The safety and efficacy of investigational products or uses has not been determined and is not approved by the United States Food and Drug Administration (FDA)

Pulmonary Alveolar Proteinosis

• Inhaled GM-CSF reduces the need for whole lung lavage and improves gas exchange in autoimmune PAP patients
  Campo I, Carey BC, Paracchini E, Kadija Z, De Silvestri A, Rodi G, De Amici M, Torre C, Zorzetto M, Griese M, Meloni F, Corsico AG, Trapnell BC, Mariani F. Eur Respir J. 2024;63(1):2301233. doi: 10.1183/13993003.01233-2023

• Inhaled GM-CSF for pulmonary alveolar proteinosis
  Tazawa R, Ueda T, Abe M, Tatsumi K, Eda R, Kondoh S, Morimoto K, Tanaka T, Yamaguchi E, Takahashi A, Oda M, Ishii H, Izumi S, Sugiyama H, Nakagawa A, Tomii K, Suzuki M, Konno S, Ohkouchi S, Tode N, Handa T, Hirai T, Inoue Y, Arai T, Asakawa K, Sakagami T, Hashimoto A, Tanaka T, Takada T, Mikami A, Kitamura N, Nakata K. N Engl J Med. 2019;381(10):923-932. doi:10.1056/NEJMoa1816216

• Whole lung lavage followed by inhaled sargramostim as therapy of autoimmune pulmonary alveolar proteinosis
  Campo I, Mariani F, Paracchini E, Kadija Z, Zorzetto M, Tinelli C, Rodi G, Meloni F, Griese M, Trapnell BC, Scheuch G, Muellinger B, Luisetti M. Poster presented at American Thoracic Society (ATS) International Conference; May 18, 2016; San Francisco, CA. Abstract published in Am J Respir Crit Care Med. 2016;193:A6438

• Duration of benefit in patients with autoimmune pulmonary alveolar proteinosis after inhaled granulocyte-macrophage colony-stimulating factor therapy
  Tazawa R, Inoue Y, Arai T, Takada T, Kasahara Y, Hojo M, Ohkouchi S, Tsuchihashi Y, Yokoba M, Eda R, Nakayama H, Ishii H, Nei T, Morimoto K, Nasuhara Y, Ebina M, Akira M, Ichiwata T, Tatsumi K, Yamaguchi E, Nakata K. Chest. 2014;145(4):729-737. doi:10.1378/chest.13-0603

• Inhaled granulocyte/macrophage-colony stimulating factor as therapy for pulmonary alveolar proteinosis
  Tazawa R, Trapnell BC, Inoue Y, Arai T, Takada T, Nasuhara Y, Hizawa N, Kasahara Y, Tatsumi K, Hojo M, Ishii H, Yokoba M, Tanaka N, Yamaguchi E, Eda R, Tsuchihashi Y, Morimoto K, Akira M, Terada M, Otsuka J, Ebina M, Kaneko C, Nukiwa T, Krischer JP, Akazawa K, Nakata K. Am J Respir Crit Care Med. 2010;181(12):1345-1354. doi:10.1164/rccm.200906-0978OC

• The role of GM-CSF autoantibodies in infection and autoimmune pulmonary alveolar proteinosis: A concise review
  Ataya A, Knight V, Carey BC, Lee E, Tarling EJ, Wang T. Front Immunol. 2021;12:752856. doi:10.3389/fimmu.2021.752856.

Infection

• Recombinant human granulocyte-macrophage colony-stimulating factor (rhu GM-CSF) as adjuvant therapy for invasive fungal diseases
  Chen TK, Batra JS, Michalik DE, Casillas J, Patel R, Ruiz ME, Hara H, Patel B, Kadapakkam M, Ch’Ng J, Small CB, Zagaliotis P, Ragsdale CE, Leal LO, Roilides E, Walsh TJ. Open Forum Infect Dis. 2022;9(11):ofac535. doi:10.1093/ofid/ofac535

• Opportunistic infection associated with elevated GM-CSF autoantibodies: A case series and review of the literature
  Lee E, Miller C, Ataya A, Wang T. Open Forum Infect Dis. 2022;9(5):ofac146. doi:10.1093/ofid/ofac146

• GM-CSF: Orchestrating the pulmonary response to infection
  McCormick TS, Hejal RB, Leal LO, Ghannoum MA. Front Pharmacol. 2022;12:735443. doi:10.3389/fphar.2021.735443

• Recombinant human granulocyte macrophage-colony stimulating factor expressed in yeast (sargramostim): A potential ally to combat serious infections
  Damiani G, McCormick TS, Leal LO, Ghannoum MA. Clin Immunol. 2020;210:108292. doi:10.1016/j.clim.2019.108292

