Streamlining drug development for amyotrophic lateral sclerosis: towards a cure
In this project, the UMC Utrecht and Julius Clinical collaborate to fight the debilitating and deadly disease amyotrophic lateral sclerosis (ALS). As a consortium we have the scientific and operational expertise to develop a core protocol for drug research. This core protocol will guide future studies to improve the chance of success, make research more cost-effective, and attract more pharmaceutical companies.
Despite testing over 100 different drugs, no cure for ALS has been found. The unpredictable course of ALS, the unclear disease mechanisms, and the variability in clinical trial setup causes drug research to fail. It is often unclear whether the drug truly didn’t work or that a treatment effect was missed due to poor study design. This leads to a waste of funding and time. As a result, the life expectancy of people with ALS remains grim, with most people dying within three years after the first disease signs appear.
The project consists of three phases: (1) evidence synthesis to inform computer models, (2) investigating stakeholder perspectives and conducting market research, and (3) creating an evidence-based, stakeholder-supported core protocol. By using computer models, we can study the best strategies for finding new drugs as efficiently as possible. This data is then linked to patient preferences, industry preferences, cost, and requirements set by regulatory authorities. From this, we determine the best strategy for each phase in the drug development path (the "core protocol”).
This core protocol will serve as a standardized platform for faster and more efficient drug research, called the Methodical ALS Global Network of Efficacy Trials (MAGNET). The end product will consist of a set of regulatory-accepted tools, guidance documents and consensus standards, with the goal to make ALS research more attractive to pharmaceutical companies and help to speed up the search for a cure.
