GPP78 is a potent inhibitor of Nampt (IC50: 3.0 nM for nicotinamide adenine dinucleotide (NAD) depletion). GPP78 is cytotoxic to neuroblastoma cell line SH-SY5Y cells (IC50: 3.8 nM by inducing autophagy). GPP78 has anti-cancer and anti-inflammatory effects.

GPP78 appears truly cytotoxic in melanoma cell lines, while in the others it is mainly cytostatic. GPP78 inhibits the growth of most cell lines tested, with nanomolar potency (GI50) in cell lines derived from leukemia, lung, CNS, colon, melanoma, ovarian, renal, and prostate cancers. GPP78 (10 nM; 24-40 hours; SH-SY5Y cells) treatment with cells, punctate staining of LC3-II, and the formation of autophagolysosomes are observable. LC3-II is membrane-bound and is present in autophagosomes [1].

GPP78 (i.p.; daily; 10 mg/kg; 1 hour or 6 hours after SCI; for 19 days; male adult CD1 mice) treatment decreases the severity of spinal cord trauma in SCI mice [2].