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214 Current Medicinal Chemistry, 2015, 22, 214-236
Peptide Deformylase: A New Target in Antibacterial, Antimalarial and
Anticancer Drug Discovery
Jaiprakash N. Sangshetti
1,*
, Firoz A. Kalam Khan
1
and Devanand B. Shinde
2
1
Y. B. Chavan College of Pharmacy, Dr. Rafiq Zakaria Campus, Rauza Baugh, Aurangabad-431001;
2
Department of Chemical Technology, Dr. B. A. M. University, Aurangabad-431004, India
Abstract Peptide deformylase (PDF) is a class of metalloenzyme responsible for catalyzing the removal of
the N-formyl group from N-terminal methionine following translation. PDF inhibitors are moving into new
phase of drug development. Initially, PDF was considered as an important target in antibacterial drug dis-
covery; however genome database searches have revealed PDF-like sequences in parasites ( P. falciparum)
and human, widening the utility of this target in antimalarial and anticancer drug discovery along with anti-
bacterial. Using structural and mechanistic information together with high throughput screening, several types of chemical
classes of PDF inhibitors with improved efficacy and specificity have been identified. Various drugs like, GSK-1322322
(Phase II), BB-83698 (Phase I), and LBM-415 (Phase I) have entered into clinical developments. Developments in the
field have prompted us to review the current aspects of PDFs, especially their structures, different classes of PDF inhibi-
tors, and molecular modeling studies. In nut shell, this review enlightens PDF as a versatile target along with its inhibitors
and future perspectives of different PDF inhibitors.
Keywords: Antibacterial, anticancer, antimalarial, clinical developments, peptide deformylase.
1. INTRODUCTION
The successful development of antibacterial drugs gener-
ated a misconception in the late 1960s and early 1970s that
infectious diseases had been conquered, resulting in a de-
crease in academic and industrial research in antibacterial
area. However, 40 years later, infectious diseases remain the
second-leading cause of death worldwide [1]. During the
past decades, many epidemiologists have reported that
pathogenic bacteria and parasites rapidly develop resistance
to medicines currently used for treatment in human infec-
tions [2]. The emergence of multi-drug resistance is of great
concern and has created a situation in which there are few or
no treatment options for infections with certain microorgan-
isms [3]. Gram-negative bacteria like P. aeruginosa, K.
pneumoniae, S. maltophila and A. baumannii are posing the
most serious treatment challenges with increasing incidence
of drug resistant isolates, and also there are fewer active an-
tibiotics available to treat infections caused by such organ-
isms [4, 5]. Among Gram-positive pathogens, the drug resis-
tant pathogens of greatest concern are S. aureus, S. epider-
midis, E. faecium and E. faecalis [6, 7]. Also, the emergence
of multi-drug resistant of M. tuberculosis strains has made
many of the currently available antitubercular drugs (ATDs)
ineffective [8].
Due to the current global resistance crisis, search for new
antibacterial agents with activity against drug-resistant
pathogens should be a high priority for the academic and
industrial researchers. It is now widely accepted that the tra-
ditional screening methods are unlikely to generate new
*Address correspondence to this author at the Y. B. Chavan College of
Pharmacy, Dr. Rafiq Zakaria Campus, Rauza Baugh, Aurangabad-431001,
India; Tel:/Fax: +91-240-23801129; E-mail: jnsangshetti@rediffmail.com
promising molecules. Alternative strategies must therefore
be developed to find new drugs. One possible strategy is to
identify a molecular target at the outset and then to screen
the available libraries of chemical compounds, looking for
“hits” with potent inhibitory capacities in-vitro. In order to
address this strategy, the identification of good and novel
target is vital [2]. Therefore, identification of a new target
which is previously untapped will play a critical role in the
development of new antibacterial agents active against resis-
tant pathogens [9]. It is important that such new target
should: (i) be present in most human pathogens (i.e., the in-
hibitors should have broad spectrum activity); (ii) be absent
in human cells; (iii) be part of an essential pathway in the
pathogens; (iv) not be inhibited by widely used anti-infective
agents; (v) be easy to assay in-vitro and in-vivo; (vi) be
highly specific for the pathogens and be non toxic to human
being; and (vii) not be result in the rapid acquisition of resis-
tance [10]. The analysis of microbial genomes has revealed
an abundance of novel and potentially useful targets, but, so
far, little has been achieved from these efforts [11]. One tar-
get that has not received much attention until recently is pep-
tide deformylase (PDF). PDF has been a possible target that
may fulfill all the above criteria essential for good target to
develop new antibacterial agents with novel mechanism of
action.
Recently, genome database searches have revealed eu-
karyotic PDF-like sequences in parasites, plants, and mam-
mals [12]. Malaria is the world’s most serious tropical para-
sitic disease and accounts for 1-3 million deaths each year
[13]. The increasing incidences of malaria reflect the devel-
opment of drug resistant strains of Plasmodium and justify
referring to malaria as a re-emerging disease [14, 15]. Rec-
ognizing the potential importance of P. falciparum peptide
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