Description du projet
Un nouveau médicament contre la sclérose en plaques
La sclérose en plaques (SP) est une maladie neurodégénérative chronique qui se caractérise par des rechutes débilitantes associées à une perte de la fonction neurologique. La SP touche des millions de personnes dans le monde, mais les options de traitement existantes sont principalement de nature palliative. Le projet ENHANCIDO, financé par l’UE, vise à évaluer de petites molécules innovantes potentialisant l’activité de l’enzyme IDO1 immunosuppressive capable de réduire la sévérité de la SP dans des modèles animaux. En évaluant l’efficacité ainsi que les aspects pharmacocinétique et pharmacodynamique de ces composés, les scientifiques espèrent parvenir à un médicament innovant unique en son genre susceptible d’améliorer la vie des patients atteints de SP. Plus important encore, cette molécule pourrait potentiellement être utilisée pour traiter d’autres maladies auto-immunes.
Objectif
ENHANCIDO will assess the commercial and technical feasibility of novel immune tolerance-inducing small molecules as superior treatments for multiple sclerosis (MS). MS is a chronic neurodegenerative disease affecting 2.3 million people worldwide. 85% of MS cases belong to the relapsing-remitting subtype (RRMS) characterised by bouts of recurring and debilitating relapses followed by unstoppable loss of neurological functions. Unfortunately, current treatments for RRMS are ineffective, poorly tolerated, and expensive. They cannot prevent disease progression and only provide symptomatic relief. Furthermore, severe adverse effects and inconvenient administration routes contribute to high rates of treatment discontinuation amongst patients (30-80%). To tackle these issues, Prof. Ursula Grohmann has developed a set of novel, orally administered, effective, and safe small molecules that induce immune tolerance to alter the previously inexorable course of the disease. These first-in-class molecules are positive allosteric modulators (PAMs) of the immunoregulatory enzyme IDO1. Prof. Grohmann’s PAMs increase IDO1 activity, reduce disease severity in mouse models of acute MS and show no off-target effects. Within ENHANCIDO, we will assess whether IDO1 PAMs are feasible as breakthrough treatments for RRMS. Firstly, we will validate their technical feasibility by evaluating their efficacy, pharmacokinetics and pharmacodynamics in RRMS mouse models. Subsequently, commercial feasibility will be determined by verifying IP position and strategy, performing in-depth market and competitor analyses, and finally consolidating these into a business plan to establish the best path to commercialisation. Successful commercialisation of IDO1 PAMs could reduce the profound socioeconomic burden of MS, extend and improve the lives of patients, and provide the pharmaceutical industry with a profitable, first-in-kind drug that can potentially be used for other autoimmune diseases.
Champ scientifique
Programme(s)
Régime de financement
ERC-POC-LS - ERC Proof of Concept Lump Sum PilotInstitution d’accueil
06123 Perugia
Italie