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From the Director: Stem Cell Research: Unlocking the Mystery of Disease

Dr. Zerhouni

Photo courtesy of NIH

When he testified earlier this year before the U.S. Senate Appropriations Subcommittee on Labor, Health and Human Services, and Education, Dr. Elias Zerhouni, NIH Director, described the need for expanding stem cell research. Recently, he spoke about stem cell research with MLP coordinator Christopher Klose.

Klose: You have said the greatest risk to scientific progress is to stop taking risