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Stem Cells

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Group Four Project:

Embryonic Stem Cells

Source: http://www.nectareal.com

Embryonic Stem Cells:

Background

 Retrieved from inner mass of an early-stage

embryo

 4-5 days following fertilization by sperm

 Consists of 50-150 cells

 Isolating the the inner cell mass results in the

death of the human embryo

 Embryonic stem cells are mainly valuable

because they are:

 Pluripotent (ability to differentiate to become any

type of cell)

 Able to replicate indefinitely to any kind of the

over 220 type of cell in the adult human body

Embryonic Stem Cells: History

 In 1981, embryonic stem cells were independently

derived from embryos

 Martin Evans and Matthew Kaufman of the University of

Cambridge pioneered a new technique for culturing mouse

embryos in the uterus to allow for an increase in cell

number, allowing for the derivation of embryonic cells from

these embryos

 In 1981 Gail Martin, of the University of California,

San Francisco, published a paper in December and

coined the term “Embryonic Stem Cell”

 In 1998, a breakthrough occurred when

James Thomson and his group of

researchers at the University of Wisconsin-

Madison, developed a technique to isolate

and cultivate human embryonic stem cells in

cell culture

Social Concerns



 It’s more of an ethical issue as it has to do

with one’s morality.

 Some believe embryonic stem cells for

research is an activity involves deliberate

attempt to destroy human life.

 Embryonic stem cells can cause cancer when

and if they become malignant.

Economic Concerns



 Many Clinics in the US have been set up to collect cells from the

people

 The cells are stored to be used in the future

 they can be used to cure health problems like organ transplant,

spinal cord injuries, birth defects, etc.

 To run these clinics, millions and millions of dollars are used.

 This money is mainly funded by the government.

 But the thing is researchers are not even sure if this will ever be

possible

 Now the question is: Is the money worth being spent for this?

 Even if we are able to find a cure

Clinical Trials

Although embryonic stem cell research has not

progressed to the stages of completed clinical

research, the FDA have recently approved of a

couple of clinical trials on human embryonic

stem cells. The successes of these trials are

not certain, but the hopes are high. At this

stage, the main priority is to make sure the

patients are kept safe. As well, there are a

couple of preclinical trials that have been

completed with mostly good, but the occasional

varying result

Geron: The Hope

 To use oligodendrocyte progenitor cells

(GRNOPC1),

derived from

human embryonic

stem cells (hESC),

to help people

that have suffered

from spinal cord injuries by injecting the cells

directly into the spinal cord lesion

Geron: The Plan

 Patients in the trial cannot have been

injured for more than 14 days prior to

receiving GRNOPC1

 Patients will be AIS (the ASIA

 Impairment Scale ) – Grade A (most severe

loss of locomotor and sensory activity)

thoracic spinal cord injuries

 Patients will be monitored for 15 years after

the injection

 The trial is to be conducted at seven different

medical centres in the U.S.

Geron: Preclinical Trial

 Animals both suffering from spinal cord injuries

and uninjured were monitored for 12 months

after receiving and injection of GRNOPC1 to see

if teratomas (a type of germ cell tumor, can be

benign or malignant) formed.

 Animals injected with clinical grade GRNOPC1 did not

form any teratomas

 Rats and mice with spinal cord injuries were

given GRNOPC1, acute and chronic toxicology

studies were performed, as well as multiple

hematology, clinical chemistry, urinalysis, and

gross and microscopic pathology tests

 No significant toxicity were found

 GRNOPC1 was not found outside of central nervous

system

Geron: Preclinical Trials

 Rats with spinal cord injuries were

administered with GRNOPC1 and checked

for allodynia (Pain from things that are not

usually painful

 No allodynia was found

 in vitro studies were used to test if the

allograft will be rejected (Direct Allogeneic

Immune Response to GRNOPC1)

 No Direct Allogeneic Immune Response were

found

 Cysts were found

Geron: Problems





 As stated before, animals studies have

shown GRNOPC1 to be ineffective if used

more than three months after the injury.

 Cysts might form as a result of the injection

ACT: MA09-hRPE



 A type of drug derived from embryonic stem

cells in hopes of treating Stargardt’s Macular

Dystrophy, a type of blindness

 Approved earlier this year, there is not much

more information available about this

particular clinical trial

 Although, ACT has in place the Blastomere

Program, a way to generate hESC without

killing the embryo

International Consequences:

Geron

 If this trial succeeds, then people around the

world with spinal injuries that are unable to

walk could have the opportunity again

 However, as many nations are opposed to

stem cell research, especially embryonic

stem cell research, people in those nation

may lose out the chance, creating more

tension between nations.

International Consequences:

ACT

 If the drug works, there could be new hope

for all the blind people in the world. The

treatment for Stargardt’s Macular Dystrophy

can lead to breakthroughs for other kinds of

blindness

 If the Blastomere Program works, then there

will be one less objection for using embryonic

stem cells for medical advancements.

Embryonic Stem Cells: clinical

uses

 It is mainly argued that because the embryonic stem cells can only be

obtained from a human embryo, it is morally wrong to treat diseases.

Due to this arguable factor, countries like Canada and America aren’t

studying in depth about this particular type of stem cell.

 Embryonic stem cells are “pluripotent”, meaning they are capable of

forming embryonic tissue.

 Researchers extract stem cells from 5-7 day old blastocyst.

 Stem cells can divide in culture to form more of their own kind,

thereby creating a stem cell line.

 These cells help generate healthy tissues needed by patients.

 More than 100 human embryonic stem cell lines have been derived

worldwide.

