IMBA: Mini-brains on their way to the market

Once again the European Research Council (ERC) has awarded a competitive research grant to the Viennese Top - scientist Jürgen Knoblich. The aim of the ERC Proof of Concept is to explore the commercial potential of cerebral organoids, small developing human brains Jürgen Knoblich’s group has been able to grow from human stem cells.

The ERC recently announced the names of 44 scientists who will receive a Proof of Concept grant. Each recipient will be awarded € 150,000 to explore the commercial potential of research results obtained through a previous ERC advanced grant, the most competitive funding line of the ERC.

Among this year's awardees is Jürgen Knoblich, senior scientist and deputy director at the Institute of Molecular Biotechnology (IMBA) of the Austrian Academy of Sciences. Knoblich received an ERC Advanced Grant in 2009 already. In 2013, his research team announced a breakthrough technology, named cerebral organoids, in the journal Nature. For the first time, they managed to generate 3D cultures from human stem cells that resemble the early steps of human embryonic brain development. The cultures start from pluripotent stem cells that can be obtained from any human individual and therefore can be used to model neurological brain disorders in a culture dish. Cerebral organoids resemble various different brain regions and grow into structures strikingly similar to human brain areas.

In the future, organoids derived from patients, will provide new insights into the mechanisms that lead to neurological disorders. At the moment, investigation of those diseases relies heavily on animal models but transferring those animal results to humans is often quite challenging. "Particularly for neurological disorders animal models fail to reflect the complexity of the human brain and suitable disease models for preclinical studies are not yet available", says Jürgen Knoblich. It is therefore difficult or sometimes impossible to study brain diseases and to develop appropriate medications. "Cerebral organoids offer an exciting possibility to transfer results from animal experiments to humans, allowing for more targeted clinical studies to reduce costs and improve the reliability of results", says Knoblich.

Even today, medical treatment of diseases like schizophrenia, largely relies on findings made in the 1950s. Pharmaceutical companies around the world have recognized an essential need to develop models representing those diseases to develop new strategy for treatment and diagnosis. "We were already able to show that cerebral organoids can be used as a model of human disease such as microcephaly." Microcephaly is a severe neurodevelopmental disorder where brain size is reduced, leading to severe mental impairment. In mice and rats, microcephaly cannot easily be investigated as these animal models do not display the same severe symptoms seen in humans.

Supported by the ERC, Jürgen Knoblich and his team want to investigate the commercial possibilities of cerebral organoids as a new and cost-effective application, to contribute to the development of urgently needed treatments for neurological disorders. In this regard, leading pharmaceutical companies have already expressed their interest.

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