Research on stem cells is advancing knowledge about how an organism develops from a single cell and how healthy cells replace damaged cells in adult organisms. This promising area of science is also leading scientists to investigate the possibility of cell-based therapies to treat disease, which is often referred to as regenerative or reparative medicine.

Stem cells are one of the most fascinating areas of biology today. But like many expanding fields of scientific inquiry, research on stem cells raises scientific questions as rapidly as it generates new discoveries.

There are many areas in medicine where stem cell research could have a significant impact. For example, there are a variety of diseases and injuries in which a patient's cells or tissues are destroyed and must be replaced by tissue or organ transplants. Stem cells may be able to generate brand new tissue in these cases, and even cure diseases for which currently there is no adequate therapy. Diseases that could see revolutionary advances include Alzheimer's and Parkinson's disease, diabetes, spinal cord injury, heart disease, stroke, arthritis, cancer, and burns.

Parkinson's Disease

Parkinson's disease (PD) is a very common neurodegenerative disorder that affects more than 2% of the population over 65 years of age. PD is caused by a progressive degeneration and loss of dopamine (DA)-producing neurons, which leads to tremor, rigidity, and hypokinesia (abnormally decreased mobility). It is thought that PD may be the first disease to be amenable to treatment using stem cell transplantation. Factors that support this notion include the knowledge of the specific cell type (DA neurons) needed to relieve the symptoms of the disease. In addition, several laboratories have been successful in developing methods to induce embryonic stem cells to differentiate into cells with many of the functions of DA neurons.

In a recent study, scientists directed mouse embryonic stem cells to differentiate into DA neurons by introducing the gene Nurr1. When transplanted into the brains of a rat model of PD, these stem cell-derived DA neurons reinnervated the brains of the rat Parkinson model, released dopamine and improved motor function.

Regarding human stem cell therapy, scientists are developing a number of strategies for producing dopamine neurons from human stem cells in the laboratory for transplantation into humans with Parkinson's disease. The successful generation of an unlimited supply of dopamine neurons could make neurotransplantation widely available for Parkinson's patients at some point in the future.

Alzheimer's

Stem cell research may provide useful insights that could be helpful in finding ways to prevent and treat Alzheimer's disease, even if it is not likely to lead to specific treatments in the near future. Science is the only place we can turn for clues and eventual answers to Alzheimer's. We have every reason to believe we are approaching a breakthrough in understanding and treating the disease, if Congress continues its support and increases funding for general research into Alzheimer's and if scientists are allowed to pursue all the promising leads that are beginning to develop.

An estimated 4 million Americans have Alzheimer's disease today. Because Alzheimer's is so closely linked to age, demographic changes alone mean that by the middle of this century, 14 million Americans - one in ten over age 65 and one in two persons who live to be 85 - will have the disease. Today, there is absolutely nothing anyone can do to prevent the onset of Alzheimer's or the inevitable degeneration and eventual death that Alzheimer's causes in every person who gets the disease.

Huntington's Disease

HD is a genetically caused brain disorder that causes uncontrollable bodily movements and robs people's ability to walk, talk, eat, and think. An estimated 30,000 Americans have it, and at least 150,000 more are at risk. HD affects both sexes and all ethnic groups and strikes in the prime of life. Everybody carries the Huntingtin gene, but in some people it goes awry, leading to an attack on the brain. The final result is a slow, ugly death. Children of a parent with HD have a 50-50 chance of inheriting the condition, and the disease can start as early as childhood. There is no cure of treatment.

HD causes immense suffering and loss of productivity. In San Diego alone as many as six families per month find out that HD has struck them. Thousands of caregivers across America must help the ill do the simplest tasks. Affected individuals lose jobs and insurance. But there is hope. In 1993 scientists found the HD gene. HD is on the cutting edge of biotechnology. HD research provided a boost to the Human Genome Project, and progress in HD research has generated many new investigative techniques. As scientists move towards a cure, they are increasing our understanding of the brain and having a tremendous impact on the study of cystic fibrosis, Lou Gehrig's disease, Alzheimer's, Parkinson's, certain forms of diabetes, and other maladies. Stopping HD will be a historic breakthrough leading to a better life for all.



Special thanks to Amy for providing us with HD information!


Human Embryonic Stem Cell Video

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