Descrizione del progetto
Un nuovo farmaco contro la sclerosi multipla
La sclerosi multipla (SM) è una malattia neurodegenerativa cronica caratterizzata da recidive debilitanti associate a perdita della funzione neurologica. La SM colpisce milioni di persone in tutto il mondo, ma le opzioni terapeutiche esistenti sono principalmente palliative. Il progetto ENHANCIDO, finanziato dall’UE, si propone di valutare nuove piccole molecole che potenziano l’attività dell’enzima immunosoppressore IDO1 noto per ridurre la gravità della SM in modelli animali. Valutando l’efficacia, la farmacocinetica e la farmacodinamica di questi composti, gli scienziati sperano di fornire un farmaco innovativo di prima classe in grado di migliorare la vita dei pazienti con SM. Ancora più importante, questo farmaco ha il potenziale per essere utilizzato per trattare anche altre malattie autoimmuni.
Obiettivo
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.
Campo scientifico
Programma(i)
Argomento(i)
Meccanismo di finanziamento
ERC-POC-LS - ERC Proof of Concept Lump Sum PilotIstituzione ospitante
06123 Perugia
Italia