Immune Dysfunction

• Recombinant GM-CSF for diseases of GM-CSF insufficiency: Correcting dysfunctional mononuclear phagocyte disorders
  Lazarus HM, Pitts K, Wang T, Lee E, Buchbinder E, Dougan M, Armstrong DG, Paine R 3rd, Ragsdale CE, Boyd T, Rock EP, Gale RP. Front. Immunol. 2023;13:1069444. doi:10.3389/fimmu.2022.1069444

• Invasive fungal disease and the immunocompromised host including allogeneic hematopoietic cell transplant recipients: Improved understanding and new strategic approach with sargramostim
  Chu S, McCormick TS, Lazarus HL, Leal LO, Ghannoum MA. Clin Immunol. 2021;228:108731. doi:10.1016/j.clim.2021.108731

Immune Paralysis and Sepsis

• Utility of monocyte HLA-DR and rationale for therapeutic GM-CSF in sepsis immunoparalysis
  Joshi I, Carney WP, Rock EP. Front Immunol. 2023;14:1130214. doi:10.3389/fimmu.2023.1130214

• Immune modulation in pediatric sepsis
  Hall MW. J Pediatr Intensive Care. 2019;8(1):42–50. doi:10.1055/s-0038-1676607

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

• Lower respiratory tract delivery, airway clearance, and preclinical efficacy of inhaled GM-CSF in a postinfluenza pneumococcal pneumonia model
  Umstead TM, Hewage EK, Mathewson M, Beaudoin S, Chroneos ZC, Wang M, Halstead ES. Am J Physiol Lung Cell Mol Physiol. 2020;318(4):L571-L579. doi:10.1152/ajplung.00296.2019

• GM-CSF overexpression after influenza A virus infection prevents mortality and moderates M1-like airway monocyte/macrophage polarization
  Halstead ES, Umstead TM, Davies ML, Kawasawa YI, Silveyra P, Howyrlak J, Yang L, Guo W, Hu S, Hewage EK, Chroneos ZC. Respir Res. 2018;19(1):3. doi:10.1186/s12931-017-0708-5

• Lung epithelial GM-CSF improves host defense function and epithelial repair in influenza virus pneumonia-a new therapeutic strategy?
  Rosler B, Herold S. Mol Cell Pediatr. 2016;3(1):29. doi:10.1186/s40348-016-0055-5

• Inhaled granulocyte/macrophage colony-stimulating factor as treatment of pneumonia-associated acute respiratory distress syndrome
  Herold S, Hoegner K, Vadász I, Gessler T, Wilhelm J, Mayer K, Morty RE, Walmrath HD, Seeger W, Lohmeyer J. Am J Respir Crit Care Med. 2014;189(5):609-611. doi:10.1164/rccm.201311-2041LE

• GM-CSF in the lung protects against lethal influenza infection
  Huang FF, Barnes PF, Feng Y, Donis R, Chroneos ZC, Idell S, Allen T, Perez DR, Whitsett JA, Dunussi-Joannopoulos K, Shams H. Am J Respir Crit Care Med. 2011;184(2):259-268. doi:10.1164/rccm.201012-2036OC

COVID-19

• 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
  Joshi I, Garner F, Roychowdhury D, Simms L, Ahuja S, McManus J, Rock EP, Perez R, Bacchelli S, Paine R 3rd. Poster presented at IDWEEK; October 11-15, 2023; Boston, MA. Abstract published in Open Form Infect Dis. 2023;10(S2):ofad500.35.1. doi:10.1093/ofid/ofad500.351

• 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)
  Paine R, Chasse R, Halstead ES, Nfonoyim J, Park DJ, Byun T, Patel B, Molina-Pallete G, Harris ES, Garner F, Simms L, Ahuja S, McManus JL, Roychowdhury DF. Mil Med. 2023;188(7-8):e2629-e2638. doi:10.1093/milmed/usac362

• Loss of GM-CSF-dependent instruction of alveolar macrophages in COVID-19 provides a rationale for inhaled GM-CSF treatment
  Bosteels C, Van Damme KFA, De Leeuw E, Declercq J, Maes B, Bosteels V, Hoste L, Naesens L, Debeuf N, Deckers J, Cole B, Pardons M, Weiskopf D, Sette A, Weygaerde YV, Malfait T, Vandecasteele SJ, Demedts IK, Slabbynck H, Allard S, Depuydt P, Van Braeckel E, De Clercq J, Martens L, Dupont S, Seurinck R, Vandamme N, Haerynck F, Roychowdhury DF, Vandekerckhove L, Guilliams M, Tavernier SJ, Lambrecht BN. Cell Rep Med. 2022;3(12):100833. doi:10.1016/j.xcrm.2022.100833

• Immune modulation in coronavirus disease 2019 (COVID-19): Strategic considerations for personalized therapeutic intervention
  Hall MW, Joshi I, Leal L, Ooi EE. Clin Infect Dis. 2022;74(1):144-148. doi:10.1093/cid/ciaa904

