Repurposing of existing drugs for a new indication can save many years of the developmental timeline for new entities since safety and tolerability studies, as well as other PK and PD analyses, are already completed. The goal of this proposal is to finalize a preclinical trial on small rodents using a selected number of repurposed molecules that pass stringent in vitro test. In the long term, if the preclinical trial is successful, the next step will be to embark in a phase I/II clinical trial, recruiting PMS patients from different centers in Europe and in North America. In the next phase the team could pursue two different strategies (i) either contacting companies that are leaders in contract research organization (Quintiles, Parexel…) and develop with them and with pharma companies a clinical trial; and, (ii) seeking for public funds from large organizations (EU, fund raising organization alliance) to start a trial led by joint efforts of research institutes and hospitals. As the project develops and data are available, the consortium will establish collaboration and exchange progresses and results with companies that are taking similar approaches for neurodegenerative diseases or with other scientists that could be developing similar drug screening strategies with focus on other cell types.

The consortium is committed to improve competitiveness and market opportunities for the participants through the best possible exploitation and commercialization of the results within the legal constrain regulating the activities of each partner.

The MS International Alliance project could be running in parallel with a European clinical trial on PMS patients in which selected repurposed molecules (and their combination) with reported neuroprotective and promyelinating potential are planned to be tested in human. Some partners are in both consortia.

The objectives and the topics of this proposal are of great importance for the ongoing current and future research of most of the partners. The members of the consortium are committed to demonstrate feasibility of robust drug search on biological proxies of human brain cells (differentiated hiPSCs). Certainly, among the foreseen plans that the partners will engage we can mention: (i) patient specific hiPSC cell lines to assess subjects specific drug response; (ii) identification of molecular biomarkers that could be traced in vivo in biological fluids of patients to evaluate whether the treatment is effective; (iii) development of new patentable compounds (QSAR analysis) starting from active molecular backbone; (iv) development of transgenic animal model(s) pinpointing specific target that could better resemble the pathophysiological progressive profile of MS patients.

Comparison of drug discovery approaches