NIH director wants more clinical science research on conditions like diabetes, cancer, autism

From Reuters:

WASHINGTON -- As he puts together his budget proposal for the coming year, National Institutes of Health director Dr. Francis Collins plans to do something a little different -- emphasizing the "health" in the name of his U.S. agency.

A stronger focus on the potential immediate impacts of research will not only help justify the NIH's $30 billion budget but may energize the work of the largest funder of U.S. medical research, Collins says.

"It's tough with the economy, the deficit growing. The administration is feeling enormous pressure to rein in spending," Collins told Reuters in an interview.

"We have to be clear here -- this is not an effort to derail basic science," added Collins, himself an M.D./PhD whose life has been focused on genetic research.

Much of the work done at NIH is so-called basic science, looking at the function of cells and molecules and years away from ever benefiting patients.

But without this kind of work, new drugs would never be invented. What Collins wants to do is connect the dots between basic and clinical science a little sooner.

One example -- discoveries about mutations linked to conditions ranging from diabetes to prostate cancer.

"Those are drug targets. Most basic scientists don't think about it," Collins said. "If you've just discovered the molecular basis of a rare disease, you can turn it into an assay."

Having a science-friendly boss may help, said Collins, praising President Barack Obama's affinity for science.

"It is wonderful to work for a president who has a sense of what science can do," he said.

One of Obama's first acts as president was to loosen restrictions on federal funding for embryonic stem cell research. Collins said the big research hospital at NIH, called the clinical center, might host some of the trials treating patients with human embryonic stem cells.

"Wouldn't this be a very important place to push that agenda?" Collins asked. "Health delivery research seems like an area for NIH to get more involved in."

The day after he took over, Collins told reporters he would press Congress for more stable funding of the agency, which has a 2009 budget of $30.9 billion.

Flat funding in recent years translates to 17 percent less spending power since 2003, Collins said, leaving researchers demoralized and hurting the U.S. lead in medical research.

Collins said the drug pipeline is slowing down for a range of diseases as companies tweak existing drugs instead of making big new discoveries.

"There are lots of 'me-too' drugs," Collins said.

But a National Cancer Institute project to sequence all the mutated genes in tumors might show how to turn this trend.

This Cancer Genome Atlas network had its first payoff last week, with a study showing that the brain tumor glioblastoma multiforme is, genetically, in fact four different diseases.

Such genome-wide studies may help transform the understanding of disease. "I think we will re-classify cancer in major ways," Collins said.

"Out of this is going to come, I am sure, dozens of new drug targets," he added. "We can replace the carpet-bombing approach of chemotherapy with smart bombs."

Another area that will get more scrutiny is autism. Just last month the U.S. Centers for Disease Control and Prevention reported that one in every 110 U.S. 8-year-olds have autism.

"Clearly, we have an enormously frustrating mystery on our hands," Collins said. "We have to figure out what is going on here."

Collins, former director of the NIH's National Human Genome Research Institute, says genetics may answer many of these questions. The NIH's National Institute of Mental Health is mounting an effort to sequence the entire genomes of hundreds of children and their parents.

"By 2012, we should know a lot more about the heritable part of autism," he said.