x2) and romiplostim (administered once), compared to filgrastim and sargramostim (administered daily x5 and x14, respectively). Formal assessment of the published evidence is urgently needed to provide categorical guidance regarding cytokine use for patient management, and to public health officials involved in establishing a national or shared regional radiation stockpile for immediate use in a mass casualty radiological/nuclear (R/N) emergency."

Journal • Hematological Disorders • Oncology

A Phase II/III Trial of Nivolumab, Ipilimumab, and GM-CSF in Patients With Advanced Melanoma (clinicaltrials.gov) - P2/3 | N=600 | Active, not recruiting | Sponsor: National Cancer Institute (NCI) | Recruiting ➔ Active, not recruiting

Enrollment closed • Breast Cancer • Cutaneous Melanoma • Melanoma • Oncology • Solid Tumor • BRAF

Sunitinib or Cediranib for Alveolar Soft Part Sarcoma (clinicaltrials.gov) - P2 | N=34 | Completed | Sponsor: National Cancer Institute (NCI) | Active, not recruiting ➔ Completed

Monotherapy • Trial completion • Alveolar Soft Tissue Sarcoma • Oncology • Sarcoma • Solid Tumor

Bone Marrow Colony Stimulating Factors (CSF) and Stem Cell Therapy as Medical Countermeasures (MCM) for Treatment of Hematopoietic-Acute Radiation Syndrome: A Systematic Review. (PubMed, Disaster Med Public Health Prep) - "While studies looking at efficacy of CSFs given after 24 hours were limited, 2 studies included in this review showed that delayed administration of CSFs up to 120 hours may be beneficial compared to no treatment. Lack of standardization in experimental study design (e.g. radiation doses, animal species, interventions) between studies prevented direct comparisons using meta-analytic statistical approach."

Journal • Review

Phase IIa Study of SurVaxM Plus Adjuvant Temozolomide for Newly Diagnosed Glioblastoma. (PubMed, J Clin Oncol) - P2 | "SurVaxM appeared to be safe and well tolerated. The combination represents a promising therapy for nGBM. For patients with nGBM treated in this manner, PFS may be an acceptable surrogate for OS. A large randomized clinical trial of SurVaxM for nGBM is in progress."

Clinical • Journal • P2a data • Brain Cancer • CNS Tumor • Glioblastoma • Immunology • Oncology • Solid Tumor • BIRC5 • CD8

Autoimmune Pulmonary Alveolar Proteinosis Triggered by Salazosulfapyridine in a Patient With Rheumatoid Arthritis: A Case Report. (PubMed, Cureus) - "The development of aPAP is considered extremely rare in patients with RA, as the disease is typically characterized by elevated GM-CSF activity, creating a pathological paradox. Previous reports have revealed that almost all patients with aPAP and RA receive SASP treatment. We hypothesized that aPAP associated with RA developed due to a dual suppression of GM-CSF: drug-induced inhibition of GM-CSF at the alveolar level and acquired production of anti-GM-CSF antibodies."

Journal • Immunology • Inflammatory Arthritis • Respiratory Diseases • Rheumatoid Arthritis • Rheumatology

A pilot induction regimen incorporating dinutuximab and sargramostim for the treatment of newly diagnosed high-risk neuroblastoma: A report from the Children's Oncology Group. (ASCO 2022) - P2 | "Chemoimmunotherapy including irinotecan, temozolomide, DIN and sargramostim (GM-CSF) in patients with recurrent or refractory neuroblastoma results in robust objective clinical responses. The administration of DIN and GM-CSF to COG Induction Cycles 3-5 for patients with newly-diagnosed high-risk neuroblastoma was tolerable and feasible. The objective response rate at EOI appears encouraging. This therapeutic regimen will be studied in a randomized phase 3 trial to further evaluate the efficacy of Induction phase chemoimmunotherapy for high-risk neuroblastoma."

Clinical • Hypotension • Neuroblastoma • Oncology • Pain • Solid Tumor • CSF2

A pilot study of chemoimmunotherapy in the postconsolidation setting for high-risk neuroblastoma (ANBL19P1): A report from the Children's Oncology Group. (PubMed, Cancer) - "The administration of chemoimmunotherapy in the postconsolidation setting after tandem ASCT is feasible and tolerable. Future studies will be needed to define the population most likely to benefit from this augmented postconsolidation therapy."

