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Accession NumberDB00020  (BTD00035, BIOD00035)
GroupsApproved, Investigational
DescriptionSargramostim is a human recombinant granulocyte macrophage colony-stimulating factor (GM-CSF) expressed in yeast. It is a glycoprotein that is 127 residues. Substitution of Leu23 leads to a difference from native protein.
Protein structureDb00020
Related Articles
Protein chemical formulaC639H1006N168O196S8
Protein average weight14434.5 Da
>DB00020 sequence
Download FASTA Format
External Identifiers Not Available
Approved Prescription Products
NameDosageStrengthRouteLabellerMarketing StartMarketing End
LeukineInjection, powder, for solution250 ug/mLIntravenous; SubcutaneousSanofi Aventis1991-05-01Not applicableUs
LeukineLiquid500 ug/mLIntravenous; SubcutaneousSanofi Aventis1996-12-01Not applicableUs
LeukineInjection, powder, for solution250 ug/mLIntravenous; SubcutaneousGenzyme Corporation2010-03-152016-06-30Us
Approved Generic Prescription ProductsNot Available
Approved Over the Counter ProductsNot Available
Unapproved/Other Products Not Available
International Brands
Brand mixturesNot Available
SaltsNot Available
CAS number123774-72-1
IndicationFor the treatment of cancer and bone marrow transplant
Structured Indications
PharmacodynamicsSargramostim is used in the treatment of bone marrow transplant recipients or those exposed to chemotherapy an recovering from acut myelogenous leukemia, Leukine or GM-CSF is a hematopoietic growth factor which stimulates the survival, clonal expansion (proliferation) and differentiation of hematopoietic progenitor cells. GM-CSF is also capable of activating mature granulocytes and macrophages. After a bone marrow transplant or chemotherapy, patients have a reduced capacity to produce red and white blood cells. Supplementing them with external sources of GM-CSF helps bring the level of neutrophils back to normal so that they can better fight infections.
Mechanism of actionSargramostim binds to the Granulocyte-macrophage colony stimulating factor receptor (GM-CSF-R-alpha or CSF2R) which stimulates a JAK2 STAT1/STAT3 signal transduction pathway. This leads to the production of hemopoietic cells and neutrophils
TargetKindPharmacological actionActionsOrganismUniProt ID
Granulocyte-macrophage colony-stimulating factor receptor subunit alphaProteinyes
HumanP15509 details
Interleukin-3 receptor subunit alphaProteinyes
HumanP26951 details
Cytokine receptor common subunit betaProteinyes
HumanP32927 details
HumanP34741 details
Bone marrow proteoglycanProteinunknownNot AvailableHumanP13727 details
Related Articles
AbsorptionNot Available
Volume of distributionNot Available
Protein bindingNot Available
MetabolismNot Available
Route of eliminationNot Available
Half lifeNot Available
  • 420 mL/min/m2 [Normal people with liquid LEUKINE (IV)]
  • 431 mL/min/m2 [Normal people with lyophilized LEUKINE (IV)]
  • 549 mL/min/m2 [Normal people with liquid LEUKINE (SC)]
  • 529 mL/min/m2 [Normal people with lyophilized LEUKINE (SC)]
ToxicityNot Available
Affected organisms
  • Humans and other mammals
PathwaysNot Available
SNP Mediated EffectsNot Available
SNP Mediated Adverse Drug ReactionsNot Available
Drug Interactions
DrugInteractionDrug group
BleomycinThe risk or severity of adverse effects can be increased when Sargramostim is combined with Bleomycin.Approved
CyclophosphamideThe risk or severity of adverse effects can be increased when Cyclophosphamide is combined with Sargramostim.Approved, Investigational
Food InteractionsNot Available
Synthesis Reference

Kaname Sugimoto, “Process for the production of human colony-stimulating factor.” U.S. Patent US4621050, issued May, 1983.