• G-CSF and GM-CSF are different. Which one is better for COVID-19?
  Lazarus HM, Gale RP. Acta Haematol. 2021;144(4):355-359. doi:10.1159/000510352

Immune Oncology

• Sargramostim for prophylactic management of gastrointestinal immune-related adverse events of immune checkpoint inhibitor therapy for cancer
  Dougan M, Nguyen LH, Buchbinder EI, Lazarus HM. Cancers (Basel). 2024;16(3):501. doi:10.3390/cancers16030501

• Real-world outcomes of 18,186 metastatic solid tumor outpatients: Baseline blood cell counts correlated with survival after immune checkpoint inhibitor therapy
  Goldschmidt JH, Chou LN, Chan PK, Chen L, Robert N, Kinsey J, Pitts K, Nestor M, Rock EP, Lazarus HM. Cancer Med. 2023;12(22):20783-20797. doi:10.1002/cam4.6645.

• Granulocyte-macrophage colony-stimulating factor (GM-CSF) influence on soluble and membrane bound ICOS in combination with immune checkpoint blockade
  Li X, Li J, Zheng Y, Lee SJ, Zhou J, Giobbie-Hurder A, Butterfield LH, Dranoff G, Hodi FS. Cancer Immunol Res. 2023;11(8):1100-1113. doi:10.1158/2326-6066.CIR-22-0702

• Sargramostim and immune checkpoint inhibitors: Combinatorial therapeutic studies in metastatic melanoma
  Tarhini AA, Joshi I, Garner F. Immunotherapy. 2021;13(12):1011-1029. doi:10.2217/imt-2021-0119

• Sargramostim (rhu GM-CSF) as cancer therapy (systematic review) and an immunomodulator. A drug before its time?
  Lazarus HM, Ragsdale CE, Gale RP, Lyman GH. Frontiers in Immunol. 2021;12:706186. doi:10.3389/fimmu.2021.706186

• Ipilimumab plus sargramostim vs ipilimumab alone for treatment of metastatic melanoma: A randomized clinical trial
  Hodi FS, Lee S, McDermott DF, Rao UN, Butterfield LH, Tarhini AA, Leming P, Puzanov I, Shin D, Kirkwood JM. JAMA. 2014;312(17):1744–1753. doi:10.1001/jama.2014.13943

Neuroblastoma

• GM-CSF, G-CSF, or no cytokine therapy with anti-GD2 immunotherapy for high-risk neuroblastoma
  Mora J, Modak S, Kinsey J, Ragsdale CE, Lazarus HM. Int J Cancer. 2024;154(8):1340-1364. doi: 10.1002/ijc.34815.

• Naxitamab treatment for relapsed or refractory high-risk neuroblastoma: Outcomes from the first prespecified analyses of the Pivotal 201 Trial
  Mora J, Bear M, Chan G, Morgenstern DA, Nysom K, Tornoe K, Sorenson PS, Kushner B. Poster presented at European Society for Medical Oncology (ESMO) Congress; September 9-13, 2022; Paris, France. Abstract published in Ann Oncol. 2022;33(S7):S956. doi:10.1016/j.annonc.2022.07.1017

• 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
  Mora J, Chan GC, Morgenstern DA, Nysom K, Bear MK, Tornøe K, Kushner BH. Cancer Rep (Hoboken). 2023;6(1):e1627. doi:10.1002/cnr2.1627

• Naxitamab combined with granulocyte-macrophage colony-stimulating factor as consolidation for high-risk neuroblastoma patients in complete remission
  Mora J, Castañeda A, Gorostegui M, Santa-María V, Garraus M, Muñoz JP, Varo A, Perez-Jaume S, Mañe S. Pediatr Blood Cancer. 2021;68(10):e29121. doi:10.1002/pbc.29121

• Humanized 3F8 anti-GD2 monoclonal antibody dosing with granulocyte-macrophage colony-stimulating factor in patients with resistant neuroblastoma: A phase 1 clinical trial
  Kushner BH, Cheung IY, Modak S, Basu EM, Roberts SS, Cheung NK. JAMA Oncol. 2018;4(12):1729-1735. doi:10.1001/jamaoncol.2018.4005

• Anti-GD2 antibody with GM-CSF, interleukin-2, and isotretinoin for neuroblastoma
  Yu AL, Gilman AL, Ozkaynak MF, London WB, Kreissman SG, Chen HX, Smith M, Anderson B, Villablanca JG, Matthay KK, Shimada H, Grupp SA, Seeger R, Reynolds CP, Buxton A, Reisfeld RA, Gillies SD, Cohn SL, Maris JM, Sondel PM; Children’s Oncology Group. N Engl J Med. 2010;363(14):1324-1334. doi:10.1056/NEJMoa0911123

