Wednesday, June 20, 2012

New stem cell findings point to future therapies for spinal musclar atrophy

Clive Svendsen
In the past few years, stem cell researchers have been taking advantage of the ability to create embryonic-like stem cells from skin to develop laboratory models of disease. The idea is simple: first, take skin from someone with a genetic disease (the skin cells will have the same genetic mutation as the cells effected in the disease). Next, turn those skin cells into embryonic-like stem cells called iPS cells. Then mature those stem cells into the type of cell that goes awry in the disease.

So far, researchers have followed this procedure to create cells in the lab dish with symptoms of Parkinson’s disease, autism, schizophrenia, Alzheimer’s disease, ALS and others. Researchers at Cedars-Sinai used the technique to create a laboratory model of the fatal childhood disorder spinal muscular atrophy, or SMA, back in 2009. Now, in work published in the June 19 online issue of PLoS ONE they have created more lab models of SMA but with newer techniques that are less likely to cause unwanted changes in the cells.

The point of all this work is to figure out what goes wrong in the disease, and to find drugs to fix the problem. Since all of these diseases occur in living neurons, which in general people are loath to part with, scientists have never had a way of studying how the disease forms in the lab. And if you don’t know what goes wrong in the first place, how can you fix it?

The latest in these disease-in-a-dish models comes from CIRM grantees led by Clive Svendsen who created motor neurons from the skin of two people with SMA. In the roughly 100,000 newborns born with the disease each year, the motor neurons that control muscle movement don’t form properly and the children are generally paralyzed by age three.

Svendsen and his group found that in the lab dish, the SMA stem cells didn’t form as many motor neurons as iPS cells from people without the disease. What they noticed is that the cells seemed to be dying off through a regulated process of cell death known as apoptosis. When they exposed the neurons to molecules that block apoptosis, the neurons lived.

A press release from Cedars-Sinai quotes Svendsen, who is director of their Regenerative Medicine Institute.
“With this new understanding of how motor neurons die in spinal muscular atrophy patients, we are an important step closer to identifying drugs that may reverse or prevent that process.”
The goal now is to start testing drugs on these SMA neurons to find ones that seem to reverse the disease. Those drugs could then become possible therapies for people with SMA.

CIRM funds $15 million in awards that could one day benefit people with SMA. You can see the complete list of those awards here.



  1. Hello,

    Nice post. Stem cell treatments have the potential to change the face of human disease and alleviate suffering. The ability of stem cells to self renew. I like your site it is very informative. Thanks a lot for creating this type of valuable site...

    1. You are missing the point to the entire article........Stem Cells have shown no success at growing out. The research community should spend $30 million and get one to grow out correctly, if they could ever get that to happen they can then focus on the roughly 1,000 other specific motor neurons and try to get each one of those to project out. Thats why the article clearly states the best use of stem cells is to model diseases to test other therapies on. Not to actually correct diseases themselves. There are "Rockstar" celebrity researchers that have done great damage to Stem Cell research. I am happy there are respected researchers like Clive that actually tell the truth.

    2. Israeli Channel 2 reports: Dramatic Change in Patient with ALS and MG following Compassionate Use of BrainStorm's NurOwn(TM) Cell Therapy

      PETACH TIKVA, Israel, Jul 02, 2012 (BUSINESS WIRE) -- BrainStorm Cell Therapeutics Inc. BCLI +19.13% ; Israeli Channel 2 TV interviewed today a patient suffering from Myasthenia Gravis (MG) and recently diagnosed with ALS. The patient reported that he has experienced visible improvement in his speech, walking, balance, posture, muscle strength, appetite, digestion, and weight gain following compassionate treatment with BrainStorm's NurOwn cell therapy.

      "Due to the rare disease combination of MG and ALS, this patient was approved for compassionate treatment with BrainStorm's NurOwn cell therapy," says Professor Dimitrios Karussis of the Neurology Department at The Hadassah Medical Center in Jerusalem, and Principal Investigator of BrainStorm's current Phase I/II clinical trial. "Within a few weeks following injection with NurOwn cells, the patient showed dramatic improvement in a variety of functions including breathing, speech, walking, muscular strength, and overall well-being. While we cannot draw scientific conclusions based on the outcome of an individual patient, these results are extremely encouraging."

  2. Your own post answers itself

    "While we cannot draw scientific conclusions based on the outcome of an individual patient, these results are extremely encouraging."

    Anyone can make dramatic is the scientific data that speaks volumes.