July 31, 2008
Human Neurons Made From Skin Cell Of ALS Patient

Using viruses to introduce genes that are known to control pluripotency (i.e. embryonic state) of cells some Harvard researchers managed to take skin cells from a women suffering from Amyotrophic Lateral Sclerosis (ALS - Lou Gehrig's disease) and make them into first stem cells and then neurons.

Cambridge, MA, July 31, 2008 - Less than 27 months after announcing that he had institutional permission to attempt the creation of patient and disease-specific stem cell lines, Harvard Stem Cell Institute (HSCI) Principal Faculty member Kevin Eggan today proclaimed the effort a success - though politically imposed restrictions and scientific advances prompted him to use a different technique than originally planned.

The breakthrough by Eggan and colleagues at Harvard and Columbia University marks the first time scientists are known to have produced human stem cell lines coaxed from the cells of adult patients suffering from a genetically-based disease. The affected patients had Amyotrophic Lateral Sclerosis (ALS), commonly known as Lou Gehrig's disease.

The ability to induce pluripotency on demand is impressive. The research into what causes cells to be embryonic stem cells and to be other kinds of stem cells is beginning to bear fruit. As scientists learn more about the internal genetic state of cells they will find easier and safer ways to control cell state and to produce therapies for almost all diseases.

The initial intent is to use these cells to study ALS. The longer term intent is to create neural stem cells that could be used to repair brains that are decaying due to ALS.

The work, published in today's on-line edition of the journal Science, provides "proof of concept" for the belief of scientists and fervent hope of patients that in the not-too-distant future it may be possible to treat patients suffering from chronic diseases with stem cell-based treatments created from their own adult cells. However, Eggan believes that the first therapeutic use of these newly derived stem cells will in fact be to use them to study the root cause of this disease and to screen for drugs that may provide benefit in patients.

This method does not currently produce cells that are safe to use in therapy. The viruses are a rather blunt instrument for introducing genes into cells and the cells run the risk of becoming cancerous. We need improved ways to do gene therapy and to regulate the epigenetic state of cells. That will all come with time. But the faster it comes the more likely you won't die from some disease before rejuvenation therapies become available.

The cells will be studied to understand what goes wrong in ALS neurons that cause them to die.

"This is a seminal discovery," said Valerie Estess, director of research for Project A.L.S. "The ability to derive ALS motor neurons through a simple skin biopsy opens the doors to improved drug discovery. For the first time, researchers will be able to look at ALS cells under a microscope and see why they die. If we can figure out how a person's motor neurons die, we will figure out how to save motor neurons."

Share |      Randall Parker, 2008 July 31 11:35 PM  Brain Stem Cells


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