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Tackling Genomics of the Brain

This article was originally published in Start Up

Executive Summary

With the limited success of traditional approaches to CNS, scientists are betting that genomics may hold the key to a new generation of drugs that stop disease progression and possibly reverse its impact. At least half a dozen companies focusing mostly on neurogenomics have started up and raised more than $100 million in the past three years. While they use standard genomics tools and follow typical post-genomics business models, they face special challenges due to the complexity of the brain.

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Psychiatric Genomics Inc.

Psychiatric Genomics will use information generated by the Human Genome Project, along with gene expression, microarray and high-throughput technology to uncover the complex, multiple genetic factors implicated in mental illness. It will initially focus on schizophrenia, bipolar disorder and autism--underserved illnesses with strong evidence of a genetic component.

Neurome Inc.

Two-month-old Neurome has a stable of tools to help partners find new therapeutic opportunities based on gene expression patterns in the brain. Founded by three neuroscientists, it got off to a fast start and already has a strategic alliance with Elan Corp. Neurome is building standardized, quantitative databases that for the first time adequately and accurately depict the molecular, cellular and circuitry patterns of the brain region by region and circuit by circuit.

Making Progress in Stroke

Few effective treatments exist for acute ischemic stroke, but not for lack of effort. Pharmaceutical approaches have been extremely disappointing; more than a handful of companies have been forced to stop work on a drug already in Phase III trials. Device innovators are more optimistic; they believe that their devices might help improve patient outcomes when combined with drugs. The devices could be used to get the drug directly to the site of the clot that causes the stroke and to prepare the clot to receive and absorb the thrombolytic rapidly. Other device companies are working in the field of temperature management, aiming to achieve hypothermia. Challenges remain great, and include the lack of infrastructure for diagnosing patients in a timely fashion and treating them once they arrive in a hospital ER; the difficulty of recruiting eligible patients for clinical trials; and the complexities of the disease. But a small number of device companies are in early clinical trials, albeit moving at a glacial pace.

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