Motor Neurons Spared In SMA, ALS and Spinal Cord Injury Models, Progenitor Transplantation Demonstrates Motor Neurons Sparing

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“We’ve taken a critical step forward in directing stem cell differentiation, and illustrating their ability to spare spinal cord cells from loss after injury or disease," said Hans Keirstead, Ph.D. This will be a powerful treatment strategy for diseases characterized by motor neuron loss such as ALS, SMA and spinal cord injury.

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We see a future where we will have the ability to maintain dying motor neurons by delivering motor neuron-specific neurotrophic support.

Each day brings us closer to a treatment. The landmark research manuscript, "Human Motor Neuron Progenitor Transplantation Leads to Endogenous Neuronal Sparing in 3 Models of Motor Neuron Loss," by Tanya J Wyatt, Sharyn L Rossi, Monica M Siegenthaler, Jennifer Frame, Rockelle Robles, Gabriel Nistor and Hans S Keirstead, was published yesterday in the journal Stem Cells International.

Hans Keirstead, PhD, UC Irvine Professor of Anatomy and Neurobiology and Neurosurgery at the Reeve-Irvine Research Center, and Chairman of the Scientific Advisory Board of California Stem Cell (CSC), led the science teams from UC Irvine and California Stem Cell (CSC), who devised this important treatment.

“We’ve taken a critical step forward in directing stem cell differentiation, and illustrating their ability to spare spinal cord cells from loss after injury or disease. We see a future where we will have the ability to maintain dying motor neurons by delivering motor neuron-specific neurotrophic support.This will be a powerful treatment strategy for diseases characterized by motor neuron loss such as ALS, SMA and spinal cord injury. We are encouraged, and cautiously optimistic.”

The manuscript will be published in the journal Stem Cells International, which is an open access journal.

ABSTRACT

Motor neuron loss is characteristic of many neurodegenerative disorders and results in rapid loss of muscle control, paralysis, and eventual death in severe cases. In order to investigate the neurotrophic effects of a motor neuron lineage graft, we transplanted human embryonic stem cell-derived motor neuron progenitors (hMNPs) and examined their histopathological effect in three animal models of motor neuron loss. Specifically, we transplanted hMNPs into rodent models of SMA (Δ7SMN), ALS (SOD1 G93A), and spinal cord injury (SCI). The transplanted cells survived and differentiated in all models. In addition, we have also found that hMNPs secrete physiologically active growth factors in vivo, including NGF and NT-3 a, which significantly enhanced the number of spared endogenous neurons in all three animal models. The ability to maintain dying motor neurons by delivering motor neuron-specific neurotrophic support represents a powerful treatment strategy for diseases characterized by motor neuron loss.

ABOUT CALIFORNIA STEM CELL

Founded in 2005, California Stem Cell is to catalyze the efficient development of human therapies based on human embryonic stem cells. CSC has developed proprietary methods for scalable production of human motor neurons, neuronal progenitors, hepatocytes, and cardiac cells, at high purity, from hESCs.

CSC is currently developing stem cell based therapies for spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS, or Lou Gehrig's Disease). CSC also markets high-purity human cells in platforms suitable for high throughput and high content screening of conventional drug candidates using human cell substrates, predictive toxicity of conventional drugs using human cell substrates, and development of experimental research tools

CSC is a privately held Delaware Corporation with headquarters and research facilities in Irvine, CA. CSC can economically supply large quantities of high purity cells for our own clinical development pipeline in specific application areas. CSC can also supply high purity, fully characterized, clinically relevant human cell populations to companies or other institutions for development of therapies, efficacy screening or creation of toxicity profiles for candidate drugs, or experimental research.

To learn more about California Stem Cell please visit: http://www.californiastemcell.com
Follow us on Twitter: http://twitter.com/castemcell

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Kelly Rice, Partner
QuantumMethod
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Chris N. Airriess, Ph.D; Chief Operating Officer