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Send Orders for Reprints to reprints@benthamscience.ae 1381-6128/16 $58.00+.00 © 2016 Bentham Science Publishers Systems Pharmacology: A Unified Framework for Prediction of Drug-Target

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Send Orders for Reprints to reprints@benthamscience.ae

1381-6128/16 $58.00+.00 © 2016 Bentham Science Publishers

Systems Pharmacology: A Unified Framework for Prediction of Drug-Target Interactions

Duc-Hau Le*a and Ly Leb

a School of Computer Science and Engineering, Water Resources University, 175 Tay Son, Dong Da, Hanoi,

Vietnam; b School of Biotechnology, International University, Vietnam National University, Vietnam

Abstract: Background: Drug discovery is one important issue in medicine and pharmacology area Traditional

methods using target-based approach are usually time-consuming and ineffective Recently, the problems are

ap-proached in a system-level view and therefore it is called systems pharmacology This research field deals with the

problems in drug discovery by integrating various kinds of biomedical and pharmacological data and using

ad-vanced computational methods Ultimately, the problems are more effectively solved One of the most important

problem in systems pharmacology is prediction of drug-target interactions Methods: In this review, we are going

to summarize various computational methods for this problem Results: More importantly, we formed a unified

framework for the problem In addition, to study human health and disease in a more systematically and

effec-tively, we also presented an integrated scheme for a wider problem of prediction of disease-gene-drug associations

Conclusion: By presenting the unified framework and the integrated scheme, underlying computational methods for problems in systems

pharmacology can be understood and complex relationships among diseases, genes and drugs can be identified effectively

Keywords: Drug-target interaction, network-based approach, machine learning-based approach, drug-disease association, disease-gene

association, drug-gene-disease association

1 INTRODUCTION

The development of a drug from an original idea to the market

usually takes ten to seventeen years and costs about billion US

dol-lar on average [1, 2] In addition, several of them have been

with-drawn due to adverse/side effects The major reason is that the

drugs do not only interact to therapeutic targets but also to

off-targets In addition, drugs can interact with other drugs or chemicals

from food when they are used in the same patients Therefore, drug

discovery are needed to be considered at system-level, in which

many kinds of data for biomedical and pharmacological instances

are investigated simultaneously [3] In addition, computational

methods are also used to recognize interactions and mutual effects

among the instances Therefore, system pharmacology, which is a

combination between systems biology and pharmacology, becomes

promising approach to deal with this problem In which, systems

biology is the way to investigate interactions among cellular

com-ponents at system-level Therefore, systems pharmacology is a

biological and chemical approach for health and diseases [4-6]

More specifically, systems pharmacology studies how drugs work

(i.e., mode of action) at different levels such as whole human body,

organs, tissues, or cellular components [7] Computational methods

in systems pharmacology are usually used along with various kinds

of data such as –omics (genomics, transcriptomics, proteomics,

metabolomics, phenomics, interactomics) as well as

pharmacologi-cal data, etc.… [8] The final goals is to elucidate therapeutic

mechanisms of drugs (i.e., how drug work on different pathways,

cell types and tissues) and enable the process of drug discovery and

development to become more effective Based on these

computa-tional methods, many state-of-the-art technologies in modern

com-puter-aided drug design have been developed [9]

There is a number of problems in drug discovery such as

identi-fication of targets, drug repositioning, drug efficacy (e.g., safety,

*Address correspondence to this author at School of Computer Science and

Engineering, Water Resources University, 175 Tay Son, Dong Da, Hanoi,

Vietnam; Tel/Fax: +84-912324564; E-mails: hauldhut@gmail.com

adverse effects and responses) [10] Many computational methods have been proposed to solve these problems [11-16] In this study,

we focus on the most important problem in drug discovery, which

is prediction of novel drug-target interactions [17-19] To identify targets, experimental studies usually approach the problem based on either target or phenotype [20] Meanwhile, computational methods for this problem are very diverse, in which novel interactions are predicted based on known drug-target interactions using various kinds of supporting biomedical and pharmacological data [21, 22] Irrespectively to this diversity, computational methods are classi-fied into two main approaches: i) network-based, ii) machine learn-ing-based There has been a number of studies which review the computational methods for predictions of drug-target interactions [15, 23, 24] However, these studies have simply listed proposed methods but not yet proposed a unified framework of the computa-tional methods for the problem For instance, studies [23, 24] fo-cused on only machine learning-based methods Recently, Chen

et al [15] has reviewed both network-based as well as machine

learning-based methods However, this study only enumerated pro-posed methods but not yet propro-posed any general framework for this problem In this study, we are going to review the computational methods as well as propose a unified framework for the prediction

of drug-target interactions In addition, to study human health and disease in a more systematically and effectively, we present an integrated scheme for a wider problem of prediction of disease-gene-drug associations

