Schulte, J. H., Schulte, S., Heukamp, L. C., Astrahantseff, K., Stephan, H., Fischer, M., Schramm, A. and Eggert, A. (2013). Targeted Therapy for Neuroblastoma: ALK Inhibitors. Klinische Padiatr., 225 (6). S. 303 - 309. STUTTGART: GEORG THIEME VERLAG KG. ISSN 1439-3824

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Abstract

Treatment for neuroblastoma, the most common extracranial childhood tumor, spans a broad range of aggressiveness that mirrors the risk profiles of disease subtypes, with high-risk neuroblastoma still presenting a clinical challenge. Currently, most patients with relapsed neuroblastoma die of disease and present a major challenge for treatment. New therapeutic options are urgently needed to improve patient survival. Activating mutations in the gene encoding the anaplastic lymphoma kinase (ALK) remain the most frequent druggable mutations identified in neuroblastomas to date. Preclinical data support an oncogene addiction of neuroblastoma cells to mutated ALK and demonstrate that ALK inhibitory therapy strongly combats tumor models. Most recently, pediatric phase I testing has been completed for the first approved ALK inhibitor, Crizotinib, showing very encouraging antitumoral results in neuroblastoma patients. Subsequently, an international phase I study with the second generation ALK inhibitor, LDK-378, will be launched that makes ALK inhibitory therapy also available to pediatric patients in Germany.

Item Type: Journal Article
Creators:
CreatorsEmailORCIDORCID Put Code
Schulte, J. H.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Schulte, S.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Heukamp, L. C.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Astrahantseff, K.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Stephan, H.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Fischer, M.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Schramm, A.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Eggert, A.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
URN: urn:nbn:de:hbz:38-472663
DOI: 10.1055/s-0033-1357132
Journal or Publication Title: Klinische Padiatr.
Volume: 225
Number: 6
Page Range: S. 303 - 309
Date: 2013
Publisher: GEORG THIEME VERLAG KG
Place of Publication: STUTTGART
ISSN: 1439-3824
Language: English
Faculty: Unspecified
Divisions: Unspecified
Subjects: no entry
Uncontrolled Keywords:
KeywordsLanguage
ANAPLASTIC LYMPHOMA KINASE; HIGH-RISK NEUROBLASTOMA; CELL LUNG-CANCER; SOLID TUMORS; ACTIVATING MUTATIONS; ONCOGENIC MUTATIONS; N-MYC; GENE; EXPRESSION; IDENTIFICATIONMultiple languages
PediatricsMultiple languages
URI: http://kups.ub.uni-koeln.de/id/eprint/47266

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