>From Wired:
http://www.wired.com/news/technology/medtech/0,72206-0.html?tw=rss.politics

NIH Funds Are for Research

By Brandon Keim| Also by this reporter
02:00 AM Dec, 11, 2006

A favorite argument as to why the federal government should not fund embryonic 
stem cell research is that the science is unproven. It has not led to any cures 
or FDA-approved treatments.

That happens to be true. But that doesn't make it a good argument. In fact, 
most of the science funded by the federal government is not successful yet, 
since proven science doesn't usually need funding.

Scientists say people who argue against funding unproven stem-cell research 
miss the point. Science takes time. Almost every major advance in health care 
took decades of research -- often using millions in federal funding -- before 
being declared safe and effective in humans. Years are spent on research that 
is, by definition, unproven, if not far-fetched and hypothetical.

Addressing an audience at the conservative Heritage Foundation in 2005, biotech 
consultant and cellular pharmacologist Kelly Hollowell said embryonic stem 
cells were a medical bust and deserved no federal research funding.

"There are no human trials -- despite all the hype of media," she said. "After 
20 years of research, embryonic stem cells haven't been used to treat people 
because the cells are unproven and unsafe."

But what if the government had adopted that attitude when it came to the cancer 
drug paclitaxel? In 1970, researchers at the National Cancer Institute, part of 
the National Institutes of Health (the country's clearinghouse for medical 
research funding) discovered the compound. The NCI spent $700 million 
developing Taxol (paclitaxel's brand name), and clinical trials dragged on 
through the 1980s before the drug was approved in 1992. It has since saved 
hundreds of thousands of lives.

Also in the 1980s, NIH scientists spent hundreds of millions of dollars 
developing unproven vaccines for rotavirus, which kills half a million children 
every year, and human papilloma virus, which causes cervical cancer and 
annually kills more than 250,000 women. Commercial versions of both vaccines 
only appeared in 2006.

"It's a mistake not to fund the long-term research," said Elisa Eiseman, a 
senior scientist at the nonprofit RAND Corporation. "It's that blue-sky, 
high-risk research that yields very amazing discoveries."

Private-sector scientists tend to focus on quick payoffs, so it's up to the NIH 
to support research that may take decades to yield results. And while many 
scientists say too much NIH money goes to safe, short-term research, there's 
still enough left over for the cutting edge. Much experimental work involves 
making new tools for inspecting living bodies at the cellular level, where 
processes remain mysterious.

Below is a summary of promising science that, like stem cell research, is 
utterly unproven. The difference is the federal government is spending hundreds 
of millions of dollars to find out if one day it might ease pain or bring cures 
to suffering patients.
Proteomics

The 10-year, $600 million Protein Structure Initiative is another so-called 
high risk project. Scientists have identified the structures of more than a 
thousand proteins whose functions are not yet understood. With luck, a few 
might end up signaling the presence of a disease before it emerges, as with a 
telltale Alzheimer's compound found this year by National Heart, Lung and Blood 
Institute researchers. Of course, they might not -- but scientists say the only 
way to find out is to try.

National Cancer Institute researchers also used an artificial intelligence 
program to analyze the protein patterns of finger-prick blood samples for early 
signs of ovarian cancer -- an endeavor that private companies wouldn't likely 
have the luxury of pursuing. Commercial scientists typically concentrate on 
leads provided by government-funded scientists, said Ken Dill, a University of 
California, San Francisco, biophysicist.

"It's academics who explore the biology," Dill said. Experimental research into 
the human genome is another heavily-funded NIH field. Scientists now study gene 
expression at levels of complexity hardly imagined a decade ago.

"The paradigm for the last 20 or 30 years has been to choose one particular 
protein or one gene and follow it," said Alan Schechter, chief of molecular 
medicine at the National Institute of Diabetes & Digestive & Kidney diseases. 
"Now we appreciate that these processes are the results of interactions of 
dozens or hundreds of proteins and genes. One's guess is that, ultimately, 
these kinds of approaches will give us a new level of thinking about biological 
and medical processes. But, right now, the methods are controversial."
Gene Therapy

Another $200 million in NIH funding goes to the unproven but promising field of 
gene therapy. The long-anticipated technique has progressed slowly for more 
than 20 years, and could take decades more to become common. But gene therapy 
recently started showing potential. "It was in the same place that embryonic 
stem cells are now," said Eiseman. "It was hypothetical, pie-in-the-sky. But 
many trials are coming to fruition."

NIH-funded scientists have used gene therapy to treat serious diseases and 
disabilities in animals, and in August reported success using gene therapy to 
treat two people with cancer. Research on humans slowed after the death of 
Jesse Gelsinger, and remains shadowed by serious safety concerns, but early 
clinical trials are ongoing.
Next-generation Imaging

"Standard imaging isn't good enough to see microscopic detail in the human 
body," said Alan McLaughlin, director of applied science at the National 
Institute of Biomedical Imaging and BioEngineering, which spent $2.6 million 
this year on next-generation imagers. "We'd like to look at the chemical 
information in a tumor or special kind of cell, such as beta cells in the 
pancreas," McClaughlin said. "(They) are important to diabetes, but only 
present in the islets of Langerhans, which are about 100 microns wide. We can't 
look at that resolution now."
Nanotechnology

The NIH also spends nearly $200 million annually on nanotechnology and 
nanomedicine, which involves the atom-scale design of molecules that might 
someday repair or deliver drugs into cells. Again, no one knows if it will work.

Whether many of these advances will turn into cures or treatments remains to be 
seen. But scientists say that history counsels patience.

"With these kinds of approaches, one has to have the perspective that practical 
applications are likely to take decades," said Schechter. "The short-term 
results of new technologies are generally much less than people expect, but the 
long-term effects are greater."

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