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GENOMICS

NIH Targets $275M for Rare Disease Research

Program aimed at achieving a high impact in a short time.

DANIEL S. LEVINE

The Burrill Report

“The program will open up new avenues for therapeutic delivery of RNAs that could transform basic science and clinical practice.”

The National Institutes of Health announced a total of $275 million in funding for two new research programs—one focused on undiagnosed rare disease and the other on the role RNA outside of a cell plays in cellular communication.

The programs are being funded under NIH’s Common Fund, which is intended to target strategic investments that have the potential for creating a significant impact in a short amount of time. Both programs are scheduled to being during fiscal 2013.

The Undiagnosed Diseases Program, backed with about $145 million in funding over the next seven years, will provide a new network of medical research centers focused on the discovery, diagnosis, and care of undiagnosed patients.

Though individually these diseases are by definition rare, an estimated 6 percent of the U.S. population suffers from an undiagnosed rare disorder. The new program hopes to build on the success of NIH’s intramural research into rare diseases and will test whether its type of cross-disciplinary approach to disease diagnosis is feasible to implement in academic medical centers throughout the country.

It will promote the use of genomic data in disease diagnosis and will engage basic researchers to identify the underlying mechanisms so that therapies may be rapidly identified. The program will also train clinicians in the use of contemporary genomic approaches so that these methods can be used to fight other diseases.

“This program will spawn new medical discoveries and accelerate clinical investigations that will ultimately improve the lives of thousands of patients living with undiagnosed diseases,” said Collins. “Establishing a national network of clinical research centers is a critical first step towards addressing the need for more rapid and coordinated approaches to diagnose and manage rare undiagnosed diseases.”

The Extracellular RNA Communication program will be backed with about $130 million over five years. It will explore new ways in which cells communicate with each other using ribonucleic acids or RNAs that are present in the outside of and between cells. The program seeks to conduct foundational research about the synthesis, distribution, uptake, and function of extracellular RNAs that are involved in cell-to-cell communication critical for basic cell function and health.

“Because cell-to-cell communication is critical to so many different diseases, the program will open up new avenues for therapeutic delivery of RNAs that could transform basic science and clinical practice,” says James Anderson, director of the Division of Program Coordination, Planning, and Strategic Initiatives that guides the NIH Common Fund’s programs. “Through development of new technologies, research tools, and data, the program will lay the groundwork for new investigations into the role of RNAs in health and disease.”



July 06, 2012
http://www.burrillreport.com/article-nih_targets_275m_for_rare_disease_research.html

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