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Investigating Heterogeneity of Growth and Drug Response in Mesotheliomas. By Roxanne Mae Pangilan Otadoy Dr. Cleo Robinson Primary Supervisor Tumour Immunology Group National Centre for Asbestos Related Diseases School of Medicine and Pharmacology University of Western Australia 4 th Floor, G Block, Queen Elizabeth II Medical Centre Nedlands, Western Australia, 6000 Assoc. Prof. Wayne Greene Co-Supervisor Molecular Genetics School of Veterinary and Biomedical Sciences Murdoch University Room 3.041, Veterinary Biology, 90 South Street Murdoch, Western Australia, 6150 This thesis is presented for the Honours degree in Biomedical Science at Murdoch University November 2013.

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Page 1: Investigating Heterogeneity of Growth and Drug Response in ... · noncommunicable diseases, including cancers (Alwin et al. 2011). Cancer is found in many sites of the human body

Investigating Heterogeneity of Growth

and Drug Response in Mesotheliomas.

By

Roxanne Mae Pangilan Otadoy

Dr. Cleo Robinson Primary Supervisor Tumour Immunology Group National Centre for Asbestos Related Diseases School of Medicine and Pharmacology University of Western Australia 4th Floor, G Block, Queen Elizabeth II Medical Centre Nedlands, Western Australia, 6000 Assoc. Prof. Wayne Greene Co-Supervisor Molecular Genetics School of Veterinary and Biomedical Sciences Murdoch University Room 3.041, Veterinary Biology, 90 South Street Murdoch, Western Australia, 6150

This thesis is presented for the

Honours degree in Biomedical Science at Murdoch University November 2013.

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Declaration

I declare this thesis is my own account of my research and contains as its main content, work which has not been previously submitted for a degree at any tertiary educational institution. Signed: _________________________ Name: Roxanne Mae Pangilan Otadoy Date: 4/ November / 2013

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Abstract

The prognosis for patients with malignant mesothelioma is very poor with patient

response to therapy highly variable. This project was aimed at investigating the

degree of heterogeneity in the responses to treatment amongst mesothelioma cell

lines which have all arisen due to asbestos exposure. Despite the use of the same

carcinogen, a number of variabilities amongst these cancers are still present.

Avenues of investigation initially tried to determine if the variability in response was

due to heterogeneity in the proliferation of malignant mesothelioma cells. Different

growth rates were observed for cell lines grown in culture and in vivo. Cells showed

different degrees of response to treatment with different chemotherapeutic agents.

An attempt was made to determine the mode of cell death undergone by cells in their

response to the treatments. Further investigation is warranted into the validity of

these results because if the differences found in the phenotype of the cells were

accurate, it could direct individualised therapeutic strategies to target the uniqueness

of malignant mesothelioma.

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Table of Contents

Declaration I

Abstract II

Acknowledgments VII

Chapter One: Introduction 1

1.0.0 Mesothelioma 2-4

1.1.0 Disease risk associations 4-6

1.2.0 Diagnostic aids 6-9

1.3.0 Therapies 10-13

1.3.1 Classic chemotherapy 13-22

1.4.0 Cell culture 22-24

1.4.1 The in vitro assay 25-29

1.5.0 Animal models 29-30

1.5.1 The MexTAg mouse model 30-31

1.5.2 The transplantation model 31-33

1.6.0 The genetic dimension 33-36

1.6.1 CGH arrays 36-37

1.6.2 DNA microarrays 37

1.6.3 Next generation sequencing 38

1.7.0 Project hypotheses 39

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Chapter Two: Materials and methods 40

2.0.0 Cell culture 41-43

2.1.0 Proliferation 43-45

2.2.0 In vivo tumour growth 46-47

2.3.0 MTT assay 47-53

2.4.0 ATP assay 54-57

2.5.0 Caspase assay 57-58

2.6.0 DNA extraction 58-59

Chapter Three: The growth rate of cell lines in vitro 60

3.0.0 Proliferation rates of mesothelioma cell lines 61-63

3.1.0 Proliferation comparisons 64

3.2.0 A summary of the proliferation assay results 64-66

Chapter Four: The growth rate of cell lines in vivo 67

4.0.0 Establishing cell growth in vivo 68-71

4.1.0 Fast growing wild-types 72-73

4.2.0 A summary of the in vivo cell growth results 73-74

Chapter Five: The heterogeneic response of mesothelioma cell lines

to chemotherapy

75

5.0.0 Assessment of the cell line response to chemotherapy 76-85

5.1.0 The heterogeneity of response of mesothelioma cell lines

to chemotherapy treatment

85-86

5.2.0 A summary of the MTT assay 87-88

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Chapter Six: Investigating the death of cells in response to

chemotherapy

89

6.0.0 The release of ATP and caspase 3/7 90

6.1.0 Establishing a reading for ATP luminescence 90-94

6.2.0 A summary of ATP assay issues and results 95-96

6.3.0 Establishing a reading for caspase luminescence 97-98

6.4.0 Caspase signal detected 99-100

6.5.0 High purity in the extraction of DNA 100-101

Chapter Seven: Discussion 102

7.0.0 Discussion 103-109

References 110-135

Appendix I: List of tables 136

Appendix II: List of figures 137-138

Appendix III: Data analysis 139-140

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Acknowledgements

I would like to thank my Primary Supervisor, Dr. Cleo Robinson, for her continual

guidance and support throughout such a rewarding project. Major thanks also

go to my Co-supervisor Assoc. Prof. Wayne Greene and the Honours Chair

Assoc. Prof. Alan Lymbery, without whom, this project would not have even begun.

I have been so fortunate to receive such a huge amount of help from a long list of

generous people with regards to equipment, reagents, advice and general good

company for keeping one sane, that I can’t thank these people enough (listed in no

particular order): The NCARD/T.I.G. team; fellow honours students: Steph, Wayne,

Alice, Keyuri and Clara; the School of Medicine and Pharmacology department at

UWA which includes administration staff as well as laboratory staff; the baking

club: for providing the necessary tea breaks in between; the LIWA team; the UWA

and Murdoch Universities; The Asbestos Diseases Society of WA and, of course, our

suppliers.

A special thank you goes to Prof. George Yeoh and Mr. Ken Woo for the access and

training of their Cellavista system.

Finally, to my mum Lilia, brother Rommel, sister Jena, extended family, very special

friends Ben and Desiree and everyone else who has been so patient with me during

this year of honours, “THANK YOU!” a million times for all your love and support.

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Chapter One:

Introduction

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1.0.0 Mesothelioma

The World Health Organisation (WHO) recorded 57 million deaths for the year 2008

with a reported 36 million of these, approximately 63%, being caused by

noncommunicable diseases, including cancers (Alwin et al. 2011). Cancer is found in

many sites of the human body and is an increasing burden on human health globally

(Jemal et al. 2011). There are emotional costs on families and other members of

society, costs for patient treatment, subsidy costs from health systems and in some

cases, insurance companies must payout claims (Greenberg et al. 2010).

Mesothelioma, or malignant mesothelioma (MM), is a cancer of the serosal surfaces

that was once considered to be a rare disease, but it is one that is still growing in

incidence. However, it does have a huge economic and health burden, due to

hospitalisation and compensation claims (Robinson & Lake 2005). MM is a disease

in which governments have the option to take action, in order to curb the number of

resulting deaths, by removing human exposure to known carcinogens, such as

asbestos fibres, that are associated with the development of MM in exposed

individuals (Carbone et al. 2012).

In many countries, mining and the use of asbestos has been banned. For example, the

Wittenoom mine in Western Australia was closed down in 1966 when the

association of asbestos exposure with disease was first proven and a complete ban of

asbestos use in Australia was enforced in 2003 (Leigh & Driscoll 2003). However,

there are countries where asbestos is still being mined and manufactured into

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products, countries such as Russia, China, Canada, India and Kazahkstan

(LaDou 2010). Reportedly, there are also individuals who develop the disease

without first being exposed to asbestos fibres, thus indicating that other factors may

be involved (Kinoshita et al. 2013).

The serosal surfaces in which MM arises are made up of simple squamous

epithelium, a class of epithelium that is typically located in vascular systems, body

cavities, and respiratory spaces with a major function of exchange and lubrication

(Kumar et al. 2010). It produces serous fluid for smooth lining of the body cavities,

namely those of the pleura, pericardium and peritoneum. These linings, in which

both visceral and parietal surfaces are affected (Figure 1.1) (Kumar et al. 2010), are

referred to as the body’s mesothelium (Saladin 2010). A cancer arising from these

linings, monolayers of mesothelial cells (Mutsaers 2004), has profound effects on the

individual. They usually present with severe chest pains and respiratory problems, in

the case of malignant pleural mesothelioma, or incur intestinal obstruction that can

lead to death, as in the case of malignant peritoneal mesothelioma

(Kumar et al. 2010). A big indicator of malignant pleural mesothelioma is combined

chest pain and unexplained pleural effusions (Robinson & Lake 2005).

Disparity in prognosis and responses observed in clinical treatments is likely to be

due to the heterogeneity observed in MMs (Szulkin et al. 2013) which are already

histologically sub-typed into 3 main groups: epitheliod, sarcomatoid and biphasic

(Raja, Murthy & Mason 2011). Sarcomatoid mesotheliomas are more aggressive

than epitheloid (Grigoriu et al. 2007). These mesothelial cells are also known to

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change their phenotype depending on their environment, fibroblast like in culture but

epithelial like back in vivo (Mutsaers 2004).

As a cancer, MM exhibits the distinguishing feature of cancers including sustaining

proliferative signalling, evading growth suppressors, activating invasion and

metastasis, enabling replicative immortality, inducing angiogenesis and resisting cell

death (Hanahan & Weinberg 2011). Recent evidence suggests it is most likely that

there are a series of mutations that occur during tumorigenesis

(Mossman et al. 2013). The importance of fully characterising MM in order to aid in

distinguishing it from other cancers is highlighted by the fact that the epitheliod

subtype already histologically resembles adenocarcinoma, a cancer of the epithelium

(Kumar et al. 2010). Diagnosis is usually 2-3 months from the start of symptoms, a

late stage of diagnosis (Robinson, Musk & Lake 2005). It is only with correct

diagnosis that patients may receive the proper treatment (Webb & Pass 2004).

Undeniably, the prognosis for patients with MM is still very poor, just a matter of

months (Musk et al. 2011).

1.1.0 Disease risk associations

The latency period of MM, the time between initial exposure to a carcinogen and the

diagnosis of the disease, is varied and an approximate range is 25-71 years with a

median latency of 40 years (Carbone et al. 2012). The associated risks for the

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development of MM are also varied and can come from occupational, i.e. working

with, (Leigh & Driscoll 2003) or geographical exposures, i.e. working around and

simply living in an area where there is that exposure to asbestos (Pan et al. 2005).

These include people who have worked in asbestos mines, such as the Wittenoom

Gorge in Western Australia (Figure 1.2) (de Klerk et al. 1996; Asbestos Diseases

Society of Australia Inc. 2012), or are working with asbestos materials in factories at

present in China (Wang et al. 2013) as well as those who have lived nearby naturally

occurring erionite (Carbone et al. 2012).

Exposure to asbestos fibres is a confirmed risk factor, but only about 10-20% of

highly exposed individuals will develop the disease (Jasani & Gibbs 2012). Low

levels of exposure to erionite, a naturally occurring material that bears similar

fibrous morphology to asbestos, as observed in Turkey, could be more adept in

instigating the development of the disease (Carbone & Yang 2012).

The monkey simian virus 40 (SV40) can cause MM in rodents but its role in humans

for contributing to asbestos carcinogenicity is still unproven. This is despite the

SV40 T-antigen oncogene having once been confirmed as present in human MM

samples (Rizzo et al. 2001). Evidence has since emerged that false positives were

brought about by contamination of PCR samples with laboratory plasmids when

examining the link between SV40 deoxyribonucleic acid (DNA) sequences and MM.

This makes exposure to asbestos still the more likely cause of MM

(López-Ríos et al. 2004). Nonetheless, the SV40 T-antigen has been shown to

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interact with replication at the replication forks of DNA (Murakami & Hurwitz

1993), especially in the unwinding of the DNA to make the DNA more accessible to

transcription machinery (Foster & Simmons 2010).

Other aetiologies for MM have emerged. In one study, radiotherapy treatment has

been reported to cause MM. The study sample size is small but the disease is present

and the study is relatively new so more investigation is still needed to confirm its

role in being a risk factor for the development of MM (De Bruin et al. 2009;

Goodman, Nascarella & Valberg 2009). Similarly, more data on the various types of

carbon nanotubes and the way in which reactive species that may be produced by

carbon nanotubes to cause breaks in the DNA is still needed (Jaurand 2009;

Donaldson et al. 2010).

Acquisition is not only dependant on a patient’s level of exposure to a carcinogen.

Factors such as patient gender, age, MM subtype and location, the facilities for early

diagnosis and the level of therapeutic management that is at the patient’s disposal

also have a bearing on the patient’s quality of life (van der Bij et al. 2012).

1.2.0 Diagnostic aids

Negative and positive histological markers for MM are in existence. This includes

carcinoembryonic antigen (CEA) as a negative marker and calretinin, Wilms’

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tumour gene (WT1) and mesothelin as positive markers (Ordóñez 2003). More

recently, the overexpressed and hypoglycosylated variety of MUC1/EMA has been

identified as a useful marker of diagnosis for MM (Creaney et al. 2008). However,

the use of histology in the hands of an experienced pathologist is not the only means

for diagnosis nowadays (Jaklitsch, Grondin & Sugarbaker 2001).

Diagnostic tools include computed tomography (CT) scans, magnetic resonance

imaging (MRI) and positron emission tomography (PET) for visualising and

distinguishing malignancy from benign disease without being too invasive.

Ultrasound guided biopsies (Figure 1.3) (Stigt, Boers & Groen 2012), a form of

surgery, is useful to obtain a sample from a patient in order to conduct diagnostic

tests to confirm the presence of MM (Jaklitsch, Grondin & Sugarbaker 2001).

Adequately acquired specimens are required for accuracy of classification as

indicated by a study on the sensitivity, 93%, and specificity, 31%, of biopsies from

MM (Kao et al. 2011). However, samples can be obtained from tumour resections,

pleural effusions (Relan et al. 2013) as well as ascites fluid, which commonly builds

up due to the cancer (Hassan, Bera & Pastan 2004), for culturing and testing in the

laboratory.

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a)

b) c)

Figure 1.1. Malignant mesothelioma in a bisected lung. a) The white tumour is

spread out in the pleural space to encase the lung. b) An epitheliod histological

subtype of malignant mesothelioma. c) A biphasic histological subtype of malignant

mesothelioma stained to show calretinin under the immunoperoxidase method. These

images are from Kumar et al. (2010).

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Figure 1.2. Wittenoom Asbestos Mine in Western Australia. The mine was

closed down in 1966 when asbestos became known to be associated with malignant

mesothelioma. This image is from the Asbestos Diseases Society of Australia Inc.

website (2012).

Figure 1.3. Ultrasound guided biopsy of malignant pleural mesothelioma. A

needle biopsy of the tissue core of malignant pleural mesothelioma visualised via

ultrasound with the arrow on the right indicating the needle and the larger arrow on

the left the expanded pleura. This image is from Stigt, Boers & Groen (2012).

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1.3.0 Therapies

Therapies used to treat mesothelioma include surgery, radiotherapy, immunotherapy,

chemotherapy (Grégoire 2010), photodynamic therapy (Friedberg 2012) and gene

therapy (Vachani, Moon & Albelda 2011), either alone or in a multimodal fashion

(Liu et al. 2010).

The role of radical surgery in treatment is complete resection of the disease but the

determination of survival benefit is difficult, so whether this is the best option is still

being debated (Kaufman & Flores 2011). There is little evidence for the effective

treatment of MM using radiotherapy, but rather, this technique is seen to have a role

in pain reduction for the patient when other pain controllers, such as opiates, no

longer have an effect (Price 2011; Patel et al. 2013). Strategies for enhancing

radiotherapy efficacy are being looked into (Sudo et al. 2012). Extrapleural

pneumonectomy, a surgery for resecting tumour burdened parts of the lung, pleura,

ipsilateral diaphragm and pericardium, in conjunction with the use of hemithoracic

intensity modulated radiotherapy, has shown improved local control of MM

(Rice et al. 2007).

Immunotherapy strategies can involve dendritic cells, an antigen presenting cell and

unique protein markers expressed on the surfaces of MM cells, such as WT1

(Scattone et al. 2012) which in the case of WT1 would not normally be highly

expressed in normal adult tissues. It involves the identification of tumour associated

antigens (Cornelissen et al. 2012) and stimulation of the body’s own immune

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response for killing off tumour cells (Mossman et al. 2013) whilst bearing in mind

that in the development of a therapy, be it viral or non-viral based,

a major factor for consideration in immunotherapy is autoimmunity

(Ireland, Kissick & Beilharz 2012).

Systemically, mesothelin is a target for immunotherapy due to the over expression of

it in epithelioid mesothelioma when compared to expression levels in normal adult

cells (Grosso & Scagliotti 2012). Programmed death-1 (PD-1) and cytotoxic

T lymphocyte antigen 4 (CTLA-4), which are inhibitory co-receptors on activated

T and B cells, are additional focal points of study (Lesterhuis, Haanen & Punt 2011).

Immunotherapy can be used as an adjunct to chemotherapy. For instance,

Gemcitabine depletes lymphocytes of the humoural immune response but the

adaptive immune system’s antigen-specific CD4+ and CD8+ T-cell responses are

enhanced (Nowak, Robinson & Lake 2002). However, the acquired immune

response works better, in the setting of immunotherapy with antigen presenting cells

being loaded with cancer specific antigens after apoptosis because the immune

system “sees” cells as being dead and “act” towards them in a certain way which is

dependent on the many feedback events that lead up to the induction of apoptosis

(Lake & Robinson 2005).

Immunotherapy has been tested as an adjuvant in combination with chemotherapy

post partial resection of the tumour to yield an 80% cure rate in mice

(Broomfield et al. 2005). Additionally, IL-2 injected intratumorally at high doses has

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been shown to boost cytotoxic T lymphocyte activity whilst hindering angiogenesis

associated with tumours so that the body rejects the tumour (Jackaman et al. 2003).

With respect to chemotherapy, only 20-40% of patients respond to chemotherapy

and, at best, median survival is prolonged 5-9 months (Jakobsen & Sørensen 2011).

