The changing biomedical research landscape and cutbacks in industry R&D programs are encouraging more public support for pharmaceutical
research around the world. The Innovative Medicines Initiative in Europe is building a schizophrenia database of industry-sponsored
clinical trials to better identify signals of patient response to test drugs. The United Kingdom's Medical Research Council
has established the Developmental Pathway Funding Scheme to support basic research on drugs and medical devices, and the Wellcome
Trust's Seeding Drug Discovery initiative is funding efforts to take drug candidates through early clinical trials. Both US
and EU scientists are wary of being left behind by soaring Chinese investment in R&D.
At home, NIH director Francis Collins has launched a high-profile campaign to promote translational medicine as a way to spur
development of new medical treatments that can benefit patients. In December, an NIH advisory committee recommended establishing
a new NIH National Center for Advancing Translational Sciences (NCATS), a move engineered by Collins to bring together a number
of NIH programs that provide resources for translating basic discoveries into new medicines and diagnostics. These include
a program that supports development of therapies for rare and neglected diseases, along with NIH's national network of research
sites at academic medical centers supported by Clinical and Translational Science Awards.
As the former director of NIH's Human Genome Project, Collins is optimistic that new genetic discoveries can chart pathways
for discovering new medical treatments, and that the emergence of more well-validated genes will be useful in "identifying
drug targets in unprecedented numbers," he said in an interview. The scientific enterprise is yielding up a lot of new ideas
about therapeutics, he observed, yet "traditional private sector efforts to capitalize on that are taking a hammering." NCATS
aims to bolster the funding of research projects at a time when biotech and pharma companies face serious financial challenges.
Along these lines, the initiative also will encourage more collaboration between academic researchers and biopharmaceutical
companies and to strengthen ties with FDA to ensure that NIH-sponsored studies provide the data needed to support registration
of new products.
The project envisioned by Collins will help re-engineer the drug-development pipeline by investing in new assays that can
screen thousands of molecules to find ones that will hit defined disease targets. Another objective is to improve assessment
of toxicity, which may involve shifting from the use of animal testing to identify potential problems.
As part of the process, Collins' translational science campaign aims to convince Congress and the American public that the
federal investment in biomedical research can pay off in terms of new, life-saving therapies (see sidebar, "Public funds yield
more medicines"). The Obama administration has proposed a very slight increase in the NIH budget for fiscal year 2012, which
would just barely maintain current funding levels. Even during the Republican budget cutting campaign of the mid-1990s, NIH
retained several strong GOP advocates on Capitol Hill and largely escaped the chopping block; that kind of support seems to
be lacking among current Republican leaders.
Public funds yield more medicines
Collins believes that today there is greater private sector interest in NIH-funded preclinical and clinical testing, as well
as in compound rescuing or "repurposing." Pharmaceutical companies have long lists of compounds that have been abandoned along
the way, maybe because a business plan changed or the money ran out or clinical trials failed to show efficacy, Collins notes.
"We have been talking with leaders in the pharmaceutical industry about an opportunity to open the freezers and make such
compounds available, with appropriate intellectual property protection for them," Collins explains. "This isn't a giveaway,
but could be a win-win if such a compound were found to be active for a different application than originally considered."
NIH will not move into drug development per se, Collins emphasizes, but will hand promising compounds off to private sector sponsors.
Jill Wechsler is Pharmaceutical Technology's Washington editor, 7715 Rocton Ave., Chevy Chase, MD 20815, tel. 301.656.4634, firstname.lastname@example.org