Safety and tolerability of intravenous liposomal GM1 in patients with Parkinson disease: A single center open-label clinical phase I trial (NEON trial)

Background: Parkinson disease (PD) is a chronic progressive neurodegenerative disorder leading to motor and non- motor impairment often resulting in severe loss of quality of life. There are symptomatic treatments without effect on the progression of PD. A disease-modifying treatment that could ideally stop the neurodegenerative process is direly needed. Monosialotetrahexosylganglioside (GM1) is a promising molecule with neuroprotective effects in preclinical models of PD and has yielded encouraging results in patients with PD in a randomized placebo-controlled trial. Talineuren (TLN) is a liposomal formulation of GM1 that has been shown to cross the blood-brain barrier in animals. We assessed the safety and pharmacokinetics of TLN in patients with PD.