Top Sector Life Sciences & Health (LSH) entails a broad scope of disciplines, from pharmaceuticals to medical technology and from healthcare infrastructure to vaccination. To realise its mission – vital citizens in a healthy economy - the Top Sector builds on the strengths of the Dutch LSH sector to address the biggest societal challenges in prevention, cure and care. By funding multidisciplinary public-private partnerships (PPPs) the Top Sector aims to facilitate innovation. Here we give an overview of  a number of funded R&D projects by Top Sector LSH. The page is updated continuously.

Patient-driven approach for drug development

Towards better preclinical prediction of drug efficacy integrating patient- mechanisms and data and innovative assays in patient-derived cells

In this project the aim is to evaluate drug efficacy using patient cells, without the use of animals. All three partners in this newly established consortium are convinced that use of patient driven, animal-free methods will contribute to a more efficient and relevant drug development process. The partners in this extraordinary consortium cover scientific, industrial and societal needs with their different expertise and motivation.

The key to successful development of tomorrow’s medicine is to decrease the attrition rate in the expensive clinical phase by increasing the level of translatability of preclinical efficacy models. In practice, this is often limited in (animal) disease models as well as poorly understood. Also, in various disease areas no suitable animal models are available. This situation causes more than 40% of potential drugs to fail in clinical phase due to lack of efficacy  leading to a tremendous cost burden (failure in Phase 3 clinical trial costs 1B$ a year), which will conceivably worsen in the near future.

In this project the consortium partners want to come to a proof of concept of an approach that establishes the efficacy of potential therapeutics, with the use of patient data and patient-derived cells limiting the use of experimental animals. Their aim is to connect in vitro, in silico modelling and systems biology technologies in such a way that this will provide sufficient information to select the best investigational medicines for treatment in the disease area of fibrosis. Organ fibrosis is a fast growing health problem and to date there are no approved therapies to halt fibrosis progression or reverse advanced fibrosis.

The result of this project will be a description of a proof of concept without the use of animals that establishes and predicts the efficacy of potential therapeutics in humans.

 “Image courtesy of Galapagos”