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How Medicines Are Born: The Imperfect Science Of Drugs

How Medicines Are Born: The Imperfect Science Of Drugs

Vozza Lisa | D'incalci Maurizio | Gescher Andreas

(2017)

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Book Details

Table of Contents

Section Title Page Action Price
Contents xiii
Foreword — Improving the Culture of Medicine v
About the Authors vii
Precautions and Warnings before Using this Book xi
Chapter 1 To Cure, Heal or Repair 1
Unresolved Medical Problems 2
Drugs which Cure 2
Drugs which Can Heal 5
From Animal Insulin to the Biotechnologically Generated Hormone 6
“Anything but Chemistry!” 8
The Inheritance of the Willow Tree 9
From Aspirin to Baby Aspirin 10
Drugs which Break Up Preformed Blood Clots 11
When Serendipity Leads to New Drugs 12
Drugs Discovered to a Plan 13
Towards Drugs which Can Repair? 19
Chapter 2 Hunting for Drugs 21
The Human Body — Is there an Instruction Manual? 22
The Cell — A Great Chemical Laboratory 23
The Most Common Target of Drugs 25
Catalogues of Genes and Proteins 25
Towards the Molecular Causes of Diseases 27
Is this Target “Druggable”? 30
Screening of Chemicals 30
Biologicals 33
Molecular Targeted Drugs 36
Fashion, Advertisement and some Thoughts 37
How to Overwhelm Barriers against Poisons 38
Poison or Remedy? Depends on the Dose 39
Routes and Formulations 43
And now — Do we Have a New Drug? 45
Chapter 3 Laboratory Studies 47
We Are Not All the Same — And Mostly Do Not Know Why That Is 47
Experiments Using Cells 48
Why Are Experiments in Animals Necessary to Evaluate a Drug? 50
A Model for (Almost) any Disease 52
Toxic or Harmless? 54
How the Organism Modifies a Drug: Pharmacokinetics 54
How the Drug Alters the Organism: Pharmacodynamics 55
The Therapeutic Index 56
Towards the First Tests in Humans: Does the Traffic Light Display Green, Red or Yellow? 56
Chapter 4 First Time in Humans 59
The First Controlled Clinical Trial 60
The Participants 62
Informed Consent 63
Randomised, Controlled, Blinded — Three Fundamental Rules of a Rigorous Clinical Study 64
Placebo and Nocebo Effects 66
The Four Phases of a Clinical Study 67
Phase one 68
The initial dose is small 71
A phase one trial is not necessarily the last resort 72
Towards phase two: Drugs which just make it 74
Phase two 75
Phase three 76
Endpoints 77
Not Inferior Drugs 78
“One Can’t Predict the Weather...” 79
Chapter 5 Only Probabilities, Never Certainties 81
Measurements are Never without Errors 82
Is the Study Sufficiently Powered? 83
Is the Result a False Negative? 84
Is the Result a False Positive? 85
Can We Exclude Chance? 85
Cause and Effect or Correlation? 86
Rare Diseases 88
Extrapolation can be Dangerous! 89
Chapter 6 Approval or Rejection 91
Who Does Approve New Drugs? 92
Stories of Rejections 94
Which Criteria are Important for Drug Approval? 96
Good Practices 96
Has a New Drug Got to Be Innovative? 97
Transparency: Needs to be Improved 98
Unpublished Studies and a Treatment for Depression 99
The Patent — Branded and Generic Drugs 101
Are Generic Drugs Trustworthy? 101
What are Biosimilar Drugs? 103
Shapes and Colours 104
Finally in the Pharmacy! 105
Chapter 7 Vigilance, Revisions, New Indications 107
Pharmacovigilance Can Dig Up Dishonesty 108
Pharmacovigilance Can Improve Therapy 109
Pharmacovigilance Can Discover Resistance 109
Indications Beyond Those in the Patient Information Leaflet 112
Drugs Against Non-existent Diseases 114
Drug Pipelines 115
Chapter 8 Predicting the Drugs of the Future 119
Nanovehicles for Drugs 120
Selecting the Right Patients for the Right Therapy 122
New Indications for Old Drugs 123
Biobanks — Sources of Priceless Treasures 126
Digesting Big Data Sets 129
Simulations and Imaging 131
Drugs Which Repair Rather Than Treat 131
Dreaming of the Molecular Instruments of Tomorrow 134
Who Will Invent the Drugs of Tomorrow? 135
Chapter 9 Not Every Pill is a Proper Drug 137
Imagining a World without Drugs 139
Pills Which Are not Drugs 140
The Odd World of Homoeopathy 142
Be Careful! 144
If You Want to Know More… 145
Books 145
Websites and Articles 145
Myths to be Dispelled 147
1. A natural substance is less toxic than a chemically synthesised one 147
2. Each pill is a proper drug 147
3. Homoeopathic remedies may be less efficacious than traditional drugs but at least they do not cause harm 148
4. Drug experimentation using animals is a waste of time 148
5. We know the mechanisms of action of all drugs 149
6. Drug effects are the same in all persons who take them 149
7. The human body is like a machine and we know exactly how it functions 149
8. Molecular targeted drugs are better than traditional drugs 149
9. Biological drugs are better than chemically synthesised ones 150
10. Poisons and drugs are different from each other 150
11. A new drug against a particular disease is always better than an old one 150
12. Results of studies of new drugs constitute absolute certainties 151
13. Drug safety is always advantageous for the patient 151
14. Generic drugs are worse than branded ones 151
15. Identification of a gene facilitates the understanding of its role in a disease and engenders the discovery of a new drug 152
16. A tablet is always better than an injection 152
Did You Know That… 153
Many drugs treat, a few cure, almost none repairs 153
Insulin is generated by a genetically modified organism 153
The ancient legacy of the willow tree 153
“Anything but chemistry” 154
Baby aspirin 154
The effects of viagra were discovered by accident 154
Erythropoietin (EPO) is perhaps the best known biological drug 154
In nature, poisons are the norm rather than the exception 155
The first clinical trial 155
Participants in clinical trials are always volunteers 156
The placebo effect 156
The nocebo effect 157
The reality about some novel anticancer drugs 157
Recruitment of volunteers is difficult 158
Many new drug dossiers are more voluminous than an encyclopaedia 158
New drug approval in Europe 158
Thalidomide: disaster and rebirth 159
To what extent are results of clinical studies made public? 159
Do drugs against depression act like a placebo? 160
Individual preferences of medicines 160
Only one in 60,000 experimental drug molecules goes on to obtain regulatory approval 160
Pharmacovigilance can improve therapy 161
Any drug targeted against a microorganism is destined to fail 161
Many drugs are prescribed for off-label indications, for purposes not listed in the information leaflet 162
The medical world pre-anaesthesia was brutal 162
A healthy life style is always better than any pill 163
Index 165