With a $650M gift and a trove of genomic data, Broad sets out to innovate in schizophrenia

Edward Scolnick, founding director of the Broad Institute's Stanley Center for Psychiatric Research

It has been nearly 60 years since researchers developed the last fundamentally new treatment for schizophrenia, a debilitating psychiatric disorder that affects about 1% of the world's population. Now, scientists from the Broad Institute are unveiling new genomic findings from thousands of schizophrenia patients that could lead to new molecular targets. And, thanks to philanthropist Ted Stanley, they'll have $650 million to help find out.

Stanley's multiyear commitment is the largest ever in psychiatric research, according to Broad, and it'll fund ongoing work by the institute's investigators, drawn from MIT, Harvard University and elsewhere. The plan is to fully flesh out the wide range of genetic variants that cause mental illness, map the pathways of those genes, develop new animal models that accurately mimic psychiatric disorders and, eventually, churn out new compounds that might treat them.

And, in light of a sweeping study just published in Nature, the researchers have some solid ground from which to get started.

Coinciding with Stanley's gift, investigators from Broad and institutions around the world are trumpeting the results of a global study through which they've identified 108 spots on the genome that play a role in schizophrenia. The researchers studied more than 80,000 genetic samples from patients with and without the disorder, nearly quadrupling the known number of schizophrenia-associated genes in the process, Broad said.

Now, with so many genetic loci to track, scientists can go from feeling around in the dark to charting patterns between them, study author Stephan Ripke said, opening up new lines of inquiry into the root causes of psychiatric disorders.

"We can group them into identifiable pathways--which genes are known to work together to perform specific functions in the brain," Ripke said in a statement. "This is helping us to understand the biology of schizophrenia."

From there, they hope to develop pathway-modulating chemicals that could become drug candidates, potentially ending the decades-long drought in therapeutic development for the disorder.

For now, it's early days in what will likely be a long process, fraught with false leads and dead ends, as is always the case in drug development. But the combination of Stanley's long-term gift and some actionable genomic signals in schizophrenia is cause for renewed optimism, Broad leader and Merck ($MRK) veteran Edward Scolnick said, especially in a field whose complexity has frightened off many in the industry.

"I'd be bold enough to say that in 5 years, all the drug companies that got out of psychiatric research will be getting back in," Scolnick said in a statement. "The coming decades of psychiatric research will yield new science and a needed parallel effort to increase resources for services that can help patients and their families."

- read more about the donation
- here's the release on the study