2 A UNIFIED FRAMEWORK FOR PREDICTION OF DRUG-TARGET INTERACTIONS

Drug discovery by systems pharmacological approaches re-quires the integration of various kinds of data [8, 25] This is sys-tems biology and chemistry-based approach [5] for human health and diseases [4] Therefore, all computational methods integrate different kinds of data such as –omics as well as pharmacological data [26, 27] Many computational methods have been proposed for the drug discovery [8], however, a majority of them are based on

Duc-Hau Le

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networks of biomedical and pharmacological instances (e.g., drugs,

targets and diseases), and therefore called network pharmacology

[10, 28-32] The network-based methods start with construction of

heterogeneous networks of biomedical and pharmacological

in-stances by combining homogeneous ones (i.e., drug similarity

net-works, target similarity networks and phenotypic disease similarity

network) and bipartite ones (i.e., drug-target networks, drug-disease

networks and disease-gene networks) In addition, network-based

methods consider action modes of drugs in the context of

interac-tions among cellular components [28] After heterogeneous

net-works are constructed, computational methods are then proposed to

identify novel interactions between drugs and targets Besides the

network-based approaches, other major approaches are based on

machine learning techniques [23, 24] The machine learning-based

approaches also require integrating various kinds of data to build

feature vectors for biomedical and pharmacological instances as

well as calculate similarity matrices among the instances After that,

a suitable machine learning algorithm is proposed to build a predic-tive model for identification of novel drug-target interactions

In general, these two main approaches are both based on simi-larity measures because they are all under an assumption that chemically or pharmacologically similar drugs target similar target proteins [33] Network-based approaches use similarity measures to construct the heterogeneous networks and calculate relative similar-ity between candidate interactions and known ones Meanwhile, machine learning-approaches use them for building kernel matrices

as well as calculating similarity among feature vectors/interaction profiles In addition, three main types of data spaces are used in these approaches, they are genomic space (i.e., general proteins as well as target ones), chemical space (i.e., chemical structures of drugs) and pharmacological space (i.e., general phenotypes as well

as drug-affected ones) However, in order to build kernel matrices,

Fig (1) A unified framework for prediction of drug-target interactions

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Systems Pharmacology: A Unified Framework for Prediction Current Pharmaceutical Design, 2016, Vol 22, No 23 3571

similarity networks and feature vectors, many types of data have

been used such as chemical structure of drugs, sequence of proteins,

network of instances and text (e.g., ontologies and literature) This

requires different similarity measures to effectively estimate how

similar two instances are In addition, the proposed methods are not

only application of a network/machine learning-based algorithm on

a raw data, but they are a processes containing sequential steps such

as data preparation, model building, algorithm selection, model

assessment and result analysis Therefore, the categorization of the

studies into two main approaches (i.e., network-based and machine

learning-based) are simply based on algorithm selection An

exam-ple for the intersection between the two main approaches is that the

kernel matrices in machine learning-based methods can be

con-structed using kernel functions on graph/network In addition,

ma-chine learning-based methods can be started by building a bipartite

network such as known drug-target interaction networks [34] After

that, learning algorithms were used to predict novel ones [35-37]