General ReferencesNot Available
External Links
ATC CodesL03AA09
AHFS CodesNot Available
PDB Entries
FDA labelNot Available
MSDSNot Available
Clinical Trials
Clinical Trials
0Not Yet RecruitingTreatmentMyasthenia Gravis1
1Active Not RecruitingTreatmentAdenocarcinomas / Cancer, Breast / Cancer, Ovarian / Colorectal Cancers1
1Active Not RecruitingTreatmentDisorder Related to Transplantation / Leukemias / Lymphoid Malignancies / Lymphoma NOS / Transplantation, Stem Cell1
1Active Not RecruitingTreatmentMalignant Melanoma1
1CompletedNot AvailableDendritic Cell Deficiency1
1CompletedPreventionHuman Immunodeficiency Virus (HIV) Infections2
1CompletedTreatmentCytopenias / Human Immunodeficiency Virus (HIV) Infections4
1CompletedTreatmentFollicular Lymphoma (FL) / Lymphoma NOS / Lymphoma, B Cell1
1CompletedTreatmentHodgkins Disease (HD) / Leukemias / Multiple Myeloma (MM) / Non-Hodgkin's Lymphoma (NHL) / Other Plasma Cell Dyscrasia (Waldenstrom, Amyloidosis)1
1CompletedTreatmentHuman Immunodeficiency Virus (HIV) Infections / Kaposi s Sarcoma (KS)2
1CompletedTreatmentHuman Immunodeficiency Virus (HIV) Infections / Non-Hodgkin's Lymphoma (NHL)1
1CompletedTreatmentMelanoma (Skin)1
1CompletedTreatmentProstatic Neoplasms1
1Not Yet RecruitingTreatmentPediatric Acute Respiratory Distress Syndrome1
1RecruitingTreatmentAcute Myelogenous Leukaemia (AML) / Multiple Myeloma (MM) / Myelodysplastic Syndromes (MDS) / Smoldering Multiple Myeloma (SMM)1
1RecruitingTreatmentAdvanced Malignancies / Cancer, Advanced1
1RecruitingTreatmentCancer, Breast / Gastric Cancers1
1RecruitingTreatmentParkinson's Disease (PD)1
1TerminatedTreatmentExtensive Stage Small Cell Lung Cancer / Recurrent Colon Cancer / Recurrent Non-small Cell Lung Cancer / Recurrent Rectal Cancer / Recurrent Small Cell Lung Cancer / Stage IV Colon Cancer / Stage IV Non-Small Cell Lung Cancer / Stage IV Rectal Cancer1
1TerminatedTreatmentHIV-1 Infections1
1, 2Active Not RecruitingTreatmentAdvanced Renal Cell Cancer / Renal Cell Cancer1
1, 2Active Not RecruitingTreatmentRecurrent Prostate Cancer1
1, 2Active Not RecruitingTreatmentRenal Cancers1
1, 2CompletedBasic ScienceCritical Illness / Immuno-suppression / Sepsis 1
1, 2CompletedTreatmentCarcinoma, Colorectal1
1, 2CompletedTreatmentLymphoma NOS / Non Hodgkin Lymphoma (NHL)1
1, 2CompletedTreatmentMalignant Melanoma1
1, 2RecruitingTreatmentMetastatic Melanoma1
1, 2TerminatedTreatmentCrohn's Disease (CD)1
2Active Not RecruitingTreatmentCancer, Breast1
2Active Not RecruitingTreatmentCancer, Ovarian / Fallopian Tube Cancer / Primary Peritoneal Cancer1
2Active Not RecruitingTreatmentCarcinoma of the Prostate2
2Active Not RecruitingTreatmentHodgkins Disease (HD) / Leukemia, Lymphocytic, Chronic, B-Cell / Leukemia, Myeloid, Acute / Multiple Myeloma (MM) / Myelodysplastic Syndromes / Non-Hodgkin's Lymphoma (NHL)1
2Active Not RecruitingTreatmentLeukemias2
2Active Not RecruitingTreatmentMetastatic Colon Cancer / Metastatic Rectal Cancer1
2Active Not RecruitingTreatmentMyelomas1
2CompletedTreatmentAcute Lymphocytic Leukemia (ALL) / Acute Myelogenous Leukaemia (AML) / Chronic Lymphocytic Leukaemia (CLL) / Chronic Myelogenous Leukemia (CML) / Hodgkins Disease (HD) / Multiple Myeloma (MM) / Myelodysplastic Syndromes / Non-Hodgkin's Lymphoma (NHL) / Peripheral T-Cell Lymphoma (PTCL)1
