Potentials of Stem Cell Therapy

Traditional medical therapy relies on the small-molecule drugs as therapeutic agents.

These compounds are obtained either by serendipity, or by screens of natural products such as

those produced by plants and fungi, or more recently, by screens of synthetic combinatorial

chemical libraries. Whereas most early efforts apply troublesome screens and determination of

the biological activity or potency of a substance, the modern version of drug discovery depends

on high amount of raw material processed within a given time. In the past 20 years, recombinant

DNA technology has authorized two new classes of therapeutics: protein and gene therapies. Of

these, protein therapy has been more successful, principally because the technical barriers are

less severe. (Kamb & Rao)

Improvements in cell culture and molecular markers offer new hope for cell and tissue

therapy. Cultured stem cells and embryonic stem cells, in particular, are among the most

promising indicator for future medical innovation. They are obvious candidates for cell and

organ therapy. Moreover, stem cells may serve as useful tools for discovery of new genes and

protein targets.

A therapeutic approach that is perfectly suited to stem cells is the culture of cells prior to

their introduction into patients for therapeutic benefit. Two different approaches can be

envisioned: direct transfer of stem cells into specific sites in the body; and culture and

manipulation in vitro of stem cells or their differentiated offspring, followed by fabrication of

tissues or organs for transplantation. (Kamb & Rao)

With direct transfer of stem cells, one hopes that the body’s regulatory systems will take

over and integrate the transplanted cells appropriately on site. If successful, this process would
be equivalent to reforestation--supplying a fresh batch of seed that produces a full-blown forest

in due course. Such transfer produces might be used to correct tissue or organ damage, for

example, to repopulate neurons lost during strokes. They might also be used to rejuvenate tissue,

for instance, to supply new brain cells to aging regions of the brain. ( Kamb & Rao)

Stem cells are cells that have the potential to develop into many different or specialized

cell types. They can be thought as primitive, “unspecialized” cells that are able to divide and

become specialized cells of the body such as liver cells, blood cells, muscle cells, and other cells

with specific functions. They are referred to as “undifferentiated” cells because they have not yet

committed to a developmental path that from a specific tissue or organ. The process of changing

into a specific cell type is known as differentiation. ( Stoppler, 1997)

Stem cell therapy helps humans with their diseases through the differentiation of stem

cells into many kinds of cells. These cells are introduced into the damaged tissue so as to treat

the disorder or the injury. To make it simpler, stem cells are extracted from the blood and

transplanted into the patient through a procedure like blood transfusion. They are also critically

involved in the normal development and maintenance of a great variety of tissues, from plants to

animals. There are a number of medical researchers who believe that the stem cell therapy has

the potential to change the treatment of human diseases and reduce the suffering people face

when they have a disease. Moreover, they believe that stem cell therapy is one of the potential

cures to replace damaged and diseased tissues in the body. ( Biehl & Russel, 2009)

In the case of a patient needing tissues or organ transplant, their common questions are:

Who is the donor? Are the organs suited for their body?
 With the use of stem cell therapy, these uncertainties or doubts can fade. The use of stem

cells can decrease the possibility of rejection since these cells will be coming from the patients

themselves. Scientists are hoping that they could “program” these stem cells to reconstruct or

replace damaged and dysfunctional cells that have been injured or destroyed by diseases because

of the abilities that stem cells have shown. They are now looking into the possibility of using

these cells to treat illnesses such as type 1 diabetes, heart disease and liver disease.

Stem cells have the ability to build every tissue in the human body; hence, they have

great potential for future uses in the tissue regeneration and repair. For cells to fall under the

definition of stem cells, they must display two essential characteristics. First, stem cells must

have the ability of unlimited self-renewal to produce progeny, a descendant or offspring, exactly

the same as the originating cell. This trait is also true for cancer cells that divide in an

uncontrolled manner, whereas stem cell division is highly regulated. Therefore, it is important to

note the second requirement for stem cells; they must be able to give rise to a specialized cell

type that becomes part of the healthy animal or human. (Biehl & Russel, 2009)

Stem cells are classified into two categories:

• based on their origin,

• and based on their potential use in research and medicine. (Abboud)

In relation to their origin, stem cells can be classified into embryonic stem cells and adult

stem cells. Embryonic stem cells are from the inner cell mass, which is part of the 5-7 day old

embryo called blastocyst. They have the ability to differentiate into any of the over two hundred

types of cells that make up the human body. Adult stem cells are from mature adults, the

numbers of cell types which they can differentiate into are limited. They, however, don’t have
the ability to differentiate into any types of cells but they differentiate into varieties of a

particular type of cells, determined by the type of tissue in which they are found. (Abboud)

Stem cells can also be classified according to their potential use in research and medicine.

