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Frequently Asked
Questions
Advanced therapies
What are "advanced
therapies"?
“Advanced therapies”
is an umbrella term
that covers the
three new techniques
that are
revolutionizing
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.
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What are the types
of advanced
therapies?
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).
A person’s own cell
and tissue can offer
a patient a wide
range of health care
solutions, from
prosthetic and
restorative to
therapeutic or even
cosmetic in nature.
Under normal
conditions damaged
joint cartilage does
not – or only poorly
- regenerates in the
body. For several
years now, cell
therapy for
restoring defects to
knee cartilage has
been available by
growing a patient's
own cartilage cells
to repair cartilage
defects. Active
research, involving
human cell-and
tissue-based
products, is being
conducted in the
regeneration and
repair of bones,
tendons, nerves and
ligaments.
Research into stem
cells result in
important cell-based
therapies to treat
serious diseases and
conditions like
Parkinson’s disease,
Alzheimer’s disease,
spinal cord
injuries, as well as
diabetes, stroke,
burns, skin
disorders and heart
disease. Researchers
work on three types
of human stem cells
– adult, foetal or
embryonic.
Despite the high
standard of today's
medical treatments,
and the number of
already available
drugs, many of the
most debilitating
human diseases do
not have a cure yet.
The molecular basis
of many inborn
disorders, such as
haemophilia, cystic
fibrosis or muscular
dystrophy, has been
exposed by the
discovery of
affected genes. In
gene therapy, genes
are introduced to
replace, repair or
switch off defective
ones.
In many forms of
cancer, genetic
predisposition plays
as important a role
as environmental
factors in tumour
growth, and
malignancy.
Identifying the gene
for such diseases
and redirecting its
course is one of the
most promising ways
to cure certain
diseases.
Gene therapy has entered a phase of active clinical investigation in
many areas of
medicine. Human
clinical trials have
been started for the
treatment of severe
immunodeficiencies,
cystic fibrosis,
hypercholesterolemia,
haemophilia,
muscular dystrophy,
many types of
tumours (e.g.
melanoma, prostate,
ovarian and lung
cancer), AIDS, and
cardiovascular
disorders.
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.
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What is the
Regulation for
Advanced Therapies
in Europe?
Advanced Therapies
refers to the
Regulation (EC) No
1394/2007 on
advanced therapy
medicinal products
that bring all
advanced therapies
(gene, cellular and
tissue-based)
together within a
single, integrated
European regulatory
framework, thereby
ensuring consistency
across member
states.
The Regulation sets
out tailor-made
technical
requirements for
these innovative
therapies and
establishes new
standards for
clinical trials in
the development of
advanced medicinal
products. A proper
legal framework for
the market
authorisation of
these products is to
stimulate increased
research in this
field.
The Regulation is
providing one single
centralized
procedure, run by
the European
Medicines Agency (EMEA),
for the whole of the
European Union for
assessing the
safety, efficacy and
quality of advanced
therapies and giving
them marketing
authorization.
The Regulation set
up a Committee for
Advanced Therapies
(CAT) within the
EMEA to ensure the
most thorough
scientific
evaluation possible.
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