CHRISTIAN BAILLY¹*, JEAN-FRANÇOIS GOOSSENS²
*Corresponding author
1. Oncowitan, Wasquehal (Lille), France 
2. Univ. Lille, CHU Lille – GRITA – Groupe de Recherche sur les formes Injectables et les Technologies Associées, Lille, France

Abstract

Amyotrophic lateral sclerosis (ALS) is a rare but severe neuro-degenerative disease. Edaravone, an FDA-approved small molecule drug, can slow functional loss and disease progression in ALS, at least during the early stage. This synthetic phenyl-pyrazolone derivative is a potent scavenger of oxygen free radicals but it is also a reactive molecule which efficiently reacts with the aldehyde moiety of reducing sugars. The drug can form adducts with bioactive aldehydes such as 6-formylpterin, a photo-product of folic acid and folates. The different faces of edaravone are underlined here.

INTRODUCTION

The Man with Two Faces was the title of a popular American melodrama, first released in 1934. The title derives also from a book of medical oddities entitled “Anomalies and Curiosities of Medicine” (1896), with reference to an article published in the Boston Sunday Post on December 8, 1895. It told the story of a man (Edward Mordrake) with a handsome, normal face and a terrifying second face on the back of his head. One man, two opposite personalities (1). By analogy, the small molecule 3-methyl-1-phenyl-2-pyrazolin-5-one could be called The Molecule with Two Faces. One the one side, the compound is a laboratory reagent, designated PMP, useful to study the composition and structure of polysaccharides, and for the analysis of carbohydrates in complex media. On the other side, the same compound is an approved drug, used to treat amyotrophic lateral sclerosis and acute stroke. In both cases, it is the same molecule, but the chemistry implicated in the mechanism of action is quite distinct, as briefly evoked here.

DISCUSSION

PMP is a reagent classically used to deri ...