Scientists unveiled a more effective approach for assessing drug response

06 November 2018

GMP News

Scientists from Eli Lilly and Company, the Icahn School of Medicine at Mount Sinai (New York, USA) and Sema4 (Stamford, USA) released results from a proof-of-concept study demonstrating that patient-derived cells offer a more effective approach for assessing drug response than conventional methods.

The Icahn School of Medicine at Mount Sinai is an international leader in medical and scientific training, biomedical research, and patient care. Sema4 is a patient-centered predictive health company dedicated to advancing the diagnosis, treatment, and prevention of disease.

The findings could pave the way for streamlined drug discovery, particularly for diseases such as schizophrenia that have seen little therapeutic innovation. The study was published in Nature Communications journal.

Drug discovery for neuropsychiatric disorders such as schizophrenia is limited due to a lack of useful models for screening candidate treatments. Currently, all antipsychotic drugs target the same dopamine receptor. Unfortunately, two-thirds of patients with schizophrenia have no response or only partial response to these treatments. New therapies are necessary, but without biologically relevant screening models there has been little progress.

In this new study, scientists theorized that more meaningful results could be generated from testing drug candidates on patient-derived cells, rather than on generic cell lines. They utilized cells isolated from 12 patients with schizophrenia and 12 healthy controls, generating induced pluripotent stem cells that were guided to become neural progenitor cells, the key cell type targeted for neuropsychiatric disorders.

Researchers prepared a series of assays from each individual’s cells, treated them with 135 different small molecules selected for their predicted or demonstrated interaction with schizophrenia-relevant biology, and analyzed gene activity. The same drugs were tested on commonly used cancer cell lines for comparison. Results clearly showed that the patient-derived cells yielded more disease-relevant information than the generic cells. In some cases, certain drugs reversed the gene expression signatures associated with schizophrenia.




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