Enterin, Inc. a CNS pharmaceutical company based in Philadelphia and developing novel compounds to treat neurodegenerative diseases, will shortly be initiating a Phase 1/2a randomized, controlled study in patients with Parkinson’s disease. The study will take place at 9-10 US sites and will enroll 50 patients over 6-9 months. Details relating to the study can be found at ClinicalTrials.gov. Contact information is available on Enterin’s website.
The study will establish the safety, tolerability and efficacy of an orally administered synthetic derivative of squalamine which is not absorbed into the blood stream. The compound acts locally on the enteric nerve cells of the gut, stimulating gut motility and substantially altering the afferent neural signaling from gut to brain. It has the potential to ameliorate the non-motor symptoms of Parkinson’s such as constipation and abnormal sleeping patterns and to modify the progression of the disease.
Synthetic squalamine was recently shown to prevent the buildup and reduce the toxicity of a lethal protein (alpha-synuclein) implicated in the pathogenesis and progression of Parkinson’s disease. The compound was shown to displace alpha-synuclein clumps from the inner wall of nerve cells and to prevent the muscular stiffness which develops in C. Elegans worms engineered to produce alpha-synuclein in its muscles. The research was performed by an international team from Cambridge University in the UK, the NIH and Georgetown University and published online in the February 7th edition of the Proceedings of the National Academy of Sciences (Perni et al, PNAS Vol 114, no 6, 2017, doi: 10.1073/pnas.1610586114). It was widely reported by the international press. Links to the article and to the press coverage can be found at the Enterin website.