2005.
Awaiting revision 2008
MEDICINE IN THE NEXT 100 YEARS
Part
2
Will
GENE THERAPY produce a FRANKENSTEIN
FUTURE?
Gene therapy is now in the ascendant. Last month I covered the
story behind the discovery of the human genome. This month - some
of the practical results of this work.
WHAT IS THE POTENTIAL OF THE MAPPING OF THE GENOME? The
government Human Genome Project completed the first draft of the
map of chromosomes 5, 16, and 19 in mid April 2000. This is
extremely useful for researchers, who know these particular
chromosomes are involved in schizophrenia, diabetes, some cancers,
and hypertension. They now know where to focus their attention in
looking for treatments for these conditions.
This does not mean that the cure for, say, diabetes is around the
corner; but it does mean that, in time, a cure will probably be
available for the disease, for most people, through gene
replacement.
To put the future in some perspective...
...LET'S TRAVEL BACK 100 YEARS... In the Spring of 1900 -
precisely 100 years before Craig Venter announced his mapping of
the human genome - one William Bateson was talking to the Royal
Horticultural Society.
The previous year, Bateson had said that detailed testing
must be carried out into how various characteristics of
plants were passed on from generation to generation.
He then discovered - funnily enough, at the same time as three
other researchers independently discovered it - the remarkable work
of an Augustinian monk called Gregor Mendel.
Mendel had observed and recorded the transmission of various
characteristics of pea plants down the generations. By 1863, Mendel
had tested 28,000 plants, and carefully recorded his results - a
treasure-store of data for the hungry researchers - Bateson, as
well as Hugo de Vries, Carl Correns, and Erik von Tschermak.
A PATTERN? Mendel had been intrigued by the differing
characteristics of peas, and felt there must be some pattern. He
had discovered that certain characteristics - such as the shape of
the seed pods - were 'dominant' ie. they took precedence over other
characteristics which were 'recessive'.
Recessive characteristics did not necessarily disappear, however;
they could remain hidden, to re-appear in a future generation.
APPRECIATED ONLY AFTER HIS DEATH Mendel's 'Experiments in
Plant Hybridisation' was published in 1865. He died in 1884, never
knowing how much his work would be appreciated. Sad, eh?
Bateson said of Mendel's discoveries, "That we are in the presence
of a new principle of the highest importance is, I think, manifest.
To what further conclusions it may lead us cannot yet be
foretold."
GENETICS 'AS IMPORTANT AS THE LAWS OF CHEMISTRY'
He declared that Mendel's work was, "Worthy to rank with those that
laid the foundation of the atomic laws of chemistry."
Bateson devoted the remainder of his scientific career to what
became known as 'Mendelism'.
Now, exactly 100 years after the re-discovery of Mendel's work, we
have made the next huge leap forward in the science of genetics -
the mapping of the genome.
WHAT DOES THE MAPPING OF THE GENOME MEAN - IN PRACTICAL
TERMS? Perhaps by 2025 - give or take a few years - you will
be able to go to a pharmacy and easily obtain a tape or disc of
your own personal genetic code.
Walter Gilbert, a key figure in the Human Genome Project,
predicts that possessing a genetic map, and the DNA sequence of an
individual will revolutionise medicine. In 20-25 years, we could be
looking back on the drugs of today in the same way that today we
look back on the barbarism of using mercury, sulphur, and other
poisons in an attempt to treat sickness in previous centuries.
"We are entering an era when disease will be predicted before it
occurs," says William Haseltine, who is also involved in the Human
Genome Project. "Medicine is basically going to change from a
treatment-based to a prevention-based discipline."
Well - hooray to that! It sounds as if gene therapy, combined with
natural therapy just may have something to offer.
WHICH DISEASES WILL BE CURABLE? It is estimated that
between about 2020-2050, a significant, and increasing, number of
diseases will be treatable through gene replacement. About 5,000
diseases are known to be capable of being inherited. Between about
2050 and 2100, the majority of these inherited diseases may be
completely avoidable thanks to genetic therapy.
This would include many cases of:
- Heart conditions
- Cancers
- Diabetes
- Mental disease, and
- Alzheimer's
disease.
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In time, nearly all of these 5,000 inherited
diseases will be treatable. A child being born in 50-100
years will grow up with this reality. Try explaining to
today's children what living without TV or microwaves was
like. It will be the same for genetic treatment.
The major causes of early death - heart disease and cancer - would
be greatly reduced.
It used to be rare to live to 100. Today it's not that unusual. By
2100 it will be common - or even usual - to break the century
barrier.
For now, scientists can identify and isolate useful strands of
genetic material DNA - quite easily. The problem lies in getting
the body to accept it.
