SFK Inhibitors as New Strategy for RMS Treatment
Luigi Bagella
1,2*
and Irene Marchesi
1
Department of Biomedical Sciences, and National Institute of Biostructures and Biosystems, University of Sassari, Sassari, Italy
2
Sbarro Institute for Cancer Research and Molecular Medicine, Center for Biotechnology, College of Science and Technology, Temple University, Philadelphia USA
*Corresponding author: Luigi Bagella, Adjunct Associate Professor Sbarro Health Research Organization Center of Biotechnology Deptartment of Biology College of
Science and Technology, Temple University, BioLife Science Building, Philadelphia, USA, Tel: 215-204-9524; Fax: 215-204-9522; E-mail: bagella@temple.edu
Received date: January 07, 2016; Accepted date: February 11, 2016; Published date: February 16, 2016
Copyright: © 2016 Bagella L, et al. This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted
use, distribution, and reproduction in any medium, provided the original author and source are credited.
Introduction
A new study investigates about the activity of a new SFK inhibitor in
RMS. SI221 is a pyrazolo [3,4-d] pyrimidine derivative able to reduce
both apoptosis and diferentiation in eRMS and aRMS. SFKs are
frequently overexpressed in RMS and their inhibition can be a good
strategy for RMS treatment.
Rhabdomyosarcoma (RMS) represents approximately half of the
total pediatric sof tissue sarcoma (STS), slightly more frequent in
males but equally distributed across racial groups [1].
RMS is characterized by the expression of genes involved in the
early myogenesis which fail to complete diferentiation and cell cycle
arrest [2]. It can be divided into 2 major histological subtypes:
embryonal (eRMS), mostly found in childhood, and alveolar (aRMS)
common in both children and adults [1].
Te outcome of RMS patients is improved in the last decades (5-
year survival rates of ~70%), however, patients with tumor relapse afer
treatment or with metastasis at diagnosis are rarely cured [3,4].
Moreover, frequent toxicity and long-term side efects associated with
the therapy, dramatically afect the quality of life [1]. Consequently,
new specifc therapeutic strategies, able to reduce toxicity and side
efects, are required for the treatment of RMS. Te induction of
diferentiation and/or cell death through the inhibition of specifc
enzyme involved in carcinogenesis and tumor progression can be a
promising strategy for RMS patients. For instance, several studies focus
on the role of epigenetic changes in RMS with the purpose to
investigate if the modulation of the activity of some epigenetic enzyme
can help to induce apoptosis or diferentiation in RMS cells [5-8].
Recently, Casini and coworkers analyzed efects of a new SRC family
kinase (SFK) inhibitor in RMS. Tis molecule called SI221 is a
pyrazolo [3,4-d] pyrimidine derivative that showed an antiproliferative
and pro-apoptotic activity in several tumors [9-11]. Casini et al.,
demonstrated that SI221 specifcally inhibited SFKs and in particular
YES, a SKK member that have an oncogenic role in eRMS and aRMS
(described below) [12]. SI221 was able to induce apoptosis, to reduce
cell migration and invasion with low cytotoxic efects on normal cells.
Moreover, treated cells showed a partial rescue of muscular phenotype
and an increase of myogenic markers. Tese efects were dependent by
the activation of p38 pathway [13].
SFK is a family of non-receptor tyrosine kinases that includes c-
SRC, FYN, YES, BLK, YRK, FGR, HCK, LCK and LYN. Tese enzymes
are involved in several biological processes as cell proliferation,
adhesion, invasion and motility; their expression or activity is
frequently altered in cancer [14]. SKFs modulate adhesion, invasion
and cell motility mainly through the disruption of adherents and focal
junctions.
Adherent junctions are responsible for cell–cell adhesion and they
are composed by E-cadherin homodimers of adjacent cells connected
to the actin cytoskeleton through the complex α-catenin-β-catenin and
p120 catenin. c-SRC activated by protein tyrosine phosphatase 1B-
dependent de-phosphorylation, associates to this complex promoting
the disassembly of adherents junction. Focal adhesions are responsible
for the binding of cells to the extracellular matrix through
heterodimers α- and β-integrin. A cytoplasmic complex connects
integrins to the cytoskeleton. SFKs activate focal-adhesion kinase
(FAK) that in turn phosphorylate several component of the junctions
promoting cytoskeleton changes and focal adhesion disruption
(reviewed in [14]). Furthermore, c-SRC induces tyrosine
phosphorylation of RRAS, suppressing integrin activity, cell-matrix
adhesion and disrupting focal adhesion [15]
Moreover, SFKs induce phosphorylation, ubiquitylation and
endocytosis of E-cadherin, promoting the release of cells from the
matrix and from each other [16].
Several papers explore the role of SFK and associated pathways in
sarcomas.
A frst screening showed that SRC is activated in human sarcomas
and sarcoma cell lines [17]. Moreover, global tyrosine phosphorylation
analysis in sarcoma cell lines and human tumor samples showed an
increase of the expression and phosphorylation of tyrosine kinases
including several members of SFKs as, for instance, c-SRC, LYN, and
FAK [18]. Furthermore, the treatment of several sarcoma cell lines with
Dasatinib, a SFK inhibitor, blocks SRC downstream pathways
inhibiting FAK and p130cas signaling. Dasatinib inhibited cell motility
and invasion, indicating SFKs as good targets for sarcoma treatment
[17] (Table 1).
Clinical phase (ClinicalTrials.gov)
Dasatinibi
b
eRMS,
aRMS
Phase 1/2 for several tumors, also in combination with
other drugs
PP2 eRMS,
aRMS
Preclinical
Saracatini
b
eRMS,
aRMS
Phase 2/3 for several tumors Phase 2 for RMS (Adult)
SI221 eRMS,
aRMS
Preclinical
Table 1: SFK inhibitors tested in RMS.
For what concern specifcally RMS, it has been shown that SFKs
have a role in both eRMS and aRMS. For instance, SFC increases
oncogenic activity of Caveolin-1 through its phosphorylation
Bagella and Marchesi, Chemo Open Access 2016,
5:2
DOI: 10.4172/2167-7700.1000189
Short Communication Open Access
Chemo Open Access
ISSN:2167-7700 CMT, an Open Access Journal
Volume 5 • Issue 2 • 1000189
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ISSN: 2167-7700
Chemotherapy: Open Access