General Information of Drug Combination (ID: DCU0JZC)

Drug Combination Name
Miglustat IT-141
Indication
Disease Entry Status REF
Chronic myelogenous leukemia Investigative [1]
Component Drugs Miglustat   DM5J64S IT-141   DMAYD7O
Small molecular drug N.A.
High-throughput Screening Result Testing Cell Line: KBM-7
Zero Interaction Potency (ZIP) Score: 5.93
Bliss Independence Score: 5.93
Loewe Additivity Score: 6.89
LHighest Single Agent (HSA) Score: 6.9

Molecular Interaction Atlas of This Drug Combination

Molecular Interaction Atlas (MIA)
Indication(s) of Miglustat
Disease Entry ICD 11 Status REF
Niemann-Pick disease type C N.A. Approved [2]
Type 1 gaucher disease 5C56.0Y Approved [3]
Gaucher disease 5C56.0Y Investigative [2]
Miglustat Interacts with 1 DTT Molecule(s)
DTT Name DTT ID UniProt ID Mode of Action REF
Ceramide glucosyltransferase (UGCG) TTPHEX3 CEGT_HUMAN Inhibitor [5]
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Miglustat Interacts with 1 DOT Molecule(s)
DOT Name DOT ID UniProt ID Mode of Action REF
Cystic fibrosis transmembrane conductance regulator (CFTR) OT6B22QH CFTR_HUMAN Increases Activity [6]
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Indication(s) of IT-141
Disease Entry ICD 11 Status REF
Solid tumour/cancer 2A00-2F9Z Phase 1 [4]
IT-141 Interacts with 1 DTT Molecule(s)
DTT Name DTT ID UniProt ID Mode of Action REF
DNA topoisomerase I (TOP1) TTGTQHC TOP1_HUMAN Inhibitor [4]
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References

1 Recurrent recessive mutation in deoxyguanosine kinase causes idiopathic noncirrhotic portal hypertension.Hepatology. 2016 Jun;63(6):1977-86. doi: 10.1002/hep.28499. Epub 2016 Mar 31.
2 Miglustat FDA Label
3 URL: http://www.guidetopharmacology.org Nucleic Acids Res. 2015 Oct 12. pii: gkv1037. The IUPHAR/BPS Guide to PHARMACOLOGY in 2016: towards curated quantitative interactions between 1300 protein targets and 6000 ligands. (Ligand id: 4841).
4 Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA)
5 Effect of miglustat on bone disease in adults with type 1 Gaucher disease: a pooled analysis of three multinational, open-label studies. Clin Ther. 2007 Aug;29(8):1645-54.
6 Proteasome-dependent pharmacological rescue of cystic fibrosis transmembrane conductance regulator revealed by mutation of glycine 622. J Pharmacol Exp Ther. 2008 Apr;325(1):89-99. doi: 10.1124/jpet.107.134502. Epub 2008 Jan 29.