The most successful method has been by putting the DNA into a
genetically altered cold virus, and injecting it. But this is far
from a perfect method - research is continuing.
For the moment, much better results are promised by using bacteria
to integrate strands of DNA with their own DNA, and producing
valuable proteins. Such proteins are often the key in disease
treatments.
WILL EVERYONE AUTOMATICALLY BE HEALTHY? No! Many people
will still be stuffing down junk foods, overeating, and carrying on
bad habits - so that doesn't mean everyone would be healthy! But we
will certainly be living much longer on average.
Some genetic treatments will be purely symptomatic - and so will
not lead to the holistic health we advocate. Producing proteins to
treat disease, for example, sounds rather like producing drugs to
treat disease. But let's see exactly what is on offer before
deciding what is, and what is not, a good idea.
Our world is soon going to be a dramatically different place.
Health will be more an area which we 'manage' rather than 'worry
about'. And it's no good saying 'I don't care - I won't be here'.
The whole point is - you are much more likely to be here!
TWO EARLY SUCCESSES OF GENE THERAPY
- Gene replacement appears to have reduced the need
of a small group of haemophiliacs for blood
transfusions.
- Two 18-month old children with 'severe combined
immunodeficiency' (SCID) appear to have developed a raised
protection against infection, and remain healthy nine months
after gene
therapy.
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These results need to be confirmed by further
testing, but they indicate the sort of successes which are
probably possible.
ONE FAILURE
- An 18-year old boy died after genetic therapy for a
rare metabolic condition. Why - it is not sure. It is likely
that his death was due to a reaction of his immune system to
the de-activated virus used to carry the gene into the
body.
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RESEARCH WILL CONTINUE Scientists need to
be cautious. But they won't stop the research.
Better methods will develop as they learn. There will be no
shortage of volunteers for treatment. Many people who have no other
choice for treatment would choose to have the gene therapy.
Some results of treatment will be absolutely fantastic. Especially
if it's for you or a loved one!
In the early days there are bound to be more failures - or even
disasters. But the successes will outnumber the failures;
and gene therapy will become commonplace.
MIGHT THERE BE ABUSE OF GENETIC THERAPY? Of course, there
are concerns regarding gene therapy. New choices will be available
to us which many will find difficult - or objectionable. Emotive
issues about whether to permit or deny life itself will have to be
confronted head-on.
But life-and-death decisions have been made by doctors - covertly -
ever since medicine began. The exact time to turn off a
life-support machine, for example, is an unenviable, and often
purely subjective, choice for the medical profession. The choice is
made even more difficult because occasional cases of completely
unexpected emergence from coma have occurred.
Soon, though, we will have the ability to manipulate human life
more than ever before. Yes, we will be able to replace
disease-producing genes before, or just after, birth. Obviously, if
we could choose between our baby having a particular birth 'defect'
or not, most of us would prefer our child to be free of it.
But also we will have the ability to introduce genes to make
'improved' humans. These could include raising IQ, choosing hair
colour, or deciding the size of particular muscles, for example.
Most governments will ban the obviously distasteful options; but
society has to decide exactly where to draw the line. And will some
of the more secretive governments of the world be more lax in their
regulations?
In the 1960s and 70s, some Eastern European countries persuaded
their sportsmen and women to use anabolic steroids from a
dangerously early age, to boost athletic performance. This risked
their lives and health. Will some athletes of the future be
genetically 'bred for success' by the unscrupulous?
THE SECRET OF ETERNAL LIFE? And what about old age? No
reason has been found for the aging process. The body re-creates
nearly all of its cells within seven years. Why doesn't it create
brand new ones each time - so that aging would not occur? We know
some of the mechanisms which produce aging - but why are these
mechanisms in place? Nobody really knows.
Some genes are certainly involved in producing aging. The genes
which produce aging of the skin have already been identified. By
manipulating these and other genes will we eventually be able to
choose the length of life we enjoy/endure? Scientists have already
made human cells live for double their normal lifespan. They have
lengthened the lifespan of simple organisms. This type of science
will certainly be applied to man.
One thing is certain - genetic therapy is going to throw up some
brand new questions. We have no choice but to confront them head
on.
GENE THERAPY IS HERE TO STAY We must focus on the good
that gene therapy will bring - while accepting that it will not all
be good. We have to take things very slowly while we decide on the
safeguards which will be necessary.
We have to keep our fingers crossed - but then the human race is
used to that.
The human race is about to take a huge step into gene therapy. It
is almost with us. There is now no going back.
LINKS: Resources about all aspects of genetics:
www.kumc.edu/gec/
Longevity: pcbs042.open.ac.uk/future/Millennium/842.htm
Aging and Genetics: www.biorap.org/rg/rgagepd.html
______________________
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