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New NIH Translational Research Center Plan Moves Forward: What It Could Mean for Neurology

ARTICLE IN BRIEF

DR. STORY C. LANDIS: “What we have been doing at NINDS will be magnified several-fold. For example, NCATS will explore strategies to expedite ways to bring small molecules that have already been identified into therapeutic trials. The hope is that trying different strategies might make it faster.”

The NINDS director and leading neurology researchers agree that a new NIH center for translational research could help advance therapies for rare neurological disorders and others that have been neglected by pharmaceutical companies.

For the first time in more than 80 years, the NIH is dismantling one of its major research centers and creating a new one geared exclusively toward getting new drugs to patients more quickly through expedited “translational” bench-to-bedside research.

After the NIH Scientific Management Review Board recommended creating the National Center for Advancing Translational Sciences (NCATS) last December and breaking up the $1.3 billion National Center for Research Resources (NCRR), NIH Director Francis S. Collins, MD, PhD, moved quickly — some say too quickly — to execute a plan to create the new center. The NCATS will have an initial budget of more than $600 million, $100 million of which is expected to fund the Cures Acceleration Network, a grant program to underwrite research on “high-need” medications.

Although 60 percent of current NCRR staff will be reassigned to other centers and institutes, the Clinical and Translational Science Awards program will be rolled over into the new center. The transition should cause little disruption in current grants or new applications, according to the NCATS task force members who discussed the plans for a new center in a Mar. 9 teleconference.

In its December report, the Scientific Management Review Board said the new center will be a catalyst for collaborative interactions and partnerships to help bring new therapeutic agents more quickly to market. The current timeline for developing a new drug is roughly seven to 10 years, the review board noted, and it can cost several hundred million dollars before a novel medication reaches patients.

Moreover, the number of new drug approvals has fallen — a recent study found new drug applications have fallen below 50 percent, and not necessarily because the FDA has raised its approval bar. Industry data show that the number of new drug applications from manufacturers has been steadily declining for a decade.

NINDS WELCOMES NEW CENTER

The new center has the potential to greatly help neurologists and their patients, especially those with less common disorders, according to NINDS Director Story C. Landis, PhD.

“NCATS should have a significant impact in this area,” she told Neurology Today in a telephone interview. “One goal is to do pre-clinical work on rare disorders, and many of those that we deal with at NINDS are considered rare.”

The center will also provide NINDS with useful information and resources, encouraging basic science work that will decrease the economic burden on pharmaceutical companies who might not otherwise find such research financially feasible.”

“What we have been doing at NINDS will be magnified several-fold,” she said. “For example, NCATS will explore strategies to expedite ways to bring small molecules that have already been identified into therapeutic trials. The hope is that trying different strategies might make it faster.”

DR. WILLIAM T. TALMAN: “Dr. Collins is making bold strides to try to affect changes in care as a result of new discoveries. I hope [NCATS] will benefit translational research, but there are many nuances involved. Will we look back in five or ten years and see this as ‘the transitional moment’? I certainly hope so.”

New strategies to improve toxicology screening will also be targeted under NCATS, for example whole-genome studies on rare diseases. The center will also be looking for alternatives to animal models such as better in vitro testing.

“I think people are very enthusiastic,” Dr. Landis said. “At first, some were concerned that NCATS was going to be like a drug company, but our goal is to de-risk [research] programs so that pharmaceutical companies will be more willing to invest because the process will move further along.”

One major ongoing concern, however, is the NIH budget, she noted. “Right now it is not looking too generous. But I think at the NINDS, as well as the NIH, there is a general feeling that this is the right time to do this, even if the budget is cut.”

NEW FOCUS FOR RESEARCH

S. Claiborne Johnston, MD, PhD, professor of neurology and epidemiology at the University of California — San Francisco School of Medicine, agreed that NCATS could be very useful to neurology.

“Rare diseases are a big part of our specialty. These have been relatively neglected by industry but are excellent targets for NCATS,” he wrote in an e-mail to Neurology Today. “There has been a huge investment over the last decade in neuroscience. If NCATS can do even a fraction of what it aspires to, we may see more of the substantial progress in neuroscience translated into health improvements.”

Pharmaceutical companies have spent billions of dollars trying to develop treatments for neurological diseases, but “NCATS must carve out focus areas where industry won't go,” he said. ”Examples are testing agents in the public domain (such as those off-patent for other indications) and studying diseases that are neglected because they are rare or financially unappealing to companies. The Center for Translational Science Awards will be the major component of NCATS, and we really need to put them to work.”

DR. S. CLAIBORNE JOHNSTON: “There has been a huge investment over the last decade in neuroscience. If NCATS can do even a fraction of what it aspires to, we may see more of the substantial progress in neuroscience translated into health improvements.”

‘A TRANSITIONAL MOMENT’?

Only time will tell how effective the change will be and whether positive effects will be felt, said William T. Talman, MD, professor and VA chief of neurology at the University of Iowa in Iowa City, and president of the Federation of American Societies for Experimental Biology.

“For neurology and neuroscience, basic research will continue to be funded through the institutes and centers — the NCATS will not eliminate that,” he told Neurology Today in a telephone interview. In discussions with Dr. Collins, he added, he has been reassured that the NIH remains committed to continuing to enhance and expedite neurological research efforts.

“I think we need to look at this with regard to intent. If we could have dissociated concerns over the speed of the proposed changes I think the effort would have been applauded.”

The transparency of the proposal and the inclusion of many voices in the research and professional communities in discussing the changes — and listening to their feedback — has already helped to assuage many of their concerns, Dr. Talman noted, and “straw models” of the plan have been revised accordingly.

“Dr. Collins is making bold strides to try to affect changes in care as a result of new discoveries. I hope [NCATS] will benefit translational research, but there are many nuances involved. Will we look back in five or 10 years and see this as ‘the transitional moment’? I certainly hope so.”