Journal • Neuroblastoma • Oncology • Solid Tumor • Transplantation

Basic Science and Pathogenesis. (PubMed, Alzheimers Dement) - "Age-associated increases in plasma measures of the rate of neuronal death/damage and astrogliosis/inflammation in people with DS compared to normosomic individuals and of neuronal apoptosis in a mouse model of DS indicate an accelerated rate of brain aging, likely caused by increased APP expression and consequent production of neurotoxic Ab and exacerbated by astrogliosis/inflammation. Clinical trials are indicated for testing whether GM-CSF (sargramostim) treatment may reduce neuronal death/damage and gliosis and improve cognition in young adults with DS and in typical normosomic aging individuals."

Biomarker • Journal • Review • CNS Disorders • Dementia • Developmental Disorders • Genetic Disorders • Inflammation • CASP3 • CSF2 • FAP • GFAP • Plasma T-Tau

SESAD: Phase II Trial to Evaluate Safety and Efficacy of GM-CSF/Sargramostim in Alzheimer's Disease (clinicaltrials.gov) - P2 | N=42 | Recruiting | Sponsor: University of Colorado, Denver | Trial completion date: Oct 2027 ➔ Nov 2026 | Trial primary completion date: Apr 2027 ➔ Nov 2026

Trial completion date • Trial primary completion date • Alzheimer's Disease • CNS Disorders

Temozolomide and Survivin Long Peptide Vaccine (SurVaxM) for the Treatment of Patients With Progressing Metastatic Neuroendocrine Carcinomas (clinicaltrials.gov) - P2 | N=60 | Suspended | Sponsor: Roswell Park Cancer Institute | Not yet recruiting ➔ Suspended

Trial suspension • Endocrine Cancer • Neuroendocrine Carcinoma • Oncology • Solid Tumor

Blood measure of neuronal death is exponentially higher with age, especially in females, and halted in Alzheimer's disease by GM-CSF treatment. (PubMed, Cell Rep Med) - "GM-CSF treatment also reduces neuronal apoptosis and astrogliosis in a rat model of AD. An exponential increase in neurodegeneration with age, accelerated by astrogliosis/inflammation, may underlie the contribution of aging to cognitive decline and AD and can be halted by GM-CSF/sargramostim treatment."

Journal • Alzheimer's Disease • CNS Disorders • Inflammation • Targeted Protein Degradation • CSF2 • GFAP • UBC

A Cancer Vaccine (STEMVAC) in Combination With Chemotherapy for the Treatment of PD-L1 Negative Metastatic Triple-Negative Breast Cancer (clinicaltrials.gov) - P2 | N=20 | Not yet recruiting | Sponsor: University of Washington | Initiation date: Dec 2025 ➔ Mar 2026

Trial initiation date • Breast Cancer • Oncology • Solid Tumor • Triple Negative Breast Cancer • ENG

Tebentafusp-tebn With LDT in Metastatic UM (clinicaltrials.gov) - P1/2 | N=109 | Recruiting | Sponsor: Thomas Jefferson University | Not yet recruiting ➔ Recruiting | Trial completion date: Mar 2032 ➔ Aug 2032 | Trial primary completion date: May 2030 ➔ Oct 2030

Enrollment open • Trial completion date • Trial primary completion date • Eye Cancer • Melanoma • Oncology • Solid Tumor • Uveal Melanoma

Intermittent Sargramostim Administration Expands Proliferating Naïve T Cells, Tregs, HLA-DR+ PD-L1+ Monocytes and Myeloid-Derived Suppressor Cells: Results from a Randomized Placebo-Controlled Clinical Trial of GM-CSF in Patients with Peripheral Artery Disease (ASH 2023) - "Our findings demonstrate that intermittent dosing with sargramostim is well-tolerated and induces significant but transient increases in the numbers and activation status of T cells in the bloodstream. Sargramostim treatment also increased numbers of proliferating naïve T cells, Tregs, and certain subsets of myelo-monocytic cells with immunosuppressive phenotypes. These results support the use of intermittent sargramostim dosing to avoid excessive leukocytosis and associated adverse effects."

