Drug Delivery in Cancer - technologies, markets and companies

0. Executive Summary ...... 15

1. Introduction to cancer therapy.......... 17

Molecular biology of cancer........ 17

The genesis of cancer .17

Normal cell cycle and growth .17

Oncogenes .....18

Tumor Suppressor Genes ......18

Role of Bub 1 gene in cell division .........19

Accumulation of random mutations .......20

Chromosomal instability........20

Aneuploidy .....21

Telomeres and cancer...........21

DNA methylation and cancer..22

Anticancer treatments based on RNA regulation of genes .............22

Hallmarks of cancer....22

Self-sufficiency of tumor proliferation .........23

Apoptosis ............23

Therapeutic implications of apoptosis in cancer ............24

Autophagy......25

Induction of angiogenesis...........25

Acquisition of a potential for unlimited replication..............27

Invasion and metastases............27

Cancer biomarkers  28

Molecular imaging of cancer .......28

Cancer genomics... 29

Gene expression profiling in cancer ............29

Cancer proteomics  30

Limitations of genomics and proteomics for understanding cancer..... 30

Cancer microenvironment .......... 30

Role of hypoxia and miRNA in cancer microenvironment ....31

Epidemiology of cancer .............. 31

Current management of cancer.. 32

Chemotherapy .....32

Limitations of cancer chemotherapy.......32

Radiotherapy .......33

Brachytherapy 33

Surgery ..............33

Basics of drug delivery in cancer 33

Historical landmarks in cancer drug delivery .. 34

2. Innovative treatments for cancer ...... 35

Introduction......... 35

Selective estrogen receptor modulators.... 36

Antiangiogenic agents. 37

Development of antiangiogenic therapies ....37

Classification of antiangiogenic agents ........37

Blocking Akt signaling pathway by rapamycin ..............38

Inhibitors of endothelial proliferation .....39

Inducers of apoptosis of endothelial cells of tumor vessels ...........39

Chemotherapy at lower than maximum tolerated dose..39

Monoclonal antibodies with vasculostatic properties......40

Matrix metalloproteinase inhibitors ........41

PPARa agonists .....41

VEGF Trap......42

Agents that decrease the permeability of tumor blood vessels ......42

Antiangiogenic agents in clinical trials ....42

Combination of antiangiogenic with cytotoxic therapy ........43

Bacterial anticancer agents........ 43

Tumor-targeted bacteria ............44

Genetically modified Salmonella typhimurium as anticancer agent.44

TAPET (Tumor Amplified Protein Expression Therapy) ...44

Bacterial protein for targeted delivery of liposomal cancer drugs ...45

Killed but metabolically active (KBMA) bacteria ..45

Bacterial toxins targeted to tumors.............45

Immunotoxins 45

Escherichia Coli toxins ..........46

- 4 -

Engineered anthrax toxin ......46

Recombinant fusion toxins.....47

Type III secretion systems .........48

Induction of apoptosis in cancer by bacterial proteins ........49

Induction of immune response by bacteriolytic therapy......49

Innovations in cell therapy for cancer....... 49

Stem cell transplantation for cancer ...........50

Cancer drug/gene delivery by mesenchymal stem cells......50

Cancer immunotherapy.............. 50

Cytokines...........51

Cancer vaccines ...51

5T4 as a target for cancer immunotherapy ...52

Anti-telomerase vaccine........53

Antigen-specific cancer vaccines. ..........53

Carcinoembryonic antigen-based vaccines....54

Dendritic cells for cancer vaccination .....54

Hybrid cell vaccination ..........55

Lymphocyte-based cancer therapies ......56

Tumor cell vaccines ..............58

Vaccines that simultaneously target different cancer antigens.......59

Concluding remarks about cancer vaccines ..59

Cancer Vaccine Consortium ........59

Innovative methods of radiation delivery . 59

Image-guided ultrasound technology for delivery of radiation ............60

Respiratory gating technology for radiation therapy...........60

Positron therapy ..60

Boron neutron capture therapy ...61

Skeletal Targeted Radiotherapy ..61

Irreversible electroporation....... 62

Methods to overcome multidrug resistance (MDR) ........ 62

P-glycoprotein-mediated MDR.....63

MDR-associated protein gene......63

Strategies for overcoming MDR...63

Blocking the action of P-glycoprotein .....63

Nitric oxide inducers .............64

Managing resistance to antiapoptotic action of anticancer agents ..64

Inhibition of DNA repair ........64

Liposome formulation of drugs..............65

Modification of the chemical structure of the anticancer drug ........65

Enzyme Catalyzed Therapeutic Activation.....65

Modulation of SPARC expression ...........66

Iron chelators that overcomes resistance to chemotherapeutics ....66

Proton pump inhibitors..........66

Combination of targeted drugs with different specificities .............66

Targeted cancer therapies ......... 67

