NATURE REVIEWS | CLINICAL ONCOLOGY ADVANCE ONLINE PUBLICATION | 1
Department of Medical
Oncology (M.A., F .A.),
INSERM Unit U981
(C.V., C.L.), Department
of Biostatistics and
Epidemiology (S.M.),
Gustave Roussy and
Université Paris Sud,
94800 Villejuif, France.
Division of Research
and Cancer Medicine,
Peter MacCallum
Cancer Centre,
University of
Melbourne,
East Melbourne,
VIC 3002, Australia
(S.L.). Department of
Medical Oncology and
INSERM U916, Institut
Bergonié, 229 Cours de
l’Argonne,
33000 Bordeaux,
France (H.B.).
Correspondence to: F.A.
fandre@igr.fr
Precision medicine for metastatic breast
cancer—limitations and solutions
Monica Arnedos, Cecile Vicier, Sherene Loi, Celine Lefebvre, Stefan Michiels, Herve Bonnefoi
and Fabrice Andre
Abstract | The development of precision medicine for the management of metastatic breast cancer is an
appealing concept; however, major scientific and logistical challenges hinder its implementation in the clinic.
The identification of driver mutational events remains the biggest challenge, because, with the few exceptions
of ER, HER2, PIK3CA and AKT1, no validated oncogenic drivers of breast cancer exist. The development of
bioinformatic tools to help identify driver mutations, together with assessment of pathway activation and
dependency should help resolve this issue in the future. The occurrence of secondary resistance, such as
ESR1 mutations, following endocrine therapy poses a further challenge. Ultra-deep sequencing and monitoring
of circulating tumour DNA (ctDNA) could permit early detection of the genetic events underlying resistance
and inform on combination therapy approaches. Beside these scientific challenges, logistical and operational
issues are a major limitation to the development of precision medicine. For example, the low incidence of most
candidate genomic alterations hinders randomized trials, as the number of patients to be screened would
be too high. We discuss these limitations and the solutions, which include scaling-up the number of patients
screened for identifying a genomic alteration, the clustering of genomic alterations into pathways, and the
development of personalized medicine trials.
Arnedos, M. et al. Nat. Rev. Clin. Oncol. advance online publication 21 July 2015; doi:10.1038/nrclinonc.2015.123
Introduction
The discovery of the oestrogen receptor (ER)
1
and
human epidermal growth factor receptor‑2 (HER2)
2
as therapeutic targets in patients with breast cancer
has enabled treatment success in terms of patient out‑
comes with ER or HER2‑blocking therapies,
3,4
and set
the stage for the development of stratified medicine.
Furthermore, progress in cancer genomics research
over the past few decades has reinforced the notion
that cancer is driven by various genomics alterations.
5
As a result of different international initiatives such as
The Cancer Genome Atlas (TCGA) or the International
Cancer Genome Consortium (ICGC), the use of next‑
generation sequencing (NGS) has helped define the
genomic landscape of early stage breast cancer.
6
These
studies have revealed the high level of tumour hetero‑
geneity for each breast tumour that consists of several
molecular subsets, which are driven by distinct molecu‑
lar alterations, indicating that tumours could be treated
according to their individual molecular landscape.
Despite the exciting potential for personalized medicine,
ER and HER2 are currently the only targetable molecu‑
lar alterations with confirmed predictive and prognostic
value.
3,4
Other targeted therapies, such as mTOR and
CDK4/6 inhibitors, have been approved on the basis of
their efficacy in subgroup populations, but no predic‑
tive biomarkers have been found.
7,8
In this Review, we
discuss the potential applications of genomics to improve
the management of metastatic breast cancer (MBC), and
consider the challenges that precision medicine must
overcome before it can be widely implemented in the
clinic—most notably, those challenges that relate to
the remarkable cellular complexity of this type of cancer.
Genomic landscape of breast cancer
Analysis of the molecular features of early stage breast
cancer using NGS has led to the description of the
genomic landscape of this disease.
6
This research has
confirmed that TP53 and PIK3CA mutations are the
most frequent genomic alterations overall in all intrinsic
subtypes (28% for both genes). Amplifications in ERBB2,
FGFR1 and CCND1 follow in frequency, being observed
in 10–20% of all breast cancer subtypes. Additional
alterations are less frequent, but could be highly clinically
relevant, including PTEN mutations and deletions, and
AKT1, RB1 BRCA1 or BRCA2 mutations. Sequencing
analyses have uncovered mutations in other genes of
interest that might have some clinical relevance in breast
cancer, including KRAS, APC, NF1, SKT11, MAP2K4,
MAP3K1 and AKT2.
6
A similar genomic study focused
on patients with triple‑negative breast cancer (TNBC)
revealed a more heterogeneous molecular profile, with
some tumours having just a few molecular alterations
whereas others harboured hundreds of alterations.
9
Competing interests
F.A. receives honourarium and has a research contract with
AstraZeneca and Novartis. M.A. receives honourarium from
Novartis. The other authors declare no competing interests.
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