Research consortium on stem cells for ALS supported by
the André-Delambre Foundation

The André-Delambre Foundation is supporting a research consortium headed by Dr. Jean-Pierre Julien (Laval Univerity) to produce and to analyse iPS stem cells derived from the skin of ALS patients. The sporadic cases of ALS constitute 95% cases of ALS and the causes of disease are unknown. The new technology of iPS stem cells offers the possibility to reconstitute motor neurons in culture that will serve as model of disease to elucidate the molecular changes associated with ALS pathogenesis. The iPS-derived motor neurons will also offer new avenues to screen for therapeutic targets and for drugs. Moreover, the iPS-derived cells might serve for future cell therapy experiments.

What is an iPS cell?
Recently, a number of laboratories reported that it is possible to reprogram somatic cells, like skin cells, with the use of specific regulatory proteins to produce pluripotent stem cells which are called iPS cells (induced pluripotent stem cells). These iPS cells can then be differentiated in different cell types according to culture conditions. Recently, a reserach team succeeded in generating iPS cells from a patient with ALS and to differentiate these cells into motor neurons.    

Why generating a bank of iPS cells from the skin of ALS patients? 
At this time, only the genetic causes of ALS have been identified for only 5% ALS cases that correspond to familial cases. The causes of ALS for sporadic ALS remain unknown and they remain very difficult to find by standard genetic approaches. The possibility to generate iPS cells and to differentiate them into motor neurons constitutes a major breakthrough which opens new avenues for elucidating the defects in gene expression and properties of motor neurons in sporadic ALS. The reconstituted motor neurons might also serve for the screening of new therapeutic targets and of drugs to rescue the molecular or cellular defects. It is also noteworthy that research on iPS cells does not raise the same ethical concerns that stem cells obtained from human embryos.  Thus, the generation of iPS cells from ALS patients and their differentiation into motor neurons will provide new powerful tools for elucidating the causes of sporadic ALS, for the discovery of new therapeutic targets and for drug screening.