Chemotherapy is still the most frequent method used for treating MM (Nowak 2012)

despite the different side effects experienced by patients (Cheok 2012).

Gene therapy strategies that have shown some efficacy include suicide gene therapy,

cytokine gene therapy and gene-modified T-cells for adoptive transfer (Vachani,

Moon & Albelda 2011). In suicide gene therapy (Figure 1.4), a gene is introduced

into a cell that is transcribed and translated into an enzyme that converts a prodrug

into a toxic drug that can kill that cell (Duarte et al. 2012; Wu 2009). Cytokine gene

therapy uses cationic liposomes to deliver the cytokine gene, such as IFN-β, into the

cell for transcription and translation so that the particular cytokine is locally

up-regulated (Ohno et al. 2012; Kruklitis et al. 2004). Gene modified T-cells are

T-cells with specific antigen receptors generated to antigens coming from the

individual tumour and engineered on the surface of cells, these are adoptively

transferred into a patient in order to initiate the body’s immune response

(Stauss & Morris 2013). Some clinical trials for these have been performed

(Vachani, Moon & Albelda 2011).

Photodynamic therapy is relatively new and is a 3 component light-based

experimental treatment for MM to be used in conjunction with surgery to cause

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physical cell damage, induction of apoptosis and immune response stimulation

(Friedberg 2009).

However, despite combined treatments being utilized, patient survival is still poor

and an optimal therapy is still elusive so the search for effective treatments continues

(Liu et al. 2010). A case of watch this space.

Figure 1.4. Suicide gene therapy. A suicide gene is introduced into a cell. When

transcribed and translated an enzyme is produced that is able to convert a prodrug

into a toxic drug to kill the cell. This image is from Duarte et al. (2012).

1.3.1 Classic chemotherapy

In consideration of the “classic” method for treating MM, that is, treatment with

chemotherapy, there are varying responses between individual MM patients for

single-agent treatment of chemotherapeutic drugs including Cisplatin,

Cyclophosphamide, Doxorubicin, Epirubicin, Etoposide, Gemcitabine, Ifosfamide,

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Methotrexate, Paclitaxel, Pemetrexed and Vincristine, whereby a percentage of

patients respond to treatment and a percentage do not (Tomek & Manegold 2004).

Chemotherapeutic drugs each have different characteristics and modes of action such

that cells respond differently to them (Baas 2002). It is important to test a wide range

of these chemotherapeutic drugs in mesothelioma cell lines to determine patterns of

efficacy. Some chemotherapeutic drugs used in the clinical setting are summarised in

Table 1.1.

Cisplatin may initially bind to plasma proteins, mainly albumin, that leads to its

inactivation (Fuertes et al. 2003). It may alternatively reach the cell surface and enter

via passive diffusion. In some cases uptake may be via facilitated or via active

transport mechanisms. Inside the cell, cations of 2 species of Cisplatin are formed

which are very reactive to nucleophile sites. However, less than 1% of Cisplatin

actually binds to DNA by chance as most end up binding to other biomolecules, such

as proteins (Fuertes et al. 2003). Once it binds DNA in the cell nucleus, it interferes

with normal transcription, or DNA replication, thus blocking the functions of

important cellular proteins, eg. Hsp90 the adenosine triphosphate (ATP) binding

chaperone, leading to deregulation of the cell cycle. There is DNA degradation in

180 base pair (bp) fragments, cell blebbing and cell shrinkage that all indicate

apoptosis. In cell lines with drug resistance one can see features of necrotic cell

death. This is evidence that both types of cell death may occur simultaneously

(Fuertes et al. 2003). Cisplatin is one of the most effective chemotherapies used

currently with treatment in testicular, ovarian, bladder, small-cell and non-small cell

lung, head and neck cancers, other solid cancers (Siddik 2003) and MM

(Ong & Vogelzang 1996; Spugnini et al. 2006). A response rate of 12% was

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observed in a study (Favoni et al. 2012). A median survival of 12.1 months when

combined with Pemetrexed has been shown on another study (Dowell et al. 2012).

Cyclophosphamide is an alkylating agent (Fleming 1997). It is also a prodrug

(Wu 2009; Fleming 1997) as it requires activation by hepatic microsomal enzymes to

be metabolized into their cytotoxic form. Cell death is caused by the inhibition of

DNA synthesis through DNA being alkylated to form DNA-DNA crosslinks

(Fleming 1997). Biological activity is dose dependent and is immunostimulatory. It

also induces the immune system to migrate to the tumour site for “mopping up” of

necrotic or apoptotic cells by inducing calreticulin to translocate to the tumour cell

surface to signal to phagocytes that it requires “mopping up” (Sistigu et al. 2011).

Leukopenia limits toxicity (Fleming 1997). The drug acts on immune cells in vivo to

treat cancer, cells such as DCs, TH1/TH2, NK and B cells (Sistigu et al. 2011). In

MM, lymphocytopenia is incited in patients by pre-treating them with

Cyclophosphamide to precondition the environment in which tumour infiltrating

lymphocytes (TILs) are to be adoptively transferred. This preconditioning has been

found to be necessary for the survival of the TILs (Lesterhuis, Haanen & Punt 2011).

In this way, Cyclophosphamide is acting in an immunotherapeutic manner, but in the

other instance, when Cyclophophamide metabolites induce the formation of DNA

intra-strand and inter-strand crosslinks (Emadi, Jones & Brodsky 2009), it is seen to

act in a chemotherapeutic manner. It is used to treat Melanoma (Sistigu et al. 2011)

and other malignant diseases such as testicular cancer, lung cancer, sarcomas,

lymphomas, breast, ovarian, cervical cancers (Fleming 1997) and MM

(Emadi, Jones & Brodsky 2009).

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Ifosfamide is an alkylating agent similar to cyclophosphamide, a prodrug (Fleming

1997; Wu 2009), which requires activation by hepatic microsomal enzymes and

metabolized into their cytotoxic form. Cell death is also caused by the inhibition of

DNA synthesis through DNA being alkylated to form DNA-DNA crosslinks

(Fleming 1997). Neurotoxicity limits toxicity. It is also more readily eliminated by

the body than Cyclophosphamide (Fleming 1997). It is used to treat neuronal and

renal cells (Brüggemann, Kisro & Wagner 1997) along with testicular cancer, lung

cancer, sarcomas, lymphomas, breast, ovarian, cervical cancers (Fleming 1997) and

MM (Tomek et al. 2003).

Doxorubicin is an anthracyline drug isolated from Streptomyces peuceitus

var. caesius which inserts into the DNA to disrupt topoisomerase-II mediated DNA

repair, along with “poisoning” the enzyme itself, as well as create free radicals that

can damage membranes and other cellular components that can lead to apoptosis

(Thorn 2011). The drug is not very effective as a single agent and thus it is rarely

given on its own as a treatment, but usually in combination with Cisplatin

(Mirarabshahii et al. 2012; Baas 2002) and has been shown to be effective in a

phase-II clinical trial in Italy (Ardizzoni et al. 1991). Its serious side effects include

cardiomyopathy and myelosuppression (Tan, Choong & Dass 2009). It is used for

malignant melanoma (Frank et al. 2005), breast, lung, gastric, ovarian, thyroid,

non-Hodgkins and Hodgkins lymphoma, multiple myeloma, scarcoma and pediatric

cancers (Thorn 2011). It has been shown to display 20% response rates in MM

patients although more studies are needed to find out why (Tomek et al. 2003).

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Epirubicin is similar to Doxorubicin in that it has a reorientation of the hydroxyl

group in the 4’ position of the daunosamine ring (Khasraw, Bell & Dang 2012). It

intercalates DNA to inhibit topoisomerase-II enzyme activity thus preventing DNA

repair. It also generates free radicals to interfere with cellular components such as in

protein synthesis and therefore leading to apoptosis (Khasraw, Bell & Dang 2012). It

is used in the treatment of breast (Khasraw, Bell & Dang 2012), uroethelial (Engeler

et al. 2012), muscle invasive cancers (Herr et al. 2007) and MM with responses

ranging from 0-20% in MM (Garland 2011).

Gemcitabine enters the cell via nucleoside transporters. Once inside, it transforms

into Gemcitabine Diphosphate and Triphosphate which are responsible for its

cytotoxic effects. They insert into the DNA to inhibit DNA polymerase

(DNA synthesis) and if a few are incorporated into the DNA it leads to termination

of DNA elongation so DNA repair enzymes are inhibited leading to cell apoptosis

(Mini et al. 2006). It is used in a broad range of solid tumours such as ovarian

cancer, non-small cell lung cancer (Mini et al. 2006) and has been shown to have

activity in MM with patient response ranges of 12-40% on one study

(Garland 2011).

Methotrexate is a competitive inhibitor of dihydrofolate reductase which prevents the

formation of folate cofactors needed for de novo purine and pyrimidine synthesis, a

cell cycle block in DNA synthesis, specifically in S-phase (Chan & Cronstein 2010;

Held-Warmkessel 2000). It is used to act on T-cells and various cancer cells such as

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breast, head, neck, bladder and lung (Held-Warmkessel 2000). In MM, systemic

treatment can yield responses of 37% in clinical trials (Ellithy et al. 2013).

Pemetrexed, more commonly known by its brand name Alimta

(Chattopadhyay, Moran & Goldman 2007), inhibits thymidylate synthase,

dihydrofolate reductase, and glycinamide ribonucleotide formyltransferase enzymes

that metabolise folate and are involved in purine and pyrimidine for the synthesis of

DNA (Adjei 2004). It is transported into cells by a carrier and is converted into its

active form by folypolyglutamate synthase, and its polyglutamated form equates to

higher intracellular retention when compared to Methotrexate (Adjei 2004). It is used

to treat non-small cell lung cancers (Hanna et al. 2004), a variety of solid tumours

(Adjei 2004) and MM with a retreatment response rate of 19% in one study of MM

(Ceresoli et al. 2011). It is currently a standard therapy for MM when combined with

Cisplatin and vitamin supplementation to yield response rates of 41% with a median

survival of 12.1 months (Garland 2011).

Etoposide Phosphate is a highly water soluble form of Etoposide. This prodrug is

converted into its active form Etoposide by alkaline phosphatase in physiological

conditions. It produces cell cycle blocks at S and early G2 phases and can produce

single as well as double stranded breaks. It prevents the strand rejoining activity by

the topoisomerase enzyme (Witterland, Koks & Beijnen 1996). It is used for treating

small cell lung cancer (Witterland, Koks & Beijnen 1996), sarcoma, melanoma,

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renal cell carcinoma (Hande 1998), and MM with 0-4% response rate as a single

agent treatment in one study on MM (Ryan, Herndon & Vogelzang 1998).

Paclitaxel is a drug that targets the microtubules of the cell. Mitosis fails due to the

destabilisation of the cell microtubule polymerisation (Alexandre et al. 2007).

Tubulin polymers are formed (McGuire et al. 1996). Transport of organelles is also

affected and hence cellular function (Alexandre et al. 2007). It is used for treatment

of squamous cervical cancer cells (McGuire et al. 1996), melanoma, leukaemia, lung

tumours (McGuire et al. 1996) and MM with a 9% response rate as a single agent in

one study of MM (Ryan, Herndon & Vogelzang 1998) and no therapeutic response

in another study (van Meerbeeck et al. 1996).

Vincristine acts to inhibit the formation and the disruption of the cell’s mitotic

spindle at metaphase (Himes et al. 1976). The target molecule is tubulin

(Himes et al. 1976). At higher concentrations, it may have effect at interphase

(Madoc-Jones & Mauro 1968). It is used for the treatment of mammary cells in

breast cancer, lymphomas, lung small cell carcinomas (Himes et al. 1976) and MM

with very little therapeutic response as a single agent treatment for MM

(Mårtensson & Sörenson 1989).

Bleomycin belongs to a family of glycopeptide antibiotics and has been isolated

from Streptomyces verticillus. There are many analogues of the drug

(Chen & Stubbe 2005). It forms a complex with Fe (II) and O2. The DNA is attacked

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by a complex formed from this initial complex. At some sites the DNA bases are

released from their glycosidic linkages and at other sites the DNA backbone is

cleaved at the C3-C4 sites (Burger, Peisach & Band Horwitz 1981). It is a

hydrophilic molecule that can also bind other transition metals. It cannot freely

diffuse through the cell membrane and its uptake may be mediated by its positively

charged tail (Chen & Stubbe 2005). The most common outcomes of this treatment

are extended cell cycle arrest, apoptosis and mitotic cell death

(Chen & Stubbe 2005). It is used in the treatment of testicular cancer

(Chen & Stubbe 2005), leukemia (Bonadonna et al. 1972), early-stage Hodgkin’s

lymphoma (Engert et al. 2010) and squamous cell carcinomas (Cai et al. 2011) just

to name a few. In MM it is used in combination with chemotherapeutic drugs such as

Cisplatin and Doxorubicin (Baas 2002).

Aside from the variation in chemotherapeutic drugs alone, variation in response not

only exists in single-agent treatments but also in combination treatments

(Berghmans et al. 2002) such as Doxorubicin combined with Cisplatin, Bleomycin

and Mytomycin C (Baas 2002). If variation in chemotherapeutic drug sensitivity

between MM cell lines is anything to go by, there is a need for personalized therapy

(Szulkin et al. 2013), be it single-agent, combination chemotherapy or in

conjunction with other forms of therapy such as immunotherapy

(McCoy, Nowak & Lake 2009). The order in which the treatments are applied in

combination (Zanellato et al. 2011) and the dosage amount (Veltman et al. 2010) are

also factors for consideration.

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Furthermore, treatment development requires consideration of the immune response

that, for example, can manifest as inflammation induced by asbestos exposure,

involving HMGB1 and the Nalp3 inflammasome, a feature that promotes the growth

and survival of the MM cells. Targeting against these in treatment could lead to

prevention or a delay in the disease onset of MM in patients

(Carbone & Yang 2012). There is a need for modelling the disease in vivo

(Carbone et al. 2004) and in vitro to thoroughly test the efficacy of any proposed

treatment either as single agents or in combination before they are introduced into

the clinical setting.

Table 1.1. Chemotherapeutic and anti-proliferative drugs. Brief summary of

chemotherapeutic drugs for treating cancer grouped according to their mechanisms

of action.

Drug Mechanism of action Reference

Cisplatin, Cyclophosphamide, Ifosfamide

Alkylating agent, stops DNA replication Favoni et al. 2012

Doxorubicin, Epirubicin

Anthracycline, DNA intercalating agent to inhibit DNA systhesis Favoni et al. 2012

Gemcitabine, Methotrexate, Pemetrexed

Anti-metabolite, terminates DNA replication Favoni et al. 2012

Etoposide Topoisomerase inhibitor, inhibit DNA syntheis Hande 1998

Paclitaxel, Vincristine

Natural alkyloid/Vinca alkyloid compound, anti-mitotic to inhibit cell division Favoni et al. 2012

Bleomycin Anti-proliferative, DNA-strand breakage Cai et al. 2011

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1.4.0 Cell culture

For research purposes, patient or animal samples can be purified and cultured in vitro

and used for both in vitro and in vivo studies. MM cell lines can be established from

ascites by placement in tissue culture flasks and provided with nutrients. They can

then be grown as a monolayer and maintained in vitro (Davis et al. 1992) for several

months (Klominek et al. 1989) in humidity with 5% CO2 at 37°C

(Liu & Klominek 2003) or preserved at freezing temperatures, such as in a -80°C

freezer or in liquid nitrogen (Kubo et al. 2011). Cells in flasks (Figure 1.5) may

utilize varying amounts of media and supplements, such as foetal calf serum (FCS)

in RPMI: 5% in one study (Liu & Klominek 2003), 10% in another (Pass et al. 1995)

and 15% FCS in yet another study (Holloway et al. 2006). There is serum

dependence for growth of cell lines in vitro which vary depending on the particular

cell’s requirement (Klominek et al. 1989). Purified cell cultures can be immortalized

by providing them with the telomerase catalytic subunit that maintains and defers the

shortening of the cell’s telomeres thus allowing the cell to proliferate indefinitely

(Alberts et al. 2008).

Cell culture in this way is of a 2-dimensional nature. The cell interacts with the cells

directly adjacent to them and their other form of contact is adhesion with the bottom

of a flask in which they are contained. In vivo, the cells exist in a

3-dimensional (3D) environment whereby cells are surrounded by other cells all

over. To better mimic this physical layout in vitro, an AlgiMatrixTM 3D alginate

scaffold plate has been devised (Godugu et al. 2013).

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Nonetheless, the monolayer is very important for studies in MM. Monolayers exist

in the body for lining cavities (Harris et al. 2012). This is especially true in the case

of the serosal surfaces that line the pleura, pericardium and peritoneum

in which MM arises. Cell culture in monolayers have been used for many years

(Alberts et al. 2008) for obtaining data on cancers and other diseases not only in

response to therapies (Carmichael et al. 1988) but for also determining the physical

properties of cell interaction to provide information about the junction molecules

between the cells that are, for instance, readily targeted by pathogens

(Harris et al. 2012).

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a)

b)

Figure 1.5. Cell lines grown in culture. Cells were grown in a BD T75 culture flask

in complete medium and viewed under a Nikon TMS inverted phase contrast

microscope at 10x ocular and 10x objective magnifications. The diameter of field of

view has not been shown. a) A MexTAg 299 208 mouse cell line in culture. b) A

wild-type BM 164 mouse cell line in culture.

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1.4.1 In vitro analyses

There are a number of assays that can be performed on cultured cells that are aimed

at quantifying how a cell will respond to an agent and how this response would then

translate to a response in humans. Viability assays, including the Alamar Blue assay

(Godugu et al. 2013) and the MTT assay (Stockert et al. 2012), are useful for fast

and simple preliminary discovery of the efficacy of cytotoxic compounds by

determining the number of viable cells at an end point (Hamid et al. 2004). The

Alamar Blue assay is dependent on the conversion of a “non-fluorescent dye to a red

fluorescent dye” (Godugu et al. 2013) whilst the MTT assay is dependent on the

conversion of MTT, 3-[4,5-dimethylthiazol-2-yl]-2,5 diphenyl tetrazolium bromide,

into formazan crystals (Figure 1.6) by the living mitochondria in a cell

(Meerloo, Kaspers & Cloos 2011) such that there is a higher optical density reading

when more cells are viable.

