Stem
cells
STEM CELL SCIENCE IN THE UK
Key facts about research, regulation and funders
Stem
cells
THE UK – THE PLACE TO BE FOR STEM CELL RESEARCH
Stem cell research is one of the most
exciting areas of 21st-century science.
It offers a potentially revolutionary way
to repair diseased and damaged body
tissues, replacing them with healthy
new cells. A huge amount of research is
needed, however, to understand exactly
how stem cells work and how their
potential can be harnessed for treatments.
Stem cells offer hope for common
conditions such as Parkinson’s disease,
diabetes, cancer and Alzheimer’s disease,
which affect vast numbers of people in the
UK and around the world, and for which
there are treatments but currently no cures.
They are found in a number of places in
the human body and occur at the very
earliest stages of development through
to adulthood. Scientists believe that the
most effective way of discovering potential
treatments is to undertake research on
all types of human stem cells obtained
from the various stages of development
at which they are found, including very
early embryos. All over the world, stem
cell research has become the subject of
scientific excitement, public curiosity, moral
reflection and regulatory challenge.
The UK is well positioned, because of
its rigorous regulatory regime and its
commitment to this research, to identify
and realise the therapeutic potential
of stem cells. It also has world-class
researchers in developmental and
reproductive biology and the UK Stem
Cell Bank. These strengths present
valuable opportunities to influence the
international agenda, drive the application
of basic research to clinical benefits and
attract skilled scientists and international
investment in stem cell research.
The UK’s lead on stem cell research began
in the 1970s when scientists at Cambridge
University, led by Sir Martin Evans, isolated
the mouse embryonic stem cell. Sir Martin,
who was supported by the Medical
Research Council (MRC) between the
1970s and 1990s, went on to be awarded
the Nobel Prize in Physiology or Medicine
for 2007. Also in Cambridge in the 1970s,
Professor Robert Edwards and Dr Patrick
Steptoe fertilised first animal and then
human eggs outside the body, a discovery
that led to the birth of the first ‘test-tube
baby’ through in vitro fertilisation or IVF.
It became apparent that moral reflection
about the science was urgently required.
A committee, chaired by Baroness
Warnock, was convened to examine
the ethical issues, including whether
medical research on human embryos
could ever be justified. Their conclusions
were embodied in a landmark piece of
legislation, the Human Fertilisation and
Embryology Act 1990, which allowed
scientists to use human embryos for
a restricted range of research. Since
2001, this range has explicitly included
research into the therapeutic potential of
stem cells. Scientists are only allowed to
obtain stem cells from very early human
embryos that have not developed
beyond 14 days and they must
demonstrate that the research cannot be
done by any other means. A licence to
conduct research using embryos must
be secured from the Human Fertilisation
and Embryology Authority (HFEA).
An update of the Act is expected by
Autumn 2008.
continued overleaf...
UK Stem Cell Initiative
The UK Government set up the UK
Stem Cell Initiative in March 2005, with
the aim of working with the public and
private sector to draw up a ten-year
vision for UK stem cell research. Led by
Sir John Pattison, the initiative involves
representatives from the Medical
Research Council, the Biotechnology
and Biological Sciences Research
Council, the Department of Health, the
UK Stem Cell Foundation, the Academy
of Medical Sciences, medical research
charities and industry. In late 2005 it
reported that the UK is a world leader
in stem cell research and development
– but that more investment is needed if
this position is to be maintained.
UK Stem Cell Funders Forum
The UK Stem Cell Funders Forum
was set up in 2006 to take forward
the recommendations of the UK Stem
Cell Initiative. Its members include
research councils, major charities
working in stem cell research, UK
health departments and the Scottish
Executive. The forum allows members
to discuss and exchange information
on their current stem cell work and
funding priorities, to work together to
identify barriers to stem cell research
and ways to overcome these, and to
recognise new areas that could benefit
from joint funding.
UK National Stem Cell Network
A further part of the forum’s remit is
to coordinate public communication
activities for stem cell research. This
has been taken on by the Stem Cell
Communication Coalition, a group made
up of representatives from the forum’s
member organisations and from the
Royal Society, the UK Stem Cell Bank,
the Human Fertilisation and Embryology
Authority and the Human Tissue
Authority (HTA). Established in 2002,
the coalition has coordinated a number
of activities including a public survey
of attitudes on the use of embryos in
medical research.
The network’s mission is to promote
research activities and events at
national level which help to speed the
translation of basic stem cell research
into therapeutic applications in the
control of degenerative diseases such as
Alzheimer’s and Parkinson’s, and other
conditions such as diabetes.
The UK Government launched the UK
National Stem Cell Network (UKNSCN) in
April 2007. It is a national body to improve
the coordination of stem cell research
and the dissemination of research results,
in addition to providing a focal point for
communication with overseas researchers,
the media and the general public.
The UKNSCN is funded through
contributions from four UK research
councils – the Biotechnology and
Biological Sciences Research Council,
the Medical Research Council, the
Engineering and Physical Sciences
Research Council and the Economic and
Social Research Council.
Injecting altered cells into early mouse
embryos. © Vasanta Subramanian,
Wellcome Trust
Stem
cells
MILESTONES IN UK STEM CELL RELATED RESEARCH AND REGULATION
1978
First IVF baby born after Cambridge scientists fertilise human eggs outside the body.
1981
Isolation of mouse embryonic stem cells by Cambridge scientists.
1982
Formation of the Warnock Committee to examine the moral questions surrounding assisted
reproduction and embryo research.
1984
Warnock Report endorses human embryo research into reproductive-related areas but advises tight regulation.
1990
Human Fertilisation and Embryology Act passed by both Houses of Parliament. Human Fertilisation
and Embryology Authority (HFEA) designated statutory body to enforce provisions of legislation and
established in 1991.
1996
Dolly the sheep is cloned by cell nuclear replacement (CNR) techniques at Roslin Institute in Edinburgh.
1998
HFEA and Human Genetics Advisory Commission working party recommends that CNR be
investigated for therapeutic purposes but not for reproductive cloning.
2000
Report by Donaldson Commission, chaired by the Chief Medical Officer, Sir Liam Donaldson,
recommends that research using human embryos (created by IVF or CNR) to increase understanding of
human disease and disorders and their cell-based treatments should be permitted subject to controls in
the 1990 HFE Act. Also that the research councils should be encouraged to establish a programme of
stem cell research and consider the feasibility of establishing collections of stem cells for research use.
2001
Human Fertilisation and Embryology (Research Purposes) Regulations designed to implement the
recommendations of the Donaldson Commission passed by Parliament; three new purposes added
to the 1990 Act. House of Lords appoints a select committee to examine the issues arising from the
new regulations, including those of human cloning and stem cell research. Parliament introduces
additional legislation prohibiting reproductive cloning.
2002
A House of Lords select committee concludes that stem cells have great therapeutic potential and
that research should be conducted on both adult and embryonic stem cells. Also that a stem cell
bank should be established and overseen by a steering committee.
2002
The UK Stem Cell Bank, funded by the Medical Research Council and the Biotechnology and
Biological Sciences Research Council established at the National Institute for Biological Standards
and Control. The bank to be a repository for stem cells derived from adult, foetal and embryonic
tissues and to be open to academics and industrialists from the UK and abroad. Steering committee
established to oversee the bank and the use of stem cell lines and to develop codes of practice.
2003
Researchers at King’s College London generate the UK’s first embryonic stem cell line.
2003
The International Stem Cell Forum (ISCF) – made up of 21 funders of stem cell research from around
the world including the MRC – is established to encourage international collaboration and funding
support for stem cell research.
continued overleaf...
2004
First stem cell lines deposited in UK Stem Cell Bank by scientists from King’s College London and the
Centre for Life in Newcastle.
2004
The ISCF starts a review of global ethics and regulation relating to stem cell research.
2005
The UK Government sets up the UK Stem Cell Initiative, with the aim of working with the public and
private sectors to draw up a ten-year vision for UK stem cell research.
2006
The Hinxton Group, a consortium of 60 researchers, ethicists, scientific journal editors and lawyers
from 14 countries, reaches a consensus on a set of international guidelines for stem cell research,
which they hope will simplify existing rules and aid collaboration.
2006
On behalf of the ISCF, the Australian National Health and Medical Research Council produces a
report on intellectual property rights related to stem cell research across the world. It will be key in
encouraging further research and development worldwide.
2006
American scientists find out how to make stem cells from embryos without destroying the embryo in
the process – an advance that could overturn ethical objections.
2006
The Biotechnology and Biological Sciences Research Council leads the research councils and
relevant government departments in establishing a UK National Stem Cell Network to encourage links between researchers and to encourage development of an integrated national stem cell research community.
2007
The EU Tissues and Cells Directives became UK law via the Human Tissue (Quality and Safety for
Human Application) Regulations 2007. Under the Regulations the Human Tissue Authority regulates
the procurement, processing, testing, storage, distribution and import/export of tissues and cells for
human application.
2007
The International Stem Cell Forum publishes the results of a study characterising 59 stem cell lines.
The research will help to ensure that future advances in stem cell research involve internationally
coordinated quality standards.
2007
Teams of researchers at Cambridge and Oxford universities independently discover a new type of
stem cell in mice and rats that is very similar to human embryonic stem cells.
2007
Two independent teams of researchers – at Wisconsin and Kyoto universities – produce human
induced pluripotent stem cells. These are thought to be very similar, if not identical, to embryonic stem
cells but are made by reprogramming an adult (nonpluripotent) cell.
2008
The Hinxton Group meets for a second time and produces a consensus statement on pluripotent
stem cell-derived gametes, which discusses social and ethical issues and makes recommendations
about policy and practice.
2008
Human Fertilisation and Embryology bill updated to allow use of hybrid embryos in research.
Stem
cells
THE SCIENCE OF STEM CELLS
Stem cells, whether they are derived
from an early embryo, a foetus or an
adult, have two key properties. Firstly,
they have the ability to reproduce almost
indefinitely and, secondly, they can be
directed to generate the specialised cells
that make up the tissues and organs of
the body. They have exciting potential for
the generation of therapies for repair and
replacement of damaged and diseased
tissues and organs, as models for the
testing of new drugs and helping us to
understand at a cellular level what goes
wrong in many conditions.
Embryonic stem cells
A fertilised egg (produced when an
egg is penetrated by a sperm cell) is
in essence a one-cell embryo. Up until
about the eight-cell stage, this very early
embryo has the capacity to develop
into every cell needed for full human
development as well as tissues such as
the placenta and umbilical cord. After
about five days of development, the
embryo consists of a ball of 50 to 100
cells called a blastocyst, which is about
the size of a full stop on this page. It is
from an inner cell mass within this ball
that the embryonic stem cell or ES cell
is derived. The cells that comprise it
have no fixed destiny – at this stage,
there is no trace of any structure such
as a nervous system which could, for
example, produce sensations of pain.
Although ES cells can be manipulated to
generate all the cell types in our bodies,
they cannot develop into a complete
embryo on their own.
The main source of embryonic stem cells
is from surplus embryos donated by
couples undergoing in vitro fertilisation
(IVF). These would otherwise be destroyed.
Developed in the 1970s, IVF is used to
treat couples who are having difficulty
becoming pregnant. Usually, the woman
takes fertility drugs to help her produce
more eggs, and these eggs are harvested
and fertilised with the man’s sperm in
the laboratory. The woman is then given
hormone drugs before the fertilised eggs
are implanted into her womb.
Adult stem cells
Adult stem cells are found, for example,
in human bone marrow, blood, the eye,
the brain and skeletal muscle. Their
purpose is to replace and replenish cells
with specialised functions, such as blood
cells. Bone marrow transplants are an
existing model of adult stem cell therapy.
continued overleaf...
STEM CELLS
adult stem
cells
sperm
fertilised
egg
totipotent
cells
blastocyst
neurons
embryonic
stem cells
bone tissue
egg
blood cells
FERTILISATION
STIMULATED
TO DEVELOP
FIVE DAYS’
GROWTH
DIFFERENTIATION INTO
ADULT STEM CELLS
SPECIALISATION INTO
FINAL CELL TYPES
For nearly 40 years, patients with blood
disorders such as leukaemia have been
treated by introducing haematopoietic
stem cells via bone marrow transplants.
This has been possible because
haematopoietic stem cells are readily
accessible, unlike many other adult
stem cell types found in our bodies, and
they are able to replenish blood cells
continuously at high rates. Adult stem
cells do not appear to have the same
capabilities as ES cells. However, under
laboratory conditions, some scientists
claim to have been able to manipulate
them to form other cell types. So it may
be possible, eventually, to direct these
cells to function in other areas of the
body and to repopulate body tissues
that have been damaged or diseased.
