Question

A phase 1, open-label, dose-ranging, safety and pharmacokinetic study to determine the maximum tolerated dose of rx-3117 administered orally as a single-agent to subjects with advanced malignancies

Answer 1

Whereas neuronal replacement in ALS patients seems a distant goal, using stem cells to prevent motor neurons from dying is a more realistic and shorter-term clinical approach. ... The efficacy of this approach could be improved by genetically modifying the stem cells to secrete molecules that promote motor neuron survival.