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Thread: Keirstead Lab Publishes Motor Neuron Paper

  1. #1
    Senior Member
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    Feb 2006
    Murrieta, Ca.

    Keirstead Lab Publishes Motor Neuron Paper

    Here's a paper published by Hans Keirsteads Lab about the Motor neuron work he's doing with the SMA community.

    It shows what a small community can do with a focused effort, $400k investment so far the article states, seems like alot of progress in a relativley short time for a small investment.

    Hans has his eye on the ball.... next stop Human Clinical Trials

  2. #2
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    Mar 2007
    This is different than the Geron trial?

  3. #3
    Senior Member Schmeky's Avatar
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    Sep 2002
    West Monroe, LA, USA

    Wow. I'm speechless. This is spectacular to say the least, and the time frame this was accomplished in is unprecedented. Keirstad is smokin' hot. I wonder if parallel SCI research has been ongoing with the SMA work? No one I am aware of goes from the lab to clinical trails as fast as Keirstad. Simply amazing.


    Entirely different scope of research. Geron's trial is based on remylination of denuded, surviving axons in the acute phase.

  4. #4
    Schmecky as opposed to remyelination in chronic stage; where are the differences annd will this be appp;icable to chronics?

  5. #5
    Wow, Families of SMA is on the ball. Raising 400,000 alone.

    If they can do it, we can do it. SCINetUSA has only raised 70,000 thus far.

  6. #6
    Quote Originally Posted by momo3 View Post
    This is different than the Geron trial?
    Yes. This would apply to chronic sci.

  7. #7
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    Mar 2007
    How will it apply and to bump keeping on's question. What is the difference?

  8. #8
    This research applies to individuals who will require motor neuron replacement therapy which has the potential to help a number of diseases and conditions.

    Hats off to the Families of SMA!

    Keirstead Laboratory Publishes Important Research Paper on Functionality of Motor Neuron Progenitor Cells Developed with Funding from Families of Spinal Muscular Atrophy.
    July 29, 2010.

    Dr. Hans Keirstead and colleagues at the University of CA at Irvine received two grant awards from Families of SMA between 2005 and 2009 for over $400,000 specifically to develop a pure population of motor neuron progenitor cells with the intent of developing possible therapeutic applications for Spinal Muscular Atrophy. The first research study assessing this cell population has recently been published in the scientific journal PLoS ONE.

    This paper outlines the first peer-reviewed demonstration of functionality for this cell population in animal models of motor neuron related disease.

    The paper is entitled, “Histological and Functional Benefit Following Transplantation of Motor Neuron Progenitors to the Injured Rat Spinal Cord.” It tests the ability of the motor neuron progenitor cells, generated with Families of SMA funding, in an animal model of spinal cord injury.
    Please click here to access the paper online at PLoS ONE.

    In the paper the authors discuss how motor neuron loss is characteristic of cervical spinal cord injury (SCI) and how that contributes to functional deficit. To investigate the feasibility of motor neuron survival and continued development in the injured adult rat spinal cord, they transplanted a high purity population of human motor neuron progenitors (hMNP), which are derived from human embryonic stem cells (hESCs), into the spinal cord of injured animals.

    In a dish, these cells displayed characteristic of motor neuron progenitor cells: 1) expressing known motor neuron markers; 2) having a typical electrophysiological profile, meaning the cells are able to produce electrical signals, which are required to stimulate muscle in living animals; 3) having the ability to functionally innervate and drive movement of human or rodent muscle in culture; and 4) secreting growth factors that cause neuronal branching and promote neuronal survival.

    Importantly, hMNP transplantation into cervical SCI sites in adult rats also resulted in greater survival of endogenous rat neurons originally present in the rat spinal cord, as well as inducing their branching. These neurotrophic (to promote neuronal health and function) effects were accompanied by significantly improved performance of the rats on a balance beam task, as compared to controls. However, hMNP transplantation did not enhance performance on a grip strength task, which assesses distal limb strength.

    Preliminary studies were also done in the, Smn-/-SMN2+/+SMNΔ7-/- mouse model of SMA, often called the Delta 7 Mouse. These mice were transplanted with hMNPs, and human cells were present in all SMA mice in the ventral horns of the spinal cord after about 2 weeks. Continued motor neuron development also occurred in the transplant population in the SMA model, although the cells did not exhibit features of fully mature motor neurons, perhaps due to the short life span of these mice and the fact that motor neuron maturation is a long process.

    These preliminary studies in an SMA mouse have laid the groundwork for testing the ability of hMNPs to enhance function and provide neurotrophic benefit to motor neurons in SMA mice. These studies are currently ongoing in the Keirstead laboratory with recent grant award from FSMA.
    Program Progress

    SMA is a disorder that results from a chronic deficiency in a protein that is essential to the proper functioning of the motor neurons in the spinal cord. CSC, a leading stem cell therapeutics company, has been working to develop a stem cell-derived motor neuron product for the treatment of SMA Type I. Families of SMA has been involved in this program, now called the MotorgraftTM Program, at UCI and CSC since the early days of the program.

    FSMA has funded projects within every stage of the program. The first round of FSMA funding for the program generated a population of human motor neurons in a manner that allowed for very large quantities of cells to be generated, in a purity that permitted human use.

    This population of cells next needed to be tested in animal models to ensure that they function well and in other animal models to ensure that they are safe. This paper is the initial published demonstration of functionality in animal models of motor neuron related disease. Currently, similar studies are underway in SMA mice with funding from FSMA.
    Finally a clinical team was assembled, and a clinical plan agreed upon. Soon the FDA will be presented for their approval with the world’s first clinical trial plan to test the safety and efficacy of a stem cell derived cellular therapy for Spinal Muscular Atrophy.
    Families of Spinal Muscular Atrophy has dedicated monetary resources to the program, which has now successfully:
    • Completed FDA required safety studies - on tumorigenicity, toxicity, biodistribution, and pain
    • Completed manufacturing protocols for clinical-grade cells
    • Completed a pre-IND meeting in October 2009
    • Obtained Orphan Drug Designation from FDA in December 2009
    According to Dr. Keirstead, “In the summer of 2010, the world’s first strategy to treat SMA using stem cells is planned to be presented to the FDA. This rate of progress is extraordinary, and is a testimony to the determination of FSMA in their efforts to conquer this insidious disease.”

    Please click here to read a recent Project Update/Press Release about the program.
    "Our lives begin to end the day
    we become silent about things that matter."
    - Martin Luther King Jr

  9. #9
    Senior Member 0xSquidy's Avatar
    Join Date
    Dec 2009
    Barcelona (Spain)
    Quote Originally Posted by antiquity View Post
    Wow, Families of SMA is on the ball. Raising 400,000 alone.

    If they can do it, we can do it. SCINetUSA has only raised 70,000 thus far.
    Let's call it an investment: SCINetUSA needs serious planning and strategy and it needs it NOW.

    Less hoping and pity and more ambition.
    Don't ask what clinical trials can do for you, ask what you can do for clinical trials.

    Fenexy: Proyecto Volver a Caminar (soon in english too)

  10. #10
    So could this particular therapy (for SMA) also provide hope for lower SCI injuries whereby gray matter (lower motor neurons) need to be replaced? If so, this is a massive stride by Keirstead also.

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