In 2006, researchers found a way to
reprogramme mouse skin cells and
turn them back into cells very similar
to embryonic stem cells. A major
breakthrough was achieved in 2007
when researchers also achieved this in
human cells, opening up a possible new
source of pluripotent stem cells, but
without the need to use actual embryos.
This technology is at a very early stage
and the techniques used to provide the
necessary reprogramming require genetic
modification and cannot currently be
used to develop therapies. Nevertheless,
scientists anticipate making rapid progress
towards better understanding and refining
approaches to generate these cells. The
short-term goal is to use them to help
model human diseases in the laboratory.
The challenge for stem cell research
As the one-cell embryo develops, cell
differentiation occurs. Differentiation is
the increasing specialisation of cells:
more specialised cells develop from
less specialised cell types. Since most
of our body cells have the same genes,
differentiation must involve switching on
(expressing) or switching off (repressing)
different subsets of genes in individual
cells. ES cells grown in the laboratory
preserve the power to become any cell
in the human body. Their destiny is to
differentiate. They are infinitely malleable
and one of the challenges for scientists
is to direct this process. A crucial task
for tapping into the therapeutic potential
for stem cells is to teach them how
to become, for example, muscle cells
for damaged hearts or neurons for
damaged brains. Another is to ensure
they do not continue to grow in an
undifferentiated way and in effect turn
into a cancer. Much basic scientific
research needs to be undertaken in
order to understand these processes.
The adult stem cells in our bodies
replenish cells and tissue in their particular
location and, in general, appear to be less
malleable than ES cells. However, recent
experiments have shown they do have the
potential to do a lot more than previously
suspected. Stem cells from bone marrow
have been induced to differentiate into
brain and kidney cells. How this happens
is not precisely understood. Research
is continuing to explore the mechanism
of such transformation and to ascertain
whether it is possible to direct adult stem
cell differentiation to produce cells
for therapies.
Cell nuclear replacement
Scientists are also exploring a technique
called cell nuclear replacement which
might potentially provide cells for
therapies. In 1996, scientists at the Roslin
Institute in Edinburgh produced Dolly the
sheep from a mammary cell of a six-yearold sheep. The nucleus of this cell was
inserted into a sheep egg cell after the
egg’s own nucleus had been removed.
This process is known as cell nuclear
replacement. Factors in the egg (the
cytoplasm) reprogrammed the nucleus of
the mammary cell and, in a sense, made
it ‘forget’ its original destiny. Instead, the
nucleus behaved as if it were inside a onecell embryo. The egg was then implanted
into a sheep’s womb. This is reproductive
cloning. It is illegal in the UK to carry out
reproductive cloning in humans.
It may be possible, however, to use
cell nuclear replacement techniques for
human therapeutic cloning. Although
the initial steps in both reproductive
and therapeutic cloning are the same,
subsequent steps and the underlying
purpose could not be more different.
In therapeutic cloning, also known as
somatic cell nuclear transfer (SCNT), the
nucleus of an adult cell, for example a
skin cell, from a patient with a condition
such as Parkinson’s, would be inserted
into a human egg whose own nucleus
had been removed. When the cloned
embryo reached the blastocyst stage, ES
cells would be derived from the inner cell
mass. These cells would be grown in the
laboratory and induced to differentiate
into the precise cells needed, for example
dopamine-producing neurons, which
are lacking in the brains of people with
Parkinson’s disease. Although much
research still needs to be undertaken, this
form of stem cell therapy could provide
new cells, likely to be genetically identical
to the patient, with a greatly reduced risk
of rejection, a common problem in any
form of transplant operation. However,
even if this technology is successful,
such personalised medicines would be
very costly.
If the patient suffers from a genetic
disease, their cells would also contain
the genetic defect, and the resulting ES
cells would not be used for cell-based
therapy. However, the ES cells could
be used to study the disease in the
laboratory to perhaps find other types of
therapy. Some ES cells are derived from
embryos that have failed the process of
preimplantation genetic diagnosis (PGD)
– which involves genetically testing an
embryo in a laboratory. Some of these
will carry the genetic defect, such as
cystic fibrosis, allowing the disease to be
studied in the laboratory.
Scientists are exploring the possibility
of creating embryos, by using human
sperm to fertilise an animal egg (hybrid),
or transferring the nuclei of human cells
into animal eggs that have had almost
all their genetic material removed. These
so-called ‘human admixed embryos’
are a type of SCNT. They could provide
a plentiful supply of stem cells and get
around the shortages of leftover IVF
embryos from which stem cells can be
harvested or of human eggs that could
be used in the same way. In late 2007,
the Human Fertilisation and Embryology
Authority granted licences to two
teams of researchers to create human
admixed embryos.
Stem
cells
STEM CELL RESEARCH: REGULATORY ISSUES
The Human Fertilisation and
Embryology Act of 1990 was enacted
to regulate the practice of in vitro
fertilisation (IVF) and the creation,
use, storage and disposal of embryos
produced in this way. It established
a regulatory authority, the Human
Fertilisation and Embryology Authority
(HFEA), which is empowered to approve
all embryo research conducted in the UK.
Such research is illegal unless it is carried
out under a licence granted by the HFEA.
In order to receive a licence the applicant
must demonstrate that embryo research
is necessary and that the proposed
research is being done for one of the five
purposes specified in the Act:
(a) promoting advances in the
treatment of infertility;
(b) increasing knowledge about the
causes of congenital disease;
(c) increasing knowledge about the
causes of miscarriages;
(d) d
eveloping more effective
techniques for contraception;
(e) developing methods for detecting the
presence of gene or chromosome
abnormalities in embryos before
implantation; or for such other
purposes as may be specified in
regulations [paragraph 3(2)].
Such other purposes are limited by the
language of the Act to projects which
“increase knowledge about the creation
and development of embryos, or about
disease, or enable such knowledge to
be applied” [schedule 2 paragraph 3(3)].
Since the Act was passed, there have
been many advances in developmental
biology but two stand out: the derivation
of human embryonic stem (ES) cells
and the cloning of Dolly the sheep in
1996 by cell nuclear replacement (CNR).
In response to the cloning of Dolly the
sheep, a working party drawn from the
HFEA and the Human Genetics Advisory
Commission was formed to undertake
a public consultation on human cloning.
In their report, ‘Cloning: Issues in
science and medicine’ (December
1998), the group recommended that the
Secretary of State for Health consider
adding two additional purposes for
which embryo research could be
licensed: the development of therapy
for mitochondrial disease and the
development of therapeutic treatments
for diseased and damaged tissues and
organs. Human reproductive cloning
was deemed unacceptable.
continued overleaf...
Egg cell manipulation.
Images courtesy of MRC Clinical Science Centre
Light micrograph of a human embryo
at the two-cell stage of development.
Richard G. Rawlins/Custom Medical Stock Photo/
Science Photo Library
An expert committee chaired by Sir
Liam Donaldson was then appointed
and the group was asked to answer two
basic questions: should this new type of
research be permitted and, if so, were
new regulations required? In its 2000
report, the committee recommended
that research on embryos (both surplus
embryos left over after IVF and embryos
created by CNR) be permitted in order
to increase understanding of human
disease and disorders, and their cellbased therapies. The Human Fertilisation
and Embryology (Research Purposes)
Regulations were brought forward by the
government; they were passed by the
House of Commons in December 2000
and by the House of Lords in January
2001. The regulations added three new
purposes to the five in the Act:
(a) increasing knowledge about the
development of embryos;
(b) increasing knowledge about serious
disease;
(c) enabling such knowledge to be
applied in developing treatments for
serious disease.
The House of Lords Select Committee
on Stem Cell Research was appointed
on 7 March 2001 “to consider and
report on the issues connected with
human cloning and stem cell research”
arising from the new regulations. On
27 February 2002, the Committee
reported. Among its conclusions was
an endorsement of the Department of
Health’s (DH) proposals to establish
the UK Stem Cell Bank, responsible for
the custody of stem cell lines, ensuring
their purity and provenance, and
monitoring their use. The Committee
also recommended that as a condition
of granting a research licence, the HFEA
should require that a sample of any
ES cell line generated in the UK in the
course of that research be deposited in
the bank.
The creation of embryos (from which
stem cells may be derived) and the
subsequent storage of these embryos
is regulated by the HFEA. This includes
embryos created from in vitro fertilisation
and cell nuclear replacement. Research
projects which involve derivation of stem
cells by the creation of embryos must
be licensed and approved by the HFEA.
Researchers must prove, to an HFEA
licence committee’s satisfaction, that
the research application fits in with the
purposes of the Human Fertilisation and
Embryology (HFE) Act 1990 and the
HFE Research Regulations 2001 and
that it is necessary to use embryos for
the research.
In addition to the two licences
subsequently granted for human
admixed embryos, the HFEA has so
far granted two licences to study the
derivation of human embryonic stem cell
lines using nuclear transfer (therapeutic
cloning). The first was in 2004 to
researchers at the International Centre
for Life at the University of Newcastle,
who are investigating new treatments
for conditions including diabetes,
Parkinson’s and Alzheimer’s disease.
The second, in the following year, was to
the Roslin Institute in Edinburgh for the
study of motor neuron disease.
The regulation of stem cells in the UK
is affected by two pieces of legislation:
the Human Tissue Act 2004 and the
Human Tissue (Quality and Safety for
Human Application) Regulations 2007,
which fully implement the EU Tissues and
Cells Directives (EUTCD). The Human
Tissue Act 2004 regulates the removal,
storage and use of human bodies,
organs, tissue and cells for a number of
purposes, including research. The EUTCD
– which was brought fully into force in
2007 – creates a common framework
that ensures high standards in the
procurement, testing, processing, storage,
distribution and import/export of tissues
and cells across the EU. The primary aim
of the Directive is to ensure the quality,
safety and traceability of tissue and cells
used for human application. It also aims to
support the exchange of tissues and cells
between member states.
Stem
cells
INTERNATIONAL ARENA
Globally, stem cell research is
becoming increasingly competitive.
Many countries, such as the ones
listed below, are progressing
considerably in the stem cell research
field, due to increasing funding and
permissive regulations.
In Asia, Japan has invested heavily
in stem cell research and Japanese
researchers have been responsible for
some important recent discoveries.
The Chinese government supports all
forms of stem cell research and Chinese
researchers are carrying out work of
international standing and publishing in
European and US journals. Stem cell
research is a major priority in Singapore,
which has attracted overseas talent with
state-of-the-art facilities and generous
research funding.
The US remains the world leader in stem
cell research. Despite restrictions on
federal funding for embryonic stem cell
research, the US National Institutes of
Health (NIH) spends very considerable
amounts on other types of stem cell
research. Individual US states, such as
California, are investing substantially
in all forms of stem cell research,
including embryonic stem cell research.
Canada spends a large amount on
stem cell research and the government
established the Canadian Stem Cell
Network to coordinate research
activity and fund major collaborations
with a concentration along product
development lines.
Sweden is a world leader in stem
cell research, with a regulatory and
ethical environment similar to the UK.
Due to its permissive laws on stem
cell research, Sweden now leads
Scandinavia, and possibly even Europe,
in this area. Germany has very tight
restrictions that have deterred its
scientists from working on embryonic
stem cells, but it is a world leader in
adult stem cell research.
Regulation
Regulation of stem cell research varies
enormously between different countries.
Some countries have no legislation at
all, and others ban all such research.
Some countries have adopted laws that
allow embryonic stem cell research,
including ‘therapeutic’ cloning and
the creation of embryos specially for
research purposes. A large number of
countries have adopted compromise
policies, permitting some types of
embryonic stem cell research and
restricting others.
The US has no national laws or
regulations to control embryonic stem
cell research. However, individual states
have introduced their own laws. Some
states, such as Florida and Pennsylvania
have prohibited embryonic stem
cell research. Other states, such as
California and Massachussetts, have
permissive laws.
continued overleaf...
Countries with permissive regulation
include the UK as well as Australia,
Belgium, China, India, Israel, Japan,
Singapore, South Korea and Sweden.
Although the UK’s regulations for
embryonic stem cell science are
permissive, the system for enforcing
them is very strict, more so than in many
other countries.
Other countries are less permissive.
Such countries allow embryonic stem
cell research using embryos left over
after IVF treatment but may not allow
cloning or the creation of embryos
specially for research purposes. These
countries include Canada, Denmark,
France, the Netherlands, New Zealand,
Portugal, Spain and Switzerland.
