PHAR1101 Lecture Notes - Lecture 18: Omics Publishing Group, Metabolomics, Transcriptomics Technologies

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25 May 2018
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The Future of Pharmaceutical Innovation:
Significant achievements of the modern pharmaceutical innovation sector:
20th century saw improvements in number and effectiveness of medicines
- i.e. cancers, viral, bacterial, cardiovascular disease etc.
Better awareness of drug toxicity and how to diminish it
Better knowledge of how drugs work, what happens in the body etc.
Developments in chemistry provided more molecules for drug testing.
Finding new drugs steadily became more challenging, complex and costly.
Post 1990s, big improvements in understanding human disease (i.e. genes, cell pathways etc.)
Powerful new OMIC“ tehnologies (measure tens of thousands of genes, proteins or metabolites in
body samples).
- big pharma an early adopter of these methods, to better find new drug targets (receptors)
Growth of government regulation made drug approval more complicated
The OMICS era (21st century medical research).
Genomics - genes
Transcriptomics message RNA
Proteomics proteins
Metabolomics metabolites
Bioinformatics data analysis
Needle in a haystack problem:
- masses of detailed experimental data very easy to find lots of disease-associated protein
changes
- uncertain over which proteins will make best drug targets
- huge cost associated with poor target choices.
Eroom’s Law – the number of new drugs approved by the FDA per billion US dollars (inflation adjusted)
spent on research and development has halved roughly every 9 years.
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Development phase has gotten more expensive.
Most classic drugs absorbed in GI tract and are small
molecules:
Older vs newer concepts of drug.
Examples of biopharmaceuticals produced by the biotechnology industry (1990s and beyond)
Therapeutic proteins:
- growth factors and cytokines
- haemopoietic factors (stimulate white blood cell growth after chemo)
- erythropoietin (EPO, stimulates red blood cell growth)
- hormones (i.e. insulin, human growth hormone)
- coagulation factors and antithrombotic factors
Therapeutic Antibodies (i.e. remicade)
Antisense nucleic acids (i.e. fomiversen)
Areas of medicine where biologics have had key impact:
- Hormone deficiencies
- Blood tumours
- Autoimmune disease
- Solid tumours
Two classic problems with protein based drugs:
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Document Summary

Significant achievements of the modern pharmaceutical innovation sector: 20th century saw improvements in number and effectiveness of medicines i. e. cancers, viral, bacterial, cardiovascular disease etc. Better awareness of drug toxicity and how to diminish it. Better knowledge of how drugs work, what happens in the body etc. Developments in chemistry provided more molecules for drug testing. Finding new drugs steadily became more challenging, complex and costly. Post 1990s, big improvements in understanding human disease (i. e. genes, cell pathways etc. ) Powerful new (cid:862)omic (cid:863) te(cid:272)hnologies (measure tens of thousands of genes, proteins or metabolites in body samples). Big pharma an early adopter of these methods, to better find new drug targets (receptors) Growth of government regulation made drug approval more complicated. Masses of detailed experimental data very easy to find lots of disease-associated protein changes. Uncertain over which proteins will make best drug targets. Huge cost associated with poor target choices.

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