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Haloperidol is a venerable antipsychotic drug that is used to treat schizophrenia, Tourette syndrome, bipolar disorder, and other conditions.

Paul A. J. Janssen at Janssen Pharmaceutica (Beerse, Belgium), discovered haloperidol in the mid-1950s. Clinical trials began in 1958, but the US Food and Drug Administration did not approve the medicine until 1967. It was first marketed by McNeil Laboratories (Fort Washington, PA) under the trade name Haldol.

Haloperidol has many side effects, some of them serious; but it is still a widely used medication. In 2018, Kristen Brennand and colleagues at the Icahn School of Medicine at Mount Sinai Hospital (New York City) and Eli Lilly (Indianapolis) used haloperidol and 134 other drugs (not all of them antipsychotics) to develop . Their impetus was that as many as 30% of schizophrenia patients are unresponsive to existing drugs, and current drug screens are not effective.

The researchers gathered skin cells from 24 individuals, 12 with schizophrenia and 12 controls. They converted those cells to stem cells and then to neural progenitor cells. When the scientists exposed the neural cells to the test drugs, the cells derived from schizophrenia patients showed sharper disease-related gene expression responses to antipsychotics than did the cells from the control studies.

This proof-of-concept experiment revealed the possibility of using similar screening methods to evaluate potential anti-schizophrenia drugs. The authors also suggest that a patient’s own cells might be used to determine which medicines are most effective against that individual’s disease.