There are also a number of countries
with tight restrictions or bans on
embryonic stem cell research. Within
Europe, countries with such laws include
Italy, Lithuania, Poland and Slovakia.
Different European countries have very
different laws on stem cell research.
The European Commission will not
fund activities that destroy the human
embryo, but may fund other research
on embryonic stem cells, provided that
this research is permissible under the
laws of the country where the research
is taking place.
There have been a number of attempts
to create international rules for stem cell
research. For example, the International
Societies for Stem Cell Research
(ISSCR) has drawn up ethical guidelines
for the embryonic stem cell research.
These guidelines are voluntary, but
scientists are coming under pressure
from international journals and funding
bodies to prove that their research is
being conducted in an ethical manner.
In 2005, the United Nations General
Assembly adopted a declaration calling
on countries to ban all forms of human
cloning, including cloning for research
purposes. However, the declaration is
non-binding and has no legal status.
The International Stem Cell Forum
The Medical Research Council
(MRC) launched the International
Stem Cell Forum (ISCF) in 2003 along
with eight other international funding
agencies with similar scientific principles
and resources. These agencies all
shared the MRC’s concerns about
the need to create standardised
global criteria for creating, storing and
maintaining stem cell lines. Today,
the ISCF has a total of 21 member
organisations in 19 countries.
The forum is working on the
International Stem Cell Initiative (ISCI).
Led by Professor Peter Andrews of
the University of Sheffield Centre for
Stem Cell Biology, the ISCI has been
developing an internationally-agreed
set of rules for growing and analysing
human embryonic stem cells. The
ISCF published a paper in Nature
Biotechnology in June 2007 analysing
in detail 59 human embryonic stem cell
lines. This international collaborative
project is now progressing with
US$2million of funding.
On behalf of the forum, the Australian
National Health and the Medical
Research Council have developed a
document about intellectual property
in stem cell science. It covers the
criteria for patenting stem cells
throughout the world, identifying
techniques that may be subject to
patenting, highlights patents already in
existence and explains how countries
are attempting to ensure ongoing access
to stem cell resources.
Led by Canada, the ISCF is also
working on a global review of ethics
and regulation of stem cell research,
which is being carried out by its Ethics
Working Party. The group includes
ethicists, research scientists, clinicians
and lawyers from the forum’s members.
Together they are reviewing the different
ethical issues and regulations in
countries that fund stem cell science,
with a particular emphasis on research
involving embryonic stem cells. The
group aims to draft best practice
guidelines for stem cell research and to
develop a global register of clinical trials
involving stem cells.
Stem
cells
UK COMMERCIALISATION OF STEM CELL RESEARCH
Although the commercial, nontherapeutic use of stem cells is already
an established part of the process
of drug discovery, the commercial
and medical benefits from stem cell
therapies are still some way off, with
the exception of bone marrow stem
cell transplants for blood disease
such as leukaemia which have been
used for many years. The delay in
further treatments is partly due to the
stringent tests for safety, quality and
efficacy required for these therapies
prior to their approval for market by
the regulatory agency - the European
Agency for the Evaluation of Medicinal
Products (EMEA). These pre-clinical
and clinical studies will typically take a
number of years to complete. It is likely
that therapies using adult stem cells will
emerge first, with embryonic stem cell
therapies following later.
also marketing their stem cells as drug
discovery tools for screening applications
in drug development, for example to test
metabolism, and in safety tests.
Support for commercialisation
Despite this, several UK public and
private stem cell research companies
have been established, such as
ReNeuron, Stem Cell Sciences,
Axordia and NovaThera. UK stem cell
companies are working on therapies for
conditions such as stroke, Parkinson’s
disease, diabetes, cardiac disease and
retinal damage of the eye. Some are
The UK Stem Cell Bank, which
provides a repository for stem cell
lines derived from adult, foetal and
embryonic tissues, will be a vital
resource to company researchers. The
bank is collaborating with commercial
partners to achieve cell lines of
sufficient stability for use by companies.
continued overleaf...
Blastocyst.
© Wellcome Trust
IVF embryo.
© Anne Falkner
The bank will ensure that cell lines which
could ultimately provide the basis for
clinical treatment are prepared under
Good Manufacturing Practice (GMP),
subjected to appropriate safety testing
and subsequently handled and stored
under quality-controlled conditions.
However, some cell lines will require
additional tests, which companies
themselves may have to carry out.
In 2004, the UK Stem Cell Bank set
up the first clinical grade GMP facility
for human stem cell banking in the
European Union. This was followed in
January 2006 by the opening of the UK’s
first GMP laboratory for the derivation
of clinical grade human embryonic
stem cell lines, at the Medical Research
Council’s Centre for Stem Cell Biology at
Sheffield University.
Companies may also benefit from the
UK Stem Cell Foundation, which was
established in 2005 to support the
transfer of stem cell techniques from the
laboratory to the clinic. The foundation
aims to raise funds from private donations
to directly fund projects where research
has indicated potential for direct clinical
benefit to patients in the short term. As
of early 2008, the MRC had approved
five joint awards with the foundation,
addressing bone and cartilage repair, liver
regeneration and brain repair.
The UK Government funds stem cell
research in companies directly via
its Technology Strategy Board. This
money is available for collaborative R&D
projects in specific areas, including
bioscience and healthcare, and is
allocated to consortia on a competitive
basis. The MRC has developed
strategies with the Technology Strategy
Board for academic and industry
cooperation, and there has already been
a joint call in regenerative medicine.
The Alzheimer’s Society is the UK’s
leading care and research charity
for people with dementia, their
families and carers. With more than
250 branches and support groups, it
provides information and support for
people with any form of dementia and
their carers through its publications,
helpline, website and local network. It
advises professionals, runs quality care
services and campaigns for improved
health and social care and greater
public understanding of dementia. The
society funds an innovative programme
of biomedical and social research on the
causes of, cures for and care for people
with the disease.
What is Alzheimer’s disease?
Alzheimer’s disease is the most common
form of dementia. More than 700,000
people in the UK have dementia, of
whom 15,000 are under the age of 65.
Dementia affects one in 14 people over
the age of 65 and one in six over the
age of 80. More than 60 per cent of the
people with dementia have Alzheimer’s
disease – a progressive, degenerative
disease of the brain, which destroys
cells and disrupts transmitters that carry
messages in the brain.
Stem cell research and
Alzheimer’s disease
In Alzheimer’s disease, nerve cells die in
a random way, interrupting the complex
interconnections of nerve cells in the
cortex, which is the outer layer of the
brain. It is this network of cells that
facilitates our memories, personalities
and behaviour patterns. Because of
the loss of many different nerve cell
types in the brain, and the impact that
the disease has on communication
between cells, developing therapies for
Alzheimer’s disease is more complicated
and challenging than for some other
neurological conditions. Until recently,
it was believed that we are born with
a certain number of nerve cells and
that once these cells die, they cannot
be replaced. However, this concept
changed after the discovery of a
population of brain cells, called neural
stem cells (NSCs), which can develop
into new nerve cells.
Experiments on animals have shown
that implanted NSCs can move
towards areas of damage and take
on a nerve cell-like structure. This
has raised hopes that NSCs may be
a useful new therapy for illnesses
where nerve cells are lost. However, in
Alzheimer’s disease, the loss of nerve
cells is very widespread and there are
doubts that the there are sufficient
NSCs in the adult brain to be able to
compensate for this loss, even if we
knew how to stimulate them.
Research on NSCs has revealed that
there may be an alternative strategy for
replacing lost nerve cells. Other parts of
the body also produce stem cells.
These stem cells seem to have
remarkable plasticity – that is, with
the appropriate chemical trigger,
they can turn into many different cell
types, including nerve cells. Indeed,
researchers have obtained bone marrow
stem cells (MSCs) from rats, grown them
in cell culture and have turned them into
cells that exhibit the characteristics of
nerve cells. This is encouraging because
bone marrow is a far more accessible
source of stem cells than the brain.
continued overleaf...
ALZHEIMER’S SOCIETY | ASSOCIATION OF MEDICAL RESEARCH CHARITIES |
BIOTECHNOLOGY AND BIOLOGICAL SCIENCES RESEARCH COUNCIL |
BRITISH HEART FOUNDATION | CANCER RESEARCH UK | DIABETES UK | HUMAN FERTILISATION AND EMBRYOLOGY AUTHORITY |
HUMAN TISSUE AUTHORITY | MEDICAL RESEARCH COUNCIL |
PARKINSON’S DISEASE SOCIETY | THE ROYAL SOCIETY |
SCOTTISH STEM CELL NETWORK | UK STEM CELL BANK | WELLCOME TRUST
MSCs are also easy to maintain in the
laboratory, can be grown easily and can
be frozen and stored for long periods
of time.
The Alzheimer’s Society has funded
stem cell research since 2002 when it
awarded a three-year fellowship. The
project involved animal models of nerve
cell loss and the replacement of nerve
cells with MSCs. Since then, we have
awarded two further fellowships and one
PhD studentship, and funded a project
in understanding how stem cells may be
useful in the treatment of dementia.
The direct benefits of this work in
terms of a possible therapy may not
be realised for some years. However, if
successful, these projects will indicate
whether we may be able to replace dead
nerve cells in a widespread and specific
manner – something that has long been
thought impossible.
Future prospects
The society sponsors research through
its Quality Research in Dementia (QRD)
programme. The focus of QRD is to fund
research that has direct benefits on the
quality of life for people with dementia
and their carers – either through
improving care, or by taking us closer to
understanding the causes and finding a
cure for dementia.
A network of 170 lay people with
personal experience of dementia is
actively involved in setting the research
agenda for society, assessing outcomes
and guiding the programme. Every
application that we seek to support
has been prioritised by this consumer
network as well as rigorously peerreviewed by leading international
academic researchers.
Stem cell research has consistently
been identified as a priority by the QRD
consumer network. The Alzheimer’s
Society hopes to receive more
applications for funding research of
this kind. We are delighted to work in
collaboration with the MRC and other
charities to support this important area
of research.
Contact
Alzheimer’s Society
Central Office
Devon House
58 St Katharine’s Way
London
E1W 1JX
Tel: 020 7423 3500
Fax: 020 7423 3501
www.alzheimers.org.uk
The Association of Medical Research
Charities (AMRC) is a membership
organisation of the leading UK charities
that fund medical and health research.
The AMRC aims to provide effective
support and leadership for its members
and the wider charity sector involved in
medical and health research through the
provision of information and guidance
and a strong and credible representative
voice. It does not conduct or provide
funding for medical research, but in this
pack you will find examples of stem cell
research work supported by several of
our member charities.
The AMRC produces policy and position
statements including on human embryo
and stem cell research, and issues
regular announcements on these topics,
which are available on our website
(www.amrc.org.uk). We also provide
web-based information about stem
cell research in our ‘issues in debate’
section covering: current debate,
regulation, legal framework, advisory
system and other guidelines, ethical
principles, charity perspective (with links
to members particularly active in this
area), public opinion, patient and carer
perspective and further information.
Reflecting public opinion – a 2007 British
Market Research Bureau survey showed
that 73 per cent of the UK public
support embryonic stem cell research
under existing or tighter Government
regulation – our statement emphasises
support for such work within a rigorous
regulatory environment, and outlines the
potential benefits of this research to the
understanding, although not necessarily
imminent treatment of a range of
conditions. These include heart disease,
diabetes, cancer, Parkinson’s disease,
stroke, arthritis and mental illness.
The AMRC has been very active in the
debate about modernising the Human
Fertilisation and Embryology Bill, in
particular the use of human-animal
hybrid embryos as a source of stem
cells for research. We provided evidence
and supported amendments to the Bill
in its draft stages and as it progressed
through Parliament. In April 2007,
the AMRC coordinated a joint letter
to the Prime Minister signed by 223
medical research charities and patient
organisations, demonstrating a strong
stance that the law regulating stem cell
research should allow the creation of
hybrid embryos. Two such projects were
licensed under current law, in January
2008 (www.hfea.gov.uk/en/377.html).
Capitalising on our award-winning
partnership with Y Touring Theatre
Company (www.ytouring.org.uk) on
‘Every Breath,’ a play about the use of
animals in medical research, the AMRC
is supporting public dialogue on the
stem cells debate through a further,
similar, partnership. With financial support
from the Medical Research Council,
Department of Health and AMRC
member Action Medical Research,
‘Nobody Lives Forever’ is a play by highly
acclaimed writer Judith Johnson which
explores the debate around stem cell
research. Y Touring’s productions present
a range of arguments, and incorporate
a live discussion between audience and
cast, who stay in role to field questions.