It is important to determine how a cell dies when exposed to an agent such as a

chemotherapeutic drug. Cells can follow an apoptotic or necrotic pathway, a normal

or an abnormal occurrence respectively. Apoptosis is programmed cell death which

can be caused by DNA damage, accumulation of misfolded proteins and atrophy

after organ blockages. Apoptotic bodies and blebbing of the cell surface are often

seen under the microscope. In necrosis the membrane integrity of cells are

compromised such that the contents of the cell tend to leak out, the cell swells and

then bursts (Kumar et al. 2010; Alberts et al. 2008).

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Agents that cause apoptosis, and not necrosis, have been shown to be more

beneficial for the efficacy of chemotherapy (McCoy, Nowak & Lake 2009). Assays

for determining the modulation of cell death for apoptosis includes methods for

morphological assessment such as the universal terminal deoxynucleotidyl

transferase-mediated dUTP nick-end labelling (TUNEL), an assay designed to take

advantage of the fact that endonucleases cleave DNA into fragments when the cell is

undergoing apoptosis such that a procedure of the assay labels the terminal ends of

the fragmented DNA for detection (Alberts et al. 2008). Another one in use is the

fluorescence-activated cell sorter (FACS), a preparation whereby antibodies to

apoptotic cell surface markers are coupled to a fluorescent dye and exposed to cells

before a machine is used to sort individual cells according to the fluorescent signal,

or lack thereof, being emitted as the cell passes in single file across laser detectors on

the FACS machine (Figure 1.7) (Kepp et al. 2011; Alberts et al. 2008). RT-PCR can

be used for measuring mRNA expression of the anti-apoptotic BCL-2 protein family

marker (Godugu et al. 2013). Necrosis can be measured by determining extracellular

HMGB-1 from ELISA based kits (Kepp et al. 2011). Alternatively, ATP levels can

help determine death by apoptosis or necrosis (Eguchi, Shimizu & Tsujimoto 1997).

Inflammation is important in the study of MM because cells exposed to asbestos

fibres, the main risk associated with MM, make pro-inflammatory cytokines along

with other host cells to initiate inflammation, a response directed against the cancer

cells themselves (Pinato et al. 2012). It has been suggested that inflammation could

be used to predict overall survival (OS) (Suzuki et al. 2011). Neutrophil-to-

lymphocyte ratios (NLR) have been associated with prognosis but further

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investigation is required (Kao et al. 2010). Inflammation occurs not only in the

development of MM but in other cancers too, such as melanoma and lung carcinoma

(Sriram Krishnamoorthy Kenneth 2006). Similarly, inflammation has been found to

precede MM in a mouse model (Hillegass et al. 2010). Inflammation markers, such

as C reactive protein (CRP), VEGF and IL-6, can be tested using commercially

available ELISA kits (Kao et al. 2013). Light microscopy

(Michael H. Ross & Wojciech 2011) and FACS (Figure 1.7) can be used for

determining the number of neutrophils and lymphocytes for the NLR analysis

(Harvey Lodish et al. 2008).

Figure 1.6. Formazan crystals formed in cells. A paper by Stockert and colleagues

(2012) showing the formation of formazan crystals in cells during an MTT assay.

The formazan crystals are labelled with MTT-F with arrows indicating their position

in the cell. The crystals have also been shown in pink.

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Figure 1.7. Fluorescence-activated cell sorter (FACS). The suspension of labelled

cells is passed in single-file for detection by a laser beam with their emitted and

scattered lights measured for discrimination between different cell types. This image

is from Harvey Lodish et al. (2008).

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1.5.0 Animal models

Animal models for studying disease in a controlled environment are a requirement

(Kane 2006) especially since the rarity of MM is a limitation when gathering useful

information. Animal welfare is of great importance however, animal use in research

is indispensable in aiding our understanding of diseases and the provision of

preclinical data about all aspects of cancer development and treatments

(Workman et al. 2010; Carbone et al. 2004).

Rats, mice and hamsters are just some of the species that have been used to reveal

the carcinogenicity of asbestos fibres as well as various other compounds and

chemicals in relation to MM (Kane 2006). Mouse models are widely used, some

genetically engineered (Kane 2006), and there are several MM mouse models

available; heterozygous Nf2 (+/-) (Altomare et al. 2005), nude

(Spugnini et al. 2006), AB1-HA (van der Most et al. 2009), BALB/c NLRP3 (-/-)

(Chow et al. 2012) and MexTAg mice (Robinson et al. 2012) just to name a few.

The replication of the onset and progression of human cancer are essential criterion

for an ideal mouse model, however, it is difficult to generate a mouse model that will

faithfully replicate all the characteristics and variants of a human cancer. Thus a

mouse model is chosen according to the specific aims of investigation, be it genetic,

aetiological and therapeutic response or insights (Hann & Balmain 2001). Among

other phenotypic issues, spontaneous tumours can develop in some of the animal

models, such that these need to be distinguished from mesothelioma development

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and can interfere with the investigation (Workman et al. 2010). For MM, the

MexTAg asbestos-induced model has the most features in common with human MM

and there is no interference from other tumours. MM only arises in the presence of

asbestos (Robinson 2011).

Animal experiments are an avenue for providing supporting evidence for the efficacy

of a novel therapy of a disease (Workman et al. 2010). How the results of testing

exactly translate to human disease cures is a line of questioning that the researcher

needs to consider when they are setting up their animal study (Workman et al. 2010).

Predicting how a patient will respond to treatment is just as important as finding

ways to kill off these MM cells (Francis et al. 2007). Rigorous preclinical testing in

response to treatments first in the animal model is still an area in which more work is

needed (Workman et al. 2010).

1.5.1 The MexTAg mouse model

The MexTAg is a transgenic (Figure 1.8) mouse model developed for mesothelial

cell expression of the SV40 T-antigen through use of the cell specific mesothelin

promoter. Four successful MexTAg mouse lines were produced with respective copy

numbers of the SV40 T-antigen of 100, 32, 15 and 1: “299h (high), 304i

(intermediate high), 270i (intermediate low) and 266s (single)”

(Robinson et al. 2006). For the development of MM in these mouse lines, asbestos

exposure is a requirement as mice without asbestos exposure do not develop MM.

The MexTAg mouse shows low incidence of other tumours (Robinson et al. 2011).

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The wild-type counterparts only show an incidence of 20-30% of developing MM

after asbestos exposure, which is low when compared to the 299h mouse lines, on

the other hand, that show an incidence of 100% (Robinson et al. 2006). The latency

period of MM in the mouse model is comparable to that in human time span along

with the location of the tumour when it does develop (Robinson et al. 2011).

Furthermore the same carcinogen is used to induce disease. This model is useful for

testing novel therapies, cancer prevention strategies, early molecular changes in

disease development and preclinical studies for optimisation of effectiveness, dose

and scheduling of chemo or immunotherapies, single use or in combination.

1.5.2 The transplantation model

Tumours can be introduced into a mouse, and have it relatively contained, by

injecting cultured cells subcutaneously into the flank of mice

(Tomayko & Reynolds 1989). Figure 1.9 shows the schematic location of the

subcutaneous layer of the skin (Alberts et al. 2008). The main advantage of this type

of model is that tumour growth can be readily measured using callipers to monitor

response to therapy. Tumour growth is typically faster compared to the carcinogen

induced models, so experimental data can be achieved more rapidly. Additionally,

chemotherapeutic agents can be administered directly into the tumour for treatment

(Tomayko & Reynolds 1989).

In MM, subcutaneous (sc) as well as intraperitoneal (ip) injections for implantation

of tumours is used in mouse models (Varghese et al. 2012). Chemotherapy

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treatments can be administered in mice in parallel with administration in human

patients, that is, via ip (Yan et al. 2009).

Figure 1.8. Creation of a transgenic mouse. To create a transgenic mouse,

embryonic stem cells (ES) are grown in culture. An altered version of a target gene is

made. This is introduced into the ES and allowed to grow. The cells are tested to find

the one where the target gene in the ES has been replaced with the altered version.

That is then injected into an early embryo that has been isolated from a female

mouse. If successful, a hybrid early embryo is formed and injected into a female

mouse. Offspring are tested for the presence of the mutant gene and those are

selectively bred to produce the required transgenic mouse. This mage is from

Alberts et al. (2008).

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Figure 1.9. Layers of the skin. The Epidermis at the top of the image, faces a “free

surface” that is not in connection with other cells. The layer labelled ‘hypodermis’,

at the bottom of the image, is the subcutaneous layer. This image is from

Alberts et al. (2008).

1.6.0 The genetic dimension

It has been proposed that a contributing factor in the variation of prognosis is genetic

predisposition (Carbone & Pass 2006) and that there exists an increased risk for

certain individuals for acquiring the disease when exposed to asbestos due to their

genetic predisposition as indicated in a genome-wide association study (GWAS)

(Cadby et al. 2013; Matullo et al. 2013). This notion is further supported by the

discovered germline BAP-1 mutation in MM whereby it is seen to gain genetic

material at a locus but then in another MM a focal deletion occurs inside of a larger

Subcutaneous injection is in this layer, the

hypodermis.

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deletion (Testa et al. 2011). However, in a Western Australian GWAS of 428 MM

cases and 1269 controls with Italian study case-controls for reference, there was not

a single nucleotide polymorphism of statistical significance detected. Although

SKD1, encoding for adhesion molecules, did show up comparably between the

Australian and Italian studies. CRTAM, for cell adhesion, and RASGRF2, converts

movements in cells from elongation to rounding, were also highlighted in the study

(Cadby et al. 2013). More genetic studies are needed.

Molecular tools such as comparative genomic hybridization (CGH) arrays

(Figure 1.10) and DNA microarrays have greatly advanced the way in which we

visualise and thus classify cancers (Pollack 2007). Microarray data enables the

researcher to identify targets for the production of potential treatments. Such targets

include those associated with energy, remodelling of the cytoskeleton and the

translation of proteins which are up-regulated during testing of MM cells in vitro

(Robinson & Lake 2005).

MM is marked by deletions rather than gains as seen when chromosomal

karyotyping is performed (Musti et al. 2006). Through the use of CGH array, gains

and losses in the “5p, 7p, 7q, 8q, and 17q” and “1p, 3p, 6q, 9p, 13q, 14q, 15q and

22q” chromosomal regions respectively with different frequencies of gains and

losses when MM are grouped into its subtypes (Musti et al. 2006).

The deletion of the CDKN2 locus is most common in MM and found in up to 80%

of MMs. Notably, deletions are observed for CDK2NA and CDK2NB that encode

for p16 and p15 (Musti et al. 2006). This region encodes cyclin dependent kinase

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inhibitors p16 and p15, which are important in cell cycle regulation and are classed

as tumour suppressor proteins. The p53 and Rb tumour-suppressor genes which are

frequently absent or inactivated by mutation in other cancers are not targeted in MM,

perhaps because the genes for p16 and p15, which are in the same pathway as p53

and Rb, are absent instead (Robinson & Lake 2005). NF2 deletions are the next most

common, in about 50% of MMs. In MM, BAP-1 is observed to have a high rate of

deletion; in 23% of malignant pleural mesothelioma cases

(Bott et al. 2011). The LATS2 gene on the 13q12 chromosome is also seen to be

deleted in MM, but is more rare (Murakami et al. 2011).

The molecular mechanism underlying development of malignancies due to exposure

to asbestos fibres are not yet fully understood (Liu, Cheresh & Kamp 2013).

However, it has been indicated that reactive oxygen species (ROS) may play a role

in the cytotoxicity and mutagenicity of asbestos induced MM such that ROS can lead

to breaks in the DNA strand as well as changes in the DNA itself with

8-OHdG causing GT modification to name one example of its effect

(Xu et al. 1999).

There is heterogeneity in the human population for MM as seen in chromosomal

aberrations in which some variants are yet to be discovered as acquiring “snap shots”

of the state of the disease at all possible time points is a big undertaking especially

due to the long latency period of MM (Musti et al. 2006). Varying levels of

expression and activation, or lack thereof, of markers and pathways linked to the

lengthy transformation and tumour progression of mesothelial cells are being

investigated (Carbone & Yang 2012). AP-1, NF-κB and Phosphoinositide 3-

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kinase/AKT pathways are just a few of the areas in which research is being

conducted (Carbone & Yang 2012; Jagadeeswaran et al. 2006). TAM67 has been

used to target the AP-1 pathway for treating cancer by impairing directed movement

that leads to a change in cell morphology via cell cycle arrest in the G1 phase of the

cell cycle (Libermann & Zerbini 2006). Bortezomib has been used in a phase-II

clinical trial with efficacy in reducing NF-κB activity in vitro and in vivo on MM

cell lines (O’Brien et al. 2013). Unfortunately, only a small portion of patients

benefit from pathway interference of these specific therapies developed from those

formally mentioned (Carbone & Yang 2012). The Wnt pathway has been found to be

disordered in MM with “secreted frizzled-related proteins” (sFRP) primarily

involved in the disruption of the pathway (Lee et al. 2004). The hippo pathway has

also been shown to be modified, or disrupted, in MM. A key player in this is NF2,

which encodes for Merlin and regulates signalling pathways involved in cell growth.

As mentioned above NF2 is frequently found to be inactivated in MM. Targeting

NF2 could lead to a treatment option (Jean et al. 2012).

1.6.1 CGH arrays

Comparative genomic hybridization (CGH) enables the user to identify unregulated,

or down regulated, parts of the genome through the use of fluorescently labelled

DNA fragments which are competitively bound to normal metaphase chromosomes

with repeating sequences, such as those blocked by Cot-1 DNA

(Inazawa, Inoue & Imoto 2004). Red signals deletion and green signals amplification

(Figure 1.10). This process reveals copy number variation and can be applied to

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tumour samples to give information on the genetic changes that could be involved in

tumourigenesis (Inazawa, Inoue & Imoto 2004). It is possible that specific genetic

changes could account for determining sensitivity or resistance of an individual’s

cancer to treatment. However, its application is limited in that prior knowledge of

regions of interest in the DNA is needed to design the array for a study

(Davies, Wilson & Lam 2005). Due to recent technological advances, applications

such as CGH array have become more affordable and accessible to researchers

(Guan, Wang & Shih 2010).

1.6.2 DNA microarrays

Specific nucleotide sequences are arranged on a special glass slide to act as probes

for hybridizing samples of fluorescent DNA along with reference samples

(Alberts et al. 2008). For instance, if gene expression is high, in the sample that has

hybridized onto the probes on the glass slide when compared to the expression level

of the reference sample, then the spot is scanned as red. The spot is green if there is

low expression of the target gene and it is yellow if there is no difference between

the target and reference expressions. All this is then combined for complex analysis,

such as the cluster analysis, that permits the researcher to look at a large number of

genes which are regulated in a coordinated manner (Alberts et al. 2008).

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1.6.3 Next generation sequencing

Next generation sequencing (NGS) overcomes the bias limitation of CGH arrays in

that more of the genome can be targeted for inspection because little knowledge of

the DNA sequence is need to provide a more comprehensive insight into the genome

with added advantage that the machines are capable of parallel processing millions

of DNA sequence reads in a single run (Mardis 2008). The cost of running a NGS is

also exponentially decreasing, making it a more affordable option for discovering

genomic variations (Koboldt et al. 2013). As always, correct analysis of the data is

required but the volume of data is so large that at present data storage and

computational infrastructures are limiting the ability of the researcher to examine the

dataset quickly (Koboldt et al. 2013).

Figure 1.10. CGH Array. Genomic DNA from tumour and normal tissues are

fluorescently labelled and hybridized onto a DNA micro array plate with the ratios of

the red and greed signals plotted. This image is from Alberts et al. (2008).

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1.7.0 Project hypotheses

1. A heterogeneic response to chemotherapy will exist and cell lines will segregate

into clear groups of good and poor responders.

2. Response to chemotherapy will correlate with genotype.

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Chapter Two:

Materials and methods

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2.0.0 Cell culture

One major area of interest in the project, from the hypothesis that response to

chemotherapy will correlate with genotype, was in the SV40 T-antigen’s interaction

with replication at the replication forks of DNA (Murakami & Hurwitz 1993),

particularly in its role in the unwinding of the DNA (Foster & Simmons 2010). As

unknown genes in the genome were made more accessible to transcriptional

machinery by this antigen (Foster & Simmons 2010), it was important to determine

whether or not this change in accessibility would actually affect the environment

within the cell, or on the cell surface, such that the response to chemotherapy was

also altered. As such the MexTAg cell lines which already have the SV40 T-antigen

DNA fragment in their genome were selected for this project along with their

transgenic negatives, the wild-type cell lines, as controls for comparison.

All cell lines (Table 2.1) were grown in complete medium composed of Roswell

Park Memorial Institute (RPMI) 1640 medium, 5% foetal calf serum (FCS), 5%

newborn calf serum (NCS), 9 mL of 10 mM hepes, 0.5 mL of 60 mg/L

benzylpenicillin and 0.66 mL of 50 mg/mLgentamicin. They were placed in

BD T75 culture flasks for in vitro assays or BD T175 culture flasks for in vivo

assays and into a humidified 37°C / 5% CO2 incubator. At about 80% - 95%

confluence they were either passaged, to continue growth of the cell line, or utilized

in an assay.

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Table 2.1. The cell lines used in this project. There were 7 wild-type and

7 MexTAg mesothelioma cell lines which originated from C57BL/6 mice which had

all been phenotyped as sarcomatoid.

For each passage of a BD T75 culture flask of cells, the following steps were taken:

1) The old complete medium was removed by suction and the cells were briefly

washed with 5 mL of phosphate buffered saline (PBS) with the PBS also

removed by suction.

a. Note that 10 mL of PBS was used for BD T175 cultures.

2) After the addition of 1.5 mL of trypsin the flask was placed in a humidified

37°C / 5% CO2 incubator for about 1 min, or until the cells were detached from

the flask surface as viewed under an inverted microscope.

a. Note that 3 mL of trypsin was used for BD T175 cultures.

Cell Line Cell Type Description

299 376299 62

C57BL/6 mice were injected with asbestos in the peritoneum. Ascites were collected from different mice and cultured as wild-type cell lines. All

previously phenotyped as sarcomatoid.

