ALSembloids
Modeling ALS to find new treatments
Faced with the challenge posed by amyotrophic lateral sclerosis (ALS), researchers at Paris Brain Institute are developing highly innovative human-derived 3D models – known as assembloids – with the aim of understanding the mechanisms of the disease and paving the way for personalized treatments.
Our challenge
Understanding a disease with many faces
ALS is a neurodegenerative disease that is currently fatal and is highly complex from both a clinical and biological perspective. It affects each patient differently, depending on the genes involved, the speed of progression, and the areas of the body affected first. This heterogeneity makes it difficult to find effective treatments.
Going beyond the limitations of traditional models
Current animal and cell models cannot recreate all of the alterations observed in human ALS. It therefore appears necessary to develop models that more closely resemble the human diseases, which represents a real scientific and technological challenge.
Reproducing a complex human disease in the laboratory
To meet this need, teams at Paris Brain Institute – experts in neurodegeneration, stem cells, and neuropathology – are developing assembloids from organoids of different structures, which they then connect.
What is an organoid?
An organoid is a 3D structure created in the laboratory from human stem cells, which can reproduce complex biological circuits.
In this project, researchers assemble brain, spinal cord, and muscle organoids derived from cells of ALS patients to recreate the complete motor unit affected by the disease. The idea is to validate whether this cutting-edge model can be used to study the progression of the disease in a realistic and individualized manner, to understand its cellular mechanisms, and to identify targeted therapeutic strategies.
“ALS is a disease of formidable complexity. Through assembloids, we aim to model its multiple aspects in a human system, an essential step forward in understanding its progression and designing tailored treatments.”
Séverine Boillée
Leader of the DecodALS: ALS Causes and Mechanisms of Motor Neuron Degeneration team at Paris Brain Institute
TARGET AMOUNT
€1,886,000
over five years
TO FUND
- The research team, consisting of a postdoctoral fellow, a research assistant, and a clinical research associate
- The use of Paris Brain Institute’s shared core facilities to conduct the complex stages of the project (experiments, cellular and molecular analyses, cell cultures necessary for the creation of these assembloids)
- Equipment and consumables
ACKNOWLEDGEMENTS
This ambitious project would not have been possible without the decisive support of LOV Group and the efforts of Olivier Goy, Ambassador of Paris Brain Institute.