New Approach to Drug Discovery Could Lead to Personalized Treatment of Neuropsychiatric Disorders

The researchers have developed a method that could significantly speed up the search for new drugs to treat mental disorders such as schizophrenia.

Mental disorders are the leading cause of disability in the world, accounting for 31% of the total number of years lived with a disability. Although our understanding of the biology behind these disorders has improved, no new neuropsychiatric drugs with improved therapeutic effects have been developed in recent decades and most of the existing treatments have have been discovered by chance.

This is mainly due to the fact that doctors can not take brain tissue samples from patients in the same way that they are able to do a biopsy of a cancerous tumor somewhere else in the body for example, it is difficult for researchers to understand exactly what to target when designing. new neuropsychiatric drugs.

At present, a team of scientists led by the University of Cambridge has shown that live blood cells from patients with mental disorders can be used to identify potential targets for drug discovery research. Their results are reported in the newspaper Progress of science.

Human blood cells contain many receptors and proteins involved in signaling, which are also found in our central nervous system and have been associated with neuropsychiatric disorders. Previous research has shown that there is a close connection between the cells of our blood and the functioning of our central nervous system. For example, patients with bacterial infections often have depressive type symptoms.

This makes blood cells an ideal environment for testing potential new drugs. There is also significant evidence that the use of primary cells of patients when developing a drug results in a higher success rate for effective drug discovery.

"Psychiatric disorders are increasingly recognized as whole-body disorders," said Sabine Bahn, a professor in the Cambridge Department of Chemical Engineering and Biotechnology, who leads the research group responsible for the work. "This study proposes a change of terrain to directly explore living cellular function as a model of disease."

Using a high-level single-cell screening process, researchers analyzed cells from 42 patients with schizophrenia and examined thousands of potential compounds for new drugs. The team focused on discovering new psychiatric uses of medications commonly prescribed for other conditions, such as hypertension.

This strategy of "reuse" of drugs can reduce the time and costs required to bring a new drug to the clinic ten times more. With an average drug development cost of $ 2 billion to $ 3 billion over 12 years, this is an effective alternative to administering potential new treatments to patients in much less time. This approach could also lead to a reduction in the number of animal tests.

They can also test existing psychiatric treatments on patients' blood cells and can predict the effectiveness of these treatments for each individual. This overcomes a major obstacle in clinical psychiatry as many patients do not respond to first-line treatments. To do this, the team tested rare blood samples from patients with schizophrenia before and after clinical treatment, collected through a network of international collaborators.

Finally, the team confirmed that the activity of the new drugs was shared between blood cells and brain cells, testing these compounds on human nerve cells.

"It is the most thorough functional exploration and so far of primary psychiatric patient tissue.It has the potential to greatly accelerate drug discovery and personalized medicine for neuropsychiatric disorders." and other human diseases, "said Dr. Santiago Lago, lead author of the project. technology with Dr. Jakub Tomasik.