A brain in a dish

One of the major challenges in drug discovery is translating promising therapies from pre-clinical models – usually mice – to clinical use. The lack of suitable human preclinical models is a major roadblock for successful translation. To bridge the gap between animal models and human clinical trials new models of myelin damage and repair using human cells are crucial.

Recent advances in brain organoid culture has shown the viability of deriving myelinating brain organoids from human-induced pluripotent stem cells. However, one of the major limitations of brain organoids is the length of time required for organoids to reach a stage at which myelination is observed – often as long as 200 days.

In a major advance, our collaborators have developed an innovative model for studying myelination in which mature myelin is observed after only 42 days. We’ll use this system as a basis for developing a sophisticated human pre-clinical model of demyelination and remyelination. Developing a human model of myelin damage and repair is a critical tool in bridging the gap between mouse models and human testing, providing an essential tool for prioritising candidates for clinical trial of remyelinating therapies.