Miller School Researchers Receive NIH Blueprint Award

The National Institutes of Health (NIH) has awarded grants to investigators across the United States, including three from The Miami Project to Cure Paralysis and the Department of Ophthalmology.

Coral Gables (August 23, 2011) — The National Institutes of Health (NIH) has awarded grants to investigators across the United States, including three from The Miami Project to Cure Paralysis and the Department of Ophthalmology for an ambitious set of projects to develop new drugs for disorders of the nervous system. The projects—aimed at treating conditions such as vision loss, neurodegenerative disease and depression—are funded through the NIH Blueprint for Neuroscience Research.

The NIH Blueprint pulls together 15 of the agency’s institutes and centers, leveraging their resources to confront major, crosscutting challenges in neuroscience research. The Blueprint Neurotherapeutics Network will serve as a resource for investigators to develop new drugs for nervous system disorders and prepare them for clinical trials. It will be funded by up to $50 million over five years.

The Miller School team that received the award is made up of principal investigators John Bixby, professor of molecular and cellular pharmacology and neuroscience at The Miami Project; Vance Lemmon, professor of neurological surgery and the Walter G. Ross Distinguished Chair of Developmental Neuroscience at The Miami Project; and Jeffrey Goldberg, associate professor of ophthalmology at Bascom Palmer Eye Institute. Their award will fund research on optic neuropathy (damage to the optic nerves), with the goal of developing compounds that help injured fibers in the optic nerve regenerate and grow through scar tissue. Damage to the optic nerves, which connect the eyes to the brain, is a common cause of vision loss.

“Our laboratories have been working together on basic science questions relating to nerve injury and repair since 2006,” Bixby said. “Both The Miami Project to Cure Paralysis and the Bascom Palmer Eye Institute are committed to efforts to translate our basic science discoveries into potential treatments for patients. We are excited that the Blueprint Neurotherapeutics Network will enable us to partner with NIH’s National Institute of Neurological Disorders and Stroke (NINDS) and with external experts in therapeutic development to move in this direction.”

For decades, public funding from NIH has helped academic labs and small businesses use their ingenuity to pursue new strategies for treating nervous system disorders. However, many labs often lack the resources – time, money, scientific staff, and regulatory expertise – to turn a promising strategy into an effective treatment. The new initiative places these investigators at the helm of an expert drug development team that includes pharmaceutical industry consultants and service contractors.

“The Blueprint Neurotherapeutics Network will pair neuroscientists with experts in therapy development and enable them to pursue their most exciting ideas for new drugs without having to redirect the focus of their laboratories,” said Story Landis, director of NINDS.

Nervous system disorders affect tens of millions of Americans, and there is a substantial unmet need for treatment. But the process for developing new drug therapies is costly and carries high risk. Only about 10 to 20 percent of candidate drugs for all disease indications survive the early phases of development and reach clinical trials. And development of treatments for disorders affecting the nervous system may face special hurdles. For example, many such disorders affect relatively few individuals, which means they present small markets for drug companies.

Before a new compound can move into clinical trials, its chemical structure must typically be redesigned to transform it into a safe and effective drug, a process called chemical optimization. Hundreds of chemical variations must be tested and retested in cell-based systems and animal models to find one with the desired effects.

Project teams supported by the Blueprint Neurotherapeutics Network receive research funding, plus access to millions of dollars worth of services normally only available to pharmaceutical companies. The pharmaceutical and biotechnology industry consultants will assist investigators throughout the drug development process, from chemical optimization, to biological testing, to advancing the drug into early-stage clinical trials. Each project team will be required to meet a set of interim goals, or milestones, to continue to receive funds and access to Blueprint resources.

The Blueprint awarded grants to seven research teams at six academic institutions and one drug discovery company.

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From left, John Bixby, Jeffrey Goldberg, and Vance Lemmon.

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