What are stem cells?

Find out more about stem cells, their abilities and how they build and repair the body.

All multicellular animals and plants rely on stem cells to grow from a single cell into
an adult. Stem cells allow our bodies to build new tissue, such as new muscle when
we exercise. Stem cells also continually replace the manyspecialised cells in our
body if they are worn out or damaged. This allows us to heal broken bones and
replace skin damaged by cuts and burns. All of this makes stem cells extremely
important in the process of development, cell renewal and healing. Researchers
hope that understanding how stem cells work will help to develop new therapies for
many different diseases, from cancer to multiple sclerosis.
Stem cells are defined by two key characteristics:

the ability to continuously divide to generate exact copies of themselves in a

process called self-renewal;

the ability to change into specialised cells in a process called differentiation.

Self-renewal and differentiation might seem like simple concepts, but

more research is needed to understand these processes.

Types of stem cells

There are many types of stem cells. Each type plays a different role in the body as
we grow and develop. Some stem cells only exist for a limited period, such as during
the development of an embryo. Others are only found in specific parts of the body,
such as in hair follicles or the liver. Depending on the purpose and location of the
stem cells, there may be limitations to what cell types the stem cell can differentiate
into. Research has also led to technologies to make different kinds of stem cells in
the laboratory. Generally, all stem cells fall into three different groups:

Embryonic stem (ES) cells and epiblast stem cells

Embryonic stem cells are pluripotent cells, which means they can differentiate into all
of the types of specialised cells in the body. ES cells are derived from cells found
within a 4 to 5 day old embryo called a blastocyst. The blastocyst is a very small ball
of about 150 cells with a diameter of ~0.2 mm. A cluster of cells within the blastocyst,
called the inner cell mass, contains stem cells that researchers isolate and grow in
the laboratory as ES cells.
Epiblast stem cells share several features with embryonic stem cells, including the
ability to differentiate into all types of specialised cells, but are derived from later
embryos that have implanted into the womb.

Tissue stem cells

Tissue stem cells can both self renew and give rise to at least one more specialised
(differentiated) cell type. They can be unipotent, meaning that they only make one
type of specialised cell (eg the spermatogonial stem cell) ormultipotent, meaning
they can generate several types of specialised cell. Under normal circumstances,
tissue stem cells generate only the cell types that make up the or organ / tissue
system they reside in.

Induced pluripotent stem (iPS) cells

Many years of researchers learning about how stem cells function has led to the
development of iPS cells. Researchers discovered that artificially activating
specific genes could induce many different cells to become pluripotent, similar to ES
cells. iPS cells have attracted a lot of attention because these cells can be generated

from adults, avoiding the ethical concerns associated with research using human
embryos.

Regenerative medicine research

Research and technological advancements are bringing regenerative medicines
closer to reality.
Aging, injuries and diseases are the result of damaged, malfunctioning or
dying specialised cells. By better understanding how stem cells are controlled and
how diseases develop, researchers hope to find new ways to treat patients. As such,
regenerative medicine aims to restore normal function by repairing or replacing
damaged or malfunctioning cells and tissues in patients.
Research in regenerative medicine is bringing tremendous advances in science,
technology, health and medicine. These advances hold promise for improving
methods of disease diagnosis and prevention, to the development of innovative
treatments for injuries and illnesses. Research by scientists hinges on learning how
different biological processes work and applying that knowledge to:

better understand the causes and progression of different diseases, such as

Parkinsons disease and multiple sclerosis, which facilitates better treatments;

create new biological tools and technologies to accelerate research, drug

discovery and the medical testing while also reducing costs and animal use;

design and develop novel treatments to both enhance natural repair

processes and use the abilities of stem cells for replacing damaged, malfunctioning
or diseased cells.
Currently, the only approved medical use of stem cells in the United Kingdom is bone
marrow transplants (also calledhaematopoietic stem cell transplants) for treating
blood and disorders in the immune system. Other stem cell treatments being used in
the UK, but not yet for general use, include emergency skin grafts using skin
(epidermal) stem cells and repairing the cornea of the eye using limbal stem cells.
However, many stem cell treatments are being researched and several show
promise in clinical trials (the official medical treatment approval process).

Why are stem cell treatments not widely used yet?

