Safety and potential efficacy of gemfibrozil as a supportive treatment for children with late infantile neuronal ceroid lipofuscinosis and other lipid storage disorders

Table 1 Effect of gemfibrozil on cells and on animals

	Treatment	Effect	Reference
Cells in vitro
Mouse & human primary astrocytes	gemfibrozi 25 μm	↑CLN2 mRNA ↑TPP1 protein	38, 39
Mouse neurons from cortex, striatum & hippocampus	gemfibrozil 25 μm	↑TPP1 protein	38
Lymphoblastic cells from CLN3 patients	gemfibrozil 25 μm	↑viability ↑autophagy recovery ↑autophagy genes	48
Human IPS models CLN2 and CLN3 mutations	gemfibrozil 25 µm	No effect on TPP1	47
Animals
Mouse	oral gemfibrozil 7.5 mg/kg for 21 d	↑TTP1 in astrocytes, cortical neurons, & non neural cells of dentate gyrus & CAI of hippocampus	38
Mouse KO LINCL	oral gemfibrozil 7.5 mg/kg for 21 d	↑longevity, ↑motor retention, ↓apoptosis, ↑anti-inflammatory molecules	49

ISSN: 1750-1172