“If you look at statistics, there is compelling evidence that medical research is leading to longer lives with NIH biomedical research spearheading this increase in life span”, said Francis S. Collins, MD, PhD, Director, of NIH speaking at a luncheon at the National Press Club on February 26th.
He continued to say that the Recovery Act with $10 billion for NIH provided much needed funds and yet it was a great challenge at NIH to figure out ways to use that amount of funding. Fortunately, the staff at NIH was able to come up with new ideas for proposals and issued a number of RFIs. Then the staff brought together 15,000 reviewers to look at all of the grant applications and after a second round of reviews, decisions were made on the projects to receive grant funding. At that time, NIH funded some 13,000 grants, 2,000 were awarded to individuals and to some small businesses that had never before been awarded a grant from NIH.
Dr. Collins discussed the recent announcement made by NIH on February 25th on what is called the “NIH Common Fund. The half a billion dollars in funds authorized for the program are part of the NIH budget with $17.8 million in FY 2010 and additional funds to be distributed in future years. The funding is to be used for projects that none of the 27 institutes and centers would be likely to fund on their own. The plan is to support scientific opportunities that do not fit easily into disease categories.
Dr. Collins mentioned several themes that cut across diseases and basic and applied science. The first theme is to develop high throughput technologies by developing bold technologies to examine how cells, organisms actually do what they do, what goes wrong, and how disease actually occurs. Also included is the need for new imaging approaches and approaches that involve very small scale efforts such as nanotechnology along with computational biology.
Another theme is to provide a scientific evidence base for decisions that may be needed to know what works and what doesn’t. This is referred to as Comparative Effectiveness Research (CER) and is a strong area of interest for NIH. It is now a high priority to pick out areas where that information is needed and to be able to provide this information to anyone that needs to make healthcare decisions.
Dr. Collins talked about a controlled trial at NIH that has been ongoing to try to assess what is the right treatment for somebody who has narrowing of the carotid artery in the neck and is therefore at risk for stroke. The standard treatment has been to use surgery called carotid endarterectomy. But recently, stents have been used and applied to the carotid artery. NIH sponsored a large controlled trial randomizing people to one treatment or the other. The result is that the success rate for treatment is about the same but stents are much less invasive.
Dr. Collins emphasized how important it is to be involved in global health because right now we’re learning about pathogens that cause diseases in low income countries. This means that we are now going to be able to develop new strategies, new drugs, new vaccines, and new diagnostics. NIH already invests $600 million a year for Global Health but he would like to see that amount increase and build some research capacity in places like sub Saharan Africa.
Another area getting attention these days is translational medicine which brings basic science discoveries into the clinic. According to Dr. Collins, it is fascinating to read about the studies to discover plasticity among cell types which could mean that we could actually take one of our skin cells and convince it to become a neuron.
As he explained, many steps would have to be taken before this could become a reality because of safety concerns. On February 25th, NIH announced that substantial new resources would be put into the creation of a Center for Induced Pluripotent Stem (IPS) cell research so that this type of research can proceed and be moved into a translational opportunity in its earliest phase.
In order to achieve success especially in treating rare diseases, you have to first know your target and then study the disease well enough to correct a cell that has gone wrong. Dr. Collins said “NIH has make this possible for academic investigators to do that kind of screening and now the investigators can come up with results in a matter of two or three days which can produce some promising starting points to further develop drugs to use for treatments.
In the last five years, NIH investigators have found 128 such compounds directed against more than several dozen targets. Next, they will want to take these promising compounds and go to FDA and ask for permission to give them to patients in a clinical trial. After the clinical trials, the compounds will need to be approved by FDA. This is a long process which can take a decade or more and is very expensive.
Dr. Collins reported that a few days ago an announcement was made that there will be a tigher relationship between FDA and NIH. A Leadership Council will be formed to focus on ways to streamline the process for evaluating new possible therapeutics and get a handle on a better way to review protocols for rare diseases or applications.
Dr. Collins stressed that although a great deal of scientific research is moving forward, we are not doing a good job of cultivating the next generation of scientists. The statistics are dismal with high school students 15 years old ranking 29th in science achievements as compared to students from 57 other countries. Almost half of 12th graders score below basic scores in science. The workforce desperately needs people with skills in science and math.
Dr. Collings is going to spend a day in a D.C. public school during national lab week with high school biology students and share the wonder of what it means to be a scientist and to be able to make vital discoveries for the first time. He wants to see the scientists at NIH and the tens of thousands of NIH funded researchers do the same during that week.