2CompletedTreatmentAcute Myeloid Leukaemias (AML) / Chronic Myeloid Leukemia (CML) / Myelodysplastic Syndromes (MDS)1
2CompletedTreatmentAdult Acute Lymphoblastic Leukemia in Remission / Adult Acute Megakaryoblastic Leukemia (M7) / Adult Acute Minimally Differentiated Myeloid Leukemia (M0) / Adult Acute Monoblastic Leukemia (M5a) / Adult Acute Monocytic Leukemia (M5b) / Adult Acute Myeloblastic Leukemia With Maturation (M2) / Adult Acute Myeloblastic Leukemia Without Maturation (M1) / Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities / Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) / Adult Acute Myeloid Leukemia With T(16;16)(p13;q22) / Adult Acute Myeloid Leukemia With T(8;21)(q22;q22) / Adult Acute Myelomonocytic Leukemia (M4) / Adult Erythroleukemia (M6a) / Adult Pure Erythroid Leukemia (M6b) / Chronic Myelomonocytic Leukemia / De Novo Myelodysplastic Syndromes / Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable / Previously Treated Myelodysplastic Syndromes / Recurrent Adult Acute Lymphoblastic Leukemia / Recurrent Adult Acute Myeloid Leukemia / Refractory Anemia / Refractory Anemia With Excess Blasts / Refractory Anemia With Ringed Sideroblasts / Refractory Cytopenia With Multilineage Dysplasia / Secondary Acute Myeloid Leukemia / Secondary Myelodysplastic Syndromes / Untreated Adult Acute Lymphoblastic Leukemia / Untreated Adult Acute Myeloid Leukemia1
2CompletedTreatmentBurkitt's Lymphoma / Lymphoma, Large-Cell, Diffuse / Lymphoma, Small Noncleaved-Cell1
2CompletedTreatmentCancer, Breast1
2CompletedTreatmentCancer, Ovarian / Fallopian Tube Cancer / Peritoneal Cancer1
2CompletedTreatmentCancer, Ovarian / Fallopian Tube Cancer / Primary Peritoneal Cancer1
2CompletedTreatmentCarcinoma of the Prostate2
2CompletedTreatmentChronic Hepatitis B Infection1
2CompletedTreatmentChronic Lymphocytic Leukaemia (CLL)1
2CompletedTreatmentCrohn's Disease (CD)1
2CompletedTreatmentHereditary Pulmonary Alveolar Proteinosis1
2CompletedTreatmentHuman Immunodeficiency Virus (HIV) Infections1
2CompletedTreatmentInflammatory carcinoma of the breast / Locally Advanced Breast Cancer (LABC)1
2CompletedTreatmentLeishmaniasis, Cutaneous1
2CompletedTreatmentLeukemias / Lymphoma NOS1
2CompletedTreatmentLiver Metastases / Uveal Melanoma1
2CompletedTreatmentLymphoma NOS3
2CompletedTreatmentMalignant Melanoma2
2CompletedTreatmentMelanoma (Skin)1
2CompletedTreatmentMetastatic Melanoma2
2CompletedTreatmentPeripheral Arterial Disease (PAD)1
2CompletedTreatmentProstatic Neoplasms2
2CompletedTreatmentRecurrent Melanoma / Stage III Melanoma / Stage IV Melanoma1
2CompletedTreatmentRefractory Plasma Cell Neoplasm / Stage II Multiple Myeloma / Stage III Multiple Myeloma1
2CompletedTreatmentRenal Cell Carcinoma (RCC)1
2CompletedTreatmentRespiratory Distress Syndrome, Adult1
2CompletedTreatmentRhabdomyosarcomas / Sarcoma, Ewing's1
2Not Yet RecruitingTreatmentAcute Myeloid Leukaemias (AML) / Myelodysplastic Syndrome1
2Not Yet RecruitingTreatmentAcute Myeloid Leukemia NOS1
2Not Yet RecruitingTreatmentBiliary Cancer1
2Not Yet RecruitingTreatmentEffects of Immunotherapy / Neoplasm, Residual / Neuroblastomas1
2RecruitingPreventionCancer, Breast2
