NIH Translational Center To Study Cell-Based Toxicity Tests, Offer Industry Grants
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Toxicity testing is one area in which NIH Director Francis Collins expects the proposed National Center for Advancing Translational Science to help industry speed up drug development. Current animal testing is not very reliable as a predictor of human response, Collins explained in an interview with “The Pink Sheet” to discuss his vision for NCATS and the roles he foresees for industry and FDA in the new center. Solving problems that cut across drug development programs, such as finding better toxicity testing methods, is the goal of NCATS, Collins said. Inaccurate conclusions from animal testing may cause work to proceed on a compound that later proves unsafe, or a drug to be dropped from the pipeline because there is “a worrisome signal in a mouse that actually would be irrelevant to humans,” he noted. “Why are we doing it this way when science is giving us new opportunities?”
Those opportunities include the ability to differentiate stem cells down different paths. With an engineering attitude, Collins suggested, scientists might “come up with a cell-based system using human tissues that would be a better predictor of what would happen in a human person. That feels like an idea whose time has come scientifically, and yet there is no home for that right now.” Collins envisions NCATs as that home, a center differentiated from the other NIH institutes, which largely have a disease focus, by its ability to address problems that are not product-specific (Also see "Consolidating Translational Research Programs Is Top FY '12 Priority For NIH" - Pink Sheet, 21 Feb, 2011.). Biomarkers are another area for which NCATS would be a “natural home,” he suggested. But while furthering science in this area, the proposed center is not intended to duplicate work being done by NIH and industry through the Biomarker Consortium (“Biomarker Consortium To Investigate Lung, Cancer, Non-Hodgkins Lymphoma,” “The Pink Sheet,” Oct. 6, 2006). NCATS To Aid Industry, Not Compete Because the center would focus on pre-competitive issues, Collins disagrees with some early complaints that NCATS will intrude into the territory of drug companies. “The real goal of NCATS is to look at the pipeline the way an engineer would and to try to identify steps in going from target identification to successful clinical trial application that could be sped up, optimized for a higher success, a more speedy achievement of success and a lower cost.”
Pharmaceutical companies concentrate on getting their products approved and individually are not going to invest in this type of activity, Collins pointed out, while NIH is well-positioned to manage this type of endeavor. In response to initial industry concerns about NCATS, Collins said he “spent a lot of time in the last three months meeting with high level [executives], particularly R&D chiefs, of pharmaceutical companies and … at this point I know of no high-level R&D chief who is opposed to this. In fact, I would say they’re almost uniformly enthusiastic.” They know that “we’re trying to solve these problems in the process development pipeline and that this is not a competitive effort.” Getting The Industry Point Of View Collins is bringing industry into his efforts through an advisory board that includes members of biotech and pharmaceutical companies as well as venture capitalists. “A lot of the expertise resides in the industry. We would be crazy not to listen closely to it.” He also expects industry advice to carry through into implementation of NCATS. The center will require an advisory council and that probably will include heavy representation from industry, he said. Collins also wants an NCATS director who is well-versed in industry’s point of view. “I would expect this person would have substantial experience in therapeutics development in both the public and private sectors because part of the goal is to pull those together.” NIH has only begun to search for the director and will wait to make the appointment until after congressional appropriators have approved funding for NCATS. All the budget materials have been sent to Congress, Collins said, so the appropriations panels now have a full grasp of the proposal (Also see "Collins Promises To Send NCATS Funding Proposal To Congress Within Weeks" - Pink Sheet, 16 May, 2011.). Industry also can expect to participate in the research effort, Collins noted. In its grant decisions, NCATS “will certainly have the ability to pull in expertise where it happens to exist, including in companies. So this will be an effort where the grants will at times be very much taking advantage of where the expertise is in the private sector.” NIH's increased interactions with industry come as the agency is tightening its conflict-of-interest regulations. Within a few months, NIH expects to issue revised conflict-of-interest policies that would increase the amount of information that institutions and investigators must reveal about their financial ties to industry, Collins said . Collins does not expect those rules to deter researchers from working collaboratively with industry. “Most scientists are more excited about the chance for a partnership with the private sector that is going to lead to scientific advances. If that can happen and it is done in a fashion where their reputations are completely protected against any accusation of conflict, they’re delighted,” he said. FDA In On Ground Floor Collins expects FDA to be a full participant in the NCATS effort. The product of NCATS’ research will be meaningless unless FDA finds a way to include it in its drug evaluation process, he pointed out. As an example, he noted that FDA will have to validate whether new toxicity test methods produce data suitable for regulatory decision-making. It is better to have FDA involved in the effort to develop those methods “from the ground floor, instead of late in the process and have some disconnect between what the scientists are trying to do and what FDA needs to have in front of them.” Make Me A Match Collins has positioned NCATS as a facilitator for spurring investigation of shelved compounds for application beyond those first studied ( (Also see "NIH Center Would Seek To Boost “Repurposing” Of Shelved Drug Candidates" - Pink Sheet, 20 Dec, 2010.) If companies are willing to release the information, NCATS would compile an inventory of compounds with known targets, he said, and serve as a conduit to link investigators with compounds that may be appropriate to their research. “Unless there was a broker, a matchmaker to make it so, [those kinds of exchanges] would never happen because of the lack of transparency in the system," Collins said. "So that would be one model in which NIH would take on that role, and also facilitate the intellectual property aspects of this by developing model agreements that both protect the companies' investment and the opportunity for a new use.” Broad-Spectrum Approach NCATS will consist primarily of translational science programs that currently are conducted at NIH. One of these is the Clinical and Translational Science Awards program, which funds research at 60 institutions that bring with them an array of projects that span a broad range of topics. The CTSA centers will bring in “talent in pre-clinical as well as clinicial trials, as well as comparative effectiveness research, implementation research in communities, even health delivery research,” Collins said. “That’s all going to be part of the NCATS agenda. So it’s translation written on a pretty wide chalkboard here, all the way from early stage to ultimate community implementation.” |
