There are several reasons why the isolation of human pluripotent stem cells might lead to better treatment, even cures, of many diseases. At the most fundamental level, pluripotent stem cells could help us to understand the complex events that occur during normal human development. By identifying the mechanisms underlying routine cell differentiation we hope to understand how disease-causing aberrations occur. Another goal of this research would be the identification of the factors involved in the cellular decision-making process that results in cell specialization -- why do some cells become heart cells, for example, while other cells become liver cells? We know that turning genes on and off is central to this process, but we do not know much about these "decision-making" genes or what turns them on or off. Some of our most serious medical conditions, such as cancer and birth defects, are due to abnormal cell differentiation and cell division. A better understanding of normal cell processes will allow us to further delineate the fundamental errors that cause these often deadly illnesses.
Human pluripotent stem cell research could also dramatically change the way we develop drugs and test them for safety. While a limited number of cultivated cell lines are currently available and provide invaluable tools for drug development and testing, pluripotent stem cells would allow expansion of this testing to more varied cell types. For example, drugs could be tested first on particular cell lines to determine toxicity, before they are tested in either animals or humans. Although this would not replace testing in animals and in human beings, it would streamline the process of drug development, and reduce potential for harm in humans and animals. 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 "cell transplantation therapies," which are aimed at diseases and disorders resulting from the destruction or dysfunction of specific cells and tissue. Although donated organs and tissues can sometimes be used to replace diseased or destroyed tissue, the number of people suffering from such disorders far outstrips the number of organs and tissues available for transplantation. Pluripotent stem cells, stimulated to develop into specialized cells and tissue, offer real hope for the possibility of a renewable source of replacement cells and tissue to treat a myriad of diseases, conditions, and disabilities for which replacement tissue is in short supply. Examples of these include neurological disorders, burns, heart disease, osteoarthritis and rheumatoid arthritis.