Moreover, network-based methods are usually started with

con-struction of heterogeneous network of instances, then a

network-based algorithm is proposed to rank candidates (e.g., candidate

targets) based on their functional similarities to known ones (e.g.,

known drug-related targets) Therefore, this can be considered as a

learning process where the model is built totally based on labeled

data (e.g., known drug-related targets) (i.e., supervised learning) or

based on both labeled and unlabeled data (e.g., other targets in the

network) (i.e., semi-supervised learning) Another common issue in

the two main approaches is data representation In general, the

com-putational approaches represent data in two ways: i) drugs and

targets are considered as separate instances (e.g., given a known

drug and its known targets, predict novel targets of this drug), and

ii) drugs and targets are consider as pairs (e.g., given known

drug-target interactions, predict novel ones) Fig (1) shows a unified

framework for the two main approaches for the prediction of

drug-target interactions In the next section, we are going to summarize

some typical studies in each approach to clarify the point

2.1 Network-Based Approaches

Typical studies in this approach usually begin by constructing a

heterogeneous network of biomedical and pharmacological

in-stances, then a network-based algorithm is proposed to identify

novel drug-target interactions For instance, Chen et al [33] built a

heterogeneous network that combines a drug similarity network and

a target protein similarity network by known drug-target

interac-tions In which, the drug similarity network was constructed based

on similarity in chemical structures of drugs and the target protein

similarity network was constructed based on similarity among

se-quences of target proteins In addition, information of known

drug-target interactions was also embedded into these two similarity

networks After that, a random walk with restart (RWR) algorithm,

which was successfully used for prediction of disease-associated

genes [38] and microRNA [39] on a heterogeneous network, was

used to identify novel target proteins related to a given drug The

underlying assumption of this method (both for the construction of

the heterogeneous network as well as the selection of RWR

algo-rithm) is that chemically similar drugs target to similar target

pro-teins Therefore, this method was proven to outperform ones, which

was solely based on target protein similarity network Using the

same algorithm as in [33], however, Seal et al [40] additionally

used an extensive drug-target network and a drug similarity

net-work with links among drugs were built based on the molecular

similarity with chemical fingerprints Another network-based

method [41] was also proposed to predict novel drug-target

interac-tions They did not specifically build the heterogeneous network of

drugs and targets However, they still based chemical similarity

between drugs and sequence similarity between target proteins to

propose predictive drug-based similarity inference (DBSI) and

tar-get-based similarity inference (TBSI) models, respectively In

addi-tion, they proposed a network-based inference (NBI) model based

on topological similarity in the drug-target bipartite network Taken together, network-based methods are usually based on the similarity between drugs or between targets (i.e., in the form of similarity networks or similarity matrices) and a known drug-target interac-tion network

2.2 Machine Learning-Based Approaches

These approaches also mainly used the three data spaces as in network-based ones (i.e., genomic, chemical and pharmacological spaces) A main strategy of these methods is to build kernel matri-ces based on similarity among drugs and similarity among targets Basically, drugs are chemical compounds, which are in the form of graph structure, therefore graph-based kernel functions [42] are usually used to calculate kernel matrices Also, target proteins can

be represented as sequences, therefore string-based kernel functions can be used to calculate kernel matrices [43] There are two main approaches for machine learning-based methods for the prediction

of drug-target interactions, they are chemogenomics and pharmaco-genomics In which, the former is based on an assumption that chemically similar drugs usually target similar target proteins [35, 44-46] Meanwhile, the latter is based on another that phenotypi-cally similar drugs interact to similar target proteins [37, 47], in which, phenotypes of drugs can be represented by effects of drugs The built kernel matrices are then used for a learning model to pre-dict novel drug-target interactions

Most of the machine learning-based methods proposed for the problem are based on kernel-based supervised learning techniques

For instance, Jacob et al [45] used a product kernel (i.e., a

combi-nation of chemical structure-based kernel matrix for ligands and sequence-based kernel matrix for target proteins) In addition to

these kinds of data, Yamanishi et al [35] utilized topological

prop-erties of the drug-target network to build a kernel-based logistic regression prediction model By combining both chemical and

pharmacological data of drugs, Yamanishi et al [37] proposed to

use a supervised learning method on the bipartite drug-target

net-work In addition, Takarabe et al [48] also used the product Kernel

as in [45] and the kernel-based regression model as in [35], how-ever, they built pharmacological kernel matrix instead of the

chemical structure-based kernel matrix as in [35] Finally, Bleakley

et al [36] used the same kernel matrices as [48] in a bipartite local

models (BLM) on the bipartite drug-target network Based on the bipartite drug-target network, some studies built interaction profiles

of drugs and targets [49-51] For instance, studies [50, 51] proposed

a method, namely GIP, by building Gaussian kernels based on these interaction profiles After that, a regularized least square-based classifier was used to predict novel drug-target interactions [50] Besides, other machine learning models have been used for the problem such as conditional random field (CRF), a probabilistic graphical model [52] and Bayesian matrix factorization [53] Taken together, these machine learning-based methods mainly used simi-larity measures to build kernel matrices These kernel matrices were usually built separately for drugs and targets After that, they were combined for predictive models

