Alzheimer's Disease and Stem Cells
By Lawrence S. Goldstein, Ph.D.

Alzheimer's disease is increasingly common and is known to act by killing brain cells. However, scientists still have only a rudimentary understanding of what causes this disease, and how it kills brain cells. Importantly, this limited understanding of the disease is a major barrier to the discovery of truly effective and potent therapies that might cure or mitigate this horrible neurodegenerative disorder.

Recently, there has been a great deal of attention focused on the value of human embryonic stem cells in the fight against Alzheimer's disease. Indeed, scientists are working hard to bring the day when stem cells might be effectively used to treat this disease. This type of therapy will require an understanding of how to use the stem cells to make, and then replace, the appropriate brain cells that are lost in disease.

At this point in time, we need additional research to identify those cells that might be most appropriate to replace. However, there is another very important and unique use that these cells can also play in better understanding and treating the disease.

Human embryonic stem cells can be used to develop a sophisticated understanding of how Alzheimer's Disease develops, what its causes are, and how new therapeutic drugs might be developed. There are several avenues of this work that are beginning in a number of different laboratories. For example, one important goal is to genetically modify these stem cells to carry the types of mutations that are involved in the development of Alzheimer's Disease. Then, in the laboratory, the modified stem cells can be directed to form the types of brain cells that die in Alzheimer's Disease.

Studies of these Alzheimer's mutant brain cells will likely yield novel insights into how Alzheimer's disease develops, the causes of the disease and new approaches for developing drugs to combat the disease. In fact, these cells may become uniquely valuable for developing and testing new drugs for treating Alzheimer's disease.

Lawrence S. Goldstein, PhD, is a Howard Hughes Medical Investigator at the UCSD School of Medicine and a specialist in Alzheimer's disease research.

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Updated: February 22, 2005