 Only 22 of these lines are eligible for federal funding in the USA.

 Stem cells can be used to generate healthy and functioning

specialized cells, which can then replace diseased or dysfunctional

cells.

Embryonic Stem Cells: clinical

uses (continued)

 It’s similar to the process of organ transplantation, only this

treatment consists of transplanting cells rather than organs.

 Bone marrow transplants is an example of cell therapy, were

the stem cells in the donor’s marrow is used to replace the

blood cells of the victims of leukemia.

 Cell therapy is also being used in experiments to graft new skin

cells to treat serious burn victims, and grow new corneas for

the sight impaired.

 The goal is for healthy cells to become integrated in the body

and start functioning like the patient’s own cells.

 Any diseases in which there is tissue degeneration can be a

potential candidate for stem cell therapies.

Applications of stem cells

 Cell replacement therapy could treat injuries

and various genetic and degenerative

conditions, including muscular dystrophies,

retinal degeneration, Alzheimer’s disease,

Parkinson’s disease, Heart disease, Severe

burns, arthritis, diabetes, spinal chord

injuries, and blood disorders such as

haemophilia.

Advantages of Embryonic

Stem Cell Usage

 They have the potential to make any body

cell.

 One cell line may be able to potentially

supply endless amounts of cells.

 Human embryos can be obtained from

fertility clinics, and are thereby easily

available to us.

Disadvantages of Embryonic

Stem Cell Use

 Inducing the cell type needed to treat a specific disease must

be defined and optimized, and this is thereby difficult to control.

 It is possible that transplanted cells would differ in their immune

profile from that of the recipient and therefore would be

rejected.

 It’s also ethically controversial for those who believe life begins

at conception.

 improper regulation of genes can lead to uncontrolled cell

division and tumor formation. This is a special concern with

cells that have been cultured in the laboratory for a period of

time, because they may regulate their genes differently than

they would in the body.

Future Use of Embryonic Stem

Cells

 The production of cardiac muscle cells, which have thus far

been evasive, would hold tremendous promise for the number

one killer: heart disease.

 Researchers could study the beneficial and toxic effects of new

medication pluripotent stem cells that have been developed to

mimic disease processes.

 Through more in-depth studies of pluripotent stem cells, it can

lead to the identifying factors responsible for differentiation of

stem cells into specialized cell types.

 Future therapies will rely on our ability to manipulate stem cells

in a way that can be accepted by the human body as normal.

 Human Embryonic Stem Cell Research offers

great promise of cures for otherwise incurable

conditions such as spinal cord injuries,

Alzheimer’s, Parkinson’s, etc.

 Research is aimed to find the appropriate source

of stem cells for a given indication (of disorders).

 Ways of expanding and perpetuating these cells in

culture.

 The best route of administrating these cells.

 Also, various methods to overcome rejection.

Potential Clinical Uses

 Human embryonic stem cells could be used to test new drugs.

 new medications could be tested for safety on differentiated cells

generated from human embryonic stem cell lines

 differentiated cells are less specialized cells that undergo a process

becoming a more specialized cell type

 Cancer cell lines used to screen potential anti-tumor drugs, with

embryonic stem cells, drug testing could be done in a larger

range of cell types

 Generation of cells and tissues could be used for cell-based

therapies.

 Treating diseases for which there are no known cures.

 Stem cells, directed to differentiate into specific cell types, could

offer a renewable source of replacement cells and tissues to treat

diseases

 ex.Alzheimer's diseases, spinal cord injury, heart disease

 Human embryonic stem cells have potential to form insulin-

producing cells that can be transplanted into diabetic patients,

curing diabetes

 Stem cell therapy could regenerate muscle and bones damaged by

injury or genetic disease

 May become possible to generate healthy heart muscle cells in

laboratory and then transplant those cells into patients with

chronic heart disease.

 Cells could repair by secreting growth factors around tissues

 Possible to direct the differentiation of embryonic stem cells or

adult bone marrow cells into heart muscle cells

 Stem cells could stimulate the growth of human tissues assisting

in healing wounds quicker

 Possible method for tissue regeneration: place adult stem cell

inside a tissue bed in a wound bed and allow stem cells to

stimulate differentiation in the tissue bed cells

 Could potentially treat Azoospermia

 Male does not have measurable level of sperm in his semen

causing infertility

Sources

 http://www.advancedcell.com/our-technology/research-programs/blastomere-program/



 http://www.geron.com/media/pressview.aspx?id=1235



 http://www.geron.com/GRNOPC1Trial/grnopc1-sec2.html



 http://www.geron.com/GRNOPC1Trial/grnopc1-sec3.html



 http://www.geron.com/GRNOPC1Trial/grnopc1-sec4.html



 http://singularityhub.com/2010/03/02/act-gains-fda-approval-for-embryonic-stem-cell-derived-therapy/



 http://machineslikeus.com/news/cautionary-warning-future-stem-cell-applications



 http://www.actionbioscience.org/biotech/pecorino2.html





 http://www.slideshare.net/doctorrao/stem-cells-and-clinical-applications-1710672

 http://stemcells.nih.gov/info/basics/basics6.asp



 http://www.reproduction-online.org/cgi/content/full/128/3/259



 http://www.aaas.org/spp/sfrl/projects/stem/report.pdf



 http://elibrary.bigchalk.com



 http://www.buzzle.com/articles/embryonic-stem-cell-research-pros-and-cons.html





 http://www.suite101.com/content/pros-and-cons-of-stem-cell-research--ethical-issues-a254568



 http://www.leaderu.com/science/promisestemcell.html



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