Other supervised approaches are feature vector-based, in which, drug-target pairs were represented as feature vectors Based on features of drugs and targets extracted from drug and target-related data as well as known drug-target interactions, classifiers were then constructed to predict novel drug-target interactions There have been several learning models used for the problem such as k-nearest neighbor (kNN) [54], logistic regression [55, 56], support vector machines (SVM) [57-59] and ensemble methods [60, 61] Interest-ingly, most of these methods are kernel-based, where kernel matri-ces are calculated from feature vectors of training instanmatri-ces [56-59, 61]

A major limitation of the above supervised learning-based methods is that the predictive models were built based on only la-beled data (i.e., known drug-target interactions) However, this kind

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of data is very limited due to the cost of labeling process In

addi-tion, some of them are binary-classification models This means

that negative training instances (i.e., non-drug-target interactions)

have to be specified in training process However, there is no such

experimentally verified interactions in literature Therefore,

semi-supervised learning methods, which learn from both labeled and

unlabeled data (i.e., unknown drug-target interactions), have been

proposed to overcome this limitations [62-64] These methods also

utilized kernel/similarity matrices constructed from chemical and

genomic spaces as well as the bipartite drug-target network

3 AN INTEGRATED SCHEME FOR PREDICTION OF

DRUG-GENE-DISEASE ASSOCIATIONS

Approaches in systems pharmacology are based on various

kinds of data and computational methods In which, three main

entities (i.e., drug, disease and gene/target protein) should be

con-sidered in the same context [65-67] There have been many

individ-ual databases for each entity, but few for all of them such as

C2Maps [68], EU-ADR corpus [69] Therefore, there is a pressing

need to build such the databases In the future, these databases can

be formed and more comprehensive with results from experimental

studies as well as text mining techniques [3, 70, 71]

The relationships among these entities pose three challenges,

one of them is the prediction of drug-target interactions The two

remaining ones are prediction of disease-associated genes/proteins

and prediction of novel drug-disease associations (also known as

drug repositioning/repurposing) Although, the three problems have

their own goals in biomedicine and pharmacology, but they have

the same target in algorithmic view, which is prediction of novel

binary interactions/associations This is also a popular problem in

bioinformatics More importantly, they are all based on a

“guilt-by-association” assumption that functionally similar drugs/diseases

target/relate to functionally similar target proteins/genes,

respec-tively Among them, prediction of novel disease-gene associations

is very popular problem in biomedicine research and well-studied

by both network- and machine learning-based methods [72-80]

Therefore, we could use or adapt the methods used for this problem

for the prediction of drug-target interactions In addition, prediction

of novel drug-disease associations have been also studied for years [16, 32, 71, 81, 82]

As aforementioned, many computational methods have been proposed for prediction of drug-target interactions However, some

of them were built and assessed based on simple settings and ulti-mately they may not be used for practical problems Therefore, computational methods are needed to take intensive consideration

on the problem modeling as well as assessment methods [83] In addition, obviously, irrespective of the network- or machine learn-ing-based methods, the calculation of similarity among instances in different data spaces to construct similarity/kernel matrices and similarity networks is the most important step Data integra-tion/fusion from various spaces is also important since the problem

is considered in multi-dimension as well as systematically There-fore, we should also take consideration on selection of integration methods, in which kernel-based data fusion methods can be suitable ones since the data is mostly represented as kernel matrices Indeed,

Wang et al [84] is a pioneer applying kernel-based data fusion for

the prediction of drug-target interactions However, it should be noted that this data fusion method has been used popularly for pre-diction of disease-gene association for years [85-87] In addition, many algorithms have been proposed for kernel-based data fusion

