Target Information

Target General Infomation
Target ID
T51499
Former ID
TTDI02169
Target Name
Ganglioside GM2 activator
Gene Name
GM2A
Synonyms
Cerebroside sulfate activator protein; GM2AP; Ganglioside GM2 activator isoform short; SAP3; Sphingolipid activator protein 3; GM2A
Target Type
Clinical Trial
Disease Multiple myeloma [ICD9: 203; ICD10: C90]
Function
The large binding pocket can accommodate severalsingle chain phospholipids and fatty acids, GM2A also exhibits some calcium-independent phospholipase activity (By similarity). Binds gangliosides and stimulates ganglioside GM2 degradation. It stimulates only the breakdown of ganglioside GM2 and glycolipid GA2 by beta-hexosaminidase A. It extracts single GM2 molecules from membranes and presents them in soluble form to beta- hexosaminidase A forcleavage of N-acetyl-D-galactosamine and conversion to GM3.
UniProt ID
P17900
Sequence
MQSLMQAPLLIALGLLLAAPAQAHLKKPSQLSSFSWDNCDEGKDPAVIRSLTLEPDPIIV
PGNVTLSVMGSTSVPLSSPLKVDLVLEKEVAGLWIKIPCTDYIGSCTFEHFCDVLDMLIP
TGEPCPEPLRTYGLPCHCPFKEGTYSLPKSEFVVPDLELPSWLTTGNYRIESVLSSSGKR
LGCIKIAASLKGI
Drugs and Mode of Action
Drug(s) BIW-8962 Drug Info Phase 1/2 Multiple myeloma [524360]
Target Expression Profile (TEP) and Drug Resistance Mutation (DRM)
TEP EXP Info
Pathways
NetPath Pathway TGF_beta_Receptor Signaling Pathway
References
Ref 524360ClinicalTrials.gov (NCT01898156) Two-Part, Open-Label, Multi-Center, Phase 1/2 Study of BIW-8962 as Monotherapy in Subjects With Lung Cancer. U.S. National Institutes of Health.
Ref 531613Genetically engineered humanized anti-ganglioside GM2 antibody against multiple organ metastasis produced by GM2-expressing small-cell lung cancer cells. Cancer Sci. 2011 Dec;102(12):2157-63.
Ref 550437National Cancer Institute Drug Dictionary (drug id 618862).

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