
Healthcare biotechnology has a tremendous
impact on meeting the
needs of patients and
their families.
Biotechnology in
healthcare not only
encompasses medicines and
diagnostics which are
manufactured using a
biotechnological process,
but also cell and tissue
engineered products, and
includes the use and the
application of key
biotechnology tools in
the research and
development of all
innovative medicines.
The majority of
innovative medicines,
whether manufactured
using biotechnology or
through a traditional
process for small
molecules, are made
available by applying
healthcare biotechnology.
From
diagnosis to treatment
and in terms of
medicines
manufactured, healthcare
biotechnology is already
delivering on its
promises, read more
about the difference
that biotech medicines
are making at our
facts and figures page.
Innovative
treatments
Healthcare Biotech is providing
patients with innovative treatments to fight disease as well as innovative diagnostics.
Biotechnology is providing doctors with more tools which are helping to move
from the treatment of diseases to prevention and cure. A whole new range of
tools are being developed to support the human body to utilize its own capacities
to fight infectious or cancerous diseases as well as injuries.
Thanks to the unravelling of the human genome, biotechnology is also increasing
the number of disease targets for conventional drug therapy. Today conventional
drugs target fewer than 500 disease targets, but in the future this is likely
to rise to between 5-10 000 targets. The pharmaceutical industry will be powered
more and more by biotechnology. The increased understanding of complex biological
processes is opening up vast new areas of possibilities to fight and cure disease.
Below are some more examples of how breakthrough biotechnologies are providing
new solutions to disease, injuries and the wear and tear of life.
Unmet medical needs
"Healthcare biotech responds to patients' unmet medical needs"
Today there are many more diseases than treatments. Just 10 000 of the 30 000
known diseases have treatments available. Greater understanding of disease and
the causes of disease is helping to produce better therapies that can more effectively
address medical needs. New insights into the biology of disease and more precise
understanding of why some people react differently lie at the heart of biotechnology.
The promise of more targeted treatments to individual groups of patients as
well as providing treatments for diseases that so far have eluded treatment
are providing us with new opportunities to meet challenging but common diseases
like heart disease, cancer and Alzheimers as well rare diseases.
Tailor-made medicines
"Healthcare biotech can tailor medical treatment to patients"
For patients, finding the right medication with less trial and error is critical.
Healthcare biotechnology is helping to bring made to measure treatments to patients.
Improvements in diagnosis
"Healthcare biotech can help prevent and better diagnose disease"
Patients often have difficulty in getting a correct diagnosis. Biotechnology
is offering new tools to doctors and patients to provide better diagnosis and
more effective but less intrusive and uncomfortable testing for patients.
To find out more about innovative biotech treatments or how biotech is helping
to meet unmet medical needs, improve diagnostics and tailor medicines to groups
of patients, click on any of the following:
Advanced Therapies
EuropaBio's Advanced
Therapies homepage
“Advanced therapies” is an umbrella term that covers the three new
techniques that are revolutionising modern medicine: cell therapy,
gene therapy and tissue engineering. These techniques are already
producing new treatments, and in the future are likely to produce
many more.
In cell therapy, new cells are introduced into tissue to treat a
disease – either from the patient’s own body (autologous), from
another person (allogenic) or from an animal (xenogenic). One
current example is the use of human adult stem cells in treating leukaemia. Around the world, researchers have been making progress
towards using stem cells to treat conditions ranging from heart
disease, spinal cord injury and brain damage to muscle damage, low
blood supply and even deafness and baldness.
In gene therapy, genes are introduced to replace, repair or switch
off defective ones. It has been used, so far with varying degrees of
success, in severe combined immunodeficiency (SCID), cystic
fibrosis, muscular dystrophy and sickle-cell anaemia, among others.
Tissue engineering is aimed at replacing or repairing defective
tissue, and is generally seen as relating to bone, cartilage and
blood vessels such as veins and arteries. This expanding field
covers the techniques of bioengineering and cell biology (and often
cell therapy itself). It is already being used to repair or replace
burnt skin, for example.