Dr Sophie Petit-Zeman, the AMRC’s
Head of External Relations, is scientific
consultant to Y Touring for the project,
which tours schools and adult audiences
through 2008 and will be the basis for
a ‘Mega-Debate’ project with the Royal
Albert Hall, involving 2,000 young people,
in spring 2009. Dr Petit-Zeman is also
a coordinating member of the project’s
advisory group.
contact details overleaf...
ALZHEIMER’S SOCIETY | ASSOCIATION OF MEDICAL RESEARCH CHARITIES |
BIOTECHNOLOGY AND BIOLOGICAL SCIENCES RESEARCH COUNCIL |
BRITISH HEART FOUNDATION | CANCER RESEARCH UK | DIABETES UK | HUMAN FERTILISATION AND EMBRYOLOGY AUTHORITY |
HUMAN TISSUE AUTHORITY | MEDICAL RESEARCH COUNCIL |
PARKINSON’S DISEASE SOCIETY | THE ROYAL SOCIETY |
SCOTTISH STEM CELL NETWORK | UK STEM CELL BANK | WELLCOME TRUST
Contact
Association of Medical
Research Charities
61 Gray’s Inn Road
London
WC1X 8TL
Tel: 020 7269 8820
Fax: 020 7269 8821
www.amrc.org.uk
The Biotechnology and Biological
Sciences Research Council (BBSRC)
is the UK’s principal funder of basic
and strategic research across the
biosciences, much of which leads
to advances and applications in the
medical, healthcare, agricultural,
veterinary and food sectors. It is funded
mainly by the Government’s Science
Budget through the Department of
Innovation, Universities and Skills (DIUS).
BBSRC-funded research has led to
several commercial developments in
stem cell science, including several spinout companies. These include Axordia novel methods for directed differentiation
of stem cells, and RegenTec - selfassembling injectable scaffold for
tissue repair.
The BBSRC works closely with the other
research councils through Research
Councils UK (RCUK), and is leading the
research councils and relevant government
departments in establishing a UK National
Stem Cell Network to encourage links
between researchers and to encourage
development of an integrated national
stem cell research community, in response
to a report from the UK Stem Cell Initiative
in 2005. The BBSRC is also leading the
research councils in providing focused
communications training for UK stem
cell scientists.
The BBSRC supports pioneering
research in the UK on the basic
biological properties and behaviour of
stem cells from both model organisms
and humans. It has played a key role in
building up the UK’s research capacity
in stem cell science, through targeted
funding initiatives and specialist training
for researchers.
The BBSRC operates a variety of
procedures to address ethical and
societal issues surrounding the
biosciences, including those associated
with stem cell research. It supports
research on both adult and embryonic
stem cells. Our position on stem cell
science is published on the BBSRC
website: http://www.bbsrc.ac.uk/
organisation/policies/position/public_
interest/stem_cells.pdf.
Funding for stem cell research
Through its response mode funding
scheme, the BBSRC supports stem
cell research through grants. It also
funds the training of stem cell scientists
through its Doctoral Training Grants
(DTG) to universities. In both of these
schemes the areas of study are chosen
by the applicants.
BBSRC spends at least £9million a
year supporting stem cell research
and training. It co-funds the UK Stem
Cell Bank with the Medical Research
Council (MRC).
Research
The focus of much BBSRC-funded
research is on providing more
knowledge about how stem cells
regulate their ability, as unspecialised
cells, to either replicate themselves
or turn into particular cell types. This
information is essential if it is to be
possible to use stem cells safely and
effectively in therapies and other
biomedical applications. This often
involves science that is supported in a
complementary way by the BBSRC and
the MRC.
In 2007, the BBSRC and the MRC
started the UK’s biggest ever public
consultation on stem cell research,
funded by the UK Government’s
Sciencewise scheme. The programme
aims to gain an insight into public
expectations, aspirations, and concerns
about this fast moving and challenging
area of science, and includes a
national programme of workshops and
discussion meetings.
Contact Chris St Pourcain, Stem Cell
Biology, BBSRC Senior Programme
Manager, email: christopher.stpourcain
@bbsrc.ac.uk.
A poster display on stem cell science,
‘Hope not Hype’, produced by the BBSRC
and the MRC is accessible at: http://www.
bbsrc.ac.uk/society/meetings/exhibition_
stem_cells/index.html.
contact details overleaf...
ALZHEIMER’S SOCIETY | ASSOCIATION OF MEDICAL RESEARCH CHARITIES |
BIOTECHNOLOGY AND BIOLOGICAL SCIENCES RESEARCH COUNCIL |
BRITISH HEART FOUNDATION | CANCER RESEARCH UK | DIABETES UK | HUMAN FERTILISATION AND EMBRYOLOGY AUTHORITY |
HUMAN TISSUE AUTHORITY | MEDICAL RESEARCH COUNCIL |
PARKINSON’S DISEASE SOCIETY | THE ROYAL SOCIETY |
SCOTTISH STEM CELL NETWORK | UK STEM CELL BANK | WELLCOME TRUST
Contact
Biotechnology and Biological
Sciences Research Council
Polaris House
North Star Avenue
Swindon
SN2 1UH
Tel: 01793 413200
Fax: 01793 413201
www.bbsrc.ac.uk
The British Heart Foundation (BHF) is
the nation’s heart charity, dedicated
to saving lives through pioneering
research, patient care, campaigning
for change and by providing vital
information. We rely on public donations
of time and money to continue our work.
The BHF is the largest independent
funder of cardiovascular science in the
UK. We make an annual investment in
research of around £50 million, and have
a research portfolio of around 1,200
studies at any one time.
In our view, if appropriate safeguards
are in place, bolstered with the stringent
ethical guidelines that exist in the UK,
stem cell research is appropriate as
there are many achievable benefits for
heart patients.
What is heart and
circulatory disease?
Cardiovascular disease – the term that
encompasses all diseases of the heart
and circulation – kills more than 200,000
people each year in the UK. The main
forms are coronary heart disease and
stroke, which together cause around
three quarters of these deaths.
Coronary heart disease is the UK’s
single biggest killer. It occurs when fatty
plaques, known as atherosclerosis,
build up in the vital coronary arteries that
feed the heart. This can be caused by a
combination of environmental, lifestyle
and biological factors such as smoking,
high blood pressure, or the genes we’ve
inherited from our parents. The plaques
can narrow our coronary arteries,
causing chest pain (‘angina’) and they
can rupture and form a blood clot that
completely blocks the artery, causing a
heart attack.
Thanks to research – a significant
part of it BHF-funded – advances in
understanding of the disease have
enabled effective treatment and
prevention strategies to steadily reduce
death rates from heart disease since the
1970s. However, more than 2.6 million
people in the UK are now living with
heart disease, which can be frightening
and debilitating.
How might stem cells help people
with heart disease?
Heart attack can cause loss of heart
muscle cells and the development
of scar tissue, leading to long-term
changes in the heart’s size and shape.
This can leave people vulnerable to
dangerous irregular heart beats and
may lead to heart failure, when the heart
doesn’t pump effectively.
Our hearts have a limited ability to repair,
so heart damage is usually irreversible.
There are currently no treatments to
‘patch up’ damaged tissue with healthy
heart muscle.
Stem cell therapy could be part of the
solution. We hope that in the future,
stem cells could prevent the long-term
consequences of heart attack injury by
repairing or replacing damaged tissue.
We may also be able to use a person’s
own stem cells to grow heart valves,
blood vessels, or even whole hearts
for transplant. These replacements
would be an exact match to the patient
– avoiding immune rejection.
Our understanding of stem cells is still
in its infancy so the BHF is funding
research across many aspects of stem
cell biology to help us understand
this exciting potential therapy. This
knowledge will equip us to move forward
and give the best chance of success for
large studies with patients in the future.
continued overleaf...
ALZHEIMER’S SOCIETY | ASSOCIATION OF MEDICAL RESEARCH CHARITIES |
BIOTECHNOLOGY AND BIOLOGICAL SCIENCES RESEARCH COUNCIL |
BRITISH HEART FOUNDATION | CANCER RESEARCH UK | DIABETES UK | HUMAN FERTILISATION AND EMBRYOLOGY AUTHORITY |
HUMAN TISSUE AUTHORITY | MEDICAL RESEARCH COUNCIL |
PARKINSON’S DISEASE SOCIETY | THE ROYAL SOCIETY |
SCOTTISH STEM CELL NETWORK | UK STEM CELL BANK | WELLCOME TRUST
Progress made by BHF
research teams
Some researchers have suggested
that injection of a patient’s own bone
marrow stem cells into their heart after
heart attack may improve the heart’s
function. BHF researchers in Leicester
investigated and found that, rather than
transforming into healthy heart cells,
bone marrow stem cell therapy may
work by protecting vulnerable heart
tissue after heart attack.
A team at the UCL Institute of Child
Health revealed that stem cells in the
outermost layer of the heart can be
guided by a specific protein to move
deeper inside and help to repair a failing
adult heart. Research funded by the
BHF and the Medical Research Council
(MRC) showed that a protein called
‘thymosin ß4’ can stimulate new blood
vessel generation from these cells in
adult mice. Previously it was thought that
cells within the adult heart are dormant
and that all cells that contribute to heart
repair travel from the bone marrow.
A team at King’s College, London, has
suggested a possible future role for stem
cells in protecting heart bypass grafts
against atherosclerosis. The researchers
found that after surgery the cells lining
the grafted blood vessel are lost leaving it vulnerable to plaque build-up before being replaced by stem cells from
the bone marrow and blood. Also, in
mice engineered to have high cholesterol
levels, there were fewer stem cells
and accelerated atherosclerosis. This
suggests that increasing our stores of
stem cells might help to protect patients
from atherosclerosis after heart surgery.
The team is currently investigating in
detail the signals that determine how
stem cells can transform into blood
vessel wall cells.
Scientists at the National Heart and
Lung Institute in London have developed
a biological ‘scaffold’ on which human
stem cells can survive, grow and begin
to develop into heart valves. Engineered
human valves may in future replace
metal and tissue replacements that have
limitations in treating children and adults
with valve defects.
Contact
British Heart Foundation
14 Fitzhardinge Street
London
W1H 6DH
Tel: 020 7935 0185
bhf.org.uk
Cancer Research UK is the world’s
largest non-governmental organisation
dedicated to cancer research. We
are committed to tackling cancer
by understanding its causes and
investigating how best to diagnose, treat
and prevent it. We also fund research
aimed at providing the best possible
support and information to cancer
patients and their families. Cancer
Research UK is the largest single funder
of cancer research in the UK, funding
more than 4,250 scientists, doctors and
nurses throughout the country. We are
also the European leader in anti-cancer
drug development. In the financial year
of 2006/07, our total scientific spend
was £315 million.
is the cause of around a quarter of all
deaths in the UK. In 2005, there were
around 153,490 deaths from cancer.
Over one-fifth of these were from lung
cancer, and a quarter from cancers of
the large bowel, breast and prostate.
Cancer is highly complex and is still
only partly understood. There are more
than 200 different types of cancer that
can occur anywhere in the body, all with
different causes and symptoms and
requiring different types of treatment. It is
only through a greater understanding of
the biology of the disease and its causes
that better treatments, and better
diagnosis and prevention strategies, will
be developed for the future.
Stem cells are ‘starter cells’ that have the
potential to develop into many different
cell types in the body. When a stem cell
divides, the resulting cells can either
remain as stem cells or, under the correct
conditions, become another type of cell
with a more specialised function, such as
a muscle cell, a red blood cell or a brain
cell. Scientists believe that stem cells may
also play a direct role in the development
of cancer, as some tumours are thought to
develop when normal stem cells become
faulty. As a stem cell’s fate is controlled by
a number of molecular factors, increasing
our understanding of these could help the
development of new anti-cancer drugs.
Our research
Stem cell research and cancer
Cancer in the UK
Cancer is a major disease in the UK.
There are around 285,000 new cases of
cancer registered each year and around
one in three of us will develop cancer at
some time in our lives. The disease is
more likely to develop later in life, with
around 65 per cent of cases diagnosed
in people over the age of 65. Cancer
Cancer occurs when cells start to
behave abnormally and the regular
mechanisms that control cell growth and
division are faulty. Cancer cells share
many characteristics with stem cells.
Consequently a greater understanding
of stem cells may be vital to the eventual
control of cancer.
Many patients suffering from serious
diseases, including cancer, could
potentially benefit from carefully
regulated research on embryonic stem
cells. Although Cancer Research UK
does not currently use human embryos
for research purposes, we recognise the
potential of using embryonic stem cells
for research projects which otherwise
could not be accomplished.
continued overleaf...
