Mount Sinai, NYSCF receive NIH grant for Alzheimer's research

NEW YORK—A team of researchers from the Icahn School ofMedicine at Mount Sinai, in partnership with the New York Stem Cell Foundation(NYSCF) and other organizations, have received a multi-year grant from theNational Institutes of Health (NIH) for the study of Alzheimer's disease. Byapplying analytical methods to large-scale molecular, cellular and clinicaldata from Alzheimer's patients, the partners will seek to construct biologicalnetwork models, learn more about the mechanisms of Alzheimer's and identifypotential therapeutic targets. This award is one of several that make up the$45 million the NIH has announced it will dole out to advance the National Planto Address Alzheimer's Disease, an initiative that seeks to identify effectiveinterventions for the disease by 2025.

"We are delighted to support Dr. Schadt and his team intheir important work of applying novel analytical methods to build models ofthis complex disorder," Dr. Neil Buckholtz, director of the Division ofNeuroscience at the National Institute on Aging, said in a press release."Additionally, this funding supports their computational approach investigatingthe repurposing of existing drugs as treatment for Alzheimer's—a key objectiveset forth in the Alzheimer's Plan."

Among the specific areas the team will be exploring is anetwork of genes identified earlier in 2013 as a key driver of Late OnsetAlzheimer's Disease through its involvement in the inflammatory response in thebrain. As a main source of data, Mount Sinai scientists will be studying geneexpression in brains from the Mount Sinai Alzheimer's Disease Research CenterBrain Bank, which specializes in the earliest stages of the disease and isconsidered one of the best such resources globally.

Several cellular and animal models will be used to validatethe activity of individual genes and molecular networks believed to driveAlzheimer's disease. A computational approach will also be used to determine ifexisting drugs, indicated for other conditions, are capable of affectingAlzheimer's networks and being repurposed to treat or prevent the disease.

"With this grant, we can continue to build and refine ourpredictive model of Alzheimer's disease to yield valuable insights into thecomplex mechanism of the disease and potential therapies. In the same way thatsophisticated predictive mathematical models drive decision making in theglobal financial markets, our field of medical research has begun to rely onnetwork models to derive meaning from vast amounts of patient data, enablingbetter understanding and treatment of human disease," commented Eric Schadt,Ph.D., Jean C. and James W. Crystal Professor of Genomics at the Icahn Schoolof Medicine at Mount Sinai and director of the Icahn Institute for Genomics andMultiscale Biology.

Schadt will be a principal investigator for this study, aswill Sam Gandy, M.D., Ph.D., director of the Center for Cognitive Health atMount Sinai. Scott Noggle, Ph.D., director of the NYSCF Laboratory and theNYSCF – Charles Evans Senior Research Fellow for Alzheimer's Disease, isanother principal investigator. He and his team at NYSCF will be the lead stemcell partner for this work, generating stem cell lines from the samples ofAlzheimer's patients and producing Alzheimer's neurons for use as a platformfor validating drug targets.

"This research is of paramount importance. Currently, noeffective disease-modifying or preventive drugs exist for common, late onsetAlzheimer's disease," said Gandy. "Despite decades of intensive conventionalresearch, the causal chain of mechanisms behind sporadic Alzheimer's diseasehas remained elusive. This multi-scale, computational strategy, combined withtarget validation in mouse brain, in fly brain and in stem cell models, isalready providing clues to unanticipated pathways and new drug discoveryopportunities."

SOURCE: Icahn School of Medicine press release