A core facility for human induced pluripotent stem cells for neurological disease

This award will allow Selina to purchase equipment, including robotics, to establish a core facility that provides reprogrammed human induced-pluripotent stem cell lines and organoids at scale to researchers across UCL, helping to replace some animal studies for research into neurological disorders. The funding also supports work to standardise operating protocols to ensure the stem cell lines and organoid models are high quality and robust, and hands-on training courses for postdoctoral researchers and students to enable wider roll-out of the models. 

This award was made as part of the 2024 non-animal methods infrastructure grants supported with funding from the Department for Science, Innovation and Technology (DSIT).

Our understanding of neurological disease and the development of new treatments has been hampered by the inaccessibility of the human central nervous system during life and the lack of available tools to culture the cell types of the human brain and spinal cord in vitro. The development of human induced pluripotent stem cells (iPSC) has revolutionised disease modelling for neurological disease. Readily accessible cells like fibroblasts can be reprogrammed to pluripotency, and the resulting iPSC can be differentiated into multiple cell types including different classes of neurons, astrocytes, microglia, oligodendrocytes and 3D models such as cerebral organoids. Importantly, this provides a fully renewable source of human brain material for study, enabling 2D and 3D modelling and reducing the need for live animal studies.

Barriers to adopting these approaches include the availability of cells from patients with a defined phenotype/genotype of interest, and the technical expertise to differentiate iPSC into disease relevant cell types. At UCL Queens Square Institute of Neurology, we have unrivalled access to clinical cohorts across the neurological disease spectrum, including Alzheimer’s disease (AD), Parkinson’ disease (PD), Motor Neuron Disease (MND), Huntington’s disease (HD) and epilepsy. Thus, we are in a privileged position to generate iPSC from individuals with rare forms of disease, which we use extensively and share with local, national and international collaborators. However, the current lack of an in-house core facility for reprogramming means that, so far, we have only scratched the surface of the resource available to us.

In early Summer 2025, we will open our new state-of-the-art translational neuroscience facility, with dedicated laboratory space to establish an iPSC core technology platform. This proposal seeks funds to establish this core, with the following objectives:

1. Enable iPSC research across UCL by establishing pipelines for the generation, characterisation and differentiation of iPSC from a range of neurological disease cohorts.
2. Facilitate translational research by developing automated culture pipelines to enhance scalability and reproducibility, thus facilitating translational approaches such as drug screening.
3. Develop a high-quality repository of patient iPSC and isogenic controls for use by the broader research community.

This facility will significantly enhance our capacity to create patient iPSC from diverse disease groups, expand iPSC usage across UCL, and establish a repository of lines available to the international research community for neurological disease research. This will promote the replacement of animal models and accelerate the development of new treatments for neurological diseases.