ALZHEIMER’S SOCIETY | ASSOCIATION OF MEDICAL RESEARCH CHARITIES |
BIOTECHNOLOGY AND BIOLOGICAL SCIENCES RESEARCH COUNCIL |
BRITISH HEART FOUNDATION | CANCER RESEARCH UK | DIABETES UK | HUMAN FERTILISATION AND EMBRYOLOGY AUTHORITY |
HUMAN TISSUE AUTHORITY | MEDICAL RESEARCH COUNCIL |
PARKINSON’S DISEASE SOCIETY | THE ROYAL SOCIETY |
SCOTTISH STEM CELL NETWORK | UK STEM CELL BANK | WELLCOME TRUST
Cancer Research UK–funded scientists
carried out pioneering research using
adult stem cells. Previously, scientists
believed that only embryonic stem
cells are capable of giving rise to other
cell types in the body. However, our
scientists showed that adult stem cells
in the bone marrow are capable of
developing into kidney, liver and epithelial
cells. Such findings raise the possibility
that doctors will be able to use blood
cells to repair kidney and liver damage
caused by cancer and other diseases.
Our scientists are also studying
molecules that might be important in
stem cell behaviour. For example, a
study published in 2003 used mouse
embryonic stem cells to discover
important information about how cancer
spreads. The study found key similarities
between how cancer cells spread out
from a tumour to move around the body
and the movement of stem cells as they
form new tissues during an embryo’s
development. The study identified a
molecule involved in this process that
could potentially be used as a drug
target to help stop cancer spread.
Some of our current research is focused
on how stem cells in the skin decide
to become specialised skin cells called
keratinocytes. When this process goes
wrong skin cancer can develop, therefore
this work could help scientists find ways
to prevent and treat skin cancer.
Future research
Ongoing studies that increase our
understanding of how stem cells
respond to signals from their local
environment and decide whether to selfrenew, specialise or die could ultimately
provide insights into the process by
which cells become cancerous. These
studies will also help scientists identify
specific targets for drug development to
remove ‘cancer stem cells’.
Future stem cell research could also
uncover ways of improving outcomes
after treatment for cancer. The ultimate
application in cancer may be in the
ability to regenerate or replace normal
tissue following surgical removal of
cancerous tissue or its destruction by
chemotherapy or radiotherapy.
Contact
Cancer Research UK
P.O. Box 123
Lincoln’s Inn Fields
London
WC2A 3PX
Tel (Supporter Services):
020 7121 6699
Tel (Switchboard):
020 7242 0200
Fax: 020 7269 3100
www.cancerresearchuk.org/
Registered charity number 1089464.
Diabetes UK is the leading UK charity
for people with diabetes. With more than
170,000 members, we are one of the
largest patient organisations in Europe.
Our mission is to ‘improve the lives of
people with diabetes and work towards a
world without diabetes’. The human and
health burden of diabetes is staggering,
as is the economic cost. The only way
to tackle this is to improve the treatment
of diabetes, prevent it from developing
in those at risk and, ultimately, find a
cure. For this, research is essential and
therefore at the heart of the organisation.
Diabetes research has a distinguished
history in the UK, and Diabetes UK
(formerly the British Diabetic Association)
has played a key role since its formation
in 1934. Over this time, with the exception
of the pharmaceutical industry, Diabetes
UK has been one of the major providers
of funds for diabetes research in the UK.
Research funded by Diabetes UK has
been of the highest scientific quality with
national and international impact.
What is diabetes?
Diabetes is a serious condition where
the amount of glucose in the blood is
much higher than it should be because
the body cannot use it properly. Broadly
speaking, there are two major types.
Type 1 diabetes occurs when the body
destroys its own insulin producing
cells in the pancreas leaving the body
completely without insulin. It tends to
appear before the age of 40 in children
or young people and injection of insulin
is needed from diagnosis.
Type 2 diabetes develops when the
body can still make some insulin, but
not enough, or when the insulin that
is produced does not work as well
because the body is less sensitive to
it. It tends to appear in people over the
age of 40 but more and more people are
being diagnosed with it at earlier ages
with some as young as seven. Type
2 diabetes is progressive – over time
people may have to shift from managing
the condition by diet and exercise and/or
tablets to using a combination of diet,
exercise, tablets and insulin injections to
achieve control.
Diabetes – the challenge
The number of people with diabetes
is increasing at an alarming rate and
it represents one of the biggest public
health problems for the 21st century.
• Diabetes occurs in men, women, the
young and old and in all races. No
group is spared.
• There is no known cure for diabetes
and available treatments are limited
in controlling the devastating
consequences of the condition.
• People with diabetes (or their carers)
are responsible for the day-to-day
management of their condition.
• Diabetes affects 5 per cent of the
world’s population and its prevalence
is doubling every generation.
• The International Diabetes Federation
estimates that in 2005 around 333
million people in the world aged 20-79
had diabetes.
• More than 2 million people in the UK
have been diagnosed with diabetes.
This number is predicted to reach 3
million by 2010.
• It is estimated that up to another
500,000 people in the UK have
diabetes but do not know they have it.
• There are up to 345,000 people in
the UK with Type 1 diabetes. This is
caused by an absolute lack of the
hormone insulin, resulting from loss of
the body’s pancreatic islet beta cells.
• Around 2 million people have Type
2 diabetes, representing about
85 per cent of diabetes cases.
Type 2 diabetes is due to varying
combinations of insulin deficiency and
insulin resistance.
• The incidence of Type 1 diabetes in
children is rising at a rate of 3-4 per
cent a year. We do not know why.
continued overleaf...
ALZHEIMER’S SOCIETY | ASSOCIATION OF MEDICAL RESEARCH CHARITIES |
BIOTECHNOLOGY AND BIOLOGICAL SCIENCES RESEARCH COUNCIL |
BRITISH HEART FOUNDATION | CANCER RESEARCH UK | DIABETES UK | HUMAN FERTILISATION AND EMBRYOLOGY AUTHORITY |
HUMAN TISSUE AUTHORITY | MEDICAL RESEARCH COUNCIL |
PARKINSON’S DISEASE SOCIETY | THE ROYAL SOCIETY |
SCOTTISH STEM CELL NETWORK | UK STEM CELL BANK | WELLCOME TRUST
• The increase in Type 2 diabetes is
closely linked to an ageing population
and rapidly rising numbers of obese or
overweight people.
• Diabetes is the leading cause of
kidney failure, blindness in adults, and
amputations. It can lead to impotence,
can affect mental health and wellbeing,
and is a major risk factor for heart
disease, stroke and birth defects.
• On average, life expectancy is reduced
by 15 years in people with Type 1
diabetes and by up to seven years
in people with Type 2 diabetes. In
the next 10 years there will be a 25
per cent increase in the number of
diabetes-related deaths.
• Diabetes has a large and growing
financial impact on the NHS. The
clinical supervision and care of people
with diabetes is estimated to consume
around 10 per cent of the NHS budget
(about £25 million a day) and 10 per
cent of hospital in-patient resources.
The NHS spend on diabetes will rise to
around 10 per cent of the NHS budget
by 2011.
• A National Service Framework outlining
the standards of care that should be
expected by people with diabetes has
been in place since 2003 but its full
implementation across the NHS has
yet to be achieved.
Diabetes UK research
Diabetes UK is committed to funding
research which has the potential to
make a difference to the lives of people
with diabetes in the short term (three to
five years), medium term (five to seven
years) and longer term (10 years and
beyond). Research applications are
considered for funding on the basis
of their scientific merit, the potential
difference the research could make to
the lives of people with diabetes and
value for money.
Contact
Research team
Diabetes UK
10 Parkway
London
NW1 7AA
Tel: 020 7424 1076
Fax: 020 7424 1001
Email: research@diabetes.org.uk
www.diabetes.org.uk
Registered charity number 215199.
Stem cell research is a key area of interest
worldwide as it offers hope to many that
in the not too distant future, we may be
able to offer exciting new treatments
for a wide range of conditions including
diabetes. Consultation exercises led by
Diabetes UK have shown that, particularly
on ethical issues, people with diabetes
and their carers are overwhelmingly
in support of stem cell research. In
December 2001, Diabetes UK’s Board
of Trustees agreed to support stem cell
research both publicly and financially.
Diabetes UK remains committed to
stem cell research and to ensuring it
does everything it can to maximise the
opportunities for working towards novel
therapies and a cure for diabetes.
The Human Fertilisation and
Embryology Authority (HFEA) regulates
centres carrying out licensed fertility
treatment and human embryo research
throughout the UK. In stem cell
research, the HFEA regulates those
projects that use human embryos to
create stem cell lines, scrutinising the
steps from the creation of the embryo
until the creation of the stem cell line.
As the UK’s regulator, the HFEA’s role
is to provide ethical and procedural
scrutiny of the creation and handling
of human embryos to maintain public
confidence in the treatment and
research sectors. The framework for
this is set out in the Human Fertilisation
and Embryology Act 1990 (HFE Act).
This states that no research may be
conducted on human embryos unless it
is licensed by the HFEA.
The Act was amended in 2001 to allow
the use of embryos for stem cell research
and consequently the HFEA has the
responsibility for regulating all human
embryonic stem cell research in the UK.
Anyone wishing to use human embryos
for stem cell research must apply to the
HFEA for a licence before they carry
out any work. The researchers must
demonstrate to the HFEA’s satisfaction
that the work proposed is legal,
necessitates the use of human embryos
and is a desirable use of human embryos.
The current law
The authority and licence committees
Under the HFE Act (as amended) any
research using human embryos
must relate to one or more of the
following purposes:
•T
o promote advances in the treatment
of infertility
•T
o increase knowledge about the
causes of congenital diseases
•T
o increase knowledge about the
causes of miscarriage
•T
o enhance knowledge in the
development of more effective
contraception
•D
etection of genetic or chromosomal
abnormalities before implantation
•T
o increase knowledge about the
development of embryos
•T
o increase knowledge about serious disease
•T
o enable any such knowledge to be
applied in developing treatment for
serious disease.
Decisions on research applications are
made by a licence committee made up
of members of the HFEA. Members bring
to the HFEA a broad range of expertise,
from medicine to law and religion to
philosophy. The members determine
HFEA policies and review treatment
and research licence applications. They
are appointed by UK health ministers in
accordance with the guidance from the
Commissioner for Public Appointments
(the ‘Nolan’ guidelines).
To ensure that the HFEA has an objective
and independent view, the HFE Act
requires that the chair, deputy chair and
at least half of the HFEA members are lay
members (neither doctors nor scientists
involved in human embryo research or
providing infertility treatment).
Applying for a research licence
The law also explicitly prohibits
some key activities
•T
he genetic structure of the cell must
not be altered while it forms part of an embryo
•R
esearch embryos must be destroyed
on or before 14 days of development
•N
o embryo created or used in research
may be implanted in a woman (or any animal).
The HFEA has the power to grant
research licences for up to three
years for individual research projects.
All research licence applications and
renewals are evaluated by an HFEA
Research Licence Committee.
The majority of researchers contact
the HFEA to discuss their proposed
research before they submit an
application for a licence.
continued overleaf...
ALZHEIMER’S SOCIETY | ASSOCIATION OF MEDICAL RESEARCH CHARITIES |
BIOTECHNOLOGY AND BIOLOGICAL SCIENCES RESEARCH COUNCIL |
BRITISH HEART FOUNDATION | CANCER RESEARCH UK | DIABETES UK | HUMAN FERTILISATION AND EMBRYOLOGY AUTHORITY |
HUMAN TISSUE AUTHORITY | MEDICAL RESEARCH COUNCIL |
PARKINSON’S DISEASE SOCIETY | THE ROYAL SOCIETY |
SCOTTISH STEM CELL NETWORK | UK STEM CELL BANK | WELLCOME TRUST
Existing licence holders liaise directly with
HFEA staff on renewals and evaluations.
The HFEA is committed to processing
90 per cent of research licence
applications within three months
of receipt of a properly completed
application form. Research ethics
approval should have been sought from
a properly constituted ethics committee
before an application is made.
An administration fee of £500 (as
at March 2008) is payable for most
projects. Projects involving the
derivation of human embryonic stem
cells or cell nuclear replacement incur
an administration fee of £750 which
reflects on the increased complexity
and rigour required for the licensing of
such projects.
Following receipt of the application and
the fee, the HFEA will commission peer
reviewers for the project to determine
whether the application:
• Comes within the statutory
requirements of the Human Fertilisation
and Embryology Act.
• Requires human embryos to fulfil its
aims and objectives.
