Descripción del proyecto
Un nuevo fármaco contra la esclerosis múltiple
La esclerosis múltiple (EM) es un trastorno neurodegenerativo crónico que se caracteriza por recidivas discapacitantes asociadas con una pérdida de la función neurológica. Afecta a millones de personas en todo el mundo, pero las opciones terapéuticas disponibles en la actualidad son fundamentalmente paliativas. El proyecto financiado con fondos europeos ENHANCIDO busca evaluar nuevas moléculas pequeñas que potencian la actividad de la enzima inmunodepresora IDO1, la cual se sabe que disminuye la gravedad de la EM en modelos animales. Al evaluar la eficacia, la farmacocinética y la farmacodinámica de estos compuestos, los científicos esperan ofrecer un innovador fármaco, el primero de su clase, que mejore la vida de los pacientes con EM. Además, dicho fármaco tiene el potencial para ser usado en el tratamiento de otras enfermedades autoinmunitarias.
Objetivo
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.
Ámbito científico
Programa(s)
Régimen de financiación
ERC-POC-LS - ERC Proof of Concept Lump Sum PilotInstitución de acogida
06123 Perugia
Italia