Tara Pugliese

Mrs. Kashinsky
Biology
7 November 15
The Success and Promise of Embryonic Stem Cells
The use of stem cell therapies to treat human disease is controversial both within
and out of the scientific community. In this essay, the effectiveness of utilizing embryonic
stem cells to develop treatments for human disease will be evaluated as well as the
political realities, ethical arguments, and possible advances concerning the topic.
First coined in 1868, stem cells were not identified in living organisms until more
than a century later when embryonic stem cells were isolated in mice. Embryonic stem
cells, derived from the inner cell mass of a blastocyst, have the ability to differentiate into
any cell type in the human body (i.e. pluripotency). Some early successes have occurred
through the use of stem cell therapies to treat diabetes, macular degeneration, and
Parkinsons disease. The following paragraphs discuss several studies related to stem cell
therapies for these illnesses.
Several recent studies have identified embryonic stem cell therapies as possible
treatments for Parkinsons disease. Parkinsons disease is a nervous system disorder
commonly found in patients sixty years or older which currently affects at least one
million Americans (Paddock). Scientists have yet to identify the definitive cause of
Parkinsons; however, the disease is known to lead to a progressive decline in the
production of the neurotransmitter dopamine (Robson). This decline results in symptoms
that include bradykinesia (slowness of movement), tremors, and muscle stiffness.

Additionally, Parkinsons disease is often accompanied by depression and anxiety.

Several medications, drugs, and surgeries are available to patients, but all have substantial
side effects and wear off as the disease progresses. Furthermore, such treatments can ease
symptoms but do not stop the progression of Parkinsons (Robson). However, many
researchers are hopeful that stem cell therapy can finally result in better treatment options
for patients. Scientists have made dopamine producing nerve cells from embryonic stem
cells in laboratories. These nerve cells can be transplanted into a human brain to produce
additional dopamine to compensate for the reduced dopamine levels caused by
Parkinsons disease (Coghlan). Prof. Parmar, leader of one such study, expressed his
particular findings: The study shows that the cells that we generate from stem cells, they
function equally as well as the cells that we find in the brain (Paddock).
Embryonic stem cell based therapies have also been developed to treat macular
degeneration. Researchers have focused mainly on two specific ocular diseases: Agerelated Macular Degeneration (AMD) and Stargardts disease. Both ailments are types of
macular degeneration the leading cause of vision loss with more than three million U.S.
cases per year. Neither disease has a cure or treatments other than lifestyle changes (i.e.
dieting, exercise, etc.). Unlike AMD, which usually affects elderly patients, Stargardts
disease develops in children and teenagers. While the cause of AMD has remained
undetermined, the recessive gene ABCA4 is known to cause Stargardts disease (Hafiz).
Safe and effective embryonic stem cell therapies have been developed to treat both
ailments. In the fall of 2014, a research paper from the University of California, Los
Angeles, (in cooperation with Advanced Cell Technology) was published online and
immediately evoked a significant amount of media attention (Begley). In this new

treatment for macular degeneration, embryonic stem cells were differentiated into retinal
pigment epithelium (RPE) cells and implanted into one eye of eighteen patients (half
suffered from AMD and half suffered from Stargardts disease). The experiment resulted
in significantly improved vision in eight of the eighteen patients and pigment levels
increased in thirteen patients. More importantly, the only side effects were mild and had
been caused by the immune-suppressing drugs taken to ensure that the implanted cells
would not be rejected (Pollack). Though Dr. Steven D. Schwartz (the papers lead author)
admitted, Theres a lot of work to be done, he also stated, This is a promising study,
and it provides a lot of hope for regenerative medicine (Pollack).
Embryonic stem cell therapies are also bringing a cure for diabetes closer to
reality. Diabetics and their families are some of the biggest advocates and lobbyists for
stem cell research because the potential benefits of embryonic stem cells as a treatment
for diabetes are substantial. About three million individuals in the U.S. have type 1 (or
juvenile) diabetes although this is only about 10% of the estimated 29.1 million that
suffer from type 2 (or adult) diabetes (MacGill). In type 1 diabetes, the patients immune
system destroys the pancreatic cells responsible for producing insulin (the hormone
regulating the amount of glucose in the blood). However, in type 2 diabetes, the body can
still produce insulin but not enough for proper function (Diabetes Fact Sheet). In both
types of diabetes, patients must regulate their nutrition, physical activity, and glucose
levels as well as regularly inject themselves with insulin to make up for the inadequate
amount of insulin their bodies are producing (MacGill). Fortunately, recent studies by the
Harvard Stem Cell Institute (HSCI), ViaCyte, and BetaLogics have all successfully
created insulin-producing cells from human embryonic stem cells (Alexander). Elaine