Clinical • Myeloid-derived suppressor cells • Acute Myelogenous Leukemia • Atherosclerosis • Bone Marrow Transplantation • Cardiovascular • Hematological Malignancies • Immune Modulation • Immunology • Leukemia • Oncology • Peripheral Arterial Disease • Transplantation • CD14 • CD8 • CSF2 • FOXP3 • IL2RA • PD-1 • PD-L1

Durable Tumor Control Following Off-the-Shelf Tumor Peptide Vaccination in Metastatic Ewing Sarcoma: A Case Report (DGHO 2025) - "Each vaccination was administered intracutaneously with sargramostim and topical imiquimod. This case illustrates the safety, feasibility, and immunogenicity of fusion-specific peptide vaccination in metastatic Ewing sarcoma. The durable clinical response, paired with functional immune data, suggests a potential benefit of this off-the-shelf immunotherapeutic strategy. Further investigation in fusion-driven sarcomas is warranted, particularly regarding optimal timing, combination with chemotherapy, and prevention of T cell exhaustion."

Case report • Clinical • Metastases • Ewing Sarcoma • Oncology • Sarcoma • Solid Tumor • CD4 • EWSR1 • FLI1

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

Enrollment open • Neuroblastoma • Oncology • Solid Tumor

ANBL19P1: Treatment With Dinutuximab, Sargramostim (GM-CSF), and Isotretinoin in Combination With Irinotecan and Temozolomide After Intensive Therapy for People With High-Risk Neuroblastoma (NBL) (clinicaltrials.gov) - P2 | N=41 | Active, not recruiting | Sponsor: Children's Oncology Group | Trial completion date: Sep 2025 ➔ Sep 2027

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

GM-CSF With Post-Transplant Cyclophosphamide (clinicaltrials.gov) - P2 | N=38 | Recruiting | Sponsor: Northside Hospital, Inc. | Trial completion date: Sep 2025 ➔ Sep 2026 | Trial primary completion date: Sep 2025 ➔ Sep 2026

Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • Hematological Malignancies • Oncology • Transplantation

STEMVAC in Patients With Early Stage Triple Negative Breast Cancer (clinicaltrials.gov) - P2 | N=33 | Active, not recruiting | Sponsor: University of Washington | Recruiting ➔ Active, not recruiting | Trial completion date: Jun 2026 ➔ Jul 2028 | Trial primary completion date: Dec 2025 ➔ Jul 2026

Enrollment closed • Trial completion date • Trial primary completion date • Breast Cancer • Hormone Receptor Negative Breast Cancer • Oncology • Solid Tumor • Triple Negative Breast Cancer • ER • HER-2 • PGR

Tebentafusp-tebn With LDT in Metastatic UM (clinicaltrials.gov) - P1/2 | N=109 | Not yet recruiting | Sponsor: Thomas Jefferson University | Trial completion date: Feb 2027 ➔ Mar 2032 | Trial primary completion date: Jan 2027 ➔ May 2030

Trial completion date • Trial primary completion date • Eye Cancer • Melanoma • Oncology • Solid Tumor • Uveal Melanoma

NCI-2018-03732: Dinutuximab, Sargramostim, and Combination Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma (clinicaltrials.gov) - P2 | N=42 | Active, not recruiting | Sponsor: National Cancer Institute (NCI) | Trial completion date: Sep 2025 ➔ Sep 2026

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

Recombinant GM-CSF drug evaluation review. (PubMed, Immunotherapy) - "Molgramostim and sargramostim are rGM-CSF which can be self-administered at home by patients with aPAP either subcutaneously or using a handheld nebulizer. In all clinical studies to date, rGM-CSF has shown a favorable safety profile with no dose limiting toxicity. This supports the application of inhaled rGM-CSF for the treatment of aPAP and the possibility of WLL as a rescue therapy."

Journal • Review • Immunology • Interstitial Lung Disease • Pulmonary Disease • Rare Diseases • Respiratory Diseases • CSF2

Testing the Addition of 131I-MIBG or Lorlatinib to Intensive Therapy in People With High-Risk Neuroblastoma (NBL) (clinicaltrials.gov) - P3 | N=750 | Recruiting | Sponsor: Children's Oncology Group | Trial completion date: Sep 2026 ➔ Sep 2030 | Trial primary completion date: Sep 2026 ➔ Sep 2030

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

Testing the Addition of 131I-MIBG or Lorlatinib to Intensive Therapy in People With High-Risk Neuroblastoma (NBL) (clinicaltrials.gov) - P3 | N=750 | Recruiting | Sponsor: Children's Oncology Group | Active, not recruiting ➔ Recruiting

Enrollment open • Ganglioneuroblastoma • Neuroblastoma • Oncology • Solid Tumor • MYCN