tuMore Resources To Help You Stay Informed
The Diane LaMotte ALS Foundation does not endorse or recommend any clinical trial or study. Content on this website is to help you and guide you in your search for diagnosis and treatment of ALS. As always you should be your own advocate in the care of your ALS patient and seek professional advice for all your decisions related to this care.
SOD1 Gene Implicated in Familial and Sporadic ALS
October 17, 2010 – Until now, the mutation to the gene SOD1 has been associated with a small number of inherited cases of ALS. Now Project A.L.S. researcher Robert H. Brown, Jr., and colleagues at University of Massachusetts, have identified a potential role for the gene in sporadic forms of the disease. Discovery of this common pathology is described in the October 17 online edition of Nature Neuroscience.
The identification of this common pathology may open the door for new therapeutic approaches. For example, scientists working on SOD1 gene silencing for familial ALS can now think about applying the promising strategy to other, more common forms of the disease. From ProjectALS.org
New Drug Screens Begin at Project A.L.S. Stem Cell Lab
October 18, 2010 – For years, people have tried to develop effective drugs for ALS, with limited success. One reason for the dearth of drug discovery is that the models of ALS against which drugs and compounds are screened are not reliable—they do not mirror human ALS accurately. A platform called iPS—induced pluripotent stem cells—is changing the way we screen drugs and chemical compounds for ALS. At the Jenifer Estess Laboratory for Stem Cell Research, collaborators from Project A.L.S., Columbia University, Harvard University, and Johns Hopkins, are using iPS cells derived from ALS patient skin to recreate live-cell representations of human ALS in each of the 360 small wells on a slide. Utilizing various screening tools, including the Plate Runner HD ©, developed by the company Trophos, Project A.L.S. has undertaken an aggressive screening of potentially neuroprotective agents.From ProjectALS.org
Motor Neurons of All Kinds Generated from Stem Cells
September 3, 2010 – From head to toes, different muscles in our bodies are stimulated by dozens of different subtypes—or pools--of motor neurons. Hynek Wichterle, a Project A.L.S. scientist at Columbia University, and his group, now show that these motor neuron subtypes can be generated from stem cells.
Featured as the cover story in this month’s Cell Stem Cell, Dr. Wichterle’s groundbreaking study indicates that ES cells can be programmed in a predictive manner to acquire the molecular and functional properties that characterize these motor neuron pools.
This breakthrough makes possible several new approaches to ALS therapies. For example, if we are able to generate motor neurons that control breathing, we may better approach strategies toward prolonging respiratory function in people with ALS. From ProjectALS.org
Leading Researchers Unite for P2 ALS, a 3-Year Mission to Understand and Treat the Neurodegenerative Disease
January 29 2010 — Project A.L.S.™ (New York, NY) and the Robert Packard Center for ALS Research at Johns Hopkins University (Baltimore, MD) announced that they will partner on P2 ALS, a $15 million initiative designed to advance ALS (Lou Gehrig’s disease) research exponentially over the next three years.
Project A.L.S.™ and the Packard Center, non-profit leaders in forging productive collaborations among research scientists, will focus jointly on identifying the underlying causes of and the first effective treatments for ALS, a uniformly fatal neurodegenerative disease that is closely related to Alzheimer’s, Parkinson’s and Huntington’s diseases. Co-scientific directors of P2 ALS are Robert H. Brown, Jr., M.D., D.Phil. (University of Massachusetts), Thomas M. Jessell, Ph.D. (HHMI/Columbia University), and Jeffrey Rothstein, M.D., Ph.D. (Johns Hopkins University).
P2ALS is distinctive in that it unites key world leaders in the three disciplines that have recently transformed the landscape of ALS science: Genetics, Stem Cell Reprogramming, and Glial-Neuron Signaling. Through P2ALS, targeted research in these three areas will be performed in an interactive, collaborative, and transparent manner. As such, the implications of discoveries in one area will be rapidly transmitted and tested in complementary areas, by multiple laboratories. New observations and ideas can and will be validated or refuted with unprecedented speed. From ProjectALS.org
SCIENTISTS DISCOVER A NEW ROLE FOR INTERNEURONS: MODULATING THE PERFORMANCE OF MOTOR NEURONS AND MUSCLES
Major Implications for ALS
It is well established that motor neurons send signals directly to muscles to make them contract. In the December 10, ‘09 issue of the journal Neuron, Project A.L.S.™ scientists led by Laskaro Zagariou, Robert Brownstone, Tom Jessell and Gareth Miles, identify C boutons, aspects of spinal interneurons, as "volume dials" for motor neurons. This discovery gives scientists a more complete picture of how the brain talks to muscles all over the body.
"The more we know about normal brain circuitry, the more effectively we can strategize therapeutics for a nervous system that is under attack by ALS," says Valerie Estess, director of research for Project A.L.S.™ From ProjectALS.org