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2020
The Friedman Brain Institute Drug Discovery Institute: A Catalyst for Neurotherapeutics Research in FBI Labs
Addiction Alzheimer’s disease Autism Depression Epilepsy Obesity Parkinson’s disease Schizophrenia
Breakthrough Finding
Assay Developed to Support Chemistry
Medicinal Chemistry
In Vitro Studies
In Vivo Studies
Safety Studies to Enable Clinical Trials
Research yields new insights into brain disorders
Design assay for new targets to support chemistry
Chemistry to design new drug that acts at target
Confirm that new drug is potent and selective
Confirm that new drug acts to modify the target in animals
Establish that the new drug is safe to test in humans
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Illustration by Danny Roldan
The brain is the most complex machine in the universe, and diseases that impact the brain are by far the least understood and most difficult to treat. More than 50 million Americans experienced a brain-related disorder over the past five years, including those impacted by neurodegenerative conditions such as Alzheimer’s and Parkinson’s diseases; psychiatric disorders, such as schizophrenia, depression, or drug addiction; and neurodevelopmental disorders, such as autism; among many others. Still, there are no therapies currently available that effectively slow the progression of neurodegenerative disorders, lessen the debilitating cognitive deficits in schizophrenia, prevent relapse in drug-addicted individuals, or alleviate the core symptoms of autism. Compounding matters is the fact that virtually all major pharmaceutical companies have drastically reduced their investment in research and development for brain disorders because of their poor success rates in finding new effective therapies over the past several decades. If not from pharmaceutical companies, from where will desperately needed new treatments for brain diseases come? Stepping into this vacuum are a new breed of translationally focused academic scientists who seek to take disease-relevant findings from their laboratories and advance them to human clinical trials. These scientists are supported
by forward-looking school leadership willing to make risky investments in the infrastructure necessary to develop new drugs, and also by new sources of National Institutes of Health and other federal funding. The Drug Discovery Institute (DDI) at Mount Sinai, directed by Paul Kenny, PhD, is a prime example of this new model of medications development by academic medical centers that has emerged over the past decade as pharmaceutical company investment in brain diseases has receded. Friedman Brain Institute (FBI) scientists supported by the DDI are working to develop new therapies for a number of brain diseases, using as a springboard their own innovative brain research that often yields new fundamental insights into the molecular, cellular, and systems-levels mechanisms of brain disease—research that has already received robust support from numerous pharmaceutical and biotechnology companies. The goal is to take advantage of new biological insights into disease mechanisms coming from FBI laboratories and partner with pharmaceutical or biotechnology companies to screen small molecules, known as “biologic therapies,” such as antibodies, RNAs, and viral-gene transfer, to attack these illnesses in unique ways, becoming an in-house catalyst for a new ecosystem of innovative, brain-focused translational science.