[88] such as L p-norm MKL [89], which could be used for kernel-based problems Therefore, they could be used to improve the pre-diction of drug-target interactions

Besides, to meet practical problems, more suitable machine learning models should be considered for the prediction of drug-target interactions such as positive and unlabeled (PU) learning technique, which has been successfully applied in prediction of disease-gene associations [90] In addition, the combination be-tween the PU learning technique and kernel-based data fusion or ensemble methods could provide better way for the problem as did for prediction of disease-gene associations in [91] and [92], respec-tively For network-based approaches, the calculation of similarity between instances in constructed networks can be done with graph-based kernel functions [93, 94] Some of them have been used suc-cessfully for prediction of disease-gene associations Similarly,

Fig (2) An integrated scheme for prediction of drug-gene-disease associations

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Systems Pharmacology: A Unified Framework for Prediction Current Pharmaceutical Design, 2016, Vol 22, No 23 3573

these machine learning- and network-based methods could be used

to predict drug-target interactions more effectively

In parallel to proposal of computational methods and

construc-tion of databases, development of tools for predicconstruc-tion of novel

drug-target interactions is also needed [95, 96] There has been a

number of such the tools such as DINIES [97], iGPCR-Drug [98]

Also, many tools have been developed for prediction of

disease-gene associations [74, 99, 100] as well as several ones for

predic-tion of drug-disease associapredic-tions such as DRAR-CPI [101] and

PROMISCUOUS [102] Obviously, the above mentioned tools are

developed individually for each the problem of prediction

There-fore, an integrated tool predicting drug-gene-disease associations is

needed such as DT-Web [103], which can predict both drug-target

interactions and drug-disease associations

Taken together, to study human health and disease in a more

systematically and effectively, the drug-gene-disease associations

should be considered in the same context Therefore, all proposed

computational prediction methods, constructed databases and

de-veloped tools should meet this requirement Figure (2) shows an

integrated scheme for prediction of drug-gene-disease associations

CONCLUSION

After human genome project, it has been expected that several

new effective drugs will be developed Unfortunately, most of drug

targets are proteins which go through significant post translation

modification or involve in complex network Systems

pharmacol-ogy therefore becomes the modern approach for drug discovery and

development in post genomic era In this study, we have reviewed

various computational approaches proposed for this problem More

importantly, by analyzing the proposed methods, we recognized

that they are based on the same assumption that similar drugs target

similar target proteins Based on this assumption, they use

similar-ity measures to calculate similarsimilar-ity among instances in different

data spaces to construct similarity/kernel matrices and similarity

networks After that, these matrices and networks are used in

net-work- or machine learning-based computational models to predict

novel drug-target interactions To be more intuitively, we proposed

a unified framework of computational methods for the prediction of

drug-target interactions In addition, we extended the problem to

effectively study human health and diseases by considering the

relationships among drugs, gene/target proteins and diseases in the

same context Interestingly, we found that the drug-gene-disease

relationship pose three key challenges (i.e., prediction of

drug-target interactions, prediction of disease-associated genes/proteins

and prediction of novel drug-disease associations) with similar

underlying assumptions (i.e., guilt-by-association) and the same

algorithmic view (i.e., prediction of binary interactions) Therefore,

predictive models and data fusion methods could be used

ex-changeably among the three problems Similarly, we also presented

an integrated scheme for prediction of drug-gene-disease

associa-tions

Finally, with high-throughput technologies in next generation

sequencing, GWAS has yielded large amount of genomic data

Therefore, computational methods for the problems in drug

discov-ery should deal with this wealth data [104, 105] which open a wide

road for research in personalized medicine [106-108] In addition,

novel predicted drug-target interactions by computational methods

need to be experimentally verified in wet-laboratory

CONFLICT OF INTEREST

The authors confirm that this article content has no conflict of

interest

ACKNOWLEDGEMENTS

This research is funded by Vietnam National Foundation for

Science and Technology Development (NAFOSTED) under grant

number 102.01-2014.21

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The author has requested enhancement of the downloaded file All in-text references underlined in blue are linked to publications on ResearchGate The author has requested enhancement of the downloaded file All in-text references underlined in blue are linked to publications on ResearchGate

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