Major depressive disorder (MDD) is among the most
disabling medical illnesses worldwide and ranks first
among all mental health, substance and neurological
disorders in terms of disability-adjusted life years
1,2
. The
consequences of untreated or partially treated depres-
sion are enormous for patients, their families, health-
care systems and society
3
. A substantial proportion of
patients with MDD do not respond to current treat-
ments, despite many antidepressant trials and augmenta-
tion strategies
4,5
: up to ~20% of patients with MDD have
treatment-resistant depression (TRD; usually defined as
a failure to respond to two or more antidepressant med-
ication trials)
3
. Moreover, the peak efficacy of first-line
antidepressants, such as serotonin-selective reuptake
inhibitors (SSRIs), is delayed, with lag times of several
weeks to months before benefit. Although the mono-
amine systems (including the serotonin, noradrenaline
and dopamine systems) have long been the focus of
depression research and treatment, there is now a gen-
eral consensus that drug discovery must move beyond
the monoamine systems to improve patient outcomes. In
particular, the glutamate system has emerged as a vibrant
area of investigation
6,7
.
The ubiquity and complexity of the glutamate sys-
tem poses considerable obstacles for drug discovery
efforts, in large part owing to the potential for seizure
induction and other tolerability-related issues. However,
motivated by the recognition that glutamate and its spe-
cific receptor subtypes serve fundamental roles in the
regulation of synaptic plasticity and affect basic human
processes of mood, cognition, learning and reward, sev-
eral early-stage clinical neuropsychiatric programmes
have been initiated for compounds that target different
components of the glutamate system. Such compounds
include modulators of ionotropic receptors (including
N-methyl-d-aspartate receptors (NMDARs) and
α-amino-3-hydroxy-5-methyl-4-isoxazole propionic
acid receptors (AMPARs)), metabotropic glutamate
receptor (mGluR) modulators, glycine transporter 1
(GlyT1) inhibitors and glutamate release inhibitors
8
.
The NMDAR-blocking agent ketamine — an anaes-
thetic that has been available for human use since the
1960s — was reported in 2000 to induce profound clinical
improvements in the core symptoms of depression within
several hours of treatment
9
; this was an unexpected find-
ing that was later hailed as “arguably the most important
discovery [in mood disorders] in half a century” (REF. 10).
Moreover, this discovery triggered vigorous research in
both industry and academia to understand the role of
glutamate signalling in depression pathophysiology and
to develop novel treatments. Investigational drugs that
target components of the glutamate system have begun
to enter phase II and phase III trials. This Review eval-
uates the role of glutamate signalling in depression and
discusses the potential of ketamine and other glutamate-
signalling modulators as novel antidepressant agents. We
critically review the limitations of existing studies of the
clinical effects and hypothesized mechanisms of action
of glutamate-based antidepressant candidates, and detail
progress in this area. We evaluate evidence concerning
the role of the NMDAR versus other molecular targets
in the antidepressant mechanism of action of ketamine
and other candidate glutamate modulators, and conclude
with a discussion of the challenges of glutamate modu-
lation for novel drug development and opportunities for
new directions.
1
Mood and Anxiety Disorders
Program, Department of
Psychiatry; Fishberg
Department of Neuroscience;
and Friedman Brain Institute,
Icahn School of Medicine at
Mount Sinai, New York,
New York 10029, USA.
2
Clinical Neuroscience
Division, VA National Center
for PTSD; Department of
Psychiatry, Yale University
School of Medicine,
New Haven, Connecticut
06511, USA.
3
Mental Health Care Line,
Michael E. DeBakey VA
Medical Center; Menninger
Department of Psychiatry
and Behavioral Sciences,
Baylor College of Medicine,
Houston, Texas 77030, USA.
Correspondence to J.W.M.
james.murrough@mssm.edu
doi:10.1038/nrd.2017.16
Published online 17 Mar 2017
Synaptic plasticity
Activity- or experience-
dependent changes in synaptic
structure and function that are
relatively long-lasting (that is,
persisting beyond the initial
electrochemical event).
Targeting glutamate signalling in
depression: progress and prospects
James W. Murrough
1
, Chadi G. Abdallah
2
and Sanjay J. Mathew
3
Abstract | Major depressive disorder (MDD) is severely disabling, and current treatments have
limited efficacy. The glutamate N‑methyl‑d‑aspartate receptor (NMDAR) antagonist ketamine
was recently repurposed as a rapidly acting antidepressant, catalysing the vigorous investigation
of glutamate‑signalling modulators as novel therapeutic agents for depressive disorders. In this
Review, we discuss the progress made in the development of such modulators for the treatment
of depression, and examine recent preclinical and translational studies that have investigated the
mechanisms of action of glutamate‑targeting antidepressants. Fundamental questions remain
regarding the future prospects of this line of drug development, including questions concerning
safety and tolerability, efficacy, dose–response relationships and therapeutic mechanisms.
REVIEWS
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