• Requires the numbers and types of
embryos described in the application.
• Meets the requirements of the HFEA
Code of Practice.
If the peer reviews are satisfactory,
the HFEA initiates visits to proposed
research sites. These visits are
conducted by HFEA regulation staff
and independent scientific inspectors.
Fuller teams may take part in the visits
if the research application is of a novel
or complex nature. The primary focus of
these visits is to review proposed project
protocols, inspect research laboratories
and to meet research teams.
The application, the peer reviews and
the site visit report are then examined
by a Research Licence Committee.
Depending on the nature of the
research, the committee may also
examine research papers and public
comment obtained through consultation.
In order for a licence to be offered, the
Licence Committee has to agree that
the proposals pass three tests – are they
legal, are they necessary and are they
desirable? If a licence is granted, the
project is then inspected on a regular
basis and the researchers are required to
keep detailed records of how they
use the embryos and the progress of
their research.
Further details, including guidance on
completing a research application
and information about current and
proposed research, can be found on
the HFEA website.
Contact
HFEA
21 Bloomsbury Street
London WC1B 3HF
Tel: 020 7291 8200
Email: admin@hfea.gov.uk
www.hfea.gov.uk
The Human Tissue Authority (HTA) was
established in 2005 to regulate the
removal, storage, use and disposal of
human bodies, organs and tissue for a
number of Scheduled Purposes set out
in the Human Tissue Act 2004 (HT Act).
Informed consent, freely given, is the
cornerstone of the legislation.
The HTA is also a Competent Authority
under the EU Tissues and Cells Directive
(EUTCD), now transposed into UK law
by the Human Tissue (Quality and Safety
for Human Application) Regulations
2007 (the Regulations 2007).
As part of our remit under the HT Act,
we license the storage of tissues and
cells (excluding cell lines) used for
research, with certain exemptions. Under
the Regulations 2007 we also license a
range of activities involving tissues and
cells (including cell lines) used for human
application. The removal, storage and
use of tissue from living people as part
of diagnosis or treatment are not part of
our statutory remit.
Our strategic aim
We aim to create a regulatory system for
the removal, use and disposal of human
tissue and organs that is clear and
consistent and in which professionals,
patients, families and members of the
public have confidence.
The HTA believes that good regulation
facilitates good science which in turn leads
to improved healthcare. We work closely
with interested parties, including the
research and stem cell communities, to
develop our licensing standards, codes of
practice and other advice and guidance.
We adhere closely to the principles of
better regulation, and work with other
regulatory and funding bodies to reduce
the burdens on the sectors we regulate.
By raising standards, regulation
increases the confidence not only of
professionals, but also the public who
donate their tissue for potentially lifesaving research. We believe that, if the
public know there is regulation in this
area, their confidence and willingness to
donate will increase.
The Human Tissue Act
The Human Tissue Authority (HTA)
regulates the removal, storage and use
of relevant material for a number of
Scheduled Purposes (such as research,
anatomical examination, public display,
and education and training) set out in
the HT Act. Relevant material is defined
as material that has come from a human
body and consists of, or includes,
human cells (other than gametes and
embryos). The HTA also licenses a
number of activities under the HT Act,
such as post mortem examination.
The HT Act covers England, Wales
and Northern Ireland. There is separate
legislation in Scotland (the Human
Tissue (Scotland) Act 2006) and the
HTA performs certain tasks on behalf
of the Scottish Government. These
include approving ethical aspects of the
donation of solid organs, bone marrow
and peripheral blood stem cells from
living people.
Both the HT Act and HT (Scotland)
Act came into force on 1 September
2006. Again, consent (referred to as
‘authorisation’ in the Scottish legislation)
is the cornerstone of the legislation.
Regulation of stem cells stored
for research
In England, Wales and Northern Ireland,
the storage of relevant material for
research requires a licence under the
HT Act, unless it is used for an ethicallyapproved research project. Material
created outside the human body is
exempt from the HT Act’s licensing
requirements, so storage of cell lines
and stem cell lines for research is not
licensable. Storage of the tissues and
cells used to create these lines may
require a licence. If cell lines or stem
cell lines are to be used for human
application in the future, an HTA licence
under the Regulations 2007 is needed
(as described overleaf).
continued overleaf...
ALZHEIMER’S SOCIETY | ASSOCIATION OF MEDICAL RESEARCH CHARITIES |
BIOTECHNOLOGY AND BIOLOGICAL SCIENCES RESEARCH COUNCIL |
BRITISH HEART FOUNDATION | CANCER RESEARCH UK | DIABETES UK | HUMAN FERTILISATION AND EMBRYOLOGY AUTHORITY |
HUMAN TISSUE AUTHORITY | MEDICAL RESEARCH COUNCIL |
PARKINSON’S DISEASE SOCIETY | THE ROYAL SOCIETY |
SCOTTISH STEM CELL NETWORK | UK STEM CELL BANK | WELLCOME TRUST
The EU Tissue and Cells Directive
The EU Tissues and Cells Directive
(EUTCD) creates a common framework
that ensures high standards of tissues
and cells for human application across
the EU community. Its primary aim
is to ensure the quality, safety and
traceability of tissue and cells used for
treatment. It also aims to support the
exchange of tissues and cells between
member states.
On 5 July 2007, the EUTCD was
transposed into UK law via the Human
Tissue (Quality and Safety for Human
Application) Regulations 2007 (the
Regulations 2007). The HTA is one of
two competent authorities in the UK
(including Scotland) for implementing
the directive. The other is the Human
Fertilisation and Embryology Authority
(HFEA), which regulates the creation and
use of embryos and gametes. The use
of embryos to derive stem cells must be
licensed and approved by the HFEA.
The Regulations 2007 require the HTA
to regulate the procurement, testing,
processing, storage, distribution and
import and export of tissues and cells for
human application.
Regulation of stem cells for
human application
Since 5 July 2007, human tissue or cells
intended for human application must
comply with all the requirements of the
Regulations 2007 and HTA Directions
001/2006, 002/2007 and 004/2007. This
includes stem cell lines isolated from any
human source, created with the intention
of being used for human application.
Where an activity is already regulated by
the Medicines and Healthcare products
Regulatory Agency (MHRA), only the
donation, procurement and testing of
cells is regulated by the HTA.
The Regulations 2007 make it an offence
to carry out the following activities without
either an HTA licence or under third party
agreement with a licensed establishment
on whose behalf the third party is acting
on. The activities are procurement,
processing, testing, distribution and
import and export in relation to tissues or
cells for human application.
Any establishment storing tissues or
cells (for more than 48 hours) for human
application may lawfully carry out this
activity only if licensed by the HTA under
the Regulations 2007. Storage, unlike the
activities listed above, cannot be carried
out under a third party agreement.
Contact
Human Tissue Authority
Finlaison House
15–17 Furnival Street
London
EC4A 1AB
Tel: 020 7211 3400
E-mail: enquiries@hta.gov.uk
www.hta.gov.uk
The Medical Research Council
supports the best scientific research
to improve human health. Its work
ranges from molecular level science
to public health medicine and has
led to pioneering discoveries in our
understanding of the human body and
the diseases which affect us all.
For 90 years, MRC researchers have
been the driving force behind a stream
of remarkable advances that have
transformed medicine and are benefiting
people all over the world.
Sir Martin Evans, known as the ‘father
of stem cell research’ and supported by
the MRC between the 1970s and 1990s,
shared the 2007 Nobel Prize in Physiology
or Medicine for his work introducing
specific gene modifications into mice using
embryonic stem cells. This work was the
basis for future targeted manipulation
of genes and experimental mammalian
genetics – everyday tools used by
scientists to improve the understanding of
the influence of genes on disease.
Today, research at MRC centres,
universities and hospitals throughout the
UK extends from the laboratory to the
bedside and beyond, and is building on
past achievements to tackle the major
health challenges of the 21st century.
The MRC has a major coordinating role
both nationally and internationally. It is
the driving force behind the International
Stem Cell Forum (ISCF), which it
launched in 2003 along with eight other
international funding agencies. The MRC
is also part of the UK Stem Cell Initiative
and funds the UK Stem Cell Bank. It
collaborates with the Biotechnology
and Biological Sciences Research
Council (BBSRC) and the Parkinson’s
Disease Society (PDS) in the funding of
fellowships in stem cell research.
In 2007, the Biotechnology and
Biological Sciences Research Council
(BBSRC) and the MRC started the UK’s
biggest ever public consultation on
stem cell research, funded by the UK
Government’s Sciencewise scheme. The
programme aims to gain an insight into
public expectations, aspirations, and
concerns about this fast moving and
challenging area of science, and includes
a national programme of workshops and
discussion meetings.
The MRC is reviewing its stem cell and
regenerative medicine research strategy
with an aim to develop joint strategies
with the Technology Strategy Board
(TSB) – the executive non-departmental
public body which promotes innovation
in the UK – and the National Institute for
Health Research (NIHR), for academic
and industry cooperation. Under the
single integrated health research
strategy with NIHR, the MRC will lead
the stem cell area.
MRC research on stem cells
Diseases such as diabetes, Alzheimer’s,
Parkinson’s, cancer, diabetes and
heart disease are some of the most
devastating conditions of our time, and
are becoming an ever greater priority
with an ageing population. Stem cell
therapy is emerging as a revolutionary
new way to treat these. If scientists can
work out how to control the growth
of stem cells, the primitive cells that
generate different kinds of tissue, they
might eventually be able to use stem
cells to provide a plentiful supply of
healthy transplant material to repair any
diseased or damaged tissue or organ.
The MRC is funding research aimed at
making this a reality. Our scientists are
investigating all aspects of stem cell
biology in both embryonic stem cells (ES
cells), which have the ability to develop
into virtually any human tissue, and adult
stem cells which replace damaged or
depleted cells in mature tissues. Work
on the basics of stem cells’ identity,
propagation, and how they give rise
to specialised cell types, will help
scientists learn how to control stem cell
self-renewal and differentiation in order
to achieve safe, efficient, large-scale
production of defined cell types. They
must also develop ways of delivering
stem cells and monitoring their migration
within tissues and find out how to
combat immune rejection.
continued overleaf...
ALZHEIMER’S SOCIETY | ASSOCIATION OF MEDICAL RESEARCH CHARITIES |
BIOTECHNOLOGY AND BIOLOGICAL SCIENCES RESEARCH COUNCIL |
BRITISH HEART FOUNDATION | CANCER RESEARCH UK | DIABETES UK | HUMAN FERTILISATION AND EMBRYOLOGY AUTHORITY |
HUMAN TISSUE AUTHORITY | MEDICAL RESEARCH COUNCIL |
PARKINSON’S DISEASE SOCIETY | THE ROYAL SOCIETY |
SCOTTISH STEM CELL NETWORK | UK STEM CELL BANK | WELLCOME TRUST
Around the UK, MRC researchers are
carrying out pioneering research into
embryonic stem cells, including what
determines the type of cell they become
and how to direct this process. They are
also investigating the role of stem cells in
cancer. For example, a study of identical
twins, by Professor Tariq Enver at the
MRC Molecular Haematology Unit and
colleagues elsewhere, has for the first
time confirmed the existence of cancer
stem cells that cause the most common
type of childhood leukaemia.
MRC teams are also using stem cells to
model human disease. For instance, they
have used embryonic stem cells to create
the first mouse model of human Down’s
syndrome, which is caused by the
presence of three copies of chromosome
21 instead of the normal two.
The ability of stem cells to turn into
specific types of cells has led scientists to
attempt to use them to repair or replace
parts of the body. Stem cells may offer
hope to patients with brain and central
nervous system diseases and injuries.
Professor Steve Dunnett and colleagues
are working on developing cells for
future transplantation into patients with
Huntington’s and Parkinson’s diseases.
They use embryonic or germ line (egg
or sperm) cells or a type of foetal cell to
provide reliable and renewable sources of
nerve cells, with the aim of restoring brain
function. At the University of Nottingham,
Professor Philip Bath has discovered that
bone marrow cells may be able to repair
the damage done to the brain by a stroke.
Stem cells also have the potential to
restore sight to people with diseases and
injuries affecting their eyes. MRC-funded
scientists at the University College
London Institute of Ophthalmology
are researching stem cell transplants
to treat people with hereditary retinal
disease and age-related macular
degeneration – two major causes of
vision problems and blindness in the
UK. There is also hope for many people
with bone problems, such as arthritis
and osteoporosis patients, people with
bone injuries and those who need joint
replacement operations. MRC-funded
scientists at Imperial College London
have successfully grown cartilage cells
from human embryonic stem cells.
