GM-CSF and Sargramostim Publications

Pulmonary Alveolar Proteinosis

• Campo I, Carey BC, Paracchini E, et al. Inhaled GM-CSF reduces the need for whole lung lavage and improves gas exchange in autoimmune PAP patients. European Resp Journal. 2023. doi: 10.1183/13993003.01233-2023
• Tazawa R, Ueda T, Abe M, et al. Inhaled GM-CSF for Pulmonary Alveolar Proteinosis. N Engl J Med. 2019;381(10):923-932. doi:10.1056/NEJMoa1816216
• Campo I, Mariani F, Paracchini E, et al. Whole Lung Lavage Followed by Inhaled Sargramostim as Therapy of Autoimmune Pulmonary Alveolar Proteinosis [abstract]. Am J Respir Crit Care Med. 2016;193:A6438
• Tazawa R, Inoue Y, Arai T, et al. Duration of Benefit in Patients with Autoimmune Pulmonary Alveolar Proteinosis After Inhaled Granulocyte-Macrophage Colony-Stimulating Factor Therapy. Chest. 2014;145(4):729-737. doi:10.1378/chest.13-0603
• Tazawa R, Trapnell BC, Inoue Y, et al. Inhaled Granulocyte/Macrophage-Colony Stimulating Factor as Therapy for Pulmonary Alveolar Proteinosis. Am J Respir Crit Care Med. 2010;181(12):1345-1354. doi:10.1164/rccm.200906-0978OC
• Ataya A, Knight V, Carey BC, Lee E, Tarling EJ, Wang T. The Role of GM-CSF Autoantibodies in Infection and Autoimmune Pulmonary Alveolar Proteinosis: A Concise Review. Front Immunol. 2021;12:752856. doi:10.3389/fimmu.2021.752856.

Infection

• Chen TK, Batra JS, Michalik DE, Casillas J, Patel R, Ruiz ME, et al. Recombinant Human Granulocyte-Macrophage Colony-Stimulating Factor (rhu GM-CSF) as Adjuvant Therapy for Invasive Fungal Diseases. Open Forum Infect Dis. 2022;9(11):ofac535. doi:10.1093/ofid/ofac535
• Lee E, Miller C, Ataya A, Wang T. Opportunistic Infection Associated With Elevated GM-CSF Autoantibodies: A Case Series and Review of the Literature. Open Forum Infect Dis. 2022;9(5):ofac146. doi:10.1093/ofid/ofac146
• McCormick TS, Hejal RB, Leal LO, Ghannoum MA. GM-CSF: Orchestrating the Pulmonary Response to Infection. Front Pharmacol. 2022;12:735443. doi:10.3389/fphar.2021.735443
• Damiani G, McCormick TS, Leal LO, Ghannoum MA. Recombinant Human Granulocyte Macrophage-Colony Stimulating Factor Expressed in Yeast (Sargramostim): A Potential Ally to Combat Serious Infections. Clin Immunol. 2020;210:108292. doi:10.1016/j.clim.2019.108292

Immune Dysfunction

• Lazarus HM, Pitts K, Wang T, et al. Recombinant GM-CSF for Diseases of GM-CSF Insufficiency: Correcting Dysfunctional Mononuclear Phagocyte Disorders. Front. Immunol. 2023;13:1069444. doi:10.3389/fimmu.2022.1069444
• Chu S, McCormick TS, Lazarus HL, Leal LO, Ghannoum MA. Invasive Fungal Disease and the Immunocompromised Host Including Allogeneic Hematopoietic Cell Transplant Recipients: Improved Understanding and New Strategic Approach with Sargramostim. Clin Immunol. 2021;228:108731. doi:10.1016/j.clim.2021.108731

Immune Paralysis and Sepsis

• Joshi I, Carney WP, Rock EP. Utility of Monocyte HLA-DR and Rationale for Therapeutic GM-CSF in Sepsis Immunoparalysis. Front Immunol. 2023;14:1130214. doi:10.3389/fimmu.2023.1130214
• Hall MW. Immune Modulation in Pediatric Sepsis. J Pediatr Intensive Care. 2019;8(1):42–50. doi:10.1055/s-0038-1676607

Influenza, Pneumonia, and Acute Respiratory Distress Syndrome (ARDS)

• Umstead TM, Hewage EK, Mathewson M, et al. Lower Respiratory Tract Delivery, Airway Clearance, and Preclinical Efficacy of Inhaled GM-CSF in a Postinfluenza Pneumococcal Pneumonia Model. Am J Physiol Lung Cell Mol Physiol. 2020;318(4):L571-L579. doi:10.1152/ajplung.00296.2019
• Halstead ES, Umstead TM, Davies ML, et al. GM-CSF Overexpression After Influenza A Virus Infection Prevents Mortality and Moderates M1-Like Airway Monocyte/Macrophage Polarization. Respir Res. 2018;19(1):3. doi:10.1186/s12931-017-0708-5
• Rosler B, Herold S. Lung Epithelial GM-CSF Improves Host Defense Function and Epithelial Repair in Influenza Virus Pneumonia-a New Therapeutic Strategy? Mol Cell Pediatr. 2016;3(1):29. doi:10.1186/s40348-016-0055-5
• Herold S, Hoegner K, Vadáz I, et al. Inhaled Granulocyte/Macrophage Colony-Stimulating Factor as Treatment of Pneumonia-Associated Acute Respiratory Distress Syndrome. Am J Respir Crit Care Med. 2014;189(5):609-611. doi:10.1164/rccm.201311-2041LE
• Huang FF, Barnes PF, Feng Y, et al. GM-CSF in the Lung Protects Against Lethal Influenza Infection. Am J Respir Crit Care Med. 2011;184(2):259-268. doi:10.1164/rccm.201012-2036OC

