The ALS Association

Ice Bucket Challenge Progress

Research Summary 2005: Advances Give Renewed Hope for the Coming Year

Roberta Friedman, Ph.D., ALSA Research Department Information Coordinator

January 6, 2006

The following is a recap of key findings published in 2005 that point the way toward new therapies for the nerve wasting disease, amyotrophic lateral sclerosis (ALS, also called Lou Gehrig’s disease).

  • Research published in the April issue of Human Gene Therapy shows that stem cells taken from the outer layer of the human fetal brain can be prompted to turn into vital support cells for motor neurons and be engineered to make a factor that helps surrounding nerve cells to survive. Clive Svendsen, Ph.D., the University of Wisconsin investigator directing the research, said “We look forward to continual interactions with ALSA as we carefully take stem cells towards the clinic."
  • Reporting in the September 29 online Journal of Neurochemistry, University of Pittsburgh researcher Robert Bowser, Ph.D. and collaborators detailed findings on potential biomarkers for ALS. A predictive panel of biomarkers would allow more rapid and accurate diagnosis for patients who often undergo months of tests and uncertainty before finding out whether they have ALS. Bowser’s work is part of a consortium effort funded by ALSA to find biomarkers for ALS.
  • Findings in the October 10 Journal of Cell Biology shed new light on why ALS kills cells. Northwestern University researcher Richard Morimoto, Ph.D. and colleagues could see with a new microscope technique that the proteasomes, which work within cells to either refold defective protein or trash it, are trapped in the mesh of aggregated, mutated SOD1. Cells that visibly formed SOD1 aggregates died within a day. Certain drugs exist or are being designed that either boost the performance of the proteasome or interrupt aggregates.
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