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We
all remember the promise of stem-cell technology when scientists at the
University of Wisconsin and Johns Hopkins University first isolated and
successfully cultured human pluripotent stem cells back in 1998 – that these miracle cells would lead to products that would
revolutionize medicine.
Now
it’s thirteen years later and the collective interest in stem cells has
waned, but that doesn’t mean scientists aren’t
still plugging away trying to realize the dream that once was. Before we get
to recent advancements in stem-cell technology and why we think investors
will soon show renewed interest in it, let’s take a step back and
review what makes stem cells special, and how they could impact medicine.
Stems
cells differ from other cell types due to three main characteristics that all
stem cells share, regardless of their source:
1. They are capable of dividing and renewing themselves
for long periods;
2. They are unspecialized; and
3. They can give rise to specialized cell types.
Because
of stem cells’ potential to develop into many different cell types and
their unique ability to serve as a sort of internal repair system, dividing
essentially without limit to replenish other cells, their potential
implications for medicine are profound.
Studies
of human embryonic stem cells (hESCs) could provide
valuable information about the complex events that occur during human
development. By identifying how undifferentiated stem cells become the
differentiated cells that form our tissues and organs and developing a more
complete understanding of the genetic and molecular controls of these
processes, scientists may be able to develop new ways to treat various birth
defects and cancer, which are the result of abnormal cell division and
differentiation.
Stem
cells could also be used to test new drugs. New medications could be tested
for safety on differentiated cells created from human pluripotent stem cell
lines, allowing for drug testing in a wider range of cell types.
And
then there’s the holy grail of stem-cell research: the generation of
cells and tissues for use in cell-based therapies. It’s no secret that
the need for transplantable organs and tissues far outweighs the available
supply. With the potential to develop into specialized cells, stem cells
could be used as replacement cells and tissues to treat countless conditions,
including heart disease, diabetes, spinal cord injury, and arthritis.
For
example, take diabetes. In people with Type I diabetes, the cells of the
pancreas responsible for producing insulin are actually destroyed by their
own immune system. Studies have shown that it may be possible to direct the
differentiation of hESCs in cell culture to form
insulin-producing cells that eventually could be used in transplantation
therapy.
But
what about the ethical issues involved in stem-cell research?
Thanks
to advancements in technology, the research is becoming less controversial.
During
the early days of human stem cell research, scientists worked with two kinds
of cells: embryonic stem cells and non-embryonic “somatic” or
“adult” stem cells. Each of these types of stem cells has
advantages and disadvantages regarding potential use for cell-based
regenerative therapies, but embryonic stem cells are usually more highly
prized because they are pluripotent and have the potential to differentiate
into almost any cell in the body. Adult stem cells are thought to be limited
to differentiating into different cell types of their tissue of origin.
What’s
more, large numbers of cells are needed for stem-cell replacement therapies,
and while embryonic stem cells can be grown relatively easily in culture,
numerous challenges remain when it comes to isolating adult stem cells from
the tissue and expanding their numbers in cell culture.
But
in 2006, scientists came up with a solution that would allow research on
pluripotent stem cells while bypassing the ethical issues associated with the
production of hESCs. Researchers working at Kyoto
University in Japan identified conditions that would allow specialized adult
cells to be genetically “reprogrammed” to assume a stem-cell-like
state.
To
quote Dr. Charles A Goldthwaite’s article, The Promise of Induced Pluripotent Stem Cells (iPSCs):
These
adult cells, called induced pluripotent stem cells (iPSCs),
were reprogrammed to an embryonic stem cell-like state by introducing genes
important for maintaining the essential properties of embryonic stem cells
(ESCs). Since this initial discovery, researchers have rapidly improved the
techniques to generate iPSCs, creating a powerful
new way to “de-differentiate” cells whose developmental fates had
been previously assumed to be determined.
Although
much additional research is needed, investigators are beginning to focus on
the potential utility of iPSCs as a tool for drug
development, modeling of disease, and transplantation medicine. The idea that
a patient’s tissues could provide him/her a copious, immune-matched
supply of pluripotent cells has captured the imagination of researchers and
clinicians worldwide. Furthermore, ethical issues associated with the
production of ESCs do not apply to iPSCs, which
offer a non-controversial strategy to generate patient-specific stem cell
lines.
While
numerous technical hurdles remain – and it’s unclear whether iPSCs could ever fully replace hESCs
in the lab – the technology represents a big step forward in stem-cell
research.
Some
other recent advancements that could stoke investor interest include:
·
In January 2011, researchers at Tottori University in
Japan successfully made heart pacemaker cells using the embryonic stem cells
of mice. The achievement could lead to breakthroughs in the treatment of
arrhythmia and could reduce the need for electronic pacemakers in human
patients.
·
In November 2010, the FDA approved a trial to test an
embryonic stem-cell-derived therapy in the treatment of an inherited disease
that causes blindness in young people.
·
In October 2010, 12 years after human embryonic stem
cells were first isolated, a therapy derived from
such cells was tested in humans for the first time.
·
In August 2010, according to an article from the Daily Telegraph, scientists
created synthetic blood using hESCs, which could
aid victims of large-scale disasters when blood bank supplies are low. The
scientists, who are working with the Wellcome Trust
in Great Britain, say their ultimate goal is to create the rare O negative
blood type. It can be given to any patient without fear of rejection, but is
produced by only 7% of the population.
·
In January 2010, researchers at Stanford showed that
transplanted neurons grown from embryonic stem cells were able to form proper
brain connections in newborn mice. The discovery, which demonstrates that
stem cells can be directed to become specific brain cells, could lead to new
treatments for nervous system diseases like ALS (Lou Gehrig’s disease)
as well as for spinal cord injuries.
The
point is that advances in stem-cell research are being made every day. The
technology has finally reached the stage of human trials. And while years of
intensive research are still necessary to overcome the technical hurdles that
remain, there’s little doubt that fortunes will be made by prudent
early investors in the space.
[Stem
cells are just one of many biotech advances that may revolutionize treating
many diseases in the very near future. The Casey
Extraordinary Technology team has put together a “curing
cancer” portfolio that includes other advances that could also
revolutionize your investments. Don’t let this opportunity slip by!]
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