Transgenic mice were created from embryos of the C57BL/6 mice. The SV40 Large T antigen was introduced to embryonic stem cells grown in culture. The stem cells in which one copy of a normal gene was replaced by the introduced

DNA fragment were injected into isolated partly formed early embryos and permitted to grow in female C57 black mice. These mice were then injected with asbestos in the peritoneum and the ascites collected from different mice

and cultured as MexTAg cell lines. These cell lines have 100 copies of the SV40 Large T antigen in its genome. All previously phenotyped as sarcomatoid.

wild-type

MexTAg

299 210

AE 3

AE 16

AE 17

AE 19

BM 109

BM 163

BM 164

299 166

299 170

299 175

299 208

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3) After a few taps of the flask, 8.5 mL of complete medium was added, the

contents of the flask transferred into a 50 mL BD falcon tube, the tube

centrifuged in a Hettich Rotina 420R centrifuge, or Eppendorf 5702 centrifuge,

for 5 mins at 1200 rpm, the supernatant removed by suction.

a. Note that 7 mL of complete medium was added to the flask for spinning

down of BD T175 cultures.

4) The pellet of cells was resuspended in 5 mL of complete medium.

a. Note that the pellet of cells was not resuspended in complete medium in

the case of BD T175 cultures. Refer to section 2.5.0: In vivo tumour

growth.

5) In order to keep the cell line going, a volume of this cell suspension was

transferred into a fresh BD T75 culture flask with 13 mL of complete medium.

This flask was then placed back into a humidified 37°C / 5% CO2 incubator for

cell growth.

a. Note that cell suspensions from cell cultures that originated from

BD T175 flasks were used for in vivo cell growth experiments only.

2.1.0 Proliferation

The cell lines in complete medium were plated onto BD 96-well flat bottom plates at

4 different concentrations in triplicate wells and permitted at least 3 hrs to attach to

the bottom of the well in a humidified 37°C / 5% CO2 incubator. The plates were

then placed in the Cellavista Innovatis system (Cellavista) machine hardware, by

Roche Diagnostics GmbH, with the following image adjustments, in the Cellavista

control and evaluation software version 2.0.1.860, to focus the camera on the

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adherent cells: bright-field exposure 1, UPLFLN 10x objective, 100% intensity and

white rim focus offset setting of 2. The plates were analysed for cell confluence

percentage on the Cellavista at 2 time points each day for at least 8 time points in

1 week. The plates were placed back into a humidified 37°C / 5% CO2 incubator for

cell growth between readings.

Two variables of interest were exported from the Cellavista: the cell confluence

percentage for each cell line in each well and the number of hours since the initial

reading. As the adherent cells proliferated on the surface of a BD 96-well plate, the

cell confluence percentage, as measured by the Cellavista system, also increased. At

4 different plating densities (5.00x103, 2.50x103, 1.25x103 and 1.00x103 cells/well)

on the BD 96-well flat bottom plate, the cell confluence percentage of 3 wells for

each cell line was obtained at different time points which was as far apart in a day as

possible. These 2 bits of data were exported onto a Microsoft (MS) Excel

spreadsheet for processing.

The exponential growth phase of a cell line was determined by eye from a growth

curve that was plotted using data from the Cellavista. The mean cell confluence

percentage and the standard deviation (SD) of 3 wells for each plating density of

each cell line were plotted against a time span from the point of initial reading. The

exponential growth phase was represented by the steepest, longest and near linear

slope of each plot as determined by eye.

The line of best fit through the exponential growth phase of a cell line was then

determined. A minimum of 3 cell confluence percentage data points at 3 time spans

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with relatively small SDs between the triplicate data points were used for the linear

regression analysis in Graphpad Prism version 6.02 (Graphpad) which outputted the

equations of the lines of best fit for each well on the plate. Any point along the

equation of the line of best fit represented the points that could be used to determine

the doubling time of a cell line in each well.

The proliferation rate was defined by the doubling time (Appendix III). The

proliferation assay was repeated until at least 3 doubling times were obtained for

each cell line. All statistical data analysis performed in this project was conducted in

the Graphpad software. Levels of statistical significance as grouped in the software

have been listed in Table 2.2.

Table 2.2. Statistical significance levels. The different levels of statistical

significance in Graphpad has been listed in this table both in words and as symbols

grouped according to p-values.

P-value Words Symbol

< 0.0001 Extremely significant ****

0.0001 to 0.001 Extremely significant ***

0.001 to 0.01 Very significant **

0.01 to 0.05 Significant *

≥ 0.05 Not significant ns

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2.2.0 In vivo tumour growth

The pellet of cells prepared from BD T175 culture flasks was resuspended in 10 mL

of PBS and put into a Hettich Rotina 420R centrifuge, or Eppendorf 5702 centrifuge,

for 5 mins at 1200 rpm, the supernatant removed by suction and the pellet of cells

resuspended again in 10 mL of PBS. A sample of this was diluted in PBS and mixed

with 0.4% trypan blue stain in a ratio of 1:1, loaded onto a Countess cell counting

chamber slide and the viable cells were counted using an Invitrogen Countess

automated cell counter. The remaining cell suspension was centrifuged again for

5 mins at 1200 rpm, the supernatant removed by suction and the pellet of cells made

up to the volume required in PBS for a final cell suspension concentration of

1x107 cells/mL. The cells were double contained in a 5 mL tube and put on ice for

transport.

A BD Ultra-Fine 0.5 mL needle was used to inject each of the wild-type mice with

0.1 mL of the 1x107 cells/mL wild-type cell suspension. The MexTAg mice were

each injected subcutaneously in the right hind flank with 0.1 mL of the

1x107 cells/mL MexTAg cell suspension. Mice were weighed with an Ohaus Scout

Pro SP401 set of scales, tumour sizes were measured using the Sylvac S_Cal PRO

digital caliper and measurements recorded automatically on Studylog version 1.9.8

and manually by hand on paper as backup. Pins, tumours that were too small for the

digital calipers to measure but were the start of tumour growth detection as they had

felt like a grain of rice under the skin, were approximated to a size of 0.25 mm2 for

small pins with slightly bigger pins approximated to a size of 1.00 mm2. A

qualitative scoring system was put in place to assess the well-being of mice: activity,

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alertness, body condition and coat were checked. All mice were acclimatised to their

environments and handled for at least 1 week prior to the subcutaneous injections.

Data was recorded on Studylog version 1.9.8 and exported into MS Excel 2007

before being copied into Graphpad for the production of graphs and execution of

statistical analysis.

2.3.0 MTT assay

A sample of cell suspension was mixed in a ratio of 1:1 with trypan blue. A

haemocytometer was used to count viable cells. A cell suspension in complete

medium with a concentration of 5x104 cells/mL was made. From this suspension,

0.1 mL was pipetted onto a BD 96-well flat bottom plate to yield 5x103 cells/well for

testing. This test plate was put into a humidified 37°C / 5% CO2 incubator for 24

hrs.

The chemotherapeutic drugs (Table 2.3) were obtained from Sir Charles Gardiner

Hospital (SCGH) Pharmacy. All drugs excluding Cisplatin were frozen down and

used after the first thaw. Cisplatin was only refrigerated. All drugs were used before

1 month had elapsed after freezing. The thawed drugs were serially diluted 10-fold in

complete medium in a BD 96-well round bottom plate.

A Thermo Scientific Finnpipette multichannel pipette was used to transfer 0.1 mL

from each well of the inoculation plate into the test plate that had been incubating for

24 hrs. This test plate was put back into a humidified 37°C / 5% CO2 incubator for a

further 48 hrs after which 0.05 mL of 2 mg/mL filtered MTT was added to all the

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wells of the test plate and the plate returned to a humidified 37°C / 5% CO2

incubator for a further 4 hrs of incubation.

Table 2.3. The chemotherapeutic drugs supplied by Sir Charles Gardiner

Hospital Pharmacy. The highest concentration made available for each

chemotherapeutic drug has been listed in this table. The drugs were diluted in

complete medium as necessary.

Chemotherapy Concentration (mg/mL)

Bleomycin 1 Cisplatin 1 Cyclophosphamide 20 Doxorubicin 2 Epirubicin 2 Etoposide Phosphate 20 Gemcitabine 40 Ifosfamide 100 Methotrexate 100 Paclitaxel 6 Pemetrexed 25 Vincristine 1

The plate was taken out and put into a Hettich Rotina 420R centrifuge for 5 mins at

2000 rpm. The supernatant of each well was removed using a Thermo Scientific

Finnpipette multichannel pipette set at 0.275 mL, before 0.100 mL of dimethyl

sulfoxide (DMSO) was added and mixed into to all the wells. The plate was covered

in aluminium foil and put on a Ratek E0M5 orbital shaker for 15 mins at speed 3

before the optical density (OD) reading of the homogeneous mixture was obtained at

a wavelength of 570 nm from a Molecular Devices SpectraMAX 250

spectrophotometer.

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The data outputted from the Molecular Devices SpectraMAX 250

spectrophotometer, which was exported into MS Excel 2007, had the labels “Wells”

in one column and “Values” in another. The former column contained the row and

column coordinates, on the BD 96-well flat bottom test plate, of the well that

matched the OD reading placed in the adjacent “Values” column. The OD readings

from the blank wells on the plate had already been subtracted from each of the OD

readings for each well and this result placed in the values column by the Softmax Pro

version 2.2.1 software supplied with the Molecular Devices SpectraMAX 250

spectrophotometer.

In order to compare the responses of cell lines against a chemotherapeutic drug, to

address the hypothesis that heterogeneity will exist and cell lines will segregate into

clear groups of good and poor responders, the commonly used IC50 measure for

comparing a drug’s inhibitory effectiveness was selected. It represented the

concentration of drug that provoked a cell line response which was half way between

the minimum and maximum of the dose response modelled data in the MTT assays

conducted. The MTT assay itself was repeated for each cell line against each

chemotherapeutic drug to obtain 3 IC50 values. The mean obtained from these

triplicate IC50 values represented the response of each cell line against a

chemotherpeutic drug which was then used to compare against the responses of the

other cell lines towards treatment of the same chemotherpeutic drug. The IC50 for

each cell line against each chemotherapeutic drug was obtained through a number of

steps.

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The OD reading data was first exported from the Molecular Devices SpectraMAX

250 spectrophotometer and imported into MS Excel 2007 whereby the mean value of

the drug control, at a specific concentration, was subtracted from each of the OD

reading of the triplicate test wells which were also at the corresponding

concentration on the same test plate. The drug concentrations were converted into

their log10 values and it was these newly modified triplicate OD readings that were

put into Graphpad that then enabled the production of graphs and execution of

statistical analysis on the formatted data.

In view of the fact that there was a different range of OD values from different

experiments, the OD values were fitted to a common scale so that they were brought

into proportion with one another so that comparisons of the IC50 data were made

possible between MTT experiments. As such, the Graphpad built-in “normalize”

analysis tool was first used on the OD readings that were entered into tables of the

software. The normalized values were obtained from the division of each OD

reading by the mean OD reading of the untreated cells and then multiplied by 100.

This calculation yielded the viability percentage of the cells in the triplicate wells. A

plot was made of the viability percentage for each triplicate well against the drug

tested at the corresponding concentration used to treat the cells in the triplicate wells.

The SD of the viability percentage at each point was shown and the concentration of

the drug was converted into its log10 concentration for ease of reading on the

graphical plot. The line of best fit for the dose-response data was modelled by the

"log (inhibitor) vs. response -- Variable slope (four parameters)" dose-response

curve that was built into Graphpad. It was important that the dose-response curve

clearly defined the bounds between 0% and 100% cell growth inhibition in the wells

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so that the concentration that inhibited the growth of 50% of cells was determined

more accurately. Thus, in the plot, 0% was defined as the viability from a mean OD

reading of 0 and 100% was defined as the viability from a mean OD reading of the

triplicate wells with cells that were incubated without a chemotherapeutic drug

inhibiting their growth, that is, the negative controls on the test plate. The IC50

intersection was manually approximated from these graphical plots using the plots of

the dotted lines available in the software (Figure 2.1).

The highest concentration of drug used in the MTT assay was increased when the

response to the dose did not yield a viability percentage close to zero. However,

when the IC50 was within a 10-fold difference of the other IC50 values for a given

cell line against a given chemotherapeutic drug, it was deemed close enough and was

kept in the determination of the mean IC50 for the assay.

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a)

b)

Figure 2.1. Cell viability plots in the determination of the IC50 in the MTT

assay. The MexTAg 299 166 cell line was treated with Bleomycin in two different

MTT assays. The IC50 obtained were within a tenfold difference of one another.

a) The drug dose was not enough to create a curve that goes down to zero. The

standard error bars were small at the IC50 cross-hair intersection. b) The dose was

increased so that the curve went down to zero. The standard error bars were a bit

bigger compared to those at the IC50 cross-hair intersection in experiment 11.

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The the triplicate IC50 values for the a cell line treated with each chemotherapeutic

drug was determined and the values put into a summary table of IC50 values for

1 cell line. The mean and SDs were also worked out. A table was made for each of

the 14 mesothelioma cell lines. Then, these triplicate means were put into Graphpad

so that bar graphs were made that showed the mean and SDs of the triplicate IC50 for

each cell line against each chemotherapeutic drug. The IC50 means, SDs and number

of replicates for the wild-type and the MexTAg cell lines against each

chemotherapeutic drug was also summarized. Statistical analyses were then

performed to compare the responses of mesothelioma cell lines against each

chemotherapeutic drug so that the question of whether or not heterogeneity existed in

the response of cell lines to chemotherapy such that there were clear groups of good

and poor responders, was answered.

The question was broken down into 4 main different response comparisons from the

mean IC50 values for each chemotherapeutic drug by all 14 mesothelioma cell lines:

a) The IC50 comparisons of 7 wild-type mesothelioma cell lines against each of

the 12 chemotherapeutic drugs.

b) The IC50 comparisons of 7 MexTAg mesothelioma cell lines against each of

the 12 chemotherapeutic drugs.

c) The IC50 comparisons of the combined 7 wild-type against the combined

7 MexTAg cell line responses against each of the 12 chemotherapeutic drugs.

d) The IC50 comparisons of the combined 14 mesothelioma cell lines against

each of the 12 chemotherapeutic drugs.

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2.4.0 ATP assay

In order to validate the data from the MTT assay, it was important that another

method to compare responses to chemotherapy was utilized. A paper written in the

Cancer Research Journal, suggested that the appearance of cell death was determined

by the levels of intracellular ATP (Eguchi, Shimizu & Tsujimoto 1997). ATP is the

most widely used molecule of free energy needed by cells to drive many chemical

reactions (Alberts et al. 2008). It was suggested that extracellular ATP had caused

cell death (Zheng et al. 1991). It was found that chemotherapy induced the reduction

of intracellular ATP and an increase in extracelluar ATP from tumour cells

(Martins et al. 2009). A luciferin-based ENLITEN ATP kit was also available for

testing from Promega (Martins et al. 2009). Additionally, an extracellular ATP level

increase was also found at tumour sites in vivo (Pellegatti et al. 2008).

Thus, the question initially asked was, what information about the proliferation and

viability of cells could the levels of ATP provide on the 14 mesothelioma cell lines

treated with chemotherapy? From the papers previously mentioned, it was

determined that we expected to see a certain level of ATP before chemotherapy

treatment and a different level of ATP after chemotherapy treatment. Intracellular

ATP was expected to decrease in number whilst extracellular ATP was expected to

increase in number after exposure to chemotherapeutic drugs. A reduction in

proliferation, ie. the number of cells, due to death would affect the viability output in

the MTT assay as there would be less cells to take up the MTT and form the

formazan crystals under the assumption that cells characteristically formed the

crystals at a given rate when put through controlled environmental conditions in the

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lab. Hence, it was determined that we would first measure extracellular ATP for a

subset of cell lines and chemotherapeutic drugs.

The cell line suspensions were counted and plated out to a concentration of

5x103 cells/well for testing in a similar manner conducted in the MTT Assay. The

layout of cells on the plate was different but the type of plate used was the same.

This test plate was put into a humidified 37°C / 5% CO2 incubator for 24 hrs.

Chemotherapeutic drugs from SCGH Pharmacy were diluted to the IC50

concentrations as determined from the MTT Assay. Each well of cells were

inoculated with 0.1 mL of the relevant drug at the IC50 concentration for that cell

line. The controls for the drugs were also added to the plate and the plate put into a

humidified 37°C / 5% CO2 incubator for 48 hrs.

The luciferin-based PerkinElmer ATPLite kit, used in a research paper

(Martins et al. 2009), was first used to establish a luminescence reading with the

available PerkinElmer Victor2 V 1420 multilabel counter. The kit was used

according to manufacturer’s instructions excluding the machine used to do the

testing as the recommended luminometer was not available.

This combination of kit and hardware did not produce a strong signal. Help was

sought out from the hardware manufacturer, PerkinElmer, who immediately doubled

checked to see if the settings on the machine were correct for the luciferin-based

ATP assay, which they were. It turned out that the ENLITEN ATP kit from Promega

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only provided a flash signal, a very quick signal that the recommended luminometer

would have picked up.

Hence, an alternative luciferin-based PerkinElmer ATPLite kit was recommended by

the PerkinElmer people. This was then tested according to manufacturer’s

instructions. A reading was established so the PerkinElmer ATPLite kit was selected

to conduct the rest of the ATP assays instead as the difference in ATP levels before

and after chemotherapy treatment was what mattered. The signal just had to be

strong enough to be read by the available machine.

The contents of the PerkinElmer ATPLite kit, which was selected for this assay,

were all equilibrated to room temperature: ATP free water, ATP standard, ATP cell

lysis solution and the ATP reagent (comprised of the substrate buffer and luciferase

substrate). After 48 hrs, the incubated plate was put into a Hettich Rotina 420R

centrifuge for 3 mins at 1300 rpm and 21°C. The plate was then left in the biohazard

hood for 15 mins with the other ATP Lite kit reagents.

In a Corning Incorporated Costar 3610 96-well white assay treated culture plate with

clear bottom, the ATPLite kit reagents and the supernatant of the cells were added to

the test. The plate was covered in aluminium foil and put on a Ratek E0M5 orbital

shaker for 5 mins at speed 3. It was then left to dark adapt for 2 mins in the

PerkinElmer Victor2 V 1420 multilabel counter before the luminescence was read

using the “No filter – Slot A7” emission filter and 1 second counting time

parameters under a CPS normal aperture operation of the Wallac 1420 Manager

software supplied with the machine. The data from the PerkinElmer Victor2 V 1420

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multilabel counter was also formatted in MS Excel 2007 and copied into Graphpad

for the production of graphs and execution of statistical analysis.

