Human Brain Organoids & Cellular Models for Neurodegenerative Drug Development

The Cellular and Molecular Neurodegeneration Laboratory - web graphic

The QIMR Berghofer Cellular and Molecular Neurodegeneration Laboratory offers customised contract R&D services built on advanced human brain cell models tailored for translational drug development. Backed by years of experience supporting industry clients, we provide a broad portfolio of patient-derived, disease-relevant models (outlined in the table below) that enable drug screening and optimisation, neurotoxicity assessment, disease modelling, and blood-brain barrier penetration studies. Our high-fidelity platforms are designed to closely mirror human brain complexity, accelerating your preclinical decision-making and reducing risk in advancing drug candidates towards the clinic.

Services

Drug screening and lead optimisation

Neurotoxicity assessment

Disease modelling (e.g. Alzheimer’s disease, motor neuron disease, childhood dementia)

Blood-brain barrier modelling and drug penetration studies

Mechanistic and biomarker studies

Drug stratification and response prediction in neurodegeneration

Customised study design, execution, and reporting

Assay Capabilities

3D microglia-neuron co-culture assays

Blood-brain barrier permeability assays

Cell viability and cytotoxicity assays (e.g. LDH)

Confocal microscopy imaging

Cytokine and chemokine multiplex immunoassays

Immunocytochemistry

Live-cell imaging and morphological analysis

Phagocytosis assays

Proteomics

RNA sequencing and transcriptomic analysis

Transendothelial electrical resistance (TEER) measurement

Case Studies

Microglia-Based Pipeline for Drug Stratification and Response Prediction in Neurodegeneration (Sci Rep 2025)
Patient-Derived 3D Microglia Cell Model for Alzheimer’s preclinical Drug Testing (J Nueroinflammation 2024)
Patient-DerivedBlood-Brain Barrier Model for Evaluation of Copper-Based Therapeutics in Alzheimer’s Disease (ACS
Chemical Neuroscience 2024)
ALS Blood-Brain Barrier Model for Testing Ultrasound-Mediated Antibody Delivery (Fluid Barriers CNS 2024)
Brain Organoids to Assess SARS-CoV-2 Neurotropism and Antiviral Efficacy (Front Microbiol 2023)
Patient-Derived Olfactory Stem Cell Model for Alzheimer’s Disease (Cells 2022)
Patient-Derived ALS Microglia Model to Study Neuroinflammation and Disease Progression (J Neuroinflammation
Alzheimer’s Patient-Derived Blood-Brain Barrier Model for Testing Antibody Delivery (Theranostics 2022)

Cell models and applications

Model Type and Applications

Model Type	Applications
Organoid Models
Brain Organoids (± microglia)	Drug optimisation Ideal for preclinical assessment and validation of drug efficacy in a complex human brain model. Disease modelling Ideal for characterisation of inflammatory pathways and evaluation of targeted therapeutic responses. Neurotoxicity
Microglial Models
Monocyte-derived Microglia	Drug screening & optimisation Disease modelling Custom generation of patient-specific microglial cultures from blood samples for personalised disease modelling across a wide range of brain neurodegenerative disorders
iPSC-derived Microglia	Drug screening & optimisation Disease modelling Mechanistic studies
iPSC-derived Brain Cells
Brain Endothelial Cells	Assessment of drug penetrability across the blood-brain barrier (BBB) High-fidelity BBB model replicating human brain barrier integrity BBB modelling
Neurons	Drug screening Neurotoxicity profiling Disease modelling
Astrocytes	Drug screening Disease modelling
Nasal Models
Olfactory Cell Cultures	Drug screening Disease modelling
Other Models
Control & Diseased Cell Lines	Indications: Alzheimer’s disease Motor neuron disease Childhood dementia