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 PROJECT A.L.S.-FUNDED DISCOVERIES SPEED APPROACHES TO THERAPY
July 31, 2008 - Three just published studies move us closer to effective treatments for ALS
Project A.L.S.’s Harvard-Columbia Team Derives Millions of ALS Motor Neurons from Patient Skin Samples
Thanks to an ongoing collaboration catalyzed and funded by Project A.L.S., scientists have generated ALS motor neurons--from a patient’s own skin. Utilizing an approach called iPS, the Project A.L.S. team, led by Kevin Eggan and Chris Henderson, will now use these motor neurons to observe the ALS disease process “live and up close” for the first time, and to more accurately screen for effective drugs. A long-term goal generated by this study is the transplantation of healthy motor neurons back into ALS patients. Read the paper in Science: (link to paper)Read: The Project A.L.S. press release Read: The Paper in Science Key Gene for Making Motor Neurons Discovered
Led by Howard Hughes Medical Institute and Columbia University investigator Thomas M. Jessell, the cover story in this month’s issue of the journal Cell identifies a gene instrumental in guiding not only the early development of motor neurons, but the precise connections between the spinal cord and target muscles. The gene, Foxp1, will allow scientists to speed drug discovery for ALS. In addition, Foxp1 will help scientists to generate stem cells that give rise to specific sub-classes of motor neurons. For example, we will now be able to use various “recipes” to create stem cell-derived motor neurons that innervate the limbs, breathing muscles, and speech and swallowing muscles.Read: The paper in Cell Improved Delivery of IGF-1 in ALS
For years, doctors have been trying to treat ALS with growth factors, including IGF-1. One of the big hurdles to IGF-1 therapy has been the brain-blood-barrier (BBB), which nature built to protect the brain from harm. But the BBB also acts as a barrier that prevents potentially helpful medicine from entering the brain. Led by Project A.L.S.-funded team, a new study jumps the BBB hurdle and provides dramatic evidence of improved delivery of IGF-1 in ALS.Read: The paper in Molecular Therapy Click here for more information or to make a donation to Project A.L.S. "Through the work of the Jenifer Estess Laboratory for Stem Cell Research we now can glimpse the new age of ALS research, an age of progress and promise." --
Thomas Jessell
Howard Hughes Investigator Columbia University |
