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NIH Rare Disease Program Leans on 'Omics

NEW YORK (GenomeWeb News) – The National Institutes of Health is putting $24 million this year into a new program that will attempt to develop drugs for rare diseases that may affect millions of Americans for which there are no treatments or cures, and for 'neglected' diseases around the world that frighten away potential drug makers.

The Therapeutics for Rare and Neglected Diseases Program (TRND, pronounced 'trend'), overseen by the NIH Office of Rare Diseases Research, includes collaborators from the National Human Genome Research Institute and its NIH Chemical Genomics Center. The five-year project will be funded by $24 million per year through 2013, NIH Acting Director Raynard Kington said in a press conference today.

The TRND program will aim to develop compounds that could treat these diseases from the earliest identification stages and on toward preclinical studies, beyond the expensive and risk-laden "valley-of-death" phase that makes pharmaceutical companies shy away from many development projects.

Although rare diseases affect less than one in 200,000 Americans, NIH said, there are over 6,800 varieties of them, and together they afflict more than 25 million people in the US. Today, they present "a major health problem," Kington said.

Unlike rare diseases, many neglected diseases lack treatments in part because people in the developing regions where they are common could not afford to buy enough of the drugs produced to fight them.

"Neglected diseases," Kington continued, "tend to be rare in the US because they have been wiped out here, but they have not been wiped out worldwide.

"As the recent outbreak of influenza A H1N1 demonstrates, America is not immune from such diseases," he added.

"The federal government may be the only institution that can take the financial risks needed to jumpstart the development of treatments for these diseases, and NIH clearly has the scientific capability to do the work," Kington said in a statement.

TRND will use NCGC's robotic high-throughput screening systems and library of over 350,000 compounds to "make basic discoveries and probe cellular pathways," NIH said.

"This effort is a natural fit with NHGRI's mission, as almost all natural disorders have a genomic link," NHGRI Director Alan Guttmacher said during the press conference.

Any molecules developed through NCGC screening having potential therapeutic properties could then be run through the TRND drug development pipeline.

NCGC Director Christopher Austin said in a statement that TRND researchers will start teams to "do the hard work of optimizing chemicals that we or others discover that may treat rare diseases and turn them into actual drugs.

"Unlike traditional drug development, however, where only successes are published, we will publish our failures as well, so everyone in the drug development community can learn from them. That alone could be revolutionary," Austin added.

In addition, "TRND will not pursue a treatment under development by someone else, and it does not want to compete with the biotechnology industry," Stephen Groft, director of the NIH Office of Rare Diseases Research, said this morning.

The effort will involve collaborations with NIH-funded researchers in academia, with patient advocacy groups, and with the private sector, and will include a panel of advisers from both spheres and from NIH.

"Meaningful collaboration with external partners is a key element in NIH's approach; for example, engaging disease-specific groups in preparation for clinical trials to characterize the natural history of diseases and determine clinical trial endpoints is essential," Sharon Terry, president and CEO of Genetic Alliance, said in a statement. "All stakeholders working together will transform the drug development pipeline," she added.

The project is intended to "take a promising molecule through the most difficult phases of clinical development, and then try to find a partner to develop it," Groft explained.

The basic plan for this year, Groft added, is to find staff, outfit laboratories, and start looking for a couple of rare diseases to begin pursuing.

NCGC's Austin also said that in most cases the TRND program will seek to find a company to in-license the therapeutic candidates it develops.