Stem cells could be a valuable aid in
drug development. Large numbers of
a particular type of cell could be grown
for use by researchers when testing
new therapies. This would be especially
useful for screening potential drugs for
toxicity or their impact on a disease
and might reduce the need for animal
testing. The MRC is part of the Stem
Cells for Safer Medicines Initiative, a
new public-private partnership involving
the Government, research councils and
industry, which is encouraging the use of
stem cells in early drug discovery.
Contact
MRC
20 Park Crescent
London
W1B 1AL
Tel: 020 7636 5422
Fax: 020 7436 6179
www.mrc.ac.uk
Parkinson’s is the second most common
neurodegenerative condition in the UK,
and most of us will know someone
who has been affected. The Parkinson’s
Disease Society (PDS) is the leading
charity dedicated to supporting people
living with Parkinson’s. We believe all
people with Parkinson’s and their families
should benefit from quality care and
support regardless of where they live in
the UK. We are achieving this by building
a better understanding of the condition
and voicing the issues of people with
Parkinson’s on a national and local level.
At present, there is no cure for
Parkinson’s and the causes are unknown.
The Parkinson’s Disease Society is
working hard to bridge this gap by
stimulating innovative research into the
condition. We have a challenging agenda
that supports high-quality research at
the forefront of the international field. To
date, we have invested £35 million in
pioneering science throughout the UK.
Our goal is to improve the treatment and
care for people with Parkinson’s and to
find a cure.
What is Parkinson’s?
Parkinson’s is a progressive, neurological
condition that affects everyday activities,
such as walking, talking and writing. It
was named after the London doctor
Dr James Parkinson, whose famous
study An Essay on the Shaking Palsy
established Parkinson’s as a recognised
medical condition in 1817.
Two centuries on, we now know that
Parkinson’s develops when nerve
cells are lost from the middle of the
brain, in a region called the substantia
nigra. These cells make dopamine, a
chemical messenger that controls and
coordinates movement. When around
80 per cent of the cells die, not enough
dopamine is produced, which means the
brain cannot work properly. This is when
the symptoms of Parkinson’s appear.
Scientists are still trying to understand
how and why dopamine-producing
nerve cells die earlier than they should.
Around 120,000 people in the UK have
Parkinson’s and 10,000 new cases are
diagnosed every year. The condition
affects both young and old, and men
are slightly more likely to develop
Parkinson’s than women. Typically, the
symptoms appear after the age of 55,
although one in 20 of those diagnosed
will be under 40 years old.
Parkinson’s is a complicated disorder
that changes over a person’s lifetime.
There is a broad range of symptoms,
which affects each person differently.
No two people will have the same
symptoms or the same rate of
progression. People may have a
tremor, muscle stiffness or move slowly
– these are the key characteristics of
the condition. People with Parkinson’s
may also experience other symptoms
not related to movement, such as
problems with their bladder and bowel,
speech and swallowing, sleeping and
memory, as well as hallucinations and
depression. This has a tremendous
impact on how a person thinks and
feels and affects simple, everyday
activities and relationships – all that we
take for granted.
How can stem cells help people
with Parkinson’s?
Scientists are searching for a cure for
Parkinson’s – a treatment that allows
people to lead a normal life, free of
symptoms. The symptoms and effects of
Parkinson’s are currently managed with
a combination of drugs, physiotherapy,
occupational therapy, and speech
and language therapy. Some people
also have surgery such as deep brain
stimulation. Unfortunately, a person’s
response to anti-Parkinson’s medication
can fluctuate during the course of
treatment and eventually wear off.
These drugs also have some unpleasant
side effects and are therefore not a
permanent solution. Similarly, surgery
can be helpful for some people but
carries a number of potential risks. For
these reasons, a huge effort is currently
underway to develop new and better
treatment options.
One exciting avenue is using stem cells
to repair the brain. This is called cell
replacement therapy. Stem cells are
the building blocks of the human body
– they are ‘blank’ cells that have the
potential to turn into every type of cell
and, therefore, make every tissue and
organ. The idea behind healing the brain
is simple - we know that the symptoms
of Parkinson’s appear when the supply
of dopamine falls in the brain. Therefore,
replacing the dead dopamine-producing
nerve cells with healthy cells would raise
dopamine levels. The cells in the different
parts of the brain would then be able to
communicate, and this would relieve the
symptoms. Scientists are working hard
on two cell replacement strategies:
continued overleaf...
ALZHEIMER’S SOCIETY | ASSOCIATION OF MEDICAL RESEARCH CHARITIES |
BIOTECHNOLOGY AND BIOLOGICAL SCIENCES RESEARCH COUNCIL |
BRITISH HEART FOUNDATION | CANCER RESEARCH UK | DIABETES UK | HUMAN FERTILISATION AND EMBRYOLOGY AUTHORITY |
HUMAN TISSUE AUTHORITY | MEDICAL RESEARCH COUNCIL |
PARKINSON’S DISEASE SOCIETY | THE ROYAL SOCIETY |
SCOTTISH STEM CELL NETWORK | UK STEM CELL BANK | WELLCOME TRUST
• Using stem cells to grow new nerve
cells in the laboratory, and then
transplanting them into a patient’s brain.
• Encouraging stem cells already in
patients’ brains to move towards the
part damaged in Parkinson’s, and then
triggering them to make new nerve cells.
Over the last 20 years, researchers
have been investigating foetal tissue
transplants as a possible treatment for
Parkinson’s. In research that is strictly
regulated by the Human Fertilisation and
Embryology Authority (HFEA), tissue
that contains stem cells is taken from
the mid-brain of human foetuses and
implanted into the brains of patients with
Parkinson’s. Up to now, around 400
people worldwide have taken part in
these experimental trials. Some people
have reported improvements in their
Parkinson’s symptoms.
Scientists do not understand why foetal
tissue transplants appear to work in
some people but fail in others. They
think that it is better suited for younger
people (under 60 years old) with milder
Parkinson’s. Also, researchers now
believe that the answer may not lie with
transplanting foetal tissue but growing
new nerve cells from stem cells in the
laboratory. For that reason, scientists
are now looking at other sources of
stem cells from embryo, foetal and
adult tissues. To develop a successful
treatment, a lot more research needs to
be done to optimise and standardise cell
transplantations. For example:
• What the best source of stem cells?
• How to grow large, reliable quantities
of cells that are appropriate for use.
•H
ow to change a stem cell into the
right type of nerve cell.
•H
ow to be certain that stem cells are
put into and stay in the right part of
the brain.
•H
ow to stop transplanted stem cells
from dying or growing uncontrollably
into tumours.
•H
ow to be sure that transplanted cells
form the right connections in the brain.
Unlike cell transplantations, scientists
are still debating the second of these
strategies - whether the brain has built-in
machinery that can repair the damage
in Parkinson’s. We know the adult brain
has limited sites that house stem cells
and generate new nerve cells, albeit at
a very slow pace. The idea is to round
up these stem cells into the region of the
brain affected by Parkinson’s and trigger
them to make dopamine-producing
nerve cells at a much faster speed.
Initial animal studies appear to suggest
that it is possible to recruit stem cells
using a combination of growth factors.
However, the next challenge is to turn
the stem cells into dopamine-producing
nerve cells. To achieve this, scientists
are experimenting with different recipes
of several growth factors, drugs and
proteins that stick to genes.
The Parkinson’s Disease Society and
stem cell research
Stem cell therapy is still in its infancy and
we know that there are many hurdles to
overcome to ensure that researchers can
move as swiftly as possible towards a
cure. We have been campaigning strongly
to make sure that all avenues of stem cell
research, including use of embryonic stem
cells, cell nuclear transfer and cytoplasmic
hybrid cells, remain open within the
rigorous ethical and regulatory framework
that exists within the UK.
Since 2001, the society has invested more
than £1.6 million in 13 different stem cell
research projects throughout UK. We also
work closely with other organisations, such
as the Medical Research Council (MRC),
to develop joint research programmes.
Our scientists are investigating embryonic,
foetal and adult stem cells. They are
working to understand the basics of stem
cell biology, how they turn into nerve cells
and can be used as a treatment for people
with Parkinson’s. More details on our stem
cell projects can be found at
www.parkinsons.org.uk/research.
Contact
Parkinson’s Disease Society
215 Vauxhall Bridge Road
London
SW1V 1EJ
Tel: 020 7931 8080
Fax: 020 7233 9908
Email: research@parkinsons.org.uk
Freephone Helpline: 0808 800 0303
www.parkinsons.org.uk
Charity registered in England and
Wales number 258197 and in Scotland
number SCO37554.
The Royal Society is an independent
academy promoting the natural and
applied sciences. Founded in 1660,
the society has three roles, as the UK
academy of science, as a learned
society, and as a funding agency.
The Royal Society’s position on stem
cell and embryo research
The Royal Society regularly evaluates and
assesses scientific developments in the
area of stem cell and embryo research
through its independent stem cell group.
Background
The Royal Society believes that stem cell
derived therapies could help to improve
or save the lives of many patients
worldwide, including those suffering from
serious injury or disease.
Therapies may be developed using
adult, foetal or embryonic stem cells.
It is important that avenues of stem
cell research, and stem cell-related
technologies, are not closed until they
have been fully investigated and proven
not to be viable.
The Royal Society strongly supports the
translation of basic stem cell research
into clinical practice when the evidence
supports efficacy and safety of clinical use.
It is important to be realistic concerning the
length of time it may take to develop stem
cell research into effective treatments.
Human admixed embryos
Throughout the development and
passage of the Human Fertilisation
and Embryology Bill, the Royal Society
has emphasised the need for scientific
studies using interspecies embryos
(now referred to as Human Admixed
Embryos). The Society has given its
backing to the creation of human
admixed embryos because it feels that
the scientific evidence now justifies the
development of such techniques.
These techniques will enable scientists
to produce stem cells without needing to
use human eggs, which are in extremely
short supply. In addition, this research
will facilitate further understanding of
basic stem cell biology, for example,
how stem cells become different cells in
the body, and to understand the genetic
causes of disease. These techniques will
also enable researchers to determine the
importance of communication between
the cell nucleus and other components
of the cell, including mitochondria (the
essential ‘powerhouses’ of the cell).
Legislation and regulation
The UK has an international reputation
as a leader in stem cell science. Its
position as an innovator and world
leader in this area can be attributed, at
least in part, to the legislative structure
and regulatory process which have
overseen embryo and stem cell research
in this country. The role of the regulator
is also crucial to foster public confidence
in stem cell and embryo research
– its assessment of individual research
projects ensures that research in this
area is carried out responsibly.
Stem cell research is a rapidly advancing
field. The UK’s legislative and regulatory
framework for embryo research,
implemented by the Human Fertilisation
and Embryology Authority (HFEA), must
continue to ensure that new stem cell
techniques are justified both ethically
and by scientific need.
The UK must support the safe,
successful and rapid translation of basic
stem cell research into clinical practice,
when the time is right, to achieve
the ultimate goal of using stem cells
therapeutically.
The Royal Society has closely followed
the development and progress of the
Human Fertilisation and Embryology
Bill. We have spoken out in areas where
the Society is best placed to provide
balanced and sound scientific advice
– for example concerning the creation of
human admixed embryos for research.
We consider commenting on the
detailed regulatory requirements for the
development of therapeutic treatments
to be outside of our remit. However,
we see a benefit in ‘future-proofing’
legislation to allow for stem cells to be
used therapeutically, providing that any
new advances are fully supported by
scientific evidence to ensure they are a
safe and appropriate next step.
contact details overleaf...
ALZHEIMER’S SOCIETY | ASSOCIATION OF MEDICAL RESEARCH CHARITIES |
BIOTECHNOLOGY AND BIOLOGICAL SCIENCES RESEARCH COUNCIL |
BRITISH HEART FOUNDATION | CANCER RESEARCH UK | DIABETES UK | HUMAN FERTILISATION AND EMBRYOLOGY AUTHORITY |
HUMAN TISSUE AUTHORITY | MEDICAL RESEARCH COUNCIL |
PARKINSON’S DISEASE SOCIETY | THE ROYAL SOCIETY |
SCOTTISH STEM CELL NETWORK | UK STEM CELL BANK | WELLCOME TRUST
Contact
The Royal Society
6-9 Carlton House Terrace
London
SW1Y 5AG
Tel: 020 7451 2500
Fax: 020 7930 2170
www.royalsociety.org
Registered charity number 207043.
