Engineered stem cells carry promising ALS therapy

“The novelty is that this is a combined cell and gene therapy approach,” Suzuki explains, noting that the bone marrow stem cells on their own had a modest effect, possibly by releasing their own protective factors. “But only when we engineered the cells to release GDNF did we see a significant improvement. The cells turned out to be quite an important component. It’s this combination of cells and drug delivery that seems to be so effective.”