Fuchs, a colleague of Melton, hailed the HSCIs study: For decades, researchers have
tried to generate human pancreatic beta cells that could be cultured and passaged long
term under conditions where they produce insulin. Melton and his colleagues have now
overcome this hurdle and opened the door for drug discovery and transplantation therapy
in diabetes (Pagliuca). This major breakthrough provides optimism to the millions of
patients suffering from diabetes. Though all of the studies were conducted either in
laboratories or with mice, clinical trials are about to commence. ViaCytes Investigational
New Drug (IND) application with the United States Food and Drug Association (FDA)
was recently approved and the company plans to initiate its clinical trial within the
upcoming years (Alexander).
Research into embryonic stem cell therapies and their effect on several other
conditions is also being conducted. Currently, embryonic stem cell therapies are being
developed to repair spinal cord injuries (Doherty), restore damaged heart cells (Can
Stem Cells Repair a Damaged Heart?), and eliminate HIV infections (Vogt-James).
Other ailments that may benefit from stem cell research include autoimmune diseases,
neural degenerative diseases, and childhood tumors (Weintraub). Though embryonic stem
cells have the potential to improve the lives of millions of individuals all across the
world, several arguments can be made against their use in treating human disease.
One such argument is the ethical dispute regarding the origins of embryonic stem
cells. As previously stated, embryonic stem cells originate in an early stage of the
embryo. The moral argument is as follows: since isolating human embryonic stem cells
from a blastocyst causes the embryo to die, it is thus murder (Greene). In this particular
claim an embryo amounts to a human being, as in the argument against abortion (Smith).

This argument proved convincing enough that in 2001, the Bush administration defunded
embryonic stem cell research (Aldridge). Moreover, several unwanted side effects can
occur from the use of embryonic stem cells all resulting from what makes these cells so
useful. First, the cells ability to renew indefinitely may cause a teratoma to develop (a
type of tumor resulting from unnaturally occurring tissues). Second, the pluripotency of
embryonic stem cells means that controlling their differentiation may be difficult. The
next paragraph will present the counterarguments to the ethical dilemma and potential
side effects.
The ethical argument made against the use of embryonic stem cells in the
treatment of human disease becomes less persuasive as the reality of their handling is
disclosed. All embryonic stem cells used in research were derived from unused embryos
at in vitro fertilization clinics and were donated by the parents with the permission to be
used for research (Noble). In addition, new methods have been developed to isolate
embryonic stem cells without killing the embryo (Chung et. al.). Another increasingly
common method of obtaining embryonic stem cells without destroying embryos is the
use of induced pluripotent stem cells (iPSCs); iPSCs are created by genetically
reprogramming adult (or somatic) stem cells to exhibit the pluripotency present in
embryonic stem cells (What are Induced Pluripotent Stem Cells?). As for the side
effects and risks associated with stem cell transplant and implantation, additional research
has already resulted in safer treatment methods and procedures have become less
dangerous with every study. Consequently, President Obama refunded embryonic stem
cell research in 2009, thus reversing George W. Bushs previous executive order
(Aldridge).

In my opinion, the well-documented success and myriad of potential cures and

treatments that can result from embryonic stem cells constitutes the continuance of
research, despite the arguments presented against this position. Embryonic stem cells
have already proven to ameliorate and even eradicate symptoms of various human
diseases and ailments. The future surely appears bright for embryonic stem cell research.

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