Scientists Pushing for Breakthroughs in Lou Gehrig's Disease

by Andrew Beckner 9/11/2008 2:52:31 AM

Since claiming the life of New York Yankee legend Lou Gehrig decades ago, amyotrophic lateral sclerosis (ALS), more commonly known as Lou Gehrig’s Disease, has gone without any medical breakthroughs. However, using a new technique to reprogram cells, scientists are growing neurons from people with the illness, which may be a possible first step in understanding how it develops.

ALS is a disease that damages the nerve cells in the brain and spinal cord, eventually leading to death. The ALS Association estimates that as many as 30,000 Americans may have the disease at any given time. "What we now have in the culture dish is cells that have the same genetic makeup as the ALS patient and they are the same cells that are affected by the disease," said Dr. Chris Henderson, co-director of the Center for Motor Neuron Biology and Disease at Columbia University. This means that, for the first time, scientists hope to be able to study the development of the disease in the cells and, from that, possibly begin studies for treatments.

The cells were acquired using skin cells of two patients, ages 82 and 89. A report from the journal Science says that the scientists were able to “reprogram” the cells into a form of adult stem cells, then changed them into nerve cells. Dr. Kevin Eggan of the Harvard Stem Cell Institute, a co-author of the journal, says that now they hope to do studies of the cells in a lab and compare them to cells that do not have the disease.

Henderson says that even though the vast majority of cases are caused by different triggers, they hope to learn about the mechanism of the disease and how it develops after being triggered.

"Up until now, it's been impossible to get access to the neurons affected by ALS and, although everyone was excited by the potential of the new technology, it was uncertain that we would be able to obtain them from patients' skin cells," Henderson said.

"Our paper now shows that we can generate hundreds of millions of motor neurons that are genetically identical to a patient's own neurons. This will be an immense help as we try to uncover the mechanisms behind this disease and screen for drugs that can prolong life."

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