Rare diseases and orphan drugs
EuropaBio's Orphan
Medicines homepage
A disease is defined as
rare in Europe when it
affects less than 1 in
2,000 citizens.
Rare diseases are
life-threatening or
chronically debilitating
diseases with a low
prevalence and a high
level of complexity.
6000 to 8000 rare
diseases have been
identified, affecting
about 30 million
European citizens at
some point in their
life.
The
European Union has
defined an orphan
medicine as one that is
intended for a
life-threatening or
seriously debilitating
condition that has no
satisfactory method of
treatment and also:
-
Affects 5 people or
less for every
10,000 citizens
(known as the
“prevalence
criterion”);
or
-
Where
the treatment –
without incentives –
would not justify
the investment in
its development
(known as the
“insufficient profit
criterion”).

Proteomics
Proteomics is the
science which studies the physiological function of proteins and
their effects on diseases. Some diseases are caused if genes do not
produce the proteins (or enough proteins) the body requires or if
the body produces wrongly folded proteins. Biotechnology is using
recombinant (artificially created) DNA and cell cultures
to produce missing or defective proteins. Replacement protein therapies include
Factor VIII-a protein which is essential for the blood-clotting process and
which some haemophiliacs lack, or insulin - a protein hormone that regulates
blood glucose levels.
A great deal of research is ongoing to determine the role that proteins play
both in the cause and cure of disease.

Pharmacogenetics
Pharmacogenetics studies the effect genes may have on an individual’s
response to a drug. Pharmacogenetics uses biotechnology-based technologies to
not only better diagnose disease but also to provide new ways to match medicine
doses and medical treatments to individual groups of patients. The evolving
area of pharmacogenetics will increase both the safety and efficacy of treatments
by diminishing the trial and error for patients trying to find the optimal dose
and treatment. Pharmacogenetics holds great promise to offer more select drugs
to treat elusive variations of common as well as rare diseases and widen the
numbers of diseases that can be treated effectively as well as limiting the
occurrences of adverse drug reactions on patients.

Diagnostics
We can now detect many diseases and medical conditions more quickly and with
greater accuracy due to the sensitivity of new, biotechnology-based diagnostic
tools. A familiar example of biotechnology's benefits is the new generation
of home pregnancy tests that provide more accurate results much earlier than
previous test-generations.
Another good example is PCR technology: The Polymerase Chain Reaction (PCR)
is a technology that imitates a cell’s ability to replicate DNA by generating
multiple copies of specific sequences of DNA through amplification. In clinical
diagnostics, a specimen of genetic material weighing only one-trillionth of
a gram can be repeatedly copied by PCR to provide sufficient material to detect
the presence or absence of a virus as well as to quantify its levels in the
blood. PCR tests were the first that could accurately measure the amount of
HIV in a patient’s blood. This provides reliable information on the disease
course and shows when changes are needed in a patient’s medication.
A new blood test has been developed through biotechnology to measure the amount
of low-density lipoprotein (LDL), or "bad" cholesterol, in blood.
The new biotech test measures LDL in one test, and fasting is not necessary.
We now use biotechnology-based tests to diagnose certain cancers, such as prostate
and ovarian cancer, by taking a blood sample, eliminating the need for invasive
and costly surgery.
The human health benefits of biotechnology detection methodologies go beyond
disease diagnosis. For example, biotechnology detection tests screen donated
blood and organs for the pathogens that cause AIDS, hepatitis and a variety
of other infectious diseases. Doctors will someday be able to immediately profile
the infection being treated and, based on the results, choose the most effective
antibiotics.

Genetic testing
The wealth of genomics information made available by the Human Genome Project
is greatly assisting doctors in diagnosing hereditary diseases. There are currently
over a thousand human hereditary diseases that can be identified using genetic
tests. The majority of these tests detect the presence of a mutation or mutations
in a single gene which lead to monogenic (single gene) disorders, most of which
are relatively rare diseases.
Unlike monogenic diseases, there are many diseases that are caused by a combination
of environmental factors and one or more hereditary factors. Many common diseases
that affect many millions of people arise through complex interactions between
the environment and a number of alternative genes called susceptibility genes.
Soon doctors will have access to tests for detecting susceptibility before the
onset of clinical signs if patients so wish. The presence of disease susceptibility
does not always cause the disease but is a risk factor for that disease, just
as smoking is a risk factor for lung or heart disease. These tests will identify
patients with a propensity to diseases caused primarily by environmental factors,
such as diet, giving patients an opportunity to prevent the disease by avoiding
the environmental triggers. Genetic testing is also critical to the development
of pharmacogenetics which uses biotechnology-based diagnostics to better diagnose
disease and provide new ways to match medicine doses and treatments to the individual.

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