COVID-19

• Joshi I, Garner F, Roychowdhury D, et al. Inhaled sargramostim (rhu GM-CSF) leads to enhanced SARS-CoV-2 virus-specific immune response and viral clearance: Results of the biomarker cohort of a randomized, double-blind, placebo-controlled phase 2B trial in non-hospitalized patients with COVID-19. Presented as a Poster Presentation at IDWEEK Oct 2023 (Boston, MA). Published: Open Forum Infectious Diseases. December 2023. doi.org: 10.1093/ofid/ofad500.351.
• Paine R, Chasse R, Halstead ES, et al. Inhaled Sargramostim (Recombinant Human Granulocyte-Macrophage Colony-Stimulating Factor) for COVID-19-Associated Acute Hypoxemia: Results of the Phase 2, Randomized, Open-Label Trial (iLeukPulm). Mil Med. 2022;usac362. doi: 10.1093/milmed/usac362
• Bosteels C, Van Damme KFA, De Leeuw E, et al. Loss of GM-CSF-Dependent Instruction of Alveolar Macrophages in COVID-19 Provides a Rationale for Inhaled GM-CSF Treatment. Cell Rep Med. 2022;3(12):100833. doi:10.1016/j.xcrm.2022.100833
• Hall MW, Joshi I, Leal L, Ooi EE. Immune Modulation in Coronavirus Diseased 2019 (COVID-19): Strategic Considerations for Personalized Therapeutic Intervention. Clin Infect Dis. 2022;74(1):144-148. doi:10.1093/cid/ciaa904
• Lazarus HM, Gale RP. G-CSF and GM-CSF Are Different. Which One Is Better for COVID-19? Acta Haematol. 2021;144(4):355-359. doi:10.1159/000510352

Immune Oncology

• Dougan M, Nguyen LH, Buchbinder EI, Lazarus HM. Sargramostim for Prophylactic Management of Gastrointestinal Immune-Related Adverse Events of Immune Checkpoint Inhibitor Therapy for Cancer. Cancers (Basel). 2024;16(3):501. Published 2024 Jan 24. doi:10.3390/cancers16030501
• Goldschmidt JH, Chou LN, Chan PK, et al. Real-world outcomes of 18,186 metastatic solid tumor outpatients: Baseline blood cell counts correlated with survival after immune checkpoint inhibitor therapy. Cancer Med. 2023 doi: 10.1002/cam4.6645.
• Li X, Li J, Zheng Y, et al. Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) Influence on Soluble and Membrane Bound ICOS in Combination with Immune Checkpoint Blockade [published online ahead of print, 2023 Jun 1]. Cancer Immunol Res. 2023;CIR-22-0702. doi:10.1158/2326-6066.CIR-22-0702
• Tarhini AA, Joshi I, Garner F. Sargramostim and Immune Checkpoint Inhibitors: Combinatorial Therapeutic Studies in Metastatic Melanoma. Immunotherapy. 2021;13(12):1011-1029. doi:10.2217/imt-2021-0119
• Lazarus HM, Ragsdale CE, Gale RP, Lyman GH. Sargramostim (rhu GM-CSF) as Cancer Therapy (Systematic Review) and An Immunomodulator. A Drug Before Its Time? Frontiers in Immunol. 2021;12:3196. doi:10.3389/fimmu.2021.706186
• Hodi FS, Lee S, McDermott DF, et al. Ipilimumab Plus Sargramostim vs Ipilimumab Alone for Treatment of Metastatic Melanoma: A Randomized Clinical Trial. JAMA. 2014;312(17):1744–1753. doi:10.1001/jama.2014.13943

