Recently, a team of researchers at the Yale University School of Medicine discovered that a drug originally developed for the use of cancer therapy could be used to treat Alzheimer’s disease (March 21, 2015 Annals of Neurology; click here for NIH story). The drug is called saracatinib, originally developed by the biopharmaceutical company AstraZeneca. However, through the NIH-funded initiative to repurpose experimental drugs, the research team discovered that the drug restores memory loss and reverses brain problems in mouse models of Alzheimer’s disease.
Nearly 5 million Americans have Alzheimer’s disease, the most common form of dementia. When a person develops Alzheimer’s disease, clumps of amyloid beta protein build up in the brain, damaging brain cells and synapses. Current Alzheimer’s treatments only ease the symptoms of the disease. When saracatinib was given to mice with symptoms similar to Alzheimer’s patients, mice showed a complete reversal of spatial learning and memory loss after four weeks. The researchers found that the drug actually restored the synapses.
Normally it takes around ten years for an experimental drug to be approved for a Phase 2a human clinical trial. This process took the Yale research team only 18 months. “The investigational drug already had been developed, optimized and studied in animals as well as tested for safety in humans, so our ability to obtain this asset through NCATS and AstraZeneca gave us an incredible shortcut in the drug development process,” explained Strittmatter, the senior author on the study. This discovery serves as a testament to the importance of collaboration and knowledge sharing across groups. The human trial will involve 152 subjects, who will receive either saracatinib or a placebo for one year. The researchers expect to have results in the next two years.
Why should we follow the Appropriations process? Aside from the obvious reason—to know how big the pot of extramural money will be next year and to understand the government’s commitment (or lack thereof) to science funding—there is another important reason: If you know the language worked into Appropriations testimony you can strategically design your research and incorporate key language and ideas into your NIH proposal in order to improve your odds of funding. (In these competitive funding times, every little advantage helps.) So without further ado, here are some key concepts from the NIH FY12 Appropriation report:
NIH has requested $31.987B for FY12. In the cover letter for the report from the Office of the Budget, Francis Collins states: “The requested funding will enhance NIH’s ability to support research that prolongs life, reduces disability, and strengthens the economy. NIH-funded research contributes to economic growth, produces well-paying jobs, and helps to keep the United States competitive on the global stage.” He continues: “For the FY 2012 budget request, NIH has identified one major area of extraordinary opportunity and three other themes that are exceptionally ripe for investment and integral to improving the health of the American people.” The one major area of opportunity of course is the proposed highly-controversial National Center For Advancing Translational Science (NCAT), which Collins refers to as “a new paradigm for turning lab discoveries into cures and treatments through targeted investments in translational science and medicine.” The three themes that NIH has deemed “instrumental in paving the way for more rapid scientific advances across all areas of human health and disease, including global applications”:
1) Technologies to Accelerate Discovery. This area focuses on genes and the environment (I guess we will see more of those FOAs), and directly lists advanced technologies such as DNA sequencing, microarray technology, nanotechnology, new imaging modalities, and computational biology.
2) Enhancing the Evidence Base For Health Care Decisions. Language here includes “comparative effectiveness” and “personalized medicine.” He also cites the new HMO Research Network, which “will bring together HMOs caring for more than13 million patients for the purpose of accelerating research in the high priority areas of epidemiological studies, clinical trials, and electronic-health-record-enabled health care delivery.”
3) New Investigators, New Ideas. Here Collins mentions two programs: “the NIH Director’s New Innovator Award, which supports new investigators with potentially high-impact projects, and the Early Independence Award, which enables our most talented young scientists to move directly from a doctoral degree to an independent research career.”
If you write NIH grants, I strongly encourage you to spend some time with the full Appropriations report put out by the NIH Office of the Budget. (click here)
The Administration requested $31.829B for NIH FY12. Here are highlights that the Administration pulled from the NIH report (and therefore deem important):
*The FY12 budget proposes to support a total of 9,158 competing Research Project Grants (RPGs), a reduction of 228 from FY10. In total, NIH projects it will support 36,582 RPGs (competing and non-competing) in FY12, an increase of 43 grants from 2010.
*The budget also proposes a 4 percent increase in stipends under the Ruth L. Kirschstein National Research Service Award (NRSA) program. The goal is to “improve NIH’s ability to attract high-quality research investigators to the field of biomedical research.” This will result in an increase in NRSA funding of $19 million over FY10, for a total of $794 million.
*The Cures Acceleration Network would receive $100 million in FY12; it is included in the budget of the Office of the Director.
*As in previous years, $300 million is transferred out of the budget of the National Institute of Allergy and Infectious Diseases (NIAID) for the Global Fund to Fight HIV/AIDS, Malaria, and TB.
*Although the budget narrative specifically mentions implementation of the National Center for Advancing Translational Sciences (NCATS), the National Center for Research Resources (NCRR) remains a line item in the FY12 budget.
*At a briefing of NIH advocates, NIH Director Francis Collins said that within the next month, the agency expects to file a budget amendment detailing the movement of NCRR programs into NCATS or other NIH institutes and centers.
*The National Children’s Study would receive $194 million, the same level as FY10; the Common Fund would receive $557 million, an increase of $13 million.
*NIH intramural research would increase by $50 million, to a total of $3.382 billion, which is approximately a 1.4 percent increase.
*NIH estimates it will be able to save more than $15 million in administrative costs in FY12. The agency plans to do so through such means as using technology to save study section travel costs by holding virtual peer review sessions.
To illustrate the achievements of NIH, Dr. Collins used two particularly compelling examples at the budget briefing:
*A 21-year-old diagnosed today with HIV/AIDS has a life expectancy of 70 years, thanks to the anti-retroviral therapy made possible by NIH funding.
*Gains in life expectancy supported by NIH-funded research result in $3.2 trillion in annual savings.
NIH identified in its FY12 budget justification priority areas and initiatives related to the following diseases: autism; cancer; Alzheimer’s disease; type 1 diabetes; and HIV/AIDS.
Some scientific program areas of accomplishment or special emphasis provided by NIH include: bioinformatics and computational biology; National Technology Center for Networks and Pathways programs; epigenomics; genotype-tissue expression; global health; Gulf oil spill long-term follow-up; health economics; high-risk, high-reward investigator initiated research; the HMO research collaboratory; the human microbe project; and nanomedicine.