“NINDS has worked very hard over the last five years to look at all the programs that we run, and if one of the programs that we initiated had not met its goal, closed it down,” she said. “Anything that we were doing that was not of the highest quality and clearly going to reach the goal, we've closed [it] down….We've closed down tens of millions of dollars of programs. We've significantly increased the efficiency of our clinical trials efforts. It's not that we have things that can easily be removed from our budget.”
In fiscal year 2012, the NINDS set its “payline,” the threshold an application must clear in order to get funding, at the 15th percentile. This year, in anticipation of further cuts, the payline was set at the 12th percentile, meaning only the top 12 percent of projects receive funding, while only some applications ranking below that will. Now that the NINDS budget is final, NINDS will be able to fund to the 14th percentile.
“One of our biggest challenges is knowing whether or not we can start new initiatives,” Dr. Landis said.
Another big program now with a cloudy future is the Epilepsy Centers without Walls project that was planned to promote collaborations in the epilepsy research community — to explore questions such as unexplained death in epilepsy and developing treatments for treatment-resistant epilepsy. Dr. Landis said this program might have to be delayed.
Even if the programs are not delayed, their scope might be diminished due to the latest budget cuts, she said.
At the NIA, Richard Hodes, MD, the institute's director, said that two major trials, funded as a result of $50 million in additional money made available in the 2012 fiscal year, are examples of the kind of research that might suffer from delay, timing and “perhaps reduction in scale.” One is a major trial examining familial, early-onset Alzheimer's patients for treatment clues, and another is focused on whole-genome and whole-exome sequencing of people at low- and high-risk of Alzheimer's disease.
“What is most certainly true is we're able to do fewer studies and they will take longer to be carried out,” Dr. Hodes said. “We can pursue fewer aims or goals of possible targets at the same time, which means that, most assuredly, we will have a delay in ultimately making progress and finding a cure.”
IMPACT ON STUDY GROUPS
Randall Bateman, MD, director of the Dominantly Inherited Alzheimer's Network Therapeutic Trials Unit, said his team hasn't heard what effects the sequestration cuts will have on their funding. His project was not mentioned specifically by Dr. Hodes, but relies heavily on NIA funding.
“The effects of the sequestration are still uncertain, but we are all very concerned about significant cuts or even non-funding of the project,” Dr. Bateman said.
Puneet Opal, MD, PhD, associate professor of neurology and cell and molecular biology at Northwestern University, said that with such a small percentage of applications being funded — and possibly with that percentage growing even smaller in coming years — the funding process “becomes almost like a mechanism of chance.”
“You have these highly competitive grants which have to be sort of compared with each other,” he said. “It's very difficult to even differentiate between the very top-quality grants.” Researchers, he said, begin to wonder: “(With) great papers and good preliminary data, we still aren't getting grants, is it really possible to remain in the system? I guess that's the biggest point.”
Disease foundations and pharmaceutical grants fund research, but not to the same extent, nor do they provide overhead costs as NIH grants do, he said.
His research, which relies on NINDS funding and will have to be renewed shortly, is exploring the basic mechanisms of cerebellum degeneration and ways to try to reverse them.
“We've got some very interesting results looking at some changes in gene expression in cerebellar degeneration, but there is considerable uncertainty about future funding,” he said.
The degree of difficulty in getting funding is bound to scare off even more potential researchers, he said. “Physician-scientists, when they realize how difficult it is to get funding, many of them basically do other things,” Dr. Opal said. “Because they're physicians besides being scientists, they will leave research completely and go into clinical medicine.”
That, he added, ultimately will have an effect on training of new physicians scientists as well — there will simply be fewer physicians conducting research to show them the ropes.
THE RESEARCH PIPELINE
Dr. Landis said this potential chilling effect is not lost on her institute. “At NINDS, we've been quite concerned about the ability of first-time investigators, particularly physician scientists, to get funded and get their labs up and going and start their research,” she said. “They bring in new tools, new techniques, fresh ideas, and we've invested a lot of money in their training.”
Dr. Hodes said that the cuts are particularly tough to swallow given that research into Alzheimer's and other diseases — in part due to the growing understanding of their genetic underpinnings — have a full head of steam and seem poised for breakthroughs.
“There's the excitement, exhilaration about the scientific opportunities coupled with a great deal of concern, distress, and disappointment with the fact that we are unable to support all of the most excellent research proposals and grants,” he said.
He said the main hope to sustain support for continued research is to help inform the public about the value of biomedical research.
“If we are going to not only improve the quality of life but also address the financial viability of our health care system, we have to successfully address diseases and cause of disability,” he sad. “And investments in research, when the opportunities are so high, are really the most effective way to do that.”
This is the second article in a series about the effect the sequestration cuts will have on neurology. The first article, “Sequestration Means Fewer Medicare Reimbursement Dollars for Neurologists: How to Lessen the Blow,” appeared April 18: http://bit.ly/ZE9xZZ.
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