Prime minister’s science, engineering and innovation council sixth meeting 30 november 2000



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Molecular medicine



PRIME MINISTER’S SCIENCE, ENGINEERING
AND INNOVATION COUNCIL


SIXTH MEETING - 30 NOVEMBER 2000

MOLECULAR MEDICINE

A paper prepared by an independent working group:


Professor Nicholas Saunders PMSEIC Council member (Chair)

Professor Sue Serjeantson PMSEIC Council member (Deputy Chair)

Sister Regis Dunne Mater Medical Research Institute

Dr Alan Finkel Axon Instruments

Dr Doug Hilton Walter & Eliza Hall Institute of Medical Research

Professor John Mattick University of Queensland

Professor Loane Skene Melbourne University

Professor Ron Trent Royal Prince Alfred Hospital

Professor Alan Trounson Monash University

Professor John White Australian Academy of Science


Terms of Reference:




  • What are the emerging research areas / enabling technologies that will revolutionise medicine?

  • Explain the science of the Human Genome Project, and Therapeutic Stem Cells.

  • Provide examples of therapeutic applications from these areas.

  • Discuss commercialisation of relevant applications.

  • Flag the public policy (ethics) issues that are under consideration.


CONTENT



EXECUTIVE SUMMARY……………………………………………………….. 3

INTRODUCTION………………………………………………………………….6




GENERATING IDEAS…………………………………………………………….6




The Science of the Human Genome 6

The Science of Stem Cells 8


Impact on Clinical Medicine 10
The Future 13

ACTING ON IDEAS………………………………………………………………14

Molecular Medicine in the Australian Economy 14


Australian Molecular Medicine in the Global Market 16

CREATING AN IDEAS CULTURE……………………………………………..18

Molecular Medicine in the Community 18


Ensuring Effective Regulation 20

THE WAY FORWARD - To Health and Wealth………………………………. 22



APPENDICES……………………………………………………………………...25

1 Ethics and Safety of Research in Humans 25

2 Approaches to Gene Testing 26


  1. Use of Stem Cells in Medicine 28

4 List of Australian Molecular Medicine Firms 29
5 Some Examples of Federal Government Support 30

6 Some Key Overseas Molecular Medicine Firms 31


7 Working Group Members 32

8 Glossary 33




OVERVIEW OF PRESENTATION……………………………………………..35


EXECUTIVE SUMMARY
The Third Great Technology Revolution
We are in the early phases of one of the most important periods of discovery in human history – the exploration of the genetic basis of life and its diversity, called Genomics.
The differences between individuals, and between limes and lemons, are embedded in genetic programming, with environmental factors sometimes affecting the expression of this ‘software’.
Those who exploit this genetic information will dominate the biological industries of this century, industries, which will account for half of the world’s economy. Entirely new industries and opportunities, which cannot yet be guessed, will be created.
Genomics is now widely regarded as the third great technology revolution, after the Industrial Revolution and Information Technology.
Genomics and advanced molecular and cellular biology are attracting considerable public and private investment in other countries.
The first stage of the Human Genome Project has been completed, consisting of the sequence of over 3 billion chemical bases in human DNA. This was an international effort, this milestone being announced by both US President Clinton and UK Prime Minister Blair. Besides humans, the DNA sequences or genomes of many other species, such as infectious bacteria and viruses, have been completed.

Genes

The DNA sequence of bases provides a template to identify and analyse genes and their functions. Each gene is a blueprint that encodes one or more proteins. Proteins are the basis of cells, tissues, and organs, underlying human growth, development and physiology.


The next major challenge is identifying what all the genes code for, and involves matching genomic sequence data with other biological data. The use of computers is emerging as a feature of this area, as vast amounts of information must be assembled and analysed. Computational biology helps develop and refine various models of biological systems.
In the broadest sense, the intersection of genomics knowledge, gene manipulation, and cell manipulation, with optics, biomaterials, and information technologies is creating new vistas. It is driving the development of innovative new industries, as happened with the Internet in the information revolution. As with any innovation, the right mix of discovery, ideas, technologies, venture capital and creative well-trained people is required.

Medical Breakthroughs

Medicine will benefit from new pharmaceuticals, vaccines, antibiotics, diagnostics, and the ability to predict accurately clinical outcomes. For the first time in history, gene therapy holds promise for the treatment of genetic disorders. New approaches involving gene therapy will also treat viral diseases and cancer. Gene therapy clinical trials are underway in this country.


At the cellular level, the application of stem cell therapies to treat burns, heart disease, liver disease, neuromuscular disease, neurodegenerative diseases and other conditions associated with ageing will advance the prospects for recovery from disease and injury.

Ethical issues will be raised by these technologies. The Australian regulatory framework to handle such issues is well regarded locally and overseas. New advances, such as re-programming of our own cells, and the development of universal stem cell lines compatible with transplantation may help address some of the ethical issues that are currently encountered.





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