title: Targeting the fetal acetylcholine receptor in rhabdomyosarcoma.
abstract: BACKGROUND
  Rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma of childhood and adolescence. Recent efforts to enhance overall survival of patients with clinically advanced RMS have failed and there is a demand for conceptually novel treatments. Immune therapeutic options targeting the fetal nicotinic acetylcholine receptor (fnAChR), which is broadly expressed on RMS, are novel approaches to overcome the therapeutic resistance of RMS. Expression of the fnAChR is restricted to developing fetal muscles, some apparently dispensable ocular muscle fibers and thymic myoid cells. Therefore, after-birth fnAChR is a tumor-associated and almost tumor-specific antigen on RMS cells.
  
  
  METHODS
  This review gives an overview on nAChR function and expression pattern in RMS tumor cells, and deals with the immunological significance of fnAChR-expressing cells, including the risk of anti-nAChR autoimmunity as a potential side effect of fnAChR-directed immunotherapies. The article also addresses the advantages and disadvantages of vaccination strategies, immunotoxins and chimeric T cells targeting the fnAChR.
  
  
  CONCLUSIONS
  Finally, we suggest technical and biological strategies to improve the available immunotherapeutic tools including increasing the in vivo expression of the target fnAChR on RMS cells.
authors.affiliation: University Medical Centre Mannheim, University of Heidelberg, Institute of Pathology, Theodor-Kutzer-Ufer 1-3, D-68135 Mannheim, Germany. katja.simon-keller@medma.uni-heidelberg.de
authors.forename: Katja
authors.initials: K
authors.lastname: Simon-Keller
authors.forename: Stefan
authors.initials: S
authors.lastname: Barth
authors.forename: Angela
authors.initials: A
authors.lastname: Vincent
authors.forename: Alexander
authors.initials: A
authors.lastname: Marx
mesh_terms: D000818:Animals; D005333:Fetus / Q000276:immunology / Q000378:metabolism*; D006801:Humans; D007167:Immunotherapy; D011950:Receptors, Cholinergic / Q000276:immunology / Q000378:metabolism*; D012208:Rhabdomyosarcoma / Q000276:immunology / Q000378:metabolism* / Q000628:therapy; D012983:Soft Tissue Neoplasms / Q000276:immunology / Q000378:metabolism* / Q000628:therapy
chemical_list: D011950:Receptors, Cholinergic