Question
#4 Taken from the International Stem Cell ForumQ4.
Have stem cells been used to treat any human diseases yet?
A. Adult stem
cells such as blood-forming stem cells in bone marrow (hematopoietic stem cells
or HSCs) are currently the only type of stem cell commonly used to treat human
disease. Doctors have been transferring HSCs in bone marrow transplants for over
40 years. More advanced techniques for collecting HSCs are now used to treat leukemia,
lymphoma and several other blood disorders. The clinical potential of adult
stem cells has also been demonstrated in the treatment of other diseases, including
diabetes, Parkinson's disease and advanced kidney cancer. For example, in 1999
scientists in the USA removed 10 to 15 neural stem cells from a Parkinson's disease
patient and used them to reproduce 6 million dopaminergic neural stem cells in
culture. These were reintroduced into the patient's brain tissue, producing a
62% increase in dopamine uptake and a 40-50% improvement in certain motor tasks.
However, these newer uses of adult somatic stem cells have involved studies with
very limited numbers of patients. Embryonic stem cells have not been used to
treat any human diseases yet, but are thought to offer potential cures and therapies
for many devastating diseases. Scientists only began studying embryonic stem cells
in the late 1990s, so much of the research is still at an early stage.
This
is just one of hundreds of research papers available on www.pubmed.govLoebinger
MR, Janes SM. MSc, Centre of Respiratory Research, Rayne Building, University
College London, 5 University St, London, WC1E 6JJ, UK. s.janes@ucl.ac.uk. Respiratory
diseases remain one of the main causes of morbidity and mortality in the world.
Interest has increased as to the possibility of optimizing the repair of the lung
with the manipulation of stem cells. Embryonic and adult stem cells have been
suggested as possibilities. Adult stem cells have traditionally been thought of
as having limited differentiation ability and to be organ specific. However, a
series of exciting reports over the last 5 to 10 years have suggested that adult
bone marrow-derived stem cells may have more plasticity and are able to differentiate
into bronchial and alveolar epithelium, vascular endothelium, and interstitial
cell types, making them prime candidates for repair. This article critically reviews
the evidence for this plasticity and the use of predominantly adult stem cells
to help with lung regeneration and repair and assesses how this technology may
be utilized in clinical medicine. PMID: 17625088 [PubMed - in process]
Is this the cell that could revolutionize medicine? (Note: This was written
in January 2002 - We have come a long way since!) IT
MIGHT turn out to be the most important cell ever discovered. It's a stem cell
found in adults that can turn into every single tissue in the body. Until
now, only stem cells from early embryos were thought to be able to do this. If
the finding is confirmed, it will mean cells from your own body could one day
be turned into all sorts of perfectly matched replacement tissues and even organs.
If so, there would be no need to resort to
therapeutic cloning-cloning people to get matching stem cells from the resulting
embryos. Nor would you have to genetically engineer embryonic stem cells (ESCs)
to create a "one cell fits all" line that doesn't trigger immune rejection.
The discovery of such versatile adult stem cells will also fan the debate about
whether embryonic stem cell research is justified. "The
work is very exciting," says Ihor Lemischka of Princeton University. "They
can differentiate into pretty much everything that an embryonic stem cell can
differentiate into." The cells were found
in the bone marrow of adults by Catherine Verfaillie at the University of Minnesota.
Extraordinary claims require extraordinary proof, and though the team has so far
published little, a patent application seen by New Scientist shows the team has
carried out extensive experiments. These confirm that the cells-dubbed multipotent
adult progenitor cells, or MAPCs-have the same potential as ESCs. "It's very
dramatic, the kinds of observations [Verfaillie] is reporting," says Irving
Weissman of Stanford University. "The findings, if reproducible, are remarkable."
At least two other labs claim to have found
similar cells in mice, and one biotech company, MorphoGen Pharmaceuticals of San
Diego, says it has found them in skin and muscle as well as human bone marrow.
But Verfaillie's team appears to be the first to carry out the key experiments
needed to back up the claim that these adult stem cells are as versatile as ESCs.
Verfaillie extracted the MAPCs from the bone
marrow of mice, rats and humans in a series of stages. Cells that don't carry
certain surface markers, or don't grow under certain conditions, are gradually
eliminated, leaving a population rich in MAPCs. Verfaillie says her lab has reliably
isolated the cells from about 70 per cent of the 100 or so human volunteers who
donated marrow samples. The cells seem to
grow indefinitely in culture, like ESCs. Some cell lines have been growing for
almost two years and have kept their characteristics, with no signs of ageing,
she says. Given the right conditions, MAPCs can turn into a myriad of tissue types:
muscle, cartilage, bone, liver and different types of neurons and brain cells.
Crucially, using a technique called retroviral marking, Verfaillie has shown that
the descendants of a single cell can turn into all these different cell types-a
key experiment in proving that MAPCs are truly versatile. Also,
Verfaillie's group has done the tests that are perhaps the gold standard in assessing
a cell's plasticity. She placed single MAPCs from humans and mice into very early
mouse embryos, when they are just a ball of cells. Analyses of mice born after
the experiment reveal that a single MAPC can contribute to all the body's tissues.
MAPCs have many of the properties of ESCs,
but they are not identical. Unlike ESCs, for example, they do not seem to form
cancerous masses if you inject them into adults. This would obviously be highly
desirable if confirmed. "The data looks
very good, it's very hard to find any flaws," says Lemischka. But it still
has to be independently confirmed by other groups, he adds. Meanwhile,
there are some fundamental questions that must be answered, experts say. One is
whether MAPCs really form functioning cells. Stem cells that differentiate may
express markers characteristic of many different cell types, says Freda Miller
of McGill University. But simply detecting markers for, say, neural tissue doesn't
prove that a stem cell really has become a working neuron. Verfaillie's
findings also raise questions about the nature of stem cells. Her team thinks
that MAPCs are rare cells present in the bone marrow that can be fished out through
a series of enriching steps. But others think the selection process actually creates
the MAPCs. "I don't think there is 'a cell' that is lurking there that can
do this. I think that Catherine has found a way to produce a cell that can behave
this way," says Neil Theise of New York University Medical School. Author:
Sylvia Pagan Westphal, Boston New Scientist
issue: 26th January 2002 New
Scientist
The Bottom Line
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