2.5.0 Caspase assay

The question also arose: was the reduction in viability of the cells in the MTT assay

after treatment with chemotherapy due to death by apoptosis or necrosis? It has been

suggested that drugs have been aimed at inducing apoptosis such as Bleomycin

(Chen & Stubbe 2005), Gemcitabine (Mini et al. 2006), Doxorubicin (Thorn 2011)

and Epirubicin (Khasraw, Bell & Dang 2012). However, death by apoptosis and

necrosis may occur at the same time, such as in the case of Cisplatin

(Fuertes et al. 2003). A major component leading to apoptosis has been the members

of the caspase family ranging from initiators, such as caspase 8, to effectors, such as

caspase 3, of the caspase dependent pathway to cell death (Philchenkov 2004). The

cleavage of caspase 3 and caspase 8 but not caspase 9 in the apoptotic pathway was

observed in a study whereby Cisplatin and Pemetrexed were used in combination to

treat MM (Li et al. 2012). The caspase assay was to be used to conduct an

investigation into the mode of cell death for the single chemotherapeutic treatments

with the endpoint effector caspase 3 as the target of investigation.

The cell line suspensions were counted and plated out to a concentration of

5x103 cells/well for testing in a similar manner conducted in the MTT Assay. There

was a difference in the layout of the plate and the type of plate used. The Corning

Incorporated Costar 3610 96-well white assay treated culture plate with clear bottom

test plate was put into a humidified 37°C / 5% CO2 incubator for 24 hrs.

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After 24 hrs, each well of cells was inoculated with 0.1 mL of chemotherapeutic

drugs from SCGH Pharmacy diluted to the IC50 concentrations as determined in the

MTT Assay for each a cell line were. The controls were completed on the plate and

the plate put into a humidified 37°C / 5% CO2 incubator for 48 hrs.

The contents of the Promega Caspase Glo 3/7 kit used for this assay were all

equilibrated to room temperature. After 48 hrs, the incubated plate was left in the

biohazard hood for 15 mins before 0.025 mL of caspase reagent was added to all the

wells. The plate with its lid was covered in aluminium foil and put on the Ratek

E0M5 orbital shaker for 30 seconds at speed 2.5. The plate was incubated at room

temperature for 1 hour before the PerkinElmer Victor2 V 1420 multilabel counter

was used to read the luminescence. The “No filter – Slot A7” emission filter and

1 second counting time parameters under a CPS normal aperature operation of the

Wallac 1420 Manager software supplied with the machine were used once again.

The data was also formatted in MS Excel 2007 and copied into Graphpad for the

production of graphs and execution of statistical analysis.

2.6.0 DNA extraction

One or two BD T75 flask of cells was cultured. The cell culture passage process was

followed until step number ‘4’ whereby the pellet of cells was resuspended in 10 mL

of PBS and put into a BD 15 mL falcon tube. This was then put into an Eppendorf

5702 centrifuge for 5 mins at 1200 rpm, the supernatant removed by suction, the

pellet of cells resuspended again in 10 mL of PBS, put into an Eppendorf 5702

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centrifuge for 5 mins at 1200 rpm and frozen down as a pellet in -80°C until DNA

extraction could be performed. The Qiagen DNeasy Blood & Tissue kit was used for

DNA extraction of the frozen down pellet of cells, according to the manufacturer’s

instructions.

The eluted DNA was quantified using Thermo Scientific’s NanoDrop 2000

spectrophotometer. The nucleic acid group class was selected in the NanoDrop 2000

software. The pins of the NanoDrop 2000 were wiped clean with 70% ethanol and

permitted to dry. A blank was made with the elution Buffer AE, the pins cleaned

again and 0.001 mL of DNA sample was measured. A report was produced through

the NanoDrop 2000 software.

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Chapter Three:

The growth rate

of cell lines in vitro

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3.0.0 Proliferation rates of mesothelioma cell lines

Tumour growth and response to chemotherapy is highly variable

(Szulkin et al. 2013). To begin to understand why this was the case we first

investigated the range of growth rates of our bank of 7 wild-type and 7 MexTAg cell

lines under an inverted microscope in their culture flasks. It was noted that they took

on different shapes as they grew attached to the surface of the flask and once they

had reached maturity, a stage where they had finished dividing. For example, the

wild-type AE 17 (Figure 3.1a) cell line was small and triangular-like and the

wild-type BM 163 (Figure 3.1b) was wider and more crescent-moon-like in shape. It

was evident that coverage of the flask surface occurred at different rates. A similar

inequality in confluence percentage was observed for all 14 mesothelioma cell lines.

A quantitative measure of the proliferation of these cell lines was necessary.

a) b)

Figure 3.1. The wild-type AE 17 and BM 163 cell line grown in culture. Viewed

under a Nikon TMS inverted phase contrast microscope at 10x ocular and

10x objective magnification. This cell line was originally phenotyped as

sarcomatoid. a) The wild-type AE 17 cell line. b) The wild-type BM 163 cell line.

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The Cellavista system was used to accurately quantify the growth rate of the cell

lines with different plating densities used to test the accuracy of the proliferation

assay. The growth rate was expected to have the same value once the cell line was in

its exponential phase of growth. For example, the equation of the line of best fit for

the wild-type AE 17 cell line was determined by linear regression analysis and the

doubling times interpolated. For triplicate well 1, the time spans were approximately

20.6 hrs (40% confluence) and 44.5 hrs (80% confluence) after the initial reading of

the plate. This yielded a doubling time of about 23.9 hrs. The doubling times for the

other wells were determined in a similar manner. The mean doubling time was

calculated, 24.4 hrs and put into a summary table so that repeats of the mean

doubling times were obtained for each cell line (Table 3.1). As some cell lines did

not reach exponential phase, or had reached a plateau before enough points could be

taken to draw up the line of best fit, these plots were not included. Other conditions

for inclusions have been listed in Table 3.1.

Figure 3.2. The proliferation time determined at 40% and 80% confluence for

the wild-type AE 17 cell line at a plating density of 5.00x103 cells/well. Points

interpolated from these linear models were used for determining cell growth rates.

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Table 3.1. Inclusions and exclusions in the determination of doubling times. The

mean and standard deviations of the doubling times have been calculated for each

cell line. The plots that were included in this calculation had to meet certain

criteria listed in the table. The 4 initial concentrations were 5.00x103 cells/well,

2.50x103 cells/well, 1.25 x103 cells/well and 1.00 x103 cells/well. The proliferation

assay was conducted over a period of 1 week and repeated at least twice for each cell

line.

5.00x10^3 2.50x10^3 1.25x10^3 1.00x10^3 5.00x10^3 2.50x10^3 1.25x10^3 1.00x10^3 5.00x10^3 2.50x10^3 1.25x10^3 1.00x10^3

DT1 DT2 DT3 DT4 DT5 DT6 DT7 DT8 DT9 DT10 DT11 DT12

299 166 27.5 - 30.2 - 29.3 28.4 - - - - - - 28.9 1.2

299 170 34.9 33.6 - - - - - - 28.9 - - - 32.5 3.2

299 175 37.9 37.3 - - 31.3 36.2 - - - - - - 35.7 3.0

299 208 22.1 22.3 25.6 28.5 - 23.4 - 24.6 - - - - 24.4 2.4

299 210 - - 28.1 26.0 - - - - 30.8 - - - 28.3 2.4

299 376 33.8 35.7 - - 31.1 31.7 - - 35.7 - - - 33.6 2.2

299 62 32.4 32.0 - - - 27.9 - - - - - - 30.8 2.5

AE 3 - 30.9 30.5 - - 27.2 - - - - - - 29.5 2.0

AE 16 - 34.5 - - 28.0 31.1 - - - - - - 31.2 3.3

AE 17 24.4 24.9 25.1 26.0 32.2 26.9 - 32.1 - - - - 27.4 3.4

AE 19 - - - 22.6 22.7 21.9 23.2 22.1 - - - - 22.5 0.5

BM 109 - - - 29.6 23.4 - - - 26.7 - - - 26.6 3.1

BM 163 - 27.0 27.3 - 23.4 23.0 - - - - - - 25.2 2.3

BM 164 27.4 28.1 - - 29.3 28.1 - - - - - - 28.2 0.8

* Criteria for inclusions: The line plotted was included in the calculation of the mean doubing time when the conditionthat the line of best fit had three time points or more was met, that the line through the points had an R-squared valuegreater than 0.9, that the triplicate cell confluence readings had small standard deviation error bars and that the line wasdrawn through the established exponential phase of growth for the cell line. The line plotted was excluded in thecalculation of the doubling time when these conditions were not met. The lines that were included in the calculationswere used to then interpolate values for 40% and 80% confluence in working out the doubling time of the cell line.** This experimental week has two replicates on the same plate in which the replicate with the smallest error bars isselected for consideration. Additionally, not all the cell lines were tested in week 3.

Cell line Wk 1 Wk 2 Wk 3 ** Mean doubling

time (hrs) *

SD doubling

time (hrs)

Doubling Times (hrs) Doubling Times (hrs) Doubling Times (hrs)

Starting conc. (cells/well) Starting conc. (cells/well) Starting conc. (cells/well)

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3.1.0 Proliferation comparisons

Out of the 14 mesothelioma cell lines, the shortest doubling time was 22.5 hrs for

wild-type AE 19 and the longest doubling time was 35.7 hrs for MexTAg 299 175.

Thus the wild-type AE 19 cell line was approximately 1.67 times faster in

proliferating than the MexTAg 299 175 cell line. The doubling time of the wild-type

AE 19 had a SD of 0.5 whilst the MexTAg 299 175 had a SD of 3.0.

Comparison of proliferation amongst 7 wild-type mesothelioma cell lines showed

significantly diverse median doubling times (p-value = 0.0069). The shortest

doubling time was approximately 22.5 hrs for wild-type AE 19 and the longest

doubling time was approximately 31.2 hrs for wild-type AE 16. Thus it took

approximately 1.4 times longer for the wild-type AE 16 cell line to double in number

when compared to the wild-type AE 19 cell line. In comparing each wild-type cell

line against each one of the other wild-type cell lines, to determine which pairs had

significantly different doubling times, only 1 out of 21 comparisons yielded a

statistical difference which was between the slowest and fastest growing lines:

wild-type AE 16 and wild-type AE 19 (p-value = 0.0117). Similar statistical analysis

was made for comparisons of proliferation amongst MexTAg mesothelioma cell

lines, wild-type against MexTAg cell lines and amongst all 14 cell lines.

3.2.0 A summary of the proliferation assay results

There was statistically significant diversity in proliferation between the cell lines

investigated when comparisons were made of the doubling times amongst

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7 wild-type cell lines (p-value = 0.0069), amongst 7 MexTAg cell lines

(p-value = 0.0021), between 7 wild-type and 7 MexTAg cell lines (p-value = 0.0027)

and amongst all 14 mesothelioma cell lines (p-value <0.0001). Statistical differences

were also found between pairs of cell lines within the main comparisons (Table 3.2).

It could be seen that there were some deviation from concordance with the doubling

times for these and other cell lines which would affect the accuracy of the doubling

times for comparisons. This was probably due to plating several different cell lines

on 1 plate. The Cellavista takes images of adherent cells on the bottom of the plate

which depends on how well they were focused at the beginning of the first reading.

If cells had not fully adhered to the surface of the plate when doing the initial

focusing then it would have had difficulty reading the confluence of cells in the

bottom of the well when they had finally fully adhered. Additionally, a range of

wells were focused in the first week of this experiment so that cells were not left out

for too long outside the incubator. Taking them in and out could have caused the step

wise growth in some of the plots. A line of best fit was still made to get an

approximate doubling time.

Nonetheless, there were still two wild-type cell lines that had SDs of 0.5 hrs and

0.8 hrs with a statistically significant difference between their means. This may

change as more repeats of the experiment are made but because different passages

were used for the repeats and the repeat still showed relatively small SD between

them for the 2 wild-types AE 19 and BM 164, this proliferation assay may still yield

a result that heterogeneity exists in their growth.

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Table 3.2. The diversity in proliferation rates of 14 mesothelioma cell lines. A

summary table of the main comparisons performed on the proliferation assay data

along with the pair-wise comparisons within the 4 main proliferation comparisons.

Figure 3.3. The proliferation rates of wild-type and MexTAg mesothelioma cell

lines are heterogeneic. There was heterogeneity in growth and 4 out of 91 pair-wise

comparisons yielded a statistically significant difference in proliferation rates.

Proliferation Comparisons P-value

Amongst wild-types 0.0069wild- type AE 16 vs. wild-type AE 19 0.0117

Amongst MexTAgs 0.0021MexTAg 299 175 vs. MexTAg 299 208 0.0031MexTAg 299 376 vs. MexTAg 299 208 0.0173

Wild-types against MexTAgs 0.0027

Amongst all cell lines < 0.0001MexTAg 299 175 vs. MexTAg 299 208 0.0184MexTAg 299 175 vs. wild-type AE 19 0.0014MexTAg 299 208 vs. MexTAg 299 376 0.0366MexTAg 299 376 vs. wild-type AE 19 0.0026

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Chapter Four:

The growth rate of

cell lines in vivo

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4.0.0 Establishing cell growth in vivo

Out of the 14 mesothelioma lines, only 10 had established growth in vivo. These

10 cell lines were comprised of 3 wild-type cell lines and 7 MexTAg cell lines. The

time it took for the tumours to double in size, length x width of subcutaneous

growth, was determined by a similar manner in Graphpad as in the proliferation

assay. For example, the MexTAg 299 166 cell line was subcutaneously injected into

3 MexTAg mice and the growth in tumour size plotted (Figure 4.1).

Figure 4.1. In vivo growth of MexTAg 299 166. The subcutaneous growth of the

cell line in 3 MexTAg mice plotted and modelled with the exponential growth

equation. The doubling times were determined from tumour sizes 40 mm2 and

80 mm2 using Graphpad’s built-in function for determining doubling time.

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The automatic option for determining the doubling time was was selected in Graphad

as the workings of the function had already been derived in the proliferation assay,

and was deemed to be accurate. The determination of the doubling time was

performed in a similar manner for the other 9 cell lines. The model for the lines of

best fit changed depending on which model gave the better R-squared value in the

goodness of fit test, which was also available as a function in Graphpad (Figure 4.2).

The MexTAg 299 170 was calculated manually at 20 mm2 and 40 mm2 in size

because all 3 of the growing tumours for this cell line did not reach a size of 80 mm2.

Additionally, there was no quick feature in Graphpad to automatically work out

doubling times from a linear model of the MexTAg 299 170. The maths for doubling

time determination was still correct, but just like the cell lines in vitro, the cells may

have only just started to establish progression and so their exponential rate of growth

was not yet fully established, hence this cell line was excluded from the

comparisons. By this same reasoning, the wild-type AE 19 cell line also did not

reach the 40 mm2 and 80 mm2 mark and was therefore excluded.

The wild-type BM 109 was excluded from the comparisons when it was determined

that a mean doubling time could not be obtained because 1 of the 3 mice did not

have enough points for the exponential curve to model. Similarly, the MexTAg

299 376, MexTAg 299 210, MexTAg 299 175 and MexTAg 299 170 were excluded

from comparisons because 1 of the 3 mice did not have a tumor that reached the

80 mm2 mark. However the 2 that did grow had similar growth curves for each of the

5 cell lines mentioned (Figure 4.3). Another unknown confounding factor was most

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likely present in the third mouse that such that the tumours were seen to not grow at

the same rate.

Figure 4.2. The exponential and linear growths. Out of the 10 mesothelioma cell

lines that grew in vivo, the MexTAg 299 208 was the fastest growing exponentially

whilst the MexTAg 299 62 had a very linear growth pattern.

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Figure 4.3. The excluded growth curves from analysis. Out of the 10

mesothelioma cell lines that grew in vivo, these 6 were excluded from analysis due

to outliers in the replicates or not reaching a size of 80 mm2.

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4.1.0 Fast growing wild-types

Out of the 10 cell lines that grew in vivo, the fastest growing cell line was wild-type

BM 164 and the slowest growing cell line was MexTAg 299 62 (Table 4.1). Out of

the 3 wild-type cell lines that grew in mice, the wild-type BM 164 cell line was the

only one that was deemed okay to make doubling time comparisons with. Hence, no

comparisons in the doubling times could be performed between wild-type cell lines.

Following this, there were not enough data points to make the consistent comparison

of triplicate mean doubling times between MexTAg and wild-type cell lines.

Table 4.1. The fastest and slowest growing cell lines in vitro and in vivo. A rank

comparison of the mean doubling times for 10 cell lines. In the in vivo calculations

of the mean doubling time, the means for 2 mice were calculated in a few.

Mean SDwild-type AE 19 22.5 0.5MexTAg 299 208 24.4 2.4wild-type BM 109 26.6 3.1wild-type BM 164 28.2 0.8MexTAg 299 210 28.3 2.4MexTAg 299 166 28.9 1.2MexTAg 299 62 30.8 2.5MexTAg 299 170 32.5 3.2MexTAg 299 376 33.6 2.2MexTAg 299 175 35.7 3.0

Mean SDwild-type BM 164 3.6 0.4MexTAg 299 208 7.2 0.3MexTAg 299 210 8.5 0.9wild-type BM 109 14.2 0.9wild-type AE 19 15.1 3.7MexTAg 299 175 18.6 1.9MexTAg 299 166 20.5 3.7MexTAg 299 376 29.3 10.6MexTAg 299 170 30.7 6.0MexTAg 299 62 31.2 1.9

In Vitro Doubling Times (hrs)

Cell LineIn Vivo Doubling Times (days)

Cell Line

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Out of the 6 MexTAg cell lines that grew in mice, only 3 were determined to be

appropriate for making comparisons between MexTAgs. There was significantly

diverse median times to reach a tumour size of 80 mm2 between the 3 MexTAg cell

lines (p-value = 0.0036). The shortest doubling time was approximately 7.20 days

(168 hrs) for wild-type BM 164 and the longest doubling time was approximately

31.2 days (749 hrs) for MexTAg 299 62. Thus it took approximately 4 times longer

for the MexTAg 299 62 cell line to grow in vivo and reach a size of 80 mm2 when

compared to the MexTAg 299 208 cell line. In pair-wise comparisons of MexTAg

cell lines to determine which pairs had significantly different doubling times, only

1 out of 3 comparisons yielded a statistical difference which was between the

slowest and fastest growing lines: MexTAg 200 208 and MexTAg 299 62

(p-value = 0.0219). No significant diversity in growth was found between the 4 cell

lines investigated when comparisons were made of the growth amongst 1 wild-type

and 3 MexTAg cell lines (p-value = 0.1145).