2RecruitingTreatmentAcute Myeloid Leukaemias (AML) / Myelodysplastic Syndrome1
2RecruitingTreatmentAdrenoleukodystrophy / Alpha Mannosidosis / Aspartylglucosaminuria / Fucosidosis / Gangliosidosis / Glycoprotein Metabolic Disorders / Hunter Syndrome / Hurler's Syndrome / I-Cell Disease / Leukodystrophy, Globoid Cell / Maroteaux Lamy Syndrome / Metachromatic Leukodystrophy / Mucopolysaccharidosis II / Mucopolysaccharidosis VI / Mucopolysaccharidosis VII / Niemann Pick B / Niemann Pick C Subtype 2 / Osteopetrosis / Peroxisomal Disorders / Sly Syndrome / Sphingolipidosis / Type 1 Mucopolysaccharidosis1
2RecruitingTreatmentAlzheimer's Disease (AD)1
2RecruitingTreatmentAutoimmune Pulmonary Alveolar Proteinosis1
2RecruitingTreatmentDementia Alzheimer's Type1
2RecruitingTreatmentGlioblastomas / Gliosarcoma1
2RecruitingTreatmentNon-Small-Cell Lung Carcinoma (NSCLC)1
2RecruitingTreatmentPulmonary Alveolar Proteinosis (PAP)1
2RecruitingTreatmentRecurrent Osteosarcoma1
2TerminatedDiagnosticLymphoma NOS1
2TerminatedTreatmentCrohn's Disease (CD)2
2TerminatedTreatmentFollicular Lymphoma (FL)1
2TerminatedTreatmentHematologic Diseases1
2TerminatedTreatmentHormone Refractory Prostate Cancer1
2TerminatedTreatmentLymphoma NOS1
2TerminatedTreatmentLymphoma, B-Cell / Lymphoma, Diffuse / Non-Hodgkin's Lymphoma (NHL)1
2TerminatedTreatmentMultiple Myeloma and Plasma Cell Neoplasm1
2TerminatedTreatmentProstatic Neoplasms2
2TerminatedTreatmentPulmonary Alveolar Proteinosis (PAP)1
2WithdrawnTreatmentCarcinoma of the Prostate1
2WithdrawnTreatmentRenal Cell Carcinoma (RCC)1
2, 3Enrolling by InvitationTreatmentPulmonary Alveolar Proteinosis (PAP)1
3Active Not RecruitingTreatmentProstate Cancer Metastatic1
3CompletedTreatmentAcute Myeloid Leukaemias (AML)1
3CompletedTreatmentCrohn's Disease (CD)1
3CompletedTreatmentCytomegalovirus Retinitis / Human Immunodeficiency Virus (HIV) Infections1
3CompletedTreatmentGraft Versus Host Disease (GVHD) / Multiple Myeloma (MM)1
3CompletedTreatmentHuman Immunodeficiency Virus (HIV) Infections / Non-Hodgkin's Lymphoma (NHL)1
3CompletedTreatmentLeukemia,Myeloid, Chronic1
3SuspendedTreatmentLymphoma NOS1
3TerminatedTreatmentBladder Cancers1
3TerminatedTreatmentCrohn's Disease (CD)2
4CompletedTreatmentAcute Myeloblastic Leukemia1
4CompletedTreatmentHIV Disease1
4RecruitingTreatmentCritical Injury (Trauma) in Children1
Not AvailableCompletedTreatmentCytomegalovirus Retinitis / Human Immunodeficiency Virus (HIV) Infections1
Not AvailableCompletedTreatmentCytopenias / Human Immunodeficiency Virus (HIV) Infections2
Not AvailableCompletedTreatmentEndotoxaemia / Inflammatory processes / Multi Organ Dysfunction Syndrome / Sepsis 1
Not AvailableCompletedTreatmentHuman Immunodeficiency Virus (HIV) Infections1
Not AvailableCompletedTreatmentLeukemias1
Not AvailableRecruitingTreatmentNeuroblastomas1
Not AvailableRecruitingTreatmentPeripheral Arterial Disease (PAD)1
Not AvailableTerminatedTreatmentOsteosarcomas1
ManufacturersNot Available
Dosage forms
Injection, powder, for solutionIntravenous; Subcutaneous250 ug/mL
LiquidIntravenous; Subcutaneous500 ug/mL
Unit descriptionCostUnit
Leukine 250 mcg vial204.79USD vial
Bio-immunex capsule0.42USD capsule
DrugBank does not sell nor buy drugs. Pricing information is supplied for informational purposes only.
