First Personalized Stem Cells A Success As Lou Gehrig's Disease First Target

By Nikki Weingartner.
Published Aug 1, 2008 by  Nikki Weingartner - 13 votes, 6 comments
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A new study released in a recent online version of Science reveal that disease specific stem cells have been successfully reprogrammed. Despite some hurdles, this find opens up a wealth of doors with regards to treatments for the fatal disease.
Until now, medical researchers were limited in their ability to study the cell lines of Lou Gehrig's patients, also known as ALS. However, a recent study has opened a door of hope for the debilitating and terminal disease.

As explained in a scientific news report by Science Daily:

This is the first time that skin cells from a chronically-ill patient have been reprogrammed into a stem cell-like state, and then coaxed into the specific cell types that would be needed to understand and treat the disease.

By using a Japanese recipe for cell reprogramming, scientists from Harvard and Columbia Universities used skin samples from two sisters who shared a genetic mutation responsible for a small percentage of all ALS cases and put to test whether the skin cells could be reprogrammed back to an embryonic state.

ALS is caused by the degeneration and eventual death of motor neurons, those responsible for voluntary muscle movement in the body. Patients who suffer from the disease often die from breathing complications within a few years after initial symptoms present themselves.

Because there are currently no treatments that will reverse the course of the disease, only drugs that slightly slow its progression, this benchmarking research opens doors for drug testing and possible motor neuron replacement therapy in patients with ALS. It also opens the door for other diseases such as Parkinson's Disease and Alzheimer's Disease, both of which affect patients later in life.

The technique had previously only been successful on healthy human tissue so for stem cell experts abroad, this study does more than just open up the possibilities for those with ALS. However, additional research is needed before medical scientists get to where they would like to be.

The Los Angeles Times reported of the study's success:

The cells expressed the same markers as embryonic stem cells and were able to grow into all the body's main tissue types.

When the scientists exposed the cells to certain small molecules, the jumbles of tissue began to differentiate into motor neurons, the cells that regulate voluntary muscle movement.

They also found evidence of glial cells, a crucial component of the central nervous system.

With around 30,000 people affected in the United States alone by ALS, this study gives hope to so many.
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