4.2.0 A summary of the in vivo cell growth results

There was no consistent comparison of triplicate mean doubling times possible for in

vivo growth between wild-type cell lines or between MexTAgs and wild-types due

to insufficient data points. However, the comparison between MexTAg cell lines

revealed that there was a difference between 3 of the cell lines with the biggest

difference in growth rate between MexTAg 299 208 and MexTAg 299 62

(p-value = 0.0219). Although this is not extremely statistically significant, it is

surprising because the MexTAgs were meant to be more similar because they all had

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SV40 T-antigen and they were all injected with cell lines on the same day. The

experiment will need to be repeated with the same lines and then other cell lines for

making comparisons to confirm this finding.

Additionally, there was no statistically significant difference in growth amongst

wild-type BM 164, MexTAg 299 166, MexTAg 299 208 and MexTAg 299 62 to

indicate that there was no heterogeneity in the growth of mesothelioma cell lines in

vivo. However, from the graphs, the cell lines were clearly growing at different rates.

The disparity between the maths and the observations could be because of variability

amongst mice, a confounding factor in the experiment.

The wild-type BM 164 cell line was faster growing in vivo than 2 out of 3 MexTAg

cell lines: MexTAg 299 166 and MexTAg 299 62. The SD of the means was

approximately 0.4 days (9.6 hrs), 3.7 days (88.8 hrs) and 1.9 days

(45.6 hrs) respectively. Additionally, the wild-types were generally faster growing in

vivo and in vitro when compared to MexTAgs (Table 4.1). Overall, the doubling

times were longer in vivo compared to in vitro which was to be expected as there

were only a few factors hindering cell growth in vitro compared to in vivo.

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Chapter Five:

The heterogeneic response

of mesothelioma cell lines

to chemotherapy

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5.0.0 Assessment of the cell line response to chemotherapy

The response of a cell line to chemotherapy is typically assessed by measurement of

the IC50 value: that is the concentration at which 50% of cell growth is inhibited. In

these first experiments to test the hypothesis that “heterogeneity will exist and cell

lines will segregate into clear groups of good and poor responders”, the MTT assay

was used. The ability of the MTT assay to represent proliferation is tested and

discussed later in the thesis.

To enable comparisons to be made between cell lines, optimisation of the techniques

and data analysis was required. The MexTAg cell line 299 208 treated with the

chemotherapeutic drug Gemcitabine was used to work through the process and

analysis of the results from 3 separate experiments performed in triplicate have been

described here. These steps were eventually applied to the experimental repeats of

each cell line treated against each chemotherapeutic drug.

In experimental repeat number 1, the MexTAg 299 208 cell line was treated with

Gemcitabine at 10-fold serial concentrations from 1,000 ng/mL down to 0.01 ng/mL

and proliferation measured 48 hrs later by MTT assay. However, these

concentrations were converted into their log10 concentration from 3 ng/mL down to

-3 ng/mL respectively for ease of reference when they were put into Graphpad. In

experimental repeat 2 and 3, the maximum concentration of Gemcitabine was

increased to 10,000 ng/mL with 10-fold serial concentrations down to 0.01 ng/mL

used in both experiments and their log10 concentration was converted accordingly.

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Normalisation of raw data

The OD readings, raw data minus the blank control, obtained from the MTT assay

for each triplicate well of the test plate was normalized across each MTT experiment

so that a trend between the MexTAg 299 208 cell line against Gemcitabine, or lack

thereof, was identified. The mean OD reading of the MexTAg 299 208 untreated

cells for experiment number 1, 2 and 3 were all mid range ( 0.615, 0.502 and 0.577

respectively) and had similar accuracy (SDs were 0.020, 0.043, and 0.034

respectively). There were some negative values in the OD readings at the higher end

of Gemcitabine concentration when all values were expected to have been zero or

above (Table 5.1, 5.2 and 5.3). This was most likely due to inaccuracy in pipetting

because the majority of values at these concentrations were generally positive and

the negative values were practically zero.

The normalization function in Graphpad was used such that the viability of the

untreated cells was taken to be 100%. Each OD reading in the test wells was divided

by the mean OD reading of the control wells, the untreated cells, and then that value

was multiplied by 100. The medians between normalized OD values of the

3 experiments were not statistically significantly different (p-value = 0.7396).

Determining the IC50

The viability percentage for the cell line was obtained by taking the mean of the

3 normalized OD readings for each concentration of Gemcitabine tested and plotted

against the relevant concentrations of Gemcitabine. The line of best fit for the

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dose-response data was inserted. The IC50 intersection was manually approximated

for all 3 experimental repeats (Figure 5.1).

Table 5.1. The triplicate well OD readings for experimental repeat number 1 of

the MexTAg 299 208 treated with Gemcitabine. a) The 3 OD readings for each

Gemcitabine concentration used in the MTT assay that were put into Graphpad.

b) The normalized OD values, each representing the cell percentage viability for

each well of the MexTAg 299 208 treated with Gemcitabine.

a)

b)

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Table 5.2. The triplicate well OD readings for experimental repeat number 2 of

the MexTAg 299 208 treated with Gemcitabine. a) The 3 OD readings for each

Gemcitabine concentration used in the MTT assay that were put into Graphpad.

b) The normalized OD values, each representing the cell percentage viability for

each well of the MexTAg 299 208 treated with Gemcitabine.

a)

b)

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Table 5.3. The triplicate well OD readings for experimental repeat number 3 of

the MexTAg 299 208 treated with Gemcitabine. a) The 3 OD readings for each

Gemcitabine concentration used in the MTT assay that were put into Graphpad.

b) The normalized OD values, each representing the cell percentage viability for

each well of the MexTAg 299 208 treated with Gemcitabine.

a)

b)

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Figure 5.1. Triplicate IC50 values determined from plots of MexTAg 299 208

treated with Gemcitabine. The sigmoidal dose-response model was used for line of

best fit through the experimental points. The blue dotted cross-hairs marked the

intersection where the cell line was at 50% viability when treated with approximately

7 ng/mL of Gemcitabine. This model was affected by the accuracy of the points such

that a slight difference in the shape of the curve was shown. The top left hand plot

represents experimental repeat 1. The top right hand plot represents experimental

repeat 2. The bottom plot represents experimental repeat 3. Three different passages

of the MexTAg 299 208 cell line were used in the experiments on 3 different days

yet IC50s with very small SDs were obtained from the MTT assay.

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Gemcitabine inhibited growth of MexTAg 299 208 with a range of IC50 values:

8.7 ng/mL, 3.5 ng/mL and 7.6 ng/mL for experimental repeats 1, 2 and 3

respectively. The mean of the 3 was 6.6 ng/mL with a SD of 2.8 ng/mL. Overall

they were the same order of magnitude. Proliferation of the cells in the wells was

seen to be hindered with chemotherapeutic treatment at various high concentrations

of chemotherapeutic drugs. Hence, cell proliferation was directly proportional to the

concentration of chemotherapeutic drug it was treated with. The negative values

noted previously did not have a great impact on the shape of the curve but the values

would have affected the curve more had they been more negative and positioned at

the IC50, at the 50% viability mark on the plot.

The IC50 values for the MexTAg 299 208 cell line treated with the other

chemotherapeutic drugs were determined in the same manner (Table 5.4). There

were a few points noted about the replicates for the MexTAg 299 208 cell line. The

SDs were quite large in some of the replicate IC50 values particularly against

chemotherapeutic drugs that yielded IC50 values at the higher end of the log10

concentration spectrum. Adjustments were made to cover the optimal concentrations

such that IC50 would fall midrange where possible. However, this was not possible

for every drug.

The IC50 values of chemotherapeutic drugs grouped into their mechanisms of action

yielded higher IC50 values for alkylating agents compared to anti-metabolites.

Cisplatin yielded an IC50 which was about 300 times less than the other two

alkylating agents. Hepatic microsomal enzymes were not included in the test for

Cyclophosphamide and Ifosfamide yet these prodrugs, drugs that required aid to be

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converted into their active form (Wu 2009; Fleming 1997), yielded an IC50 of

534,000 ng/mL and 261,000 ng/mL respectively. The smallest IC50 value was from

Gemcitabine, 6.59 ng/mL, and the largest was from Cyclophosphamide,

534,000 ng/mL. Thus, for Gemcitabine, a concentration of about 81,000 times less

than Cyclophosphamide was needed to illicit a cell line response that inhibited 50%

of cell growth.

A similar table of IC50 replicates was completed for the other 13 mesothelioma cell

lines and put into Graphpad for statistical analysis. The IC50 mean and SDs for the

wild-type and the MexTAg cell lines against each chemotherapeutic drug along with

the number of repeats were summarized in Table 5.5 and Table 5.6 respectively.

Table 5.4. The IC50 replicates for the MexTAg 299 208 cell line. The replicate

values determined from dose-response curves and rounded to 3 significant figures.

Chemotherapy

1480 1450Mean IC50 = 1460 SD IC50 = 17.3

617 562Mean IC50 = 603 SD IC50 = 36.5

537000 562000Mean IC50 = 534000 SD IC50 = 30700

63.1 95.5Mean IC50 = 70.4 SD IC50 = 22.4

24.5 17.8Mean IC50 = 21.9 SD IC50 = 3.59

12600 9770Mean IC50 = 11400 SD IC50 = 1450

8.71 7.59Mean IC50 = 6.59 SD IC50 = 2.76

257000 302000Mean IC50 = 261000 SD IC50 = 39200

55.0 14.1Mean IC50 = 30.8 SD IC50 = 21.4

776 513Mean IC50 = 601 SD IC50 = 152

89.1 67.6Mean IC50 = 82.0 SD IC50 = 12.4

17.4 33.8Mean IC50 = 18.9 SD IC50 = 14.2

Vincristine5.62

Paclitaxel513

Pemetrexed89.1

Ifosfamide224000

Methotrexate23.4

Etoposide Phosphate11700

Gemcitabine3.47

Doxorubicin52.5

Epirubicin23.4

Bleomycin1450

Cisplatin631

Cyclophosphamide501000

The MexTAg 299 208 cell line IC50 replicates with sample mean and standard deviation (ng/mL)

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Table 5.5. The IC50 summary table for all the wild-type cell lines. The mean, SD

and number of replicates for each wild-type cell line is shown.

Table 5.6. The IC50 summary table for all the MexTAg cell lines. The mean, SD

and number of replicates for each MexTAg cell line is shown.

AE 3 AE 16 AE 17 AE 19 BM 109 BM 163 BM 164(Wild-Type) (Wild-Type) (Wild-Type) (Wild-Type) (Wild-Type) (Wild-Type) (Wild-Type)

3,590 2,080 311 2,040 738 394 9,850SD= 4270 ; RPT= 3 SD= 384; RPT= 3 SD= 89.3; RPT=3 SD= 850; RPT= 3 SD= 131; RPT= 3 SD= 118; RPT=3 SD= 348; RPT=3

6,470 1,100 639 2,740 693 736 1,160SD= 2870; RPT= 3 SD= 386; RPT= 3 SD= 81.4; RPT=3 SD= 1640; RPT= 3 SD= 115; RPT= 3 SD= 19.7; RPT=3 SD= 218; RPT=3

2,010,000 2,880,000 674,000 2,330,000 750,000 955,000 2,170,000SD= 435000; RPT= 3 SD= 885000; RPT= 3 SD= 199000; RPT=3 SD= 793000; RPT= 3 SD= 241000; RPT= 3 SD= 311000; RPT=2 SD= 919000; RPT=3

369 309 181 63.9 118 160 1,310SD= 199; RPT= 3 SD= 147; RPT= 3 SD= 20.9; RPT=3 SD= 21.8; RPT= 3 SD= 41.7; RPT= 3 SD= 61.0; RPT=3 SD= 410; RPT=3

175 133 69.6 43.4 69.5 91.6 680SD= 67.3; RPT= 3 SD= 50.0; RPT= 3 SD= 20.1; RPT=3 SD= 7.88; RPT= 3 SD= 38.5; RPT= 3 SD= 11.3; RPT=3 SD= 217; RPT=3

59,500 87,500 14,100 35,300 27,400 10,300 53,800SD= 25700; RPT= 3 SD= 21800; RPT= 3 SD= 5140; RPT=3 SD= 8040; RPT= 3 SD= 4990; RPT= 3 SD= 2320; RPT=3 SD= 12200; RPT=3

15.0 3.45 7.09 10.6 7.94 3.95 7.13SD= 4.25; RPT= 3 SD= 0.812; RPT= 3 SD= 1.50; RPT=3 SD= 1.09; RPT= 3 SD= 0.0; RPT= 3 SD= 0.54; RPT=3 SD= 1.73; RPT=3

748,000 719,000 636,000 631,000 194,000 365,000 739,000SD= 43600; RPT= 3 SD= 107000; RPT= 3 SD= 141000; RPT=3 SD= 0.0; RPT= 3 SD= 33000; RPT= 3 SD= 297000; RPT=3 SD= 140000;RPT=3

46,100 2,990 11.0 9,020 408 45.6 130SD= 32600; RPT= 3 SD= 2870; RPT= 3 SD= 1.36; RPT=3 SD= 8320; RPT= 3 SD= 350; RPT= 3 SD= 36.1; RPT=3 SD= 42.7; RPT=3

1,550 316 46.1 197 37.6 491 1,860SD= 741; RPT= 3 SD= 89.6; RPT= 3 SD= 25.3; RPT=3 SD= 44.3; RPT= 3 SD= 8.47; RPT= 3 SD= 160; RPT=3 SD= 237; RPT=3

3,520,000 7,090,000 20.9 269,000 7,730 7,650.0 61,400SD= 2260000; RPT= 3 SD= 3200000; RPT= 3 SD= 8.08; RPT=3 SD= 101000; RPT= 3 SD= 5290; RPT= 3 SD= 2510; RPT=3 SD= 27300; RPT=3

6,670 5,900 7.80 57,900 40.1 7.61 512SD= 3540; RPT= 3 SD= 1060; RPT= 3 SD= 4.03; RPT=3 SD= 37300; RPT= 3 SD= 37.3; RPT= 3 SD= 2.25; RPT=3 SD= 250; RPT=3

Bleomycin

Cisplatin

Cyclophosphamide

Doxorubicin

Chem

othe

rapy

Ifosfamide

Methotrexate

Paclitaxel

Pemetrexed

Vincristine

Epirubicin

Etop. Phosphate

Gemcitabine

Cell line IC50 sample means and standard deviations (ng/mL) with the number of replicates.

299 166 299 170 299 175 299 208 299 210 299 376 299 62(MexTAg) (MexTAg) (MexTAg) (MexTAg) (MexTAg) (MexTAg) (MexTAg)

261 248 2,650 1,460 1,340 1,340 2,640SD= 144; RPT=3 SD= 100; RPT=3 SD= 1000; RPT=3 SD= 19.4; RPT=3 SD= 341; RPT=3 SD= 533; RPT=3 SD= 933; RPT=3

982 555 564 603 199 474 2,660SD= 113; RPT=3 SD= 47.2; RPT=3 SD= 45.9; RPT=3 SD= 36.2; RPT=3 SD= 18.0; RPT=3 SD= 97.8; RPT=3 SD= 1680; RPT=3

531,000 452,000 640,000 534,000 183,000 395,000 2,090,000SD= 376000; RPT=3 SD= 22100; RPT=2 SD= 242000; RPT=3 SD= 30700; RPT=3 SD= 27800; RPT=3 SD= 87800; RPT=3 SD= 470000; RPT=3

134 71.2 142 70.4 116 200 366SD= 9.73; RPT=3 SD= 20.0; RPT=3 SD= 62.4; RPT=3 SD= 22.4; RPT=3 SD= 4.09; RPT=3 SD= 149; RPT=3 SD= 91.1; RPT=3

67.4 18.3 45.7 21.9 60.1 84.3 153SD= 44.1; RPT=3 SD= 12.2; RPT=3 SD= 40.4; RPT=3 SD= 3.63; RPT=3 SD= 20.2; RPT=3 SD= 16.4; RPT=3 SD= 24.5; RPT=3

9,880 3,600 11,200 11,400 5,530 17,400 31,800SD= 1260; RPT=3 SD= 47.7; RPT=3 SD= 3000; RPT=3 SD= 140; RPT=3 SD= 818; RPT=3 SD= 1080; RPT=3 SD= 5890; RPT=3

9.78 8.58 4.51 6.59 5.07 4.24 11.0SD= 1.47; RPT=3 SD= 1.27; RPT=3 SD= 1.46; RPT=3 SD= 2.76; RPT=3 SD= 2.42; RPT=3 SD= 2.05; RPT=3 SD= 1.27; RPT=3

568,000 254,000 239,000 261,000 247,000 324,000 323,000SD= 39500; RPT=3 SD= 238000; RPT=3 SD= 41300; RPT=3 SD= 39200; RPT=3 SD= 30500; RPT=3 SD= 27000; RPT=3 SD= 63500; RPT=3

59,800 43.4 37.0 30.8 14.3 19.7 261,000SD= 34100; RPT= 3 SD= 30.6; RPT=3 SD= 43.4; RPT=3 SD= 21.4; RPT=3 SD= 4.50; RPT=3 SD= 15.9; RPT=3 SD= 88900; RPT=3

372 540 878 601 866 357 3,470SD= 194; RPT=3 SD= 315; RPT=3 SD= 859; RPT=3 SD= 152; RPT=3 SD= 377; RPT=3 SD= 126; RPT=3 SD= 2460; RPT=3

14.2 17.1 63.8 82.0 79.1 151.0 4,480,000SD= 1.63; RPT=3 SD= 7.52; RPT=3 SD= 35.7; RPT=3 SD= 12.4; RPT=3 SD= 14.0; RPT=3 SD= 55.1; RPT=3 SD= 2590000; RPT= 3

41.6 43.2 48,700 18.9 35.0 1,960.00 46,700SD= 14.8; RPT=3 SD= 10.1; RPT=3 SD= 24900; RPT=3 SD= 14.2; RPT=3 SD= 24.0; RPT=3 SD= 736; RPT= 3 SD= 20900; RPT=3

Ifosfamide

Methotrexate

Paclitaxel

Pemetrexed

Vincristine

Epirubicin

Etop. Phosphate

Gemcitabine

Chem

othe

rapy

Cell line IC50 sample means and standard deviations (ng/mL) with the number of replicates.