Patent NumberPediatric ExtensionApprovedExpires (estimated)
CA1341150 No2000-12-052017-12-05Canada
Experimental Properties
isoelectric point5.05Not Available
DescriptionNot Available
KingdomOrganic Compounds
Super ClassOrganic Acids
ClassCarboxylic Acids and Derivatives
Sub ClassAmino Acids, Peptides, and Analogues
Direct ParentPeptides
Alternative ParentsNot Available
SubstituentsNot Available
Molecular FrameworkNot Available
External DescriptorsNot Available


Pharmacological action
General Function:
Receptor activity
Specific Function:
Low affinity receptor for granulocyte-macrophage colony-stimulating factor. Transduces a signal that results in the proliferation, differentiation, and functional activation of hematopoietic cells.
Gene Name:
Uniprot ID:
Molecular Weight:
46206.185 Da
  1. Wang Y, Cai D, Brendel C, Barett C, Erben P, Manley PW, Hochhaus A, Neubauer A, Burchert A: Adaptive secretion of granulocyte-macrophage colony-stimulating factor (GM-CSF) mediates imatinib and nilotinib resistance in BCR/ABL+ progenitors via JAK-2/STAT-5 pathway activation. Blood. 2007 Mar 1;109(5):2147-55. Epub 2006 Nov 7. [PubMed:17090651 ]
  2. Chen J, Carcamo JM, Golde DW: The alpha subunit of the granulocyte-macrophage colony-stimulating factor receptor interacts with c-Kit and inhibits c-Kit signaling. J Biol Chem. 2006 Aug 4;281(31):22421-6. Epub 2006 Jun 7. [PubMed:16760463 ]
  3. Lencz T, Morgan TV, Athanasiou M, Dain B, Reed CR, Kane JM, Kucherlapati R, Malhotra AK: Converging evidence for a pseudoautosomal cytokine receptor gene locus in schizophrenia. Mol Psychiatry. 2007 Jun;12(6):572-80. Epub 2007 Mar 20. [PubMed:17522711 ]
  4. Xiao R, Zhang R, Wang YL, Zhu ZL, Chen T, Yang JH: [Expression of soluble GM-CSF-Ralpha in patients with acute myeloid leukemia]. Zhongguo Shi Yan Xue Ye Xue Za Zhi. 2006 Apr;14(2):225-7. [PubMed:16638185 ]
  5. Chen X, Ji ZL, Chen YZ: TTD: Therapeutic Target Database. Nucleic Acids Res. 2002 Jan 1;30(1):412-5. [PubMed:11752352 ]
Pharmacological action
General Function:
Interleukin-3 receptor activity
Specific Function:
This is a receptor for interleukin-3.
Gene Name:
Uniprot ID:
Molecular Weight:
43329.585 Da
  1. Eksioglu EA, Mahmood SS, Chang M, Reddy V: GM-CSF promotes differentiation of human dendritic cells and T lymphocytes toward a predominantly type 1 proinflammatory response. Exp Hematol. 2007 Aug;35(8):1163-71. Epub 2007 Jun 11. [PubMed:17562355 ]
  2. Sakhno LV, Leplina OIu, Tikhonova MA, Raspai ZhM, Gileva IP, Nikonov SD, Zhdanov OA, Ostanin AA, Chernykh ER: [Characteristics of A-interferon-generated dendritic cells in patients with pulmonary tuberculosis]. Probl Tuberk Bolezn Legk. 2007;(3):42-6. [PubMed:17500228 ]
  3. Ward KA, Stewart LA, Schwarer AP: CD34+-derived CD11c+ + + BDCA-1+ + CD123+ + DC: expansion of a phenotypically undescribed myeloid DC1 population for use in adoptive immunotherapy. Cytotherapy. 2006;8(2):130-40. [PubMed:16698686 ]
  4. Lencz T, Morgan TV, Athanasiou M, Dain B, Reed CR, Kane JM, Kucherlapati R, Malhotra AK: Converging evidence for a pseudoautosomal cytokine receptor gene locus in schizophrenia. Mol Psychiatry. 2007 Jun;12(6):572-80. Epub 2007 Mar 20. [PubMed:17522711 ]
Pharmacological action
General Function:
Receptor activity
Specific Function:
High affinity receptor for interleukin-3, interleukin-5 and granulocyte-macrophage colony-stimulating factor.