The Scottish Stem Cell Network
(SSCN) was launched in 2003 with
funding from Scottish Enterprise
Edinburgh and Lothian (SEEL), to
bring together scientists, clinicians
and industry to improve the rate at
which stem cell laboratory research
in Scotland could be translated into
therapeutic benefits for patients
and tools for drug discovery. The
SSCN was established to support the
interdisciplinary exchanges needed
to translate excellent basic research
into clinical and economic benefit, and
operates with the following mission
statement to:
• Develop and consolidate Scotland’s
reputation in stem cell biology.
• Create an environment for exchanges
of scientific and clinical ideas.
• Foster collaborative links between
research scientists and clinicians in the field.
• Engage the private sector in the
development of stem cell technology.
• Lobby government and regulatory
authorities in support of the
development of the technology.
• Develop national and international links
with other centres of excellence.
• Realise the benefits to patients from effective treatment of
degenerative diseases.
The SSCN has worked with the entire
Scottish stem cell community, which
exists within a powerful life science
research and technology base including
more than 500 organisations and
more than 26,000 employees. Scottish
universities have become established
centres of excellence in medicine,
genetics and reprogramming technology,
and the country also hosts a significant
commercial presence in research and
development, including companies
such as Cellartis, CXR Biosciences,
Scottish BioMedical, Roslin Cells and
Geron. Many Scottish organisations hold
Human Fertilisation and Embryology
Authority (HFEA) licences issued by the
UK to conduct human embryonic stem
cell derivation and are working directly
with the regulatory authorities to define
clinically acceptable processes and
facilities for stem cell production.
The SSCN works with all of these
groups and organisations to create a
forum for discussion and learning, critical
for the development of the technology. A
series of workshops and training events
are held every year, covering topics
such as the management of intellectual
property in the field to new EU legislation
on clinical trials. We also encourage
our membership to meet regularly to
discuss specific disease areas, such as
diabetes, bone and muscle degeneration
and heart disease, in the context of
both basic science and clinical and
commercial development.
A major role of the SSCN has been
to engage with the general public
and inform open debate at home and
abroad. Working with the Scottish
Executive, the SSCN sees its role as
providing an informed and educated
general public to support the full
development of the technology in
Scotland. Events such as Edinburgh
International Science Festival provide an
ideal platform from which to address the
public, and SSCN have held debates at
that event and will continue to do so in
the future.
In 2006, the SSCN received a £1.8
million grant from Scottish Enterprise
to consolidate and expand its activities
over a ten year operational period.
The SSCN has formed a non-profit
company, with a board of directors
to oversee its governance and an
Executive office to deliver its objectives.
The Executive office is now located
within the Royal College of Surgeons
in Edinburgh.
contact details overleaf...
ALZHEIMER’S SOCIETY | ASSOCIATION OF MEDICAL RESEARCH CHARITIES |
BIOTECHNOLOGY AND BIOLOGICAL SCIENCES RESEARCH COUNCIL |
BRITISH HEART FOUNDATION | CANCER RESEARCH UK | DIABETES UK | HUMAN FERTILISATION AND EMBRYOLOGY AUTHORITY |
HUMAN TISSUE AUTHORITY | MEDICAL RESEARCH COUNCIL |
PARKINSON’S DISEASE SOCIETY | THE ROYAL SOCIETY |
SCOTTISH STEM CELL NETWORK | UK STEM CELL BANK | WELLCOME TRUST
Contact
Scottish Stem Cell Network
15 Hill Place
Edinburgh
EH8 9DS
Tel: 0131 527 3440
Fax: 0131 527 3441
www.sscn.co.uk
UK
StemCellBank
The UK Stem Cell Bank was established
to help realise the potential of stem
cells in cell therapy and will provide a
vital resource to support and advance
research. Although cell banks already
exist for many other types of cells such as
bone marrow and umbilical cord blood,
this initiative, with the full backing of the
UK Government, is the world’s first stem
cell bank of its type. With an international
reputation, it reflects the UK’s leading
position in stem cell research.
The Medical Research Council (MRC)
and the Biotechnology and Biological
Sciences Research Council (BBSRC)
provide the funding for the bank. Since
2002, the bank has been hosted by the
National Institute for Biological Standards
and Control (NIBSC), a publicly-funded
organisation that works within European
and international networks to assure the
quality and safety of biological medicines.
The bank provides a repository for human
stem cell lines derived from adult, foetal
and embryonic tissues and operates
under appropriate and accredited quality
systems to support the development of
research grade and clinical grade stem
cell lines. It is open to academic and
company researchers in the UK and
abroad. Staffed by personnel trained
to appropriate technical and quality
standards, the bank ensures that cell lines
which could ultimately provide the basis
for clinical treatment are prepared under
Good Manufacturing Practice (GMP),
subjected to appropriate safety testing
and subsequently handled and stored
under quality-controlled conditions.
Not only providing high-quality starting
materials for the development of stem
cell therapy, the bank provides a
centralised resource for researchers and
reduces the requirement for surplus
embryos for the development of stem
cell lines by individual research groups.
The first human stem cell lines were
approved for banking by the Steering
Committee in 2004 and the bank is now
preparing stocks of 63 human stem cell
lines from the UK, Australia, Sweden,
India and the USA.
The bank operates in accordance with
strict principles of governance laid down
by a high level steering committee, and
is administered by a local management
committee, which includes clinicians,
scientists and lay members. It was
accredited as a tissue bank for provision
of cells for therapy in 2004 by the
Medicines and Healthcare products
Regulatory Agency under the Department
of Health Code of Practice for Tissue
Banks and currently holds a license
issued by the Human Tissues Authority.
contact details overleaf...
ALZHEIMER’S SOCIETY | ASSOCIATION OF MEDICAL RESEARCH CHARITIES |
BIOTECHNOLOGY AND BIOLOGICAL SCIENCES RESEARCH COUNCIL |
BRITISH HEART FOUNDATION | CANCER RESEARCH UK | DIABETES UK | HUMAN FERTILISATION AND EMBRYOLOGY AUTHORITY |
HUMAN TISSUE AUTHORITY | MEDICAL RESEARCH COUNCIL |
PARKINSON’S DISEASE SOCIETY | THE ROYAL SOCIETY |
SCOTTISH STEM CELL NETWORK | UK STEM CELL BANK | WELLCOME TRUST
Contact
UK Stem Cell Bank
National Institute for Biological
Standards and Control
Blanche Lane
South Mimms
Potters Bar
Hertfordshire
EN6 3QG
Tel: 01707 641500
Fax: 01707 641578
Email: enquiries@ukstemcellbank.org.uk
www.ukstemcellbank.org.uk
The Wellcome Trust is the largest and
most diverse biomedical research
charity in the world, spending over
£650 million every year both in the
UK and internationally to support and
promote research with the aim of
improving the health of humans and
animals. At any one time, the Trust funds
more than 3,000 principal investigators
and their research teams in more than
40 different countries, including both
basic biomedical research to deepen
our understanding of health and disease,
and clinical research to generate
new therapies.
The Trust seeks to ensure the research
it funds is used responsibly for the
common good. It therefore supports
research into the wider ethical and social
implications of scientific research, and
funds activities that engage the public in
science and its related issues.
The Wellcome Trust and stem
cell research
The Wellcome Trust believes that stem
cell research offers great potential for
the development of new treatments for
a wide range of diseases, and has also
contributed to a greater understanding
of fundamental developmental biology.
However, an enormous amount of
research is still required to understand
how all types of stem cells function,
how they can be reliably induced to
differentiate into the various lineages
and to find out which types of cells
may offer the greatest promise for the
development of new therapies. The Trust
is therefore willing to fund ethical and
accountable research into all types of
stem cells.
Wellcome Trust Centre for Stem
Cell Research.
The world-class Wellcome Trust Centre
for Stem Cell Research is based at
the University of Cambridge, led by
Professor Austin Smith and Professor
Fiona Watt, with over £12 million funding
from the Trust.
The Wellcome Trust has commented
on a number of proposals in this area.
These include:
• The development of the UK Stem Cell Bank
• The UK Stem Cell Initiative
• The Department of Health’s (DH)
‘Review of the Human Fertilisation and
Embryology Act’, August 2005
• The DH’s request for comments on
the House of Commons Science
& Technology Select Committee’s
report on ‘Human Reproductive
Technologies and the Law’
• The Report from the Joint Committee
on the Human Tissue and Embryos
(Draft) Bill.
The centre focuses on the genetic
and biochemical mechanisms that
control how stem cells develop into
particular types of cell. This will provide
foundations for engineering of stem
cells to model particular diseases, drug
discovery and regenerative medicine.
Wellcome Trust funding for stem
cell research
The Wellcome Trust funds both
human and animal stem cell research
through a variety of grants for large
programmes, fellowships, PhD
studentships and technology transfer
initiatives. These include:
Type 1 diabetes
Professor Kevin Docherty and colleagues
at the University of Aberdeen are
attempting to generate human embryonic
stem cells that can be used to replace the
insulin-secreting cell ‘islets’ in the pancreas
of patients suffering from type 1 diabetes.
This grant was jointly funded with the
Juvenile Diabetes Research Foundation.
Manipulating embryonic stem cells
Dr Sally Lowell, University of Edinburgh,
is exploring whether it is possible to
manipulate communication between
embryonic stem cells and determine
which type of cell they become. This will
provide insights into tissue regeneration
as well as how to generate useful
cell types in a dish, which could be
transplanted to repair damaged tissues.
continued overleaf...
ALZHEIMER’S SOCIETY | ASSOCIATION OF MEDICAL RESEARCH CHARITIES |
BIOTECHNOLOGY AND BIOLOGICAL SCIENCES RESEARCH COUNCIL |
BRITISH HEART FOUNDATION | CANCER RESEARCH UK | DIABETES UK | HUMAN FERTILISATION AND EMBRYOLOGY AUTHORITY |
HUMAN TISSUE AUTHORITY | MEDICAL RESEARCH COUNCIL |
PARKINSON’S DISEASE SOCIETY | THE ROYAL SOCIETY |
SCOTTISH STEM CELL NETWORK | UK STEM CELL BANK | WELLCOME TRUST
Restoring vision
Attempts at stem cell transplantation
to restore vision in diseased retinas
have so far failed, with brain and
retina derived stem cells showing little
evidence of integrating into their new
environment. Dr Rachael Pearson,
University College London, is examining
what factors prevent transplanted cells
from connecting up and how best to
maximise transplantation.
Tissue engineering of teeth
Odontis Ltd, a spin-out company from
King’s College London, is developing
technology to enable patients to grow
natural replacement teeth by implanting
human stem cells. Benefits include less
surgical trauma on implantation, the
psychological advantage of having one’s
own teeth, and a natural appearance
and texture.
• Professor Erica Haimes from
Newcastle University, who is
investigating how the views and values
of those IVF couples who agree to
donate embryos for research and
those who refuse to donate embryos
differ on embryo experimentation and
stem cell therapies.
Public engagement
The Wellcome Trust encourages
scientists to engage with the public,
funding a number of activities and
initiatives. These have included ‘Playing
God’, in which Suzanne Lee of All
Change Arts brought together scientists,
medical ethicists and young people
to create artworks and performances
combining dance, digital images, sound
and text. The project explored topical
and controversial themes including stem
cell research and gene therapy.
Bioethics
The Trust recognises that stem cell
research raises a number of complex
social and ethical issues and funds a
range of activities to explore and debate
these questions through its Biomedical
Ethics Programme. These include:
• Dr Helen Busby from Nottingham
University, who is interviewing parents
about their views and experiences of
cord blood stem cell banking in the UK.
Further information
Funding opportunities for stem cell
research
• Biomedical Science Grants
www.wellcome.ac.uk/funding/
biomedicalscience
• Biomedical Ethics Awards
www.wellcome.ac.uk/biomedicalethics
• History of Medicine Grants
www.wellcome.ac.uk/hom
• Public Engagement
www.wellcome.ac.uk/engagingscience
Other links
• Wellcome Trust policy on stem cell research
http://www.wellcome.ac.uk/About-us/
Policy/Policy-and-position-statements/
WTX028577.htm
• Wellcome Science articles on stem
cells and cloning
http://www.wellcome.ac.uk/News/
News-archive/Browse-by-subject/
Topic/Stem-cells-and-cloning/
Contact
Wellcome Trust
215 Euston Road
London
NW1 2BE
Tel: 020 7611 8888
Fax: 020 7611 8545
Email: contact@wellcome.ac.uk
www.wellcome.ac.uk
The Wellcome Trust is a charity
registered in England, no. 210183. Its
sole Trustee is The Wellcome
Trust Limited, a company registered
in England, no. 2711000, whose
registered office is 215 Euston Road,
London NW1 2BE.