Examples of physiological response markers that have been used in early phase NDD DMT clinical trials include the evaluation of brain glucose metabolism after administration of nerve growth factor gene therapy [68] or deep brain stimulation [76,78] for Alzheimer’s disease, and CSF cytokine production after transfusion of stem cells [101] or administration of granulocyte colony-stimulating factor (G-CSF) [115] in ALS patients (see Table 2). The gene discussed is CSF3; the disease is early-onset autosomal dominant Alzheimer disease.