Bench to Biotech

The NIH translational sciences center envisioned by Collins would house a spectrum of programs now scattered among different NIH entities, according to the report to NIH's Scientific Management Review Board, which was established two years ago to examine and recommend changes in the agency's vast organization and structure. Current translational research programs cover the development waterfront, many funded by the director's Common Fund, a $500 million kitty that Collins administers to support cutting-edge, trans-NIH projects. Instead of shifting these activities to an existing NIH institute, the advisory group thought it best to establish a new organization to emphasize the importance of these changes. Under the proposal the new Center will contain NIH's:

Molecular Library and Chemical Genomics Center. A network of high-throughput screening centers that can assess thousands of possible drug targets against library compounds and provide researchers with chemical probes to study the functions of genes, cells, and biochemical pathways.

Therapeutics for Rare and Neglected Diseases (TRND) program. Supports preclinical animal and PK testing to bring potential treatments for neglected diseases through the "valley of death." One TRND pilot seeks new treatments for schistosomiasis, a disease affecting some 250 million people in Africa, but neglected by industry and researchers for decades. Another project supports preclinical testing of an orphan drug for sickle cell disease discovered by a small biotech company, AesRx of Massachusetts. TRND also is tackling very rare conditions, such as hereditary inclusion body myopathy and Neimann-Pick Type C, a disorder of lipid metabolism. The goal is to get to the point where a private sponsor could apply for an IND (investigational new drug application) with FDA to launch clinical studies.

Clinical and Translational Science Awards (CTSAs) program. The most controversial recommendation is to shift this network based at 55 academic health centers, which receives about $500 million a year from NIH, to provide translational support to clinical research efforts. The move, though, would rob the National Center for Research Resources (NCRR) of nearly half its budget, prompting heated opposition from NCRR staff and grantees.

One interesting component that would move with the CTSAs is the Pharmaceutical Assets Portal, designed to match up research scientists with abandoned or off-patent drugs available for rescue or research. The Portal wants to include compounds from pharma companies, but so far only Pfizer has become involved. The Portal organizers hope that a new discontinued clinical compound library of fully tested products will attract broader support.

Cures Acceleration Network (CAN), which was authorized by last year's health reform legislation to fund drug development projects. The new Center would provide a visible home for CAN if it is ever fully funded by Congress. An initial $50 million budget for 2011 is still on hold, and the outlook is dim that it will ever become the $500 million program originally sought by former Senator Arlen Specter.

Several NIH activities may remain outside the new Center, such as the Pharmacogenomics Research Network (PGRN), a group studying how genes affect patient response to medicines for cancer, heart disease, asthma, nicotine addiction, and other conditions. The panel also decided to leave NIH's Clinical Center as a distinct entity, but with strong links to the Center.