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@ARTICLE{BlattnerJohnson:169858,
author = {M. Blattner-Johnson$^*$ and D. T. W. Jones$^*$ and E.
Pfaff$^*$},
title = {{P}recision medicine in pediatric solid cancers.},
journal = {Seminars in cancer biology},
volume = {84},
issn = {1044-579X},
address = {London},
publisher = {Academic Press},
reportid = {DKFZ-2021-01592},
pages = {214-227},
year = {2022},
note = {#EA:B360#LA:B360# / 2022 Sep;84:214-227},
abstract = {Despite huge advances in the diagnosis and treatment of
pediatric cancers over the past several decades, it remains
one of the leading causes of death during childhood in
developed countries. The development of new targeted
treatments for these diseases has been hampered by two major
factors. First, the extremely heterogeneous nature of the
types of tumors encountered in this age group, and their
fundamental differences from common adult carcinomas, has
made it hard to truly get a handle on the complexities of
the underlying biology driving tumor growth. Second, a
reluctance of the pharmaceutical industry to develop
products or trials for this population due to the relatively
small size of the 'market', and a too-easy mechanism of
obtaining waivers for pediatric development of adult
oncology drugs based on disease type rather than mechanism
of action, led to significant difficulties in getting access
to new drugs. Thankfully, the field has now started to
change, both scientifically and from a regulatory
perspective, in order to address some of these challenges.
In this review, we will examine some of the recent insights
into molecular features which make pediatric tumors so
unique and how these might represent therapeutic targets;
highlight ongoing international initiatives for providing
comprehensive, personalized genomic profiling of childhood
tumors in a clinically-relevant timeframe, and look briefly
at where the field of pediatric precision oncology may be
heading in future.},
subtyp = {Review Article},
keywords = {Genomics (Other) / Pediatric cancer (Other) / Precision
medicine (Other) / Targeted therapy (Other)},
cin = {B360},
ddc = {610},
cid = {I:(DE-He78)B360-20160331},
pnm = {312 - Funktionelle und strukturelle Genomforschung
(POF4-312)},
pid = {G:(DE-HGF)POF4-312},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:34116162},
doi = {10.1016/j.semcancer.2021.06.008},
url = {https://inrepo02.dkfz.de/record/169858},
}