Bleomycin

Cisplatin

Cyclophosphamide

Doxorubicin

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It was important to note a few things from Table 5.5, the wild-type BM 163 cell line

only had two completed IC50 replicates against the chemotherapeutic drug

Cyclophosphamide. The IC50 values for each of the chemotherapeutic drugs were

not identical. The large difference in SD at the higher IC50 values noted for the

MexTAg 299 208 cell line had remained for all the other wild-type cell lines with

high IC50 values. Cisplatin still yielded an IC50 value of approximately 300 times

less than the other 2 alkylating agents, Cyclophosphamide and Ifosfamide, for all the

7 wild-type cell lines. Hepatic microsomal enzymes were again not included in the

test for Cyclophosphamide and Ifosfamide, yet these still yielded an IC50 value. The

smallest IC50 values were from Gemcitabine across all 7 wild-type cell lines and the

largest IC50 value was from Cyclophosphamide in 5 out of 7 wild-type cell lines.

Thus, a majority of wild-type cell lines were more sensitive to Gemcitabine than

Cyclophosphamide. Similar notes were observed for Table 5.6.

5.1.0 The heterogeneity of response of mesothelioma cell

lines to chemotherapy treatment

The 7 wild-types were first compared between one another against their response to

Bleomycin and were found to have significantly different responses to the drug: the

median IC50 varied significantly (p-value = 0.0060). The smallest IC50 was

approximately 3.11x102 ng/mL for AE 17 and the largest IC50 was approximately

9.85x103 ng/mL for BM 164. Thus the wild-type AE 17 cell line was approximately

32 times more sensitive to Bleomycin than the wild-type BM 164 cell line.

Following a comparison of each wild-type cell line against each one of the other

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wild-type cell lines to determine which pairs had significantly different IC50 values,

2 out of 21 comparisons yielded a statistical difference between the following cell

lines: wild-type AE 17 vs. wild-type BM 164 (p-value = 0.0167) and wild-type BM

163 vs. wild-type BM 164 (p-value = 0.0334). This line of reasoning was conducted

for the other chemotherapeutic drugs. There were significant differences in the

response by wild-type cell lines against 11 out of 12 chemotherapeutic drugs. In

these responses, 3 out of 11 were significant and 8 out of 11 were very significant

(Table 5.7). The response of MexTAgs were analysed, then wild-types against

MexTAgs and then the overall differences between the mesothelioma cell lines

against each of the 12 chemotherapeutic drugs.

Table 5.7. The level of difference in response to chemotherapy by the wild-type

cell lines. The level of significance in the difference of response to each

chemotherapeutic drug have been indicated in symbols and words according to their

p-values in levels derived from the Graphpad analysis software.

Level of statistical significance Chemotherapeutic

Drug P-value In Symbol In Words

Bleomycin 0.0060 ** Very significant Cisplatin 0.0140 * Significant Cyclophosphamide 0.0274 * Significant Doxorubicin 0.0096 ** Very significant Epirubicin 0.0113 * Significant Etoposide Phosphate 0.0052 ** Very significant Gemcitabine 0.0058 ** Very significant Ifosfamide 0.0899 ns Not significant Methotrexate 0.0054 ** Very significant Paclitaxel 0.0050 ** Very significant Pemetrexed 0.0041 ** Very significant Vincristine 0.0041 ** Very significant

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5.2.0 A summary of the MTT assay

When the OD values of the control wells, cells with no drug, were compared to the

test wells, cells treated with a drug, proliferation was seen to be hindered by

chemotherapy at various high concentrations in the test wells. The 4 major IC50

comparisons of the MM cell lines against the 12 chemotherapeutic drugs indicated

that there was heterogeneity in the proliferation of cell lines after treatment.

In the IC50 comparisons of 7 wild-type mesothelioma cell lines against each of the

12 chemotherapeutic drugs: statistically significant differences in response to

chemotherapy treatment were observed for 12 out of 12 chemotherapeutic drug

comparisons. In these responses, 4 out of 12 were significant and 8 out of 12 were

very significant. Multiple comparisons yielded statistically significant differences in

responses to chemotherapy between pairs of cell lines in 9 out of

12 chemotherapeutic drugs tested.

In the IC50 comparisons of 7 MexTAg mesothelioma cell lines against each of the

12 chemotherapeutic drugs: statistically significant differences in response to

chemotherapy treatment were observed for 11 out of 12 chemotherapeutic drug

comparisons. In these responses, 1 out 12 was not significant, 6 out of 12 were

significant and 5 out of 12 were very significant. Multiple comparisons yielded

statistically significant differences in responses to chemotherapy between pairs of

cell lines in 6 out of 12 chemotherapeutic drugs tested.

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In the IC50 comparisons of 7 wild-type against 7 MexTAg cell lines treated with

each of the 12 chemotherapeutic drugs: statistically significant differences in

response to chemotherapy treatment were observed for 6 out of 12 chemotherapeutic

drug comparisons. In these responses, 6 out 12 were not significant, 2 out of 12 were

very significant and 4 out of 12 were extremely significant. Multiple comparisons

yielded statistically significant differences in responses to chemotherapy between

pairs of cell lines in 6 out of 12 chemotherapeutic drugs tested. Amongst all the cell

lines compared against each chemotherapeutic drug,

In the IC50 comparisons of 7 wild-type combined with 7 MexTAg cell lines treated

against each of the 12 chemotherapeutic drugs: statistically significant differences in

response to chemotherapy treatment were observed for 12 out of

12 chemotherapeutic drug comparisons. In these responses, 6 out 12 were very

significant and 6 out of 12 were extremely significant. Multiple comparisons yielded

statistically significant differences in responses to chemotherapy between pairs of

cell lines in 11 out of 12 chemotherapeutic drugs tested. The largest statistical

difference in response was against Pemetrexed between wild-type AE 16 and

MexTAg 299 166 (7.90x106 ng/mL). Excluding Ifosfamide, which showed no

statistical difference between all 14 cell lines, the smallest statistical difference in

response was between wild-type AE 3 and wild-type AE16 against Gemcitabine

(1.16x101 ng/mL).

The MTT assay is limited in that there are so many places that variation can be

introduced: from the pipetting right through to the timing of when cells are taken out

of the incubator and any loss of volume in the media due to evaporation.

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Chapter Six:

Investigating the death of cells in

response to chemotherapy

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6.0.0 The release of ATP and caspase 3/7

When cells die, either by apoptosis or necrosis, ATP is released. It was found that

chemotherapy induced the reduction of intracellular ATP and an increase in

extracelluar ATP from tumour cells (Martins et al. 2009). When cells die by

apoptosis, caspase expression increases (Philchenkov 2004). Chemotherapeutic

drugs have been aimed at inducing apoptosis (Chen & Stubbe 2005;

Mini et al. 2006). The apoptosis pathway for cell death is preferred and the

relationship between ATP and caspase levels to give an idea of what chemotherapy

was doing to cell growth was important. The levels were expected to be different

between therapies but would it be different between cell lines for the same therapy?

We wished to find out. Additionally, the levels of ATP ought to mirror the MTT

assay as there was a link between viable and dying cells to ATP levels.

6.1.0 Establishing a reading for ATP luminescence

It was thought that more ATP in the supernatant of treated cells would correlate with

more cell death and hence less viability. The luciferin-based PerkinElmer ATPLite

kit, used in a research paper (Martins et al. 2009) was not compatible with the

PerkinElmer Victor2 V 1420 multilabel counter available in the lab. The

manufacturer of the PerkinElmer Victor2 V 1420 multilabel counter suggested an

alternative to the luciferin-based kit, the PerkinElmer ATPLite kit, because the

Promega ENLITEN ATP kit only emitted a flash signal, a very quick signal that the

recommended luminometer would detect that the PerkinElmer Victor2 V 1420

multilabel counter could not. Hence the luciferin-based PerkinElmer ATPLite kit

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was ordered. The small number of cell lines that were available at the time included:

MexTAg 299 166, MexTAg 299 208, wild-type AE 17 and wild-type BM 164.

There were also 5 chemotherapeutic drugs that had different mechanisms of actions

available: Bleomycin, Cisplatin, Doxorubicin, Gemcitabine and Paclitaxel.

Undiluted supernatant from cells that had been incubated for 48 hrs with or without

chemotherapeutic drugs, was used in the test for extracellular ATP levels. As an

example, the plate layout for the MexTAg 299 166 cell line and the luminescence

results after 1 hr of incubation with the luciferase reagent in the kit, as per the

manufacturer’s instructions, has been shown in Figure 6.1.

Figure 6.1. The ATP assay plate layout of results. The luminescence readings

were put into their respective well from the template used in the ATP experiment.

1 2 3 4 5 6 7 8 9 10 11 12

rL/L + LB + ATPFW

+ ATP

rL/L + LB + ATPFW

+ ATP

rL/L + LB + ATPFW

+ ATP

rL/L + LB + ATPFW

+ ATP

rL/L + LB + ATPFW

+ ATP

rL/L + LB + ATPFW

+ ATP

rL/L + LB + ATPFW

+ ATP

rL/L + LB + ATPFW

+ ATP

rL/L + LB + ATPFW

+ ATP

rL/L + LB + ATPFW

+ ATP

rL/L + LB + ATPFW

+ ATP

rL/L + LB + ATPFW

+ ATP

1.0E-02 1.0E-03 1.0E-04 1.0E-05 1.0E-06 1.0E-07 1.0E-08 1.0E-09 1.0E-10 1.0E-11 1.0E-12 1.0E-13

B

C

D

E

F

G

H =

1 2 3 4 5 6 7 8 9 10 11 12

A 1449960 2681967 1180700 223273 22739 3176 604 233 148 115 92 75

B 11228 16609 9356 3888 1539 762 367 215 170 123 80 70

C 1917 2240 1987 1382 1019 655 341 232 173 120 104 80

D 936 1152 901 779 650 495 348 237 203 103 74 75

E 625 598 529 501 422 324 186 156 127 90 66 67

F 394 356 392 307 266 220 169 126 103 84 91 71

G 239 253 197 245 199 145 126 93 100 74 67 64

H 190 232 182 169 174 114 90 93 84 73 64 61

The ATP assay plating layout and luminescence readings for the MexTAg 299 166 cell line.

Total volume in each well at reading

is 200μL.

Well contents are abreviated as follows: ATP = Adenosine triphosphate, ATPFW = ATP Free Water, rL/L = reconstituted Luciferase/Luciferin reagent, M = Media, LB = Lysis Buffer, CS = Cell Supernatant, Bleo = Bleomycin, Cis = Cisplatin, Cyc = Cyclophosphamide, Dox = Doxorubicin, Epir = Epirubicin, EtoP = Etoposide Phosphate, Gem = Gemcitabine, Ifos = Ifosfamide, Meth = Methotrexate, Pac = Paclitaxel, Peme = Pemetrexed, Vinc = Vincristine.

=

Luminescence readings from the PerkinElmer Victor2 V 1420 multilabel counter using the built-in Wallac software. Readings are in counts per second (CPS) with readings for each well set at 1 second. The plate is Corning Incorporated's Costar 3610: 96-well assay plate, white, clear bottom with lid, tissue culture treated and made of polystyrene.

rL/L + LB + M + EtoP + CS

rL/L + LB + M + Bleo

Drug control and drug test wells in triplicate. =

Negative controls for rL/L and cells.

rL/L + LB + M + Cis

rL/L + LB + M + Cyc

rL/L + LB + M + Dox

rL/L + LB + M + Epir

rL/L + LB + M + EtoP

ATP Standard. Dilutions in ATPFW, 10 fold serial dilutions. Additionally, 50μL of rL/L and 50μL of LB are in each well. ATP Molar concentration is indicated. Missing a negative control well of zero ATP.

=

rL/L + LB + M + Gem + CS

rL/L + LB + M + Ifos + CS

rL/L + LB + M + Meth + CS

rL/L + LB + M + Gem

rL/L + LB + M + Ifos

rL/L + LB + M + Meth

rL/L + LB + M rL/L + LB + M + CS ATPFW + LB + M + CS ATPFW + LB + M

A

rL/L + LB + M + Pac + CS

rL/L + LB + M + Peme + CS

rL/L + LB + M + Vinc + CS

rL/L + LB + M + Pac

rL/L + LB + M + Peme

rL/L + LB + M + Vinc

rL/L + LB + M + Bleo + CS

rL/L + LB + M + Cis + CS

rL/L + LB + M + Cyc + CS

rL/L + LB + M + Dox + CS

rL/L + LB + M + Epir + CS

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The readings were performed without mixing of the well contents. The RLU units

were defined as CPS 200 mL-1. Figure 6.2 shows an attempt was made to fit a model

to the curve. An exact fit was yet to be found but the Weibull model, in the red line,

was the best so far. The luminescent readings were also plotted (Figure 6.3). The

legend and the combinations of tests in the ATP assay for Figure 6.3 has been

magnified in Figure 6.4 for ease of reference. The plot has shown that a signal was

established using the recommended kit for the PerkinElmer Victor2 V 1420

multilabel counter available in the lab.

Figure 6.2. The standard curve from a 48 hr test of the ATPlite kit from

PerkinElmer against the MexTAg 299 166. The luminescence reading is as

expected with the exception of the slight dip at the highest concentration for both

curves. The reading was performed without mixing of the well contents.

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Figure 6.3. The ATP assay graphed results for the MexTAg 299 166 cell line.

The luminescence readings showing a decreasing gradient of luminescent signal

from wells that were closest to the positive ATP control. Cross-talk has been

suspected here.

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Figure 6.4. The legend for the ATP bar graph. The legend for all 4 bar graph plots

has been magnified here. The greyed out boxes from left to right represent the

position of the tests conducted for each ATP assay graphed results for the MexTAg

299 166, MexTAg 299 208, wild-type AE 17 and wild-type BM 164 cell lines. The

grey boxes represent the label position along the x-axis of the plot from left to right.

LEGENDATPFW = ATP Free Water rL/L = reconstituted Luciferase/Luciferin reagent M = MediaNCD = No chemotherapeutic drug - Media top upNCL = No cell lineWCL = With cell line LB = Lysis BufferCS = Cell SupernatantBleo = BleomycinCis = CisplatinCyc = CyclophosphamideDox = DoxorubicinEpir = EpirubicinEtoP = Etoposide PhosphateGem = GemcitabineIfos = IfosfamideMeth = MethotrexatePac = PaclitaxelPeme = PemetrexedVinc = Vincristine

LB + ATPFW + M + NCD + NCLLB + rL/L + M + NCD + NCLLB + rL/L + M + NCD + WCLLB + rL/L + M + Bleo + NCLLB + rL/L + M + Bleo + WCLLB + rL/L + M + Cis + NCLLB + rL/L + M + Cis + WCLLB + rL/L + M + Cyc + NCLLB + rL/L + M + Cyc + WCLLB + rL/L + M + Dox + NCLLB + rL/L + M + Dox + WCLLB + rL/L + M + Epir + NCLLB + rL/L + M + Epir + WCLLB + rL/L + M + EtoP + NCLLB + rL/L + M + EtoP + WCLLB + rL/L + M + Gem + NCLLB + rL/L + M + Gem + WCLLB + rL/L + M + Ifos + NCLLB + rL/L + M + Ifos + WCLLB + rL/L + M + Meth + NCLLB + rL/L + M + Meth + WCLLB + rL/L + M + Pac + NCLLB + rL/L + M + Pac + WCLLB + rL/L + M + Peme + NCLLB + rL/L + M + Peme + WCLLB + rL/L + M + Vinc + NCLLB + rL/L + M + Vinc + WCL

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6.2.0 A summary of ATP assay issues and results

With reference to the results for MexTAg 299 166 (Figure 6.3), there were a few

things to note. The wells without cells and without the rL/L were expected to have

the lowest luminescent signal over the entire plate. This premise held true. The

lowest signal should ideally have come from a well without ATP, ie ATP free water

with LB and rL/L only. However, this negative control was missing from the design

of this plate so the next best negative control has come from the wells without cells

and without rL/L.

The highest concentration of the ATP standard was expected to have the highest

luminescent signal. This premise held true. The highest luminescent signal had a

value of 1449960 cps and the lowest value was 61 cps yielding a range of

1449899 cps. The range of values between the control groups of with or without

rL/L was 232cps - 61cps = 171 cps. Despite the fact that one lot was showing a

higher signal than the other lot, it was important to note that a difference of 171 cps

was small compared to what the difference could be on a larger scale as the range in

luminescence on the plate was 1449899 cps.

The drug control wells were expected to have similar luminescent signal levels as

that of the control wells without cells but with rL/L. Half the tests indicated this and

the other half did not. Only half the tests indicated that the drug controls had a

similar luminescent signal when compared to the wells without cells. These were

wells B10-G12, the half that is furthest away from the wells expected to give out the

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highest luminescent signal. The other half of the tests have unexpected results. The

wells B1-G3 showed a gradient luminescent signal that was decreasing down the

plate away from the wells that produce the highest luminescent signal. Suspect

crosstalk was the reason for the unexpected result in one half of the tests. Cross talk

(Berthold, Herick & Siewe 2000) may have been affecting the signals of the controls

hence a repeat of this ATP assay would need a better plate design.

The standard curve turned out as expected with the exception of the slight dip at the

highest concentration. This may have been due to pipetting error or some other

factor. This data is inconclusive in terms of providing the relationship between ATP

levels and the mode of cell death due to chemotherapy but at least a signal was

obtained from the kit and the machine.

The ATP assay would need to be repeated with the changes for a better design:

Plating with gaps in between the samples being tested if retesting is to be done in the

same white 96-well plate, possible use of black 96-well plates to absorb the light and

therefore reducing the cross-talk, lowering the range of ATP Standard as the sample

test readings do not reach anywhere near the highest 3 luminescent readings and

finally, the inclusion of a negative control with no ATP.

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6.3.0 Establishing a reading for caspase luminescence

As in the ATP assay, it was important to establish a luminescent reading from the

equipment available for testing. The MexTAg 299 208 cell line was used to plate out

the cells in a Corning Incorporated Costar 3610 96-well white assay treated culture

plate with clear bottom test plate. The chemotherapeutic drugs were made up to the

IC50 concentration for the MexTAg 299 208 cell line and then added to the cells in

their corresponding wells which was a mistake in that the drugs should have been

made up to double the concentration of the IC50 determined in the MTT assay as it

was diluted when 0.100 mL of the drug was added to 0.100 mL of the contents of

each well. This fact was noticed after the cells had been incubating with the drugs for

48 hrs. Additionally, there was not enough of the caspase reagent available for this

test so the caspase reagent was not added to all the wells. The kit was used as per the

manufacturer’s instructions.