Developing new medical treatments is a long process with many steps to ensure
safety and effectiveness. New ideas for treatments must first be developed and

rigorously tested in laboratories before being tested on people in clinical trials. The
clinical trial approval process has many government rules and regulations to protect
patients. If approved in clinical trials, a treatment then needs the pharmaceutical and
biotech industries to further develop it for widespread use. Overall, making a new
treatment can easily take 15 to 20 years and is very expensive. In fact, most ideas
dont ever become approved treatments.
Unapproved stem cell treatments are sometimes offered by unregulated companies
and clinics. Unapproved procedures often lack scientific evidence showing they work
and may even be dangerous. It is important that patients discuss medical decisions
with their general practitioner (GP) before seeking out medical treatments.
TheInternational Society for Stem Cell Research has a very informative website and
patient handbook, which is an excellent starting point for learning more about stem
cell treatments. In addition, the website eurostemcell.orgprovides the latest accurate
information regarding research progress. It is also an exceptional online resource to
find out more about stem cells and their potential uses as well as limitations in
treating disease.

Ethics
All medical advancements come with ethical considerations; learn about some of the
ethical issues surrounding stem cells in medicine.

Regulation of stem cell research

Embryonic stem cells

Induced pluripotent stem cells

Research on animals

Affording stem cell treatments

Responsibility of personal health

Regulation of stem cell research

Ethical considerations and discussions have always been part of medicine and
research. These discussions can greatly influence rules and regulations that impact
the work of researchers and clinicians. A misconception about stem cell research is
that scientists can isolate stem cells whenever they like, create stem cell lines, and

use these cells in tests as they wish. In reality, the UK has put in place specific
regulations for the creation and use of stem cells. These regulations require
scientists to submit research proposals to government agencies (the HFEA and
the HTA) before creating or using human stem cells. Lawyers, clinicians, ethicists
and other scientists evaluate the research proposals to make sure the use of stem
cells is appropriate. If researchers want to carry out new experiments with stem cell
lines they already made, a new proposal must be submitted for approval.
If approval is given to make new stem cells, UK regulations require that these cells
are registered and stored in theUK Stem Cell Bank. The stem cell bank acts as a
resource for other researchers to obtain stem cells lines and as a way for the UK to
oversee what stem cell lines researchers are making. Scientists can get approval to
use cells stored in the stem cell bank rather than making new stem cells. Using
existing stem cell lines saves researchers time, labour and guarantees the cells are
of consistent quality. It also helps avoid some ethical issues surrounding making new
cell lines like embryonic stem cells.

Embryonic stem cells

Embryonic stem (ES) cells can differentiate into any other specialised cell in the
body. Studying ES cells allows researchers to learn how the human
body develops under normal conditions. Additionally, understanding how these cells
are derived and function is essential for developing regenerative medicine
treatments.
Ethical discussions surrounding ES cells focus on how ES cells are created. ES are
derived in the laboratory from cells collected from a very early embryo, called
a blastocyst. A blastocyst develops approximately 5 days after conception and has
not yet implanted itself into the mothers womb. Blastocysts are roughly 150 cells,
which is very small, about the size of a pin head. Most blastocysts for scientific and
medical research are donated by parents that have undergone an in vitro fertilization
(IVF) treatment. The process of collecting ES cells destroys the blastocyst, however,
most unused blastocysts from IVF treatments are destroyed anyway. Still, some
people ask whether it is justifiable to destroy early embryos at all, even if the
research on ES cells might help treat people with diseases and injuries. This ethical
discussion has resulted in strict regulations that monitor who can: supply the
blastocysts to make ES cells, generate ES cells, and use ES cells once made (see
the above section on regulations). The UK governments regulations on ES cells

have attempted to find a balance between different perspectives and values of the
UK people in this ES cell debate. Likewise, government rules vary from country to
country as the citizens and their governments come to different conclusions on stem
cell research.
To a certain extent technologies, such as induced pluripotent stem (iPS) cells,
provide a way to end the use of ES cells and avoid ethical issues. However, the
reality is that some research will always require the use of ES cells to understand
things such as human development and to examine/compare the ability of iPS cells
(or any other new cell technology) to differentiate. Also, it is important to realise that
iPS cells present some of their own ethical concerns, further discussed below.

