GM-CSF and sargramostim



Bosteels C, Maes B, Van Damme K, De Leeuw E, Declercq J, Delporte A, et al. Sargramostim to treat patients with acute hypoxic respiratory failure due to COVID-19 (SARPAC): A structured summary of a study protocol for a randomised controlled trial. Trials. 2020 Jun; 21(1): 491. doi:10.1186/s13063-020-04451-7

Hall MW, Joshi I, Leal L, Ooi EE. Immune modulation in COVID-19: Strategic considerations for personalized therapeutic intervention [published online ahead of print, 2020 Jul 1]. Clin Infect Dis. 2020;ciaa904. doi:10.1093/cid/ciaa904

Lazarus HM, Gale RP. G-CSF and GM-CSF Are Different. Which One Is Better for COVID-19? [published online ahead of print, 2020 Aug 13]. Acta Haelatol. 2020.  doi:10.1159/000510352

Immune Paralysis and Sepsis

Hall MW. Immune Modulation in Pediatric Sepsis. J Pediatr Intensive Care. 2019;8(1):42–50. doi:10.1055/s-0038-1676607

J. J. Presneill, T. Harris, A. G. Stewart, J. F. Cade, J. W. Wilson, A randomized phase II trial of granulocyte-macrophage colony-stimulating factor therapy in severe sepsis with respiratory dysfunction. Am J Respir Crit Care Med 166, 138-143 (2002).


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

Pneumonia and Acute Respiratory Distress Syndrome (ARDS)

B. Rosler, S. Herold, Lung epithelial GM-CSF improves host defense function and epithelial repair in influenza virus pneumonia-a new therapeutic strategy? Mol Cell Pediatr 3, 29 (2016).

S. Herold et al., Inhaled granulocyte/macrophage colony-stimulating factor as treatment of pneumonia-associated acute respiratory distress syndrome. Am J Respir Crit Care Med 189, 609-611 (2014).

E. S. Halstead et al., GM-CSF overexpression after influenza a virus infection prevents mortality and moderates M1-like airway monocyte/macrophage polarization. Respir Res 19, 3 (2018).

T. M. Umstead 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 318, L571-L579 (2020).

F. F. Huang et al., GM-CSF in the lung protects against lethal influenza infection. Am J Respir Crit Care Med 184, 259-268 (2011).

Pulmonary Alveolar Proteinosis

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

R. Tazawa et al., Inhaled granulocyte/macrophage-colony stimulating factor as therapy for pulmonary alveolar proteinosis. Am J Respir Crit Care Med 181, 1345-1354 (2010).

R. Tazawa et al., Inhaled GM-CSF for Pulmonary Alveolar Proteinosis. N Engl J Med 381, 923-932 (2019).


Immune Oncology

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

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

Lazarus HM, Ragsdale CE, et al. Sargramostim (rhu CM-CSF) as Cancer Therapy (Systematic Review) and An Immunomodulator. A Drug Before Its Time? Frontiers in Immunology. 2021;12:3196.




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.

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

Central Nervous System

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. 2021; 7:e12158.

Acute Radiation Syndrome

Clayton NP, Charpentier EJJ, LaCasse ER, Khan-Malek RC, Keutzer JM. Sargamostim accelerates leukocyte recovery and improves mortality rate at day 60 in a non-human primate model of hematopoietic acute radiation syndrome when administered 48 h after total body irradiation. Blood. 2016;128:122:2512. doi:10.1182/blood.V128.22.2512.2512

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 [published online ahead of print, 2020 Apr 2]. Blood Rev. 2020;100690. doi:10.1016/j.blre.2020.100690

Note: Not all clinical trials of sargramostim are listed.