Luminescence readings were made from the same plate at 4 different incubation time

points to empirically determine what time the reading was best obtained: 1 hr,

1.5 hrs, 2 hrs and 2.5 hrs. After a reading the plate was removed from the machine

and covered in aluminium foil at room temperature. The relative light unit (RLU)

was defined as CPS 100 mL-1 for plotting (Figure 6.5).

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Figure 6.5: The decreasing output from a Wallac 1420 manager program on

PerkinElmer’s Victor2 V multilabel counter for caspase experiment 1 shown for

1 hr and 1.5 hr time points. The readings were done after incubation with the

Promega Caspase Glo 3/7 reagent. RLU = CPS 100 mL-1. The luminescent signal

detected was decreasing as the plate was left to incubate for longer than 1 hr.

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6.4.0 Caspase signal detected

In a plot of each time point reading it could be seen that as time progressed, the

luminescent signal had decreased (Figure 6.4 and Figure 6.5). At first glance, there

appeared to be caspase activity in all the wells as there was a luminescent signal

from each sample group. However, the control wells without cells appeared to have a

very small luminescent signal when they were not expected to. This may be

attributed to background luminescence from crosstalk between the wells, which in

this case, was very small. These were therefore used as blanks.

As expected, because there were no cells for the caspase pathway to be triggered, the

media only and drug in media controls had the lowest luminescent signals. This

suggested that there is no need to control for drug in media only especially when the

media only negative control will suffice. The caspase activity in the “Cells +

Cyclophosphamide” and “Cells + Vincristine” sample groups were lower than that of

the “Control – Media and Cells”. This was unexpected as the two drugs should have

caused more death of the cells in these wells.

These tests would definitely need to be repeated. There was error in the planning of

the plate as only half the IC50 concentration was used to treat the cells. Additionally,

the media and cell controls had a higher than expected luminescence. It should have

been near the luminescence value of the “Control - Media Only” because the cells

were not treated with any drug. The cells in the wells may have become over

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confluent and this would add variability in the luminescent signal. Unfortunately,

pictures were not taken of the wells in the plate when the plate was removed from

the incubator just prior to the Promega Caspase Glo 3/7 reagent being added. If over

confluence is a factor then the doubling time from proliferation data would need to

be looked at for making sure that the plating density for this particular cell line is

appropriate in future experiments.

The first experiment established that it was possible to obtain a luminescent signal. It

also established that the 1 hr time point was a good point for reading the luminescent

signal. However, the data was inconclusive in terms of what it could reveal about the

caspase activity at the 48hr time point when the cell line was treated with

chemotherapeutic drugs at the IC50 concentration.

6.5.0 High purity in the extraction of DNA

Four early passages of cell lines were cultured and frozen down into pellets until all

were ready for DNA extraction. The Qiagen DNeasy Blood & Tissue kit, used

according to the manufacturer’s instructions, yielded high purity of DNA

(Desjardins & Conklin 2001) allowing for further CGH array and exome sequencing

analysis (Figure 6.6). High purity of DNA was obtained from using the Qiagen kit in

the first try that allowed for the samples to be sent off for CGH array analysis. Other

cell lines have already been sent off for CGH array analysis and exome sequencing.

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Figure 6.6. The purity of DNA extracted. The DNA of the wild-type AE 3,

MexTAg 299 177, MexTAg 299 69 and MexTAg 299 21 at low passages were

extracted with high purity according to their 260/280 and 260/230 ratios.

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Chapter Seven:

Discussion

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7.0.0 Discussion

The prognosis for patients with MM is very poor, and as for most cancers, patient

response to therapy is highly variable (Musk et al. 2011). This preliminary project is

aimed at investigating this by establishing the degree of heterogeneity amongst

mesothelioma cell lines which have all arisen due to asbestos exposure. Despite the

use of the same carcinogen, a number of variabilities amongst these cancers are still

present.

Could the poor prognosis for patients with MM be due to how it is acquired?

Although there are some other risk associations with mesothelioma development,

asbestos exposure remains the main cause. There is such a strong link between

asbestos exposure and mesothelioma, that in this respect, the initial development of

this cancer could be thought of as quite homogenous. In this respect, this study

mimics that, as all of the lines arose due to asbestos exposure.

Could the poor prognosis come from a problem with finding and identifying the

disease?

MM is usually diagnosed at late stage. Patients present with respiratory problems

that could be assigned to other conditions before MM is brought up for

consideration. There is currently a variety in the diagnostic aids employed. MM cells

are currently isolated from a population of cells by identifying unique exclusion,

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CEA marker (Ordóñez 2003), and inclusion, overexpressed and hypoglycosylated

MUC1/EMA (Creaney et al. 2008) markers expressed by cells. CT, PET and

ultrasounds are other tools used to aid in diagnosis (Stigt, Boers & Groen 2012). The

body itself has its own immune system for identifying and killing off tumour cells

that is present in patients to do work even before the patient seeks help from a

physician (Mossman et al. 2013). A range of diagnosis tools are available but there is

room for improvements in this area as a long and variable latency period is still

present for patients with MM.

Is the variability in growth of MM in patients due to the MM cells being so different?

This is most likely as mesothelial cells already look different under a microscope

(Raja, Murthy & Mason 2011) and their phenotype changes depending on their

environment such that pathological process are affected (Mutsaers 2004). MMs in

culture have been observed to adopt a more fibroblast phenotype after many

passages (Mutsaers 2004). The prognosis is worse for patients with sarcomatoid

mesotheliomas than those with epitheloid meotheliomas (Grigoriu et al 2007). We

have shown variable growth rates for cell lines established from inbred, thus

homogenous, mice all exposed to the exact same amount of asbestos, and all housed

in the same environment and fed the same food. In humans the diversity is likely to

be much more extensive. In this respect, it is easier to initially investigate the link

between the response of cell lines to chemotherapy, and their genetic makeup, by

first removing confounding factors, such as smoking, age, diet, viral infection and

genetic variation between populations.

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Different confluence rates of cells growing in culture flasks were clearly observed by

examination under a microscope. This was the first indication that the cell lines had

significantly different rates of proliferation. Growth was measured quantitatively

using the Cellavista. Analysis of the data showed that heterogeneity in the growth

rate of the 14 mesothelioma cell lines was observed. The result that the MexTAg cell

lines were slower to proliferate than the wild-type cell lines was surprising. The

MexTAg cell lines all expressed high levels of the well-studied SV40 T-antigen,

which has direct interactions with the tumour suppressors p53 and RB such that the

tumour suppressors are unable to arrest the cell cycle. This may indicate that once a

tumour has established its growth, the existence of certain oncogenes is irrelevant,

and other subsequent mutations become the key regulatory factors.

Is the variability in growth of MM in patients due to the difference in the patient’s

immune system for killing off tumour cells?

Animal models are used for studying disease in a controlled environment

(Kane 2006). Laboratory mice are highly in bred and thus have a very similar gene

pool and an intact immune system. Although the mesothelioma cell lines grew at

different rates in vivo, the differences in growth rates did not reach statistical

significance amongst wild-type BM 164, MexTAg 299 166, MexTAg 299 208 and

MexTAg 299 62 to indicate that there was no heterogeneity in the growth of

mesothelioma cell lines in vivo. This is different to the result found in the

proliferation assay where the cell lines were growing almost uninhibited by things

such as an intact immune system. What it does demonstrate is that when equal

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quantities of the same batch of cells are injected into in bred mice, tumour

development is variable between individual mice.

The wild-type BM 164 cell line grows faster in vivo than the MexTAg 299 166 and

MexTAg 299 62 cell lines. This correlates with the proliferation assay data. For the

majority, MexTAgs are ranked slower to grow in vitro and in vivo. Perhaps it is

worth comparing the data for these 4 cell lines, at least, with viability data when they

are subjected to treatment.

Is the treatment being used for MM the right one?

There is a variety of treatments used for MM with different levels of success in the

goal to cure patients of the disease: surgery, radiotherapy, immunotherapy,

chemotherapy (Grégoire 2010), photodynamic therapy (Friedberg 2012) and gene

therapy (Vachani, Moon & Albelda 2011), either alone or in a multimodal fashion

(Liu et al. 2010). The classic treatment of MM is through chemotherapy, each one

with a specific mode of action to target different ways in which MM can develop and

progress in the patient. When used as a single agent, some patients do respond and

some do not (Tomek & Manegold 2004). It would be good to figure out if there

really is a difference in the growth of MM cells when treated with single agent

chemotherapies and which ones they respond to best.

Overall, there was heterogeneity in the response to chemotherapy amongst the 7

wild-type and 7 MexTAg cell lines treated against each of the 12 chemotherapeutic

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drugs. In these responses, 6 out 12 were very significant and 6 out of 12 were

extremely significant. Multiple comparisons yielded statistically significant

differences in responses to chemotherapy between pairs of cell lines in 11 out of 12

chemotherapeutic drugs tested. The largest difference in response overall exists

against Pemetrexed between wild-type AE 16 and MexTAg 299 166

(7.90x106 ng/mL) whilst the smallest difference in response is between wild-type AE

3 and wild-type AE16 against Gemcitabine (1.16x101 ng/mL). Based on the overall

largest and smallest differences one cannot say that good and poor responders exist

because we need to still correlate the in vitro and in vivo data that still requires

validating despite care being taken to try and keep conditions as consistent as

possible in the MTT assay. The data requires validation from a more sensitive assay

or a number of other assays combined. The MTT assay does have the advantage that

it is relatively cheap to conduct per experiment for preliminary investigation.

What is causing the difference in response to treatments?

The MTT data needs to be verified. The phenotypes are different and a difference in

phenotype relates to genetic differences. We know for one that the MexTAgs are

different to the wild-types because of the insertion of 100 copies of the SV40

T-antigen gene, but there was variability in the response to chemotherapy between

the MexTAg cell lines as well. This suggests that other events on top of the inserted

SV40 T-antigen gene are more important in determining response to chemotherapy.

The answer may lie in the genome.

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As a cancer, MM exhibits the hallmarks of a cancer: sustaining proliferative

signalling, evading growth suppressors, activating invasion and metastasis, enabling

replicative immortality, inducing angiogenesis and resisting cell death

(Hanahan & Weinberg 2011). MM cells acquire traits in a stepwise fashion towards

tumorigenesis (Mossman et al. 2013). Perhaps asbestos could be the first ‘trigger’.

Then, together with the presence of the SV40 T-antigen, tumour development is

accelerated which may be why in MexTAg mice we see a faster rate of disease

development after asbestos exposure compared to in wild-type mice. It may explain

why there is also 100% incidence in MexTAg mice compared to 20-30% incidence

in wild-type mice (Robinson et al. 2006). In this case, the second step towards

tumorigenesis could be the SV40 T-antigen, but not so in wild-type mice. There are

clearly other steps occurring in the wild-types for tumour progression to be

established.

The ATP assay and the caspase assays are still in the process of being optimised but

detecting a signal for both is possible with the equipment available. The DNA

extraction had high purity and is currently undergoing CGH array analysis. Some of

the lines are also being exome sequenced. Hence a follow up study is to see what the

differences are, if any, between the cell lines and if there are any correlations

between drug response or growth rate in vitro or in vivo. Maybe cell lines that

respond to a particular drug by the same magnitude of diversity may have the same

gene variant in common.

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Concluding remarks on the project:

More time is needed to optimise each of the assays conducted in this study. The

results are approximate at best. The proliferation assay can be repeated given time to

more accurately plate out the cells on separate plates. The doubling times are

approximate because the removing of cells from the incubator and exposure to light

may have an effect on their growth. If images were taken where the cells were

growing, the confounding factor of a change in environmental condition would be

removed. Still, patterns of fast and slow growing cell lines can be observed, how

much of that difference is due to confounding factors is yet to be known. The

confounding factors in the mice experiments also need to be sorted out before “cause

and effect” can be implied from the results. Practically, differences in growth can be

seen between cell lines in vitro and in vivo but more accurate data needs to be

analysed for the statistics to fully correlate heterogeneity in growth.

Validating the results of the MTT assay is a priority as the rest of the work is

dependent on the fact the heterogeneity in response to chemotherapy was found.

Against Pemetrexed, MexTAg 299 166 is about 556,000 times more sensitive than

wild-type AE 16. In 4 out of 12 chemotherapeutic drugs, wild-type BM 164 is least

responsive to chemotherapy, followed by wild-type AE 16 in 3 out of 12

chemotherapeutic drugs. The most responsive cell line is MexTAg 299 170 in 3 out

of 12 drugs. If the findings of differences in the phenotype of the cells were accurate

it could lead to individualised therapeutic strategies to target the uniqueness of MM.

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Appendix I: List of tables

Table 1.1 Chemotherapeutic and anti-proliferative drugs. 22

Table 2.1 The cell lines used in this project. 42

Table 2.2 Statistical significance levels. 45

Table 2.3 The chemotherapeutic drugs supplied by Sir Charles Gardiner

Hospital Pharmacy.

48

Table 3.1 Inclusions and exclusions in the determination of doubling times. 63

Table 3.2 The diversity in proliferation rates of 14 mesothelioma cell lines. 66

Table 4.1 The fastest and slowest growing cell lines in vitro and in vivo. 72

Table 5.1 The triplicate well OD readings for experimental repeat

number 1 of the MexTAg 299 208 treated with Gemcitabine.

78

Table 5.2 The triplicate well OD readings for experimental repeat

number 2 of the MexTAg 299 208 treated with Gemcitabine

79

Table 5.3 The triplicate well OD readings for experimental repeat

number 3 of the MexTAg 299 208 treated with Gemcitabine.

80

Table 5.4 The IC50 replicates for the MexTAg 299 208 cell line. 83

Table 5.5 The IC50 summary table for all the wild-type cell lines. 84

Table 5.6 The IC50 summary table for all the MexTAg cell lines. 84

Table 5.7 The level of difference in response to chemotherapy by the

wild-type cell lines.

86

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Appendix II: List of figures

Figure 1.1 Malignant mesothelioma in a bisected lung. 8

Figure 1.2 Wittenoom Asbestos Mine in Western Australia. 9

Figure 1.3 Ultrasound guided biopsy of malignant pleural mesothelioma. 9

Figure 1.4 Suicide gene therapy. 13

Figure 1.5 Cell lines grown in culture. 24

Figure 1.6 Formazan crystals formed in cells. 27

Figure 1.7 Fluorescence-activated cell sorter (FACS). 28

Figure 1.8 Creation of a transgenic mouse. 32

Figure 1.9 Layers of the skin. 33

Figure 1.10 CGH Array. 38

Figure 2.1 Cell viability plots in the determination of the IC50 in the MTT

assay.

52

Figure 3.1 The wild-type AE 17 and BM 163 cell line grown in culture. 61

Figure 3.2 The proliferation time determined at 40% and 80% confluence

for the wild-type AE 17 cell line at a plating density of

5.00 x 103 cells/well .

62

Figure 3.3 The proliferation rates of wild-type and MexTAg mesothelioma

cell lines are heterogeneic.

66

Figure 4.1 In vivo growth of MexTAg 299 166. 68

Figure 4.2 The exponential and linear growths. 70

Figure 4.3 The excluded growth curves from analysis. 71

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Figure 5.1 Triplicate IC50 values determined from plots of MexTAg 299 208

treated with Gemcitabine.

81

Figure 6.1 The ATP assay plate layout of results. 91

Figure 6.2 The standard curve from a 48 hr test of the ATPlite kit from

PerkinElmer against the MexTAg 299 166.

92

Figure 6.3 The ATP assay graphed results for the MexTAg 299 166 cell line. 93

Figure 6.4 The legend for the ATP bar graph. 94

Figure 6.5 The decreasing output from a Wallac 1420 manager program on

PerkinElmer’s Victor2 V multilabel counter for caspase experiment

1 shown for 1 hr and 1.5 hr time points.

98

Figure 6.6 The purity of DNA extracted. 101

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Appendix III: Data analysis

Proliferation and in vivo tumour growth analysis

In the proliferation assay, it was noted that a number of cell lines had entered the

exponential phase of growth at about 40% cell confluence and was still in this

exponential phase at about 80% cell confluence. Hence, 3 time span values were

conveniently interpolated from the 3 lines of best fit for the triplicate wells of each

cell line plating densities at 40% and 80% cell confluence. The doubling time was

obtained from subtracting the time span at 40% cell confluence from the time span at

80% cell confluence. The option to “Interpolate unknowns from standard curve” was

also selected. It was noted that each cell doubling yielded an exponential increase in

numbers and as such the exponential growth equation was used. A “non-linear

regression (curve fit)” analysis using the exponential growth equation was applied to

model the growth of the cell lines from these new time span values. Having worked

out, through a linear model, how long it took for the cell lines in each well to get to

40% and 80% confluence, the doubling time for each well was then determined by

subtracting the time span at 40% cell confluence from the time span at 80% cell

confluence in Graphpad. The proliferation assay was repeated until at least

3 doubling times were obtained for each cell line and statistical analysis performed.

In the proliferation or in vivo growth experiments, where applicable, an approximate

two-tailed p-value was obtained from an unmatched and non-parametric one-way

ANOVA, the Kruskal-Wallis test, for comparing the doubling times between all the

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wild-type, or all the MexTAg, cell lines at the α = 0.05 level of statistical

significance, so that the chance of incorrectly finding a difference in mean doubling

times would occur no more than 5% of the time. The null hypothesis was that the

means were all the same and the alternative hypothesis was that the means were not

all the same. A Dunn’s correction for multiple comparisons was applied and a

two-tailed p-value was obtained for the determination of which pairs of mean

doubling times were significantly different and which pairs were not.

A D’Agostino-Pearson omnibus K2 normality test, offered in Graphpad, was used at

the α = 0.05 level of statistical signifiance to obtain a two-tailed p-value of for the

the combined mean doubling times of the wild-type, or MexTAg, cell lines. The null

hypothesis was that the sample had come from a normally distributed population and

the alternative hypothesis was that the sample did not come from a normally

distributed population. When the test was passed by both the wild-type and MexTAg

cell lines, the Gaussian distribution was assumed. An unpaired parmetric t-test with

Welch’s correction, as it was assumed that the SDs were not equal, was used in the

comparison of their mean doubling times at the α = 0.05 level of statistical

significance.

A Kruskall-Wallis test at an α = 0.05 level of statistical significance was used to

obtain an approximate two-tailed p-value in the comparison of doubling times

amongst all 14 mesothelioma cell lines. A Dunn’s correction for multiple

comparisons was also applied to the data to obtain a two-tailed p-value so that the

pairs of mean doubling times which were significantly different and those pairs that

were not were determined.