Targeting cellular pathways........67

Targeting antigens in virus-associated cancer ....68

Targeting HAAH for cancer therapy.............68

Targeting mitochondrial membranes...........68

Targeting tumor lymphatics........69

Targeting tyrosine kinase receptors ............70

Inhibitors of bcr-abl tyrosine kinase.......70

Inhibition of multiple tyrosine kinases ....70

Inhibitors of ErbB tyrosine kinase..........71

Targeting the Hedgehog signaling pathway..71

Targeting ras oncogene..............72

Targeting the transferrin receptor-mediated endocytosis pathway ......72

Targeting cancer stem cells ........72

Targeting glycoproteins..............73

Tagging cancer with sugars ...73

Anticancer agents based on glycobiology74

Targeting cell surface glycoproteins .......74

Biofusion for targeted cancer therapy ....74

Targeted drug delivery of anticancer agents with controlled activation 75

Enhancing the effects of radiation and chemotherapy ... 75

Sensitizing agents for chemotherapy ..........75

Tesmilifene for chemosensitization ........75

CoFactor to enhance the efficacy of chemotherapy .......76

Enzyme-enhanced chemotherapy..........76

Sensitizing agents for radiotherapy ............76

IPdR ..............77

- 5 -

Manipulation of tumor oxygenation.............77

Hypoxia-based methods to enhance chemotherapy and radiotherapy ..77

Hyperbaric oxygen and radiation ...........78

HIF-1 antagonists to enhance radiotherapy ..78

Nonsteroidal antiinflammatory drugs enhance tumor radiosensitivity ...78

ONCONASE as radiosensitivity enhancer 79

Hyperthermia and chemotherapy/radiation therapy ...........79

Techniques for hyperthermia .79

Trimodality therapy: radiation, chemotherapy, and hyperthermia..80

Photodynamic therapy 80

Novel anticancer agents............. 82

Anti-EphA2 antibodies.82

Antioxidants ........83

Brostallicin ..........83

Agents disrupting folate metabolism...........83

Pemetrexed....83

Cytotoxic ribonucleases..............84

DNA hypomethylating agents......84

Histone deacetylase inhibitors.....84

HSP90 inhibitors ..85

Ion channel blockers...86

IOT-101.........86

Endovion........86

LPAAT-beta inhibitors .86

PARP inhibitors ....87

Targeted destruction of BRCA2 deficient tumors by PARP inhibitors .....87

Prodrugs .............87

Enzyme-activated prodrugs ...87

Ascorbic acid as a prodrug for cancer.....88

Prolarix ..........88

Protein kinase G activation .........88

Proteasome inhibitors .89

Recombinant human insulin-like growth factor binding protein-3........89

Second generation nucleosides ...90

Targeting topoisomerase IB........90

Therapeutic strategies based on the P53 pathway .............91

Development of targeted anticancer therapies ...91

In vivo models for molecularly anticancer drugs...........91

Checkpoint activation as a strategy against cancer .......92

Deletion-specific targeting for cancer therapy ..............92

Combining novel anticancer approaches......92

Personalized therapy of cancer.. 93

Challenges of cancer classification..............96

Design of future cancer therapies96

Personalized drug development in oncology.97

Role of molecular imaging .....97

Role of molecular imaging in targeted cancer therapy ...98

Screening for personalized anticancer drugs .98

Targeting pathways for personalized cancer therapy .....98

3. Drug delivery systems for cancer .... 101

Introduction....... 101

Routes of drug delivery in cancer............ 101

Intravenous delivery systems for cancer therapy............. 102

Oral delivery of anticancer agents ............ 103

Oral UFT ...... 103

5-FU combined with eniluracil ............. 104

Oral paclitaxel..... 105

Oral fluoropyrimidines......... 105

Oral satraplatin ... 106

Oral PXD101. 106

ARRY-142886 ..... 106

High dose pulse administration of calcitrol .. 106

Transdermal drug delivery........ 106

Delivery of the photosensitizer drug d-amino levulinic acid ......... 107

Transdermal delivery of the methotrexate .. 107

Transdermal delivery of peptide cancer vaccines ........ 107

Intradermal delivery of cancer vaccines by adenoviral vectors .... 108

Pulmonary delivery of anticancer agents ... 108

Regional intra-arterial delivery of chemotherapy ............. 108

Gas embolotherapy of tumors... 109

- 6 -

Drug delivery to lymph nodes ... 109

Intraperitoneal macrophages as drug delivery vehicle...... 110

Challenges of cancer drug delivery ........... 110

Tumor blood vessel pore barrier to drug delivery............. 110

Improvement of drug transport in tumors . 110

Delivery of anticancer drugs to nuclear targets  111

Innovative formulations for drug delivery in cancer .... 112

Cancer targeting with polymeric drugs ...... 112

Linking anticancer drugs to polyglutamate.. 113

Bacterial ghosts as drug delivery systems for anticancer drugs ........ 113

Microparticles as therapeutic delivery systems in cancer .. 114