Stem cells are perhaps best understood in terms of how committed they are to becoming any

particular type of cell. The categories into which they fall include:

• Totipotent stem cells. These cells are the most versatile of all stem cell types

because they have the potential to give rise to any and all human cells such as

brain, liver, blood or heart cells. They can even give rise to an entire functional

organism.

• Pluripotent stem cells. These cells are like totipotent stem cells in which they can

give rise to all tissue types. However, they cannot give rise to an entire organism.

• Multipotent stem cells. These cells are less plastic and more differentiated stem

cells meaning they can give rise to a limited range of cells within a tissue type.

• Unipotent stem cells. A unipotent stem cell refers to a cell that can differentiate

along only one lineage. Found in adult tissues, unipotent stem cell, in comparison

with other types of stem cells, has the lowest differentiation potential because the

cell has the capacity to differentiate into only one type of cell or tissue. Despite

their differentiation potential being limited, unipotent cells have vast therapeutic

potential to treat injuries and diseases. (Carrier & Ledingham, 2004)

The possibility of using stem cells to treat diseased tissues has produced a great impact

and interest in the field of medicine. Cardiovascular, autoimmune, skeletal, and neurological are
the broad classifications of use fro stem cells in the prevention and treatment of human diseases.

(Hayes, 2006)

Here are some of the diseases that can be treated using stem cell therapy: Acute

lymphoblastic leukemia, Acute myelogenous leukemia, Fanconi’s anemia, Peta-thalassemia,

Crohn’s disease, Chronic myelogenous leukemia, Hodgkin’s disease, Multiple myeloma,

Multiple sclerosis, Myelodysplastic syndrome, Non-hodgkin’s lymphoma, Renal carcinomia,

Sarcomas, Sickle cell disease , Systemic lupus erythematosis, Wiskott-Aldrich syndrome, Type 1

diabetes, Rheumatoid arthritis, Osteoarthritis, Kidney diseases, Blindness, Muscular dystrophy,

Liver disease, Loss of teeth, Baldness, and Aplastic Anemia. (Carrier & Ledingham, 2004)

Type 1 diabetes in children is an autoimmune disease characterized by destruction of

insulin producing cells in the pancreas. Current efforts to treat these patients with human islet

transplantation in an effort to restore insulin secretory function (obtained from human pancreas)

are limited severely by the small numbers of donated pancreas available each year combined

with the toxicity of immunosuppressive drug treatments required to prevent graft rejection.

Pluripotent stem cells, instructed to differentiate into a particular pancreatic cell called a beta

cell, could overcome the shortage of therapeutically effective material to transplant. They also

afford the opportunity to engineer such cells to effectively resist immune attack as well as graft

rejection. (Scott, 2005)

Many nervous system diseases result from loss of nerve cells. Mature nerve cells cannot

divide to replace those that are lost. Thus, without a “new” source of functioning nerve tissue, no

therapeutic possibilities exist. In Parkinson’s disease, nerve cells that make the chemical

dopamine die. In Alzheimer’s disease, cells that are responsible for the production of certain
neurotransmitters die. In amyotrophic lateral sclerosis, the motor nerve cells that activate

muscles die. In spinal cord injury, brain trauma, and even stroke, many different types of cells

are lost or die. In multiple sclerosis, glia, the cells that protect nerve fibers are lost. Perhaps the

only hope for treating such individuals comes from the potential to create new nerve tissue

restoring function from pluripotent stem cells. (Scott, 2005)

Pluripotent stem cells could be used in treatment of virtually all primary

immunodeficiency diseases. Presently, there are more than 70 different forms of congenital and

inherited deficiencies of the immune system that have been recognized. These are among the

most complicated diseases to treat with the worst prognoses. Included here are diseases such as

severe combined immunodeficiency disease (the “bubble boy” disease), Wiskott-Aldrich

Syndrome, and the auto-immune disease lupus. The immune deficiencies suffered as a result of

acquired immune deficiency syndrome (AIDS) following infection with the human

immunodeficiency virus are also relevant here. These diseases are characterized by an unusual

susceptibility to infection and often associated with anemia, arthritis, diarrhea, and selected

malignancies. However, the transplantation of stem cells reconstituted with the normal gene

could result in restoration of immune function and effective normalization of life span and

quality of life for these people. (Scott, 2005)

Stem cells, once appropriately differentiated, could correct many diseases and

degenerative conditions in which bone or cartilage cells are deficient in numbers or defective in

function. This holds promise for treatment of genetic disorders such as osteogenesis imperfecta

and chondrodysplasias. Similarly, cells could be cultivated and introduced into damaged areas of

joint cartilage in cases of osteoarthritis or into large gaps in bone from fractures or surgery.

