Study results published in JAMA Neurology suggests that degenerating neurons in patients with Alzheimer's disease (AD) measurably responded to an experimental gene therapy in which nerve growth factor (NGF) was injected into their brains, Science Daily reported.
The findings are derived from postmortem analyses of 10 patients who participated in Phase I clinical trials to assess whether injected NGF might safely slow or prevent neuronal degeneration in patients with AD.
The affected neurons displayed heightened growth, axonal sprouting and activation of functional markers, said lead author Mark H. Tuszynski, adding that the findings indicate NGF is safe over extended periods and that it merits continued testing as a potential AD treatment.
Administering NGF directly into the brain was done because it is too large pass through the blood-brain barrier, making it impossible to inject elsewhere and because freely circulating NGF causes adverse effects, such as pain and weight loss.
The gene therapy approach has since progressed to Phase II trials at multiple test sites.
