Researchers from a consortium of three faculties of the University of Szeged and the ELKH Biological Research Centre (BRC) have identified new drug candidates and developed new drug delivery systems for the central nervous system. The four-year GINOP project, which has just been completed, was carried out with nearly HUF 720 million in support from the European Union.

The main objective of the project was to identify a new family of neuroprotective, anti-inflammatory drug candidates and to investigate it extensively prior to its clinical application. Further development of the best compound selected for the purpose (code 0758-S) may contribute to the development of drugs for the prevention and treatment of diseases associated with dementia and loss of mobility, such as Alzheimer’s, Parkinson’s, Huntington’s disease, amyotrophic lateral sclerosis, and prion diseases.

It is known that the processes involved in the destruction of brain cells – especially nerve cells– are not prevented by the drugs and treatments currently in use. This means there is no causal treatment for diseases associated with dementia or impaired locomotor function. It has now been recognized that all of these diseases see the development of chronic inflammation of the brain tissue (neuroinflammation) that classical anti-inflammatory drugs cannot prevent. The consortium therefore aimed to identify and design new compounds for activating a relatively new receptor protein, the sigma-1 receptor, and to investigate their physiological and pharmacological effects.

As a result of the coordinated work of the consortium, physicists, chemists, pharmacists, biologists and physicians have been able to identify and purify several new compounds from their own library of 4,000 compounds, and have also planned new molecules that have been used in a number of preclinical in vitro experiments and have been shown to be cell protective and anti-inflammatory in an in vivo experiment. These compounds also enter the brain through the blood-brain barrier. Some of the active ingredients have an inhibitory effect on cell division, while the parent compound (0758-S) also has an antiepileptic effect.

Among the drug delivery systems developed, the researchers found one that facilitated more efficient entry of the parent compound into the central nervous system. The nasal formulation allows the active substance under development to be delivered directly to the brain, thereby bypassing the blood-brain barrier.

The GINOP-2.3.2-15-2016-00060 research project started on April 1, 2017 and ended on December 31, 2021, and was led by Professor Tamás Janáky. The members of the consortium, including the Molecular Stress Biology Research Group led by Dr. Zsolt Török, the Biological Barriers Research Group led by Dr. Mária Deli, and the Chemical Biology Laboratory led by Dr. Csaba Tömböly, intend to continue to potentially prevent and treat these neurological diseases in the future through further development of their candidate substances.