Oligodendrocytes contribute to motor neuron death in ALS via SOD1-dependent mechanism

L Ferraiuolo, K Meyer, TW Sherwood… - Proceedings of the …, 2016 - National Acad Sciences
L Ferraiuolo, K Meyer, TW Sherwood, J Vick, S Likhite, A Frakes, CJ Miranda, L Braun…
Proceedings of the National Academy of Sciences, 2016National Acad Sciences
Oligodendrocytes have recently been implicated in the pathophysiology of amyotrophic
lateral sclerosis (ALS). Here we show that, in vitro, mutant superoxide dismutase 1 (SOD1)
mouse oligodendrocytes induce WT motor neuron (MN) hyperexcitability and death.
Moreover, we efficiently derived human oligodendrocytes from a large number of controls
and patients with sporadic and familial ALS, using two different reprogramming methods. All
ALS oligodendrocyte lines induced MN death through conditioned medium (CM) and in …
Oligodendrocytes have recently been implicated in the pathophysiology of amyotrophic lateral sclerosis (ALS). Here we show that, in vitro, mutant superoxide dismutase 1 (SOD1) mouse oligodendrocytes induce WT motor neuron (MN) hyperexcitability and death. Moreover, we efficiently derived human oligodendrocytes from a large number of controls and patients with sporadic and familial ALS, using two different reprogramming methods. All ALS oligodendrocyte lines induced MN death through conditioned medium (CM) and in coculture. CM-mediated MN death was associated with decreased lactate production and release, whereas toxicity in coculture was lactate-independent, demonstrating that MN survival is mediated not only by soluble factors. Remarkably, human SOD1 shRNA treatment resulted in MN rescue in both mouse and human cultures when knockdown was achieved in progenitor cells, whereas it was ineffective in differentiated oligodendrocytes. In fact, early SOD1 knockdown rescued lactate impairment and cell toxicity in all lines tested, with the exclusion of samples carrying chromosome 9 ORF 72 (C9orf72) repeat expansions. These did not respond to SOD1 knockdown nor did they show lactate release impairment. Our data indicate that SOD1 is directly or indirectly involved in ALS oligodendrocyte pathology and suggest that in this cell type, some damage might be irreversible. In addition, we demonstrate that patients with C9ORF72 represent an independent patient group that might not respond to the same treatment.
National Acad Sciences