Back in 2008, Harvard Stem Cell Institute Principal Investigator Kevin Eggan suggested that ALS patient-derived induced pluripotent stem cells (iPSCs) may be leveraged to identify novel targets and screen drugs for ALS. Two new studies, led by Kevin Eggan and Clifford Woolf’s groups at the Harvard Stem Cell Institute and published on April 3 in the online edition of Cell Stem Cells and Cell Reports, build on these earlier findings, and provide an intriguing example of the therapeutic promise of these cells. The investigators found that motor neurons carrying independent ALS mutations exhibit an increase in random firing activity, likely due to a deficit in potassium channels (see related story from December 2007), and that these overactive neurons generate misfolded proteins, which further increase neuronal firing. They hypothesized that by opening potassium channels in these cells they could reduce the hyperexcitability, and they found exactly that – retigabine, an FDA approved medication for epilepsy, normalized the activity. The team is now collaborating with Massachusetts General Hospital to begin initial safety testing of the treatment in ALS patients.
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