Subcutaneous injection of microspheres carrying anticancer drugs .... 114

Intravascular delivery systems using microparticles.... 115

Tumor embolization with drug-eluting beads.............. 115

Tumor embolization with radioactive microparticles .... 116

Microparticles heated by magnetic field 116

Magnetic targeted microparticle technology  116

Release of drugs from micelles by ultrasound ............ 116

Release of drugs from biSphere by ultrasound ........... 117

Release of drugs from microcapsules by laser ............ 117

Chemoembolization ............ 118

Anticancer drugs bound to carbon particles  118

Anticancer drugs bound to protein microspheres ........ 118

Nanoerythrosomes ............. 118

Micronized droplets of olive oil ............ 118

Nanobiotechnology-based drug delivery for cancer ..... 119

Nanoparticle formulations for drug delivery in cancer....... 120

Anticancer drug particles incorporated in liposomes.... 120

Bacterial nanoparticles for encapsulation and chemotherapy delivery. 121

Encapsulating drugs in hydrogel nanoparticles ........... 122

Exosomes .... 122

Folate-linked nanoparticles .. 123

Lipid based nanocarriers ..... 123

Micelles for drug delivery in cancer ...... 123

Nanoparticle formulations of paclitaxel. 125

Nanoparticles containing albumin and antisense oligonucleotides  125

Non-aggregating nanoparticles ........... 125

Pegylated nanoliposomal formulation... 126

Perfluorocarbon nanoparticles............. 126

Protosphere nanoparticle technology ... 126

Nanoparticles for targeted delivery of drugs into the cancer cells ..... 127

Antiangiogenic therapy using nanoparticles  128

Carbon magnetic nanoparticles for targeted drug delivery in cancer... 128

Carbon nanotubes for targeted drug delivery to cancer cells ....... 128

Fullerenes for enhancing tumor targeting by antibodies.............. 129

Gold nanoparticles for drug delivery in cancer............ 129

Iron oxide magnetic nanoparticle formulation for drug delivery ... 130

Lipoprotein nanoparticles targeted to cancer-associated receptors..... 130

Magnetic nanoparticles for remote-controlled drug delivery to tumors 131

Nanocell for targeted drug delivery to tumor.............. 131

Nanodroplets for site-specific cancer treatment.......... 132

Polymer nanoparticles for targeted drug delivery in cancer ......... 132

Polymersomes for targeted cancer drug delivery ........ 133

Targeted drug delivery with nanoparticle-aptamer bioconjugates  133

Dendrimers for anticancer drug delivery.... 134

Application of dendrimers in boron neutron capture therapy ....... 135

Application of dendrimers in photodynamic therapy .... 135

Dendrimer-based synthetic vector for targeted cancer gene therapy.. 136

Devices for nanotechnology-based cancer therapy........... 136

Convection-enhanced delivery with nanoliposomal CPT-11 ......... 136

Nanocomposite devices....... 136

Nanoengineered silicon for brachytherapy .. 137

Nanoparticles combined with physical agents for tumor ablation ...... 137

Carbon nanotubes for laser-induced cancer destruction.............. 137

Nanoparticles and thermal ablation...... 137

Nanoparticles combined with ultrasound radiation of tumors....... 138

Nanoparticles as adjuncts to photodynamic therapy of cancer..... 138

Nanoparticles for boron neutron capture therapy........ 139

RNA nanotechnology for delivery of cancer therapeutics... 139

Nanocarriers for simultaneous delivery of multiple anticancer agents 140

Combination of diagnostics and therapeutics for cancer ... 140

- 7 -

Biomimetic nanoparticles targeted to tumors ............. 140

Dendrimer nanoparticles for targeting and imaging tumors......... 140

Gold nanorods for diagnosis plus photothermal therapy of cancer 141

Magnetic nanoparticles for imaging as well as therapy of cancer . 141

pHLIP nanotechnology for detection and targeted therapy of cancer .. 142

Radiolabeled carbon nanotubes for tumor imaging and targeting. 142

Targeted therapy with magnetic nanomaterials guided by antibodies . 142

Ultrasonic tumor imaging and targeted chemotherapy by nanobubbles ............. 142

Polyethylene glycol technology.. 143

Enzon's PEG technology ...... 143

Debiopharm's PEG biconjugate drug delivery platform  143

Nektar PEGylation .............. 144

PEG Intron ... 144

Single-chain antibody-binding protein technology............ 145

Liposomes for anticancer drug delivery ..... 145

AlZA’s Stealth liposomes ..... 147

SkyePharma's DepoFoam .... 147

Hyperthermia and liposomal drug delivery.. 147

Tumor-selective targeted drug delivery via folate-PEG liposomes  148

Boron-containing liposomes. 148

Liposomal doxorubicin formulation with N-octanoyl-glucosylceramide  148

Non-pegilated liposomal doxorubicin.... 149

Emulsion formulations of anticancer drugs ... 149

Albumin-based drug carriers.... 149

Anticancer drugs that bi