CAMBRIDGE, Mass.—GNS Healthcare, Inc. and CHDI Foundation,Inc. (CHDI) have announced a new collaboration this week to create adata-driven computer model of Huntington's disease. The neurodegenerativedisease currently lacks any effective therapies, and the companies are seekingto develop novel therapies for the disorder by using GNS' supercomputer-drivenReverse Engineering and Forward Simulation (REFS) platform.
"While the cause of Huntington's disease is known, there arecurrently no effective treatments for this devastating disorder. CHDI'sresearch efforts focus on understanding the underlying biology of HD in orderto discover treatments that slow disease progression," Dr. Keith Elliston, vicepresident of Systems Biology at CHDI, said in a press release. "The HD modelthat GNS is building will allow researchers to perform simulations thatgenerate novel hypotheses to help drive this understanding. As such, thiscollaboration between CHDI and GNS represents an innovative, complementaryapproach to advancing discoveries that we hope will lead to novel therapies."
Per the terms of the collaboration, GNS will use its REFSplatform to reverse-engineer network models from available genomic, expressionand cell signaling data, and the results from in-silico simulations of the REFS models will enable newinsights into the mechanisms of Huntington's disease. The platform is ascalable, supercomputer-enabled framework that automates the discovery andextraction of causal network models from data, using high-throughputsimulations to generate knowledge. Once the REFS network models are complete,they will be transferred to CHDI and its affiliated researchers, enabling theHuntington's disease research community to have ongoing access to the models.
"GNS will apply its big data analytics platform to create adisease-specific computer model that will yield a powerful new resource to theHuntington's disease research community—with the ultimate aim of betteroutcomes for patients," Dr. Iya Khalil, GNS' executive vice president andco-founder, said in a press release regarding the deal. "This collaborationwith CHDI is representative of our focus on using big data analytics to revealnew insights into a disease and provide tools for scientists that will help transformthe tremendous amount of information coming from new technologies such asnext-generation sequencing into predictive computer models of diseaseprogression and drug response. This project serves as a model for futureprojects focused on other challenging, complex and orphan diseases."
Huntington's disease is an inherited neurodegenerativedisorder, and results from a mutation in the huntintin gene, a defect thatcauses a DNA sequence known as a CAG repeat to occur more often than normal.Each child whose parent has a mutation in the huntingtin gene has a 50 percentchance of inheriting the mutation, and an estimated 150,000 people in theUnited States alone are believed to have a 50 percent chance of developing thedisease at some point. The disease causes patients' brain cells to degenerate,which leads to behavioral, cognitive and motor skill impairments thatdrastically reduce quality of life and eventually lead to death within 15 to 25years of the onset of noticeable symptoms. Approximately 30,000 people in theUnited States are believed to be affected by Huntington's disease, but notherapies have been approved to slow the progression of the disease.
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