Neuroblastoma

• Mora J, Modak S, Kinsey J, Ragsdale CE, Lazarus HM. GM-CSF, G-CSF, or no cytokine therapy with anti-GD2 immunotherapy for high-risk neuroblastoma. International Journal of Cancer.  doi: 10.1002/ijc.34815.
• Mora J, Bear M, Chan G, et al. Naxitamab treatment for relapsed or refractory high-risk neuroblastoma: Outcomes from the first prespecified analyses of the Pivotal 201 Trial [abstract]. Ann Oncol. 2022;33(S7):S956. doi:10.1016/j.annonc.2022.07.1017
• Mora J, Chan GC, Morgenstern DA, et al. Outpatient Administration of Naxitamab in Combination with Granulocyte-Macrophage Colony-Stimulating Factor in Patients with Refractory and/or Relapsed High-Risk Neuroblastoma: Management of Adverse Events. Cancer Rep (Hoboken). 2023;6(1):e1627. doi:10.1002/cnr2.1627
• Mora J, Castañeda A, Gorostegui M, et al. Naxitamab Combined with Granulocyte-Macrophage Colony-Stimulating Factor as Consolidation for High-Risk Neuroblastoma Patients in Complete Remission. Pediatr Blood Cancer. 2021;68(10):e29121. doi:10.1002/pbc.29121
• Kushner BH, Cheung IY, Modak S, Basu EM, Roberts SS, Cheung NK. Humanized 3F8 Anti-GD2 Monoclonal Antibody Dosing with Granulocyte-Macrophage Colony-Stimulating Factor in Patients with Resistant Neuroblastoma: A Phase 1 Clinical Trial. JAMA Oncol. Dec 1 2018;4(12):1729-1735. doi:10.1001/jamaoncol.2018.4005
• Yu AL, Gilman AL, Ozkaynak MF, et al. Anti-GD2 Antibody with GM-CSF, Interleukin-2, and Isotretinoin for Neuroblastoma. N Engl J Med. 2010;363(14):1324-1334. doi:10.1056/NEJMoa0911123

Hematology

• Solh M, Solomon SR, Holland HK, Morris LE, Bashey A. A Phase II Trial Evaluating the Efficacy and Safety of Sargramostim Post Infusion of T-Replete HLA Mismatched Peripheral Blood Haploidentical Hematopoietic Stem Cells with Post Transplant Cyclophosphamide. Blood. 2019;134(suppl 1):S4479. doi:10.1182/blood-2019-131624
• Nemunaitis J, Rosenfeld CS, Ash R, et al. Phase III Randomized, Double-Blind Placebo-Controlled Trial of rhGM-CSF Following Allogeneic Bone Marrow Transplantation. Bone Marrow Transplant. 1995;15(6):949–954
• Rowe JM, Andersen JW, Mazza JJ, et al. A Randomized Placebo-Controlled Phase III Study of Granulocyte-Macrophage Colony-Stimulating Factor in Adult Patients (> 55 to 70 years of age) with Acute Myelogenous Leukemia: A Study of the Eastern Cooperative Oncology Group (E1490). Blood. 1995;86(2):457–462

Alzheimer’s Disease

• Potter, H, Woodcock, JH, Boyd, TD, et al. Safety and Efficacy of Sargramostim (GM-CSF) in the Treatment of Alzheimer’s Disease. Alzheimer’s Dement (N Y). 2021;7(1):e12158. doi:10.1002/trc2.12158

Parkinson’s Disease

• Olson KE, Abdelmoaty MM, Namminga KL, et al. An Open-Label Multiyear Study of Sargramostim-Treated Parkinson’s Disease Patients Examining Drug Safety, Tolerability, and Immune Biomarkers from Limited Case Numbers. Transl Neurodegener. 2023;12(1):26. doi:10.1186/s40035-023-00361-1
• Abdelmoaty MM, Machhi J, Yeapuri P, et al. Monocyte Biomarkers Define Sargramostim Treatment Outcomes for Parkinson’s Disease. Clin Transl Med. 2022;12(7):e958. doi:10.1002/ctm2.958
• Olson KE, Namminga KL, Lu Y, et al. Safety, Tolerability, and Immune-Biomarker Profiling for Year-Long Sargramostim Treatment of Parkinson’s Disease. EBioMedicine. May 2021;67:103380. doi:10.1016/j.ebiom.2021.103380
• Gendelman HE, Zhang Y, Santamaria P, et al. Evaluation of the Safety and Immunomodulatory Effects of Sargramostim in a Randomized, Double-Blind Phase 1 Clinical Parkinson’s Disease Trial. NPJ Parkinsons Dis. 2017;3:10. doi:10.1038/s41531-017-0013-5

• Lazarus HM, McManus J, Gale RP. Sargramostim in Acute Radiation Syndrome. Expert Opin Biol Ther. 2022;22(11):1345-1352. doi:10.1080/14712598.2022.2143261
• Zhong Y, Pouliot M, Downey AM, et al. Efficacy of Delayed Administration of Sargramostim Up to 120 Hours Post Exposure in a Nonhuman Primate Total Body Radiation Model. Int J of Radiat Biol. 2021;97(sup1):S100-S116. doi:10.1080/09553002.2019.1673499
• Gale RP, Armitage JO. Use of Molecularly-Cloned Haematopoietic Growth Factors in Persons Exposed to Acute High-Dose, High-Dose Rate Whole-Body Ionizing Radiations. Blood Rev. 2021;45:100690. doi:10.1016/j.blre.2020.100690
• Clayton NP, Khan-Malek RC, Dangler CA, et al. Sargramostim (rhu GM-CSF) Improves Survival of Non-Human Primates with Severe Bone Marrow Suppression after Acute, High-Dose, Whole-Body Irradiation. Radiat Res. 2021;195(2):191-199. doi:10.1667/RADE-20-00131.1

Note: Not all clinical trials of sargramostim are listed.