Gene Name:
Uniprot ID:
Molecular Weight:
97334.89 Da
  1. Overington JP, Al-Lazikani B, Hopkins AL: How many drug targets are there? Nat Rev Drug Discov. 2006 Dec;5(12):993-6. [PubMed:17139284 ]
  2. Imming P, Sinning C, Meyer A: Drugs, their targets and the nature and number of drug targets. Nat Rev Drug Discov. 2006 Oct;5(10):821-34. [PubMed:17016423 ]
  3. Shen Y, Baker E, Callen DF, Sutherland GR, Willson TA, Rakar S, Gough NM: Localization of the human GM-CSF receptor beta chain gene (CSF2RB) to chromosome 22q12.2-->q13.1. Cytogenet Cell Genet. 1992;61(3):175-7. [PubMed:1424804 ]
Pharmacological action
General Function:
Pdz domain binding
Specific Function:
Cell surface proteoglycan that bears heparan sulfate. Regulates dendritic arbor morphogenesis (By similarity).
Gene Name:
Uniprot ID:
Molecular Weight:
22159.62 Da
  1. Overington JP, Al-Lazikani B, Hopkins AL: How many drug targets are there? Nat Rev Drug Discov. 2006 Dec;5(12):993-6. [PubMed:17139284 ]
  2. Imming P, Sinning C, Meyer A: Drugs, their targets and the nature and number of drug targets. Nat Rev Drug Discov. 2006 Oct;5(10):821-34. [PubMed:17016423 ]
  3. Modrowski D, Basle M, Lomri A, Marie PJ: Syndecan-2 is involved in the mitogenic activity and signaling of granulocyte-macrophage colony-stimulating factor in osteoblasts. J Biol Chem. 2000 Mar 31;275(13):9178-85. [PubMed:10734053 ]
Pharmacological action
General Function:
Heparin binding
Specific Function:
Cytotoxin and helminthotoxin. Also induces non-cytolytic histamine release from human basophils. Involved in antiparasitic defense mechanisms and immune hypersensitivity reactions. The proform acts as a proteinase inhibitor, reducing the activity of PAPPA.
Gene Name:
Uniprot ID:
Molecular Weight:
25205.345 Da
  1. Menon K, Wu Y, Haas J, Sahu SK, Yang B, Zaheer A: Diminished degradation of myelin basic protein by anti-sulfatide antibody and interferon-gamma in myelin from glia maturation factor-deficient mice. Neurosci Res. 2007 Jun;58(2):156-63. Epub 2007 Feb 22. [PubMed:17383764 ]
  2. Letuve S, Lajoie-Kadoch S, Audusseau S, Rothenberg ME, Fiset PO, Ludwig MS, Hamid Q: IL-17E upregulates the expression of proinflammatory cytokines in lung fibroblasts. J Allergy Clin Immunol. 2006 Mar;117(3):590-6. Epub 2006 Feb 8. [PubMed:16522458 ]
  3. Kang JH, Lee da H, Seo H, Park JS, Nam KH, Shin SY, Park CS, Chung IY: Regulation of functional phenotypes of cord blood derived eosinophils by gamma-secretase inhibitor. Am J Respir Cell Mol Biol. 2007 Nov;37(5):571-7. Epub 2007 Jun 28. [PubMed:17600316 ]
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Drug created on June 13, 2005 07:24 / Updated on August 17, 2016 12:23