(Scott, 2005)
 At the present time, bone marrow stem cells, representing a more committed stem cell,

are used to rescue patients following high dose chemotherapy. Unfortunately, these recovered

cells are limited in their capacity to restore immune function completely in this setting. It is

hoped that injections of properly-differentiated stem cells would return the complete repertoire of

immune response to patients undergoing bone marrow transplantation. Complete and functional

restoration will be required if, for example, immune/vaccine anticancer therapy is to work. More

importantly, success would permit use of very toxic (and effective) chemotherapeutic regimens

that could not currently be utilized for lack of an ability to restore marrow and immune function.

(Scott, 2005)

Stem cell therapy offers a lot of medical benefits, showing great potential in the treatment

of a number of conditions, in the therapeutic sectors and regenerative medicine and cloning. In

the future, the stem cell research can allow the scientists to test a number of potential medicines

and drugs without carrying out any test on animals and humans. Stem cell therapy puts into use

of the patient’s body hence, the risk of rejection can be reduced because the cells belong to the

same human body. (Buban, 2009)

Stem cell research offers a lot of benefits not only in the field but also in humans.

Professor Susan Lim, together with her team of researchers of Stem Cell Technologies, is

currently working on an innovative treatment for diabetes using adult stem cells taken from our

fat. Since body fats are composed of baby fat cells, stem cells can be separated from these tissues

and can be harvested in a laboratory dish to pattern in into the type of cells required. Thinking

that all your unwanted fats can be turned into a cure for diseases, this type of creative medicine

has a lot of extensive implications. (Lim, Tan, Hian, & Sim, 2007)
 Adult stem cell research has made amazing progress. Although it will be years before it

can be used as a standard therapy, it has already produced very promising results. Here are few

of examples of what has been reported in leading scientific journals.

• In Dusseldorf, in July 2001, German doctors reported that a patient’s own bone

marrow adult stem cells were used to regenerate tissue damaged by a heart attack,

improving his heart function.

• US doctors have taken adult stem cells from the brain of a patient with

Parkinson’s disease and reimplanted them resulting in an 83% improvement in the

patient.

• Washington Medical Centre treated 26 patients with rapidly deteriorating multiple

sclerosis. Twenty patients stabilized and six improved.

• Israeli doctors inserted adult blood stem cells into a paraplegic woman’s spinal

cord. She regained bladder control and the ability to wiggle her toes and to move

her legs.

• Immune systems of children destroyed by cancer were restored using umbilical

cord blood (these are adult stem cells).

• Surgeons in Taiwan have restored vision to a patient with severe eye damage

using stem cells from the patient’s own eyes.

• In the US adult stem cells have been used to treat sickle cell anemia. Adult

pancreatic islet cells were beneficial in helping 15 patients with insulin dependent

diabetes improve.
 • A young woman rendered paraplegic by a car accident can move her toes and legs

after injection of her own immune-system cells into her severed spinal cord.

• In the UK a three-year-old boy was recently cured of a fatal disease by the use of

stem cells extracted from his sister’s placenta.

The use of embryonic stem cell for research involves the destruction of the blastocytes

that are formed from the laboratory fertilization of the human egg. Also, the cells will not be

from the same human body and these are chances of rejection. The long term side effects of the

therapy are still unknown. Also, the stem cell therapy is still under the process of research and

there are a number of things to be established before it is used as a treatment line.

If the cells used in the therapy are embryonic, then the disadvantage is that the cells will

not be from the same human body or patient, it is from a human embryo, and we all know that

the process of obtaining them is by destroying and it is unethical. Embryonic stem cells are

versatile but they can also become malignant. So there’s a chance that these cells can cause

cancer.

While many actual benefits have been obtained from adult stem cells over recent years,

they have as yet no demonstrated benefit to human patients from embryonic stem cells. The use

of adult stem cells seems to overcome the problem of immune rejection, which will be a big

problem with the use of embryonic stem cells. (Abboud)

It has been estimated that the therapeutic applications of stem cell are very expensive that

such individualized stem cell treatments could cost at least $100, 000 per patient. Institutions

estimated that egg retrieval alone would cost over $20, 000 per procedure. Observers worry that

the development of such individualized treatments could also worsen existing inequities in the
provision of health care, and question whether public funds should be devoted to this approach to

stem-cell based treatments especially during a period in which funding for public health is being

cut. Other health policy experts have raised concerns about whether the fund now being

committed to stem cell research represents the best use of scarce health research funds. (Hayes,

2006)

Stem cell’s low cycle time leading to slow replication in culture and the fact there may be

selective pressure for the faster growing but possibly abnormal cells to dominate a culture

system; the instability of embryonic cells in general and their remarkable propensity to produce

abnormal numbers of chromosomes; the difficulty in weeding out all rogue cells that might

proliferate; the risk that stem cells after forced differentiation in culture may undergo de-

differentiation or abnormalities of gene-expression after transfer to the patient with potential for

huge harm.(Scott, 2005)