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Patient stem cells help identify common problem in ALS

Patient stem cells help identify common problem in ALS | Stem Cells & Tissue Engineering | Scoop.it
Harvard stem cell scientists have discovered that a recently approved medication for epilepsy may possibly be a meaningful treatment for amyotrophic lateral sclerosis (ALS)—Lou Gehrig's disease, a uniformly fatal neurodegenerative disorder. The researchers are now collaborating with Massachusetts ...
Jacob Blumenthal's insight:

In two papers, published in Cell Reports, and in Cell Stem Cell, researchers used induced pluripotent stem cells (iPSC) derived from Amyotrophic Lateral Sclerosis (ALS) patients cells to study  aberrant molecular mechanisms related to ALS  in-vitro. In the first paper, researchers from the labs of Kevin Eggan and Clifford Woolf found that iPSC-derived motor neurons from ALS patients are hyperexcitable compared to controls, and that the drug Retigabine,  which has recently been approved as an anticonvulsive, could rescues the hyperexcitability phenotype in multiple ALS variants.

In the second paper, researchers from the lab of Su-Chun Zhang found that neurofilament aggregation is an early phenotype of ALS, and that correction of  neurofilament expression, mitigates axonal degeneration in ALS motor neurons.

http://www.cell.com/cell-reports/abstract/S2211-1247(14)00197-1

http://www.cell.com/cell-stem-cell/abstract/S1934-5909(14)00055-1

 

Learn about motor neurons development, pathologies, gene expression and related motor neurons derivation protocols:

http://discovery.lifemapsc.com/in-vivo-development/motor-neurons

 

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Motor neurone disease: how could stem cells help?

Motor neurone disease: how could stem cells help? | Stem Cells & Tissue Engineering | Scoop.it
What is motor neurone disease?The term motor neurone disease (MND) describes several different conditions that all affect nerve cells called motor neurons. Motor neurons are found in the brain and spinal cord.
Jacob Blumenthal's insight:

This is a very good summary on motor neurons disease (MND), and the therapeutic potentital os stem cells.

Here's a patient view on ALS from Stephen Hawking: http://www.hawking.org.uk/living-with-als.html

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New Use for Stem Cells Identifies a Promising Way to Target ALS | Harvard Stem Cell Institute

New Use for Stem Cells Identifies a Promising Way to Target ALS | Harvard Stem Cell Institute | Stem Cells & Tissue Engineering | Scoop.it
RT @Mayanazatz: Pesquisadores de Harvard identificam nova droga promissora para combater ELA
http://t.co/MPMbvLaUcQ
Jacob Blumenthal's insight:

This paper describes  a new small molecule survival screen carried out using motor neurons from both wild-type and mutant SOD1 mouse embryonic stem cells. A new small molecule, termed kenpaullone had a particularly impressive ability to prolong the healthy survival of both types of motor neurons that can be attributed to its dual inhibition of GSK-3 and HGK kinases.

Link to the abstract: http://www.cell.com/cell-stem-cell/abstract/S1934-5909(13)00139-2

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Stem cells for treatment of ALS at Mayo Clinic - YouTube

Researchers in the Center for Regenerative Medicine are studying the use of intraspinal deliver of mesenchymal stem cells (MSCs) to the cerebral spinal fluid...
Jacob Blumenthal's insight:

This is a nice movie illustrating the process of isolation and transplantation of growth factor secreting-mesenchymal stem cells (MSCs) for the treatment of amyotrophic lateral sclerosis (ALS), currently being held at the Mayo Clinic.

Join my Facebook group for more stem cells news and updates:

 https://www.facebook.com/groups/STEMCELLSNET/

 

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Cell Stem Cell - A Small Molecule Screen in Stem-Cell-Derived Motor Neurons Identifies a Kinase Inhibitor as a Candidate Therapeutic for ALS

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This is an open access paper published this week!

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