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Stem Cells: An Introduction

Feb 2, 2002

Successful human cloning has recently generated great excitement bringing biomedical research into limelight. The development of human stem cell offers great promises for new therapies and prevention strategies while also opening discussion for ethical issues. Stem cells are considered most important component for human cloning. In this primer on stem cells let us focus on the background and why they hold such a great promise for advances in health care. Stem cells research is a part of the larger study of genomics. Genomics is the entire process of creating gene-based drugs and therapies starting from gene sequencing to analysis and interpretation to drug development. With the recent development in gene sequencing, genomics is a new field, which describes the connection between the advancement of molecular biology, genetics, and drug discovery. Genes can be passed from one generation to the next coding for the exact same trait while remaining intact.

Development of Genetic Medicine

*Source: National Institute of Health, US

As explained earlier, stem cells are considered most important for human gene cloning as they have the ability to divide for indefinite periods in culture and to give rise to other specialized cells as per specifications. They are best described in the context of normal human development. Human development begins when a sperm fertilizes an egg and creates a single cell that has the potential to form an entire organism.

While stem cells are extraordinarily important in early human development, multipotent stem cells are also found in children and adults. For example, consider one of the best-understood stem cells, the blood stem cell. Blood stem cells reside in the bone marrow of every child and adult, and in fact, they can be found in very small numbers circulating in the blood stream. Blood stem cells perform the critical role of continually replenishing our supply of blood cells red blood cells, white blood cells, and platelets throughout life. A person cannot survive without blood stem cells.

Human pluripotent stem cell research could dramatically change the way we develop drugs and test them for safety. For example, new medications could be initially tested using human cell lines. Cell lines are currently used in this way (for example cancer cells). Pluripotent stem cells would allow testing in more cell types. This would not replace testing in whole animals and testing in human beings, but it would streamline the process of drug development. Only the drugs that are both safe and appear to have a beneficial effect in cell line testing would graduate to further testing in laboratory animals and human subjects.

Perhaps the most far-reaching potential application of human pluripotent stem cells is the generation of cells and tissue that could be used for so-called "cells therapies." Many diseases and disorders result from disruption of cellular function or destruction of tissues of the body. Today, donated organs and tissues are often used to replace ailing or destroyed tissue. Unfortunately, the number of people suffering from these disorders far outstrips the number of organs available for transplantation. Pluripotent stem cells, stimulated to develop into specialized cells, offer the possibility of a renewable source of replacement cells and tissue to treat a myriad of diseases, conditions, and disabilities including Parkinson's and Alzheimer's diseases, spinal cord injury, stroke, burns, heart disease, diabetes, osteoarthritis and rheumatoid arthritis. There is almost no realm of medicine that might not be touched by this innovation. Some details of two of these examples follow.

Let us take a practical case. Thus, stem cells can drastically alter the way therapies are given. In the many individuals who suffer from inherent diabetes, the production of insulin by specialized pancreatic cells, called islet cells, is disrupted. Giving insulin injections treats the imbalance or the need for insulin, which is a life long and painful process. There is evidence that transplantation of either the entire pancreas could mitigate the need for insulin injections. Islet cell lines derived from human pluripotent stem cells could be used for diabetes research and, ultimately, for transplantation.

While this research shows extraordinary promise, there is much to be done before we can realize potential from these innovations. Technological challenges remain before these discoveries can be incorporated into clinical practice. These challenges, though significant, are not insurmountable. The development of stem cell lines, both pluripotent and multipotent, that may produce many tissues of the human body is an important scientific breakthrough. It is not too unrealistic to say that this research has the potential to revolutionize the practice of medicine and improve the quality and length of life.


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