Table 1 Clinical trials of NGF for the treatment of AD

2020; Phase II (NCT00087789)

2 × 10¹¹ vector genomes of AAV2-NGF by stereotactic inj

NGF did not directly reach cholinergic neurons due to limited spread and inaccurate stereotactic targeting

2018; Phase II (NCT00876863)

2.0 × 10¹¹ vector genomes of AAV2-NGF delivered by bilateral stereotactic injections

No significant difference between the treatment group and placebo

2016; Phase I (NCT01163825)

Cell biodelivery expressing NGF 10 ng /device/day, targeting at the cholinergic basal forebrain

13 of 16 implants released NGF, 8 implants released NGF at the same rate or higher than before the implant procedure

2015; Phase II (NCT00017940)

Autologous fibroblasts based NGF gene therapy to the basal forebrain, 1.2 × 10¹⁰, 5.8 × 10¹⁰ or 1.2 × 10¹¹ vector particles

In the degenerating neurons in the brain, trophic response to NGF in the form of axonal sprouting toward the NGF source

2014; Phase I (NCT00087789)

1.2 × 10¹⁰, 5.8 × 10¹⁰, 1.2 × 10¹¹ vector genomes of AAV2-NGF

Long-term, targeted, gene-mediated NGF expression and bioactivity

2012; Phase I (NCT01163825)

Cell biodelivery expressing NGF 300 ng/10⁶ cells/24 h, targeting the NBM or the limb

Persistent NGF secretion was detected in half of the patients

2005; Phase I

Implanting NGF-expressing autologous fibroblasts into the forebrain, 2.5 × 10⁶ total cells to 10.0 × 10⁶ total cells

Improvement in the rate of cognitive decline