Projektbeschreibung
Ein neues Arzneimittel gegen Multiple Sklerose
Multiple Sklerose (MS) ist eine chronische neurodegenerative Erkrankung, die durch stark beeinträchtigende Schübe mit einhergehendem Verlust der neurologischen Funktion gekennzeichnet ist. MS betrifft mehrere Millionen Menschen weltweit. Dennoch sind die aktuellen Behandlungsmöglichkeiten bislang noch vorwiegend palliativer Natur. Das immunsuppressive Enzym IDO1 reduziert nachweislich die Schwere von MS. Das EU-finanzierte Projekt ENHANCIDO wird daher an Tiermodellen neu entwickelte kleine Moleküle zur Wirkungsverstärkung von IDO1 bewerten. Die Forschenden werden die Wirksamkeit, Pharmakokinetik und Pharmakodynamik dieser Verbindungen untersuchen und hoffen, durch diese Arbeit ein innovatives, hochwertiges Arzneimittel bereitstellen zu können, das die Lebensqualität von MS-Erkrankten verbessern kann. Vor allen Dingen aber hätte ein solches Arzneimittel auch das Potenzial zur Behandlung anderer Autoimmunerkrankungen.
Ziel
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.
Wissenschaftliches Gebiet
Programm/Programme
Thema/Themen
Finanzierungsplan
ERC-POC-LS - ERC Proof of Concept Lump Sum PilotGastgebende Einrichtung
06123 Perugia
Italien