Medicine: How active ingredients reach the brain better

The so-called blood-brain barrier is the physiological barrier between the fluid spaces of the bloodstream and the central nervous system. It regulates the supply of nutrients such as glucose to our brain, but also protects it from pathogens and toxins, i.e. toxic substances. The functions in the brain are highly complex and at the same time very sensitive and require an environment that is as free of disturbances as possible. This strict protective mechanism is therefore very important for the functioning and survival of nerve cells, but it also means that less than five percent of the active substances intended for the brain actually reach it. For patients suffering from diseases of the nervous system - such as epilepsy, multiple sclerosis or Alzheimer's disease - this can mean that therapies are not always successful despite the right drugs.

As part of the IM2PACT project, Winfried Neuhaus from AIT is working with 27 international partners from science and industry on methods and ways to better overcome this natural barrier in the brain in the event of disease: "The transport function of the blood-brain barrier is altered in many diseases. We want to understand this better and then exploit it to find new routes for the passage of drugs into the brain, thus enabling better therapies," says the scientist.

Neuhaus and his team are studying human tissue to find new transport routes for active substances. Cell culture models are used to mimic disease conditions as closely as possible in order to achieve better predictive power for future clinical applications. The models are based on so-called "human induced pluripotent stem cells". Stem cells are not yet committed to any particular tissue type and can potentially develop into any cell type. They are obtained from normal somatic cells, which are reconverted into stem cells by adding several stimulation factors. They will eventually be used to produce tissues that mimic the human blood-brain barrier and can be used to study the transport pathways for potential drugs.

The groundbreaking research project is made possible by funding of EUR 18 million, consisting of direct funding from the Innovative Medicines Initiative (IMI) and in-kind contributions from industry partners. The project will run for five years and is co-led by the University of Oxford and Sanofi's Central Nervous Barrier Group.

IMI is the largest public-private partnership in Europe and is jointly funded by the European Union's Horizon 2020 research and innovation program and the European pharmaceutical industry, represented by the European Federation of Pharmaceutical Industries and Associations (EFPIA).