Induced pluripotent stem cells

Technologies to create induced pluripotent stem (iPS) cells, or other pluripotent cells,
present researchers with exciting scientific possibilities as well as important ethical
responsibilities that need to be well regulated. Shinya Yamanaka, the scientist who
led the discovery of iPS cells, emphasises the need for regulation. For
example,researchers have demonstrated they are able to make eggs and sperm
from iPS cells. These reproductive cells were used to create mouse embryos that
grew into live-born mice when hosted by a surrogate mother. Should iPS
technologies be used to make human embryos from skin cells in a similar manner?
Additionally, iPS cell technologies could permit men to make eggs from skin cells,
and women to make sperm. This technology is not yet possible in humans, yet there
is concern over how these procedures should be regulated.
Another ethical issue with iPS cells (and tissue stem cells) is deciding who owns
them. Once iPS cells are created, are the cells the property of the creators, the
donor, or both? Should iPS cells be provided to other patients with or without the
consent of the original donor? These are just some ethical concerns still
unanswered.

Research on animals
Research using animals has played and continues to play a key role in the
advancement of medical, biological and veterinary science. It has made a vital
contribution towards the understanding, prevention, treatment and cure of a wide
range of major health problems in both people and animals, including cancer, heart
disease and diabetes. Presently, all new medical treatments in the UK, stem cell

based or not, are required by law to be tested on animals to make sure they are safe
before testing in people. Medical research institutions have to follow very strict rules
and regulations set and checked by the UK Governments Home Office. These
regulations aim to maximise animal welfare and minimise the use of animals as
much as possible in research. Researchers must, by law, show that their research
could not be carried out using non-animal alternative methods before it can be
approved. Additionally, licences to use animals in research are only granted where
the potential benefits of the work are likely to outweigh the effects on the animals
concerned.
In the UK, the legal and ethical use of animals in research is guided by the 3 Rs
Replacement, Reduction and Refinement. Researchers must replace animal
studies with other research methods wherever possible; reduce the number of
animals as far as possible while maintaining scientific rigor; and refine experimental
and husbandry procedures to improve animal welfare and minimise potential
distress. Scientists are developing new technologies to help them to achieve these
aims. One such example is the development of technologies that use iPS cells to
directly test drugs on a patients cells. However, new testing systems will likely never
completely replace animal testing, as there are many biological systems too complex
to be recreated in the laboratory. At present, testing the safety of treatments in
animals is vital for rapid development of better and safer medicines.

Affording stem cell treatments

Regenerative medicines, in theory, could be developed as personalised medicine,
tailored to work specifically for each patient. As wonderful as personalised medicine
sounds, this kind of medical treatment would be very costly. Stem cell technologies
being created in todays laboratories are time-consuming and very expensive. This
raises questions of: Who will be able to afford these treatments? Will public
healthcare include expensive regenerative medicines? Who will decide the balance
between cost and a patients quality of life?
Ideas are needed to address the affordability and accessibility associated with
regenerative medicines. One proposed idea is to create a Global Stem Cell Bank.
One of the largest complications of transplanting organs, tissues and cells is immune
system rejection of the transplant. The Global Stem Cell Bank could address this
complication by carrying different types of stem cells that are matched to the immune
system requirements of groups of patients, much like blood banks carry different

blood types (A, B, O & Rh blood groups). Costs for making the stem cells could then
be shared by everyone using the stem cell bank, making it much more affordable.
Also, having stem cells already made, rather than taking weeks or months to make
suitable stem cells in a lab, would allow treatments to be performed more quickly
from the time of diagnosis.

Responsibility for personal health

Regenerative medicines may one day be able to cure injuries and treat many
diseases and injuries. However, will this mean that people no longer need to worry
about behaviour that leads to diseases and injuries? Will smokers be able to keep
smoking, knowing they can just get fixed up? The same can be asked about other
health issues, like heavy drinking, poor eating habits, drug abuse, and even injuries
from extreme sports. Should costly regenerative medicine treatments be performed
only if the patient is going to change his or her behaviour? Who should pay for
treating these individuals if they will likely need to be re-treated in the future? Stem
cell treatments may also allow individuals to live longer and longer. Questions about
the costs associated with caring for an ageing population need to be asked as the
average life span continues to rise.