Hematology

• 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
  Solh M, Solomon SR, Holland HK, Morris LE, Bashey A. Poster presented at American Society of Hematology (ASH) Annual Meeting; December 7-10, 2019; Orlando FL. Abstract published in Blood. 2019;134(suppl 1):S4479. doi:10.1182/blood-2019-131624

• Phase III randomized, double-blind placebo-controlled trial of rhGM-CSF following allogeneic bone marrow transplantation
  Nemunaitis J, Rosenfeld CS, Ash R, Freedman MH, Deeg HJ, Appelbaum F, Singer JW, Flomenberg N, Dalton W, Elfenbein GJ, Rifkin R, Rubin A, Agosti J, Hayes FA, Holcenberg J, Shadduck RK. Bone Marrow Transplant. 1995;15(6):949–954.

• 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)
  Rowe JM, Andersen JW, Mazza JJ, Bennett JM, Paietta E, Hayes FA, Oette D, Cassileth PA, Stadtmauer EA, Wiernik PH. Blood. 1995;86(2):457–462

Alzheimer’s Disease

• Safety and efficacy of sargramostim (GM-CSF) in the treatment of Alzheimer’s disease
  Potter H, Woodcock JH, Boyd TD, Coughlan CM, O’Shaughnessy JR, Borges MT, Thaker AA, Raj BA, Adamszuk K, Scott D, Adame V, Anton P, Chial HJ, Gray H, Daniels J, Stocker ME, Sillau SH. Alzheimer’s Dement (N Y). 2021;7(1):e12158. doi:10.1002/trc2.12158

Parkinson’s Disease

• An open-label multiyear study of sargramostim-treated Parkinson’s disease patients examining drug safety, tolerability, and immune biomarkers from limited case numbers
  Olson KE, Abdelmoaty MM, Namminga KL, Lu Y, Obaro H, Santamaria P, Mosley RL, Gendelman HE. Transl Neurodegener. 2023;12(1):26. doi:10.1186/s40035-023-00361-1

• Monocyte biomarkers define sargramostim treatment outcomes for Parkinson’s disease
  Abdelmoaty MM, Machhi J, Yeapuri P, Shahjin F, Kumar V, Olson KE, Mosley RL, Gendelman HE. Clin Transl Med. 2022;12(7):e958. doi:10.1002/ctm2.958

• Safety, tolerability, and immune-biomarker profiling for year-long sargramostim treatment of Parkinson’s disease
  Olson KE, Namminga KL, Lu Y, Schwab AD, Thurston MJ, Abdelmoaty MM, Kumar V, Wojtkiewicz M, Obaro H, Santamaria P, Mosley RL, Gendelman HE. EBioMedicine. 2021;67:103380. doi:10.1016/j.ebiom.2021.103380

• Evaluation of the safety and immunomodulatory effects of sargramostim in a randomized, double-blind phase 1 clinical Parkinson’s disease trial
  Gendelman HE, Zhang Y, Santamaria P, Olson KE, Schutt CR, Bhatti D, Shetty BLD, Lu Y, Estes KA, Standaert DG, Heinrichs-Graham E, Larson L, Meza JL, Follett M, Forsberg E, Siuzdak G, Wilson TW, Peterson C, Mosley RL. NPJ Parkinsons Dis. 2017;3:10. doi:10.1038/s41531-017-0013-5

• Sargramostim in acute radiation syndrome
  Lazarus HM, McManus J, Gale RP. Expert Opin Biol Ther. 2022;22(11):1345-1352. doi:10.1080/14712598.2022.2143261

• Efficacy of delayed administration of sargramostim up to 120 hours post exposure in a nonhuman primate total body radiation model
  Zhong Y, Pouliot M, Downey AM, Mockbee C, Roychowdhury D, Wierzbicki W, Authier S. Int J Radiat Biol. 2021;97(sup1):S100-S116. doi:10.1080/09553002.2019.1673499

• Use of molecularly-cloned haematopoietic growth factors in persons exposed to acute high-dose, high-dose rate whole-body ionizing radiations
  Gale RP, Armitage JO. Blood Rev. 2021;45:100690. doi:10.1016/j.blre.2020.100690

• Sargramostim (rhu GM-CSF) improves survival of non-human primates with severe bone marrow suppression after acute, high-dose, whole-body irradiation
  Clayton NP, Khan-Malek RC, Dangler CA, Zhang D, Ascah A, Gains M, Gardner B, Mockbee C, Keutzer JM, McManus J, Authier S. Radiat Res. 2021;195(2):191-199. doi:10.1667/RADE-20-00131.1

Note: Not all clinical trials and publications of sargramostim are listed.

The safety and efficacy of investigational products or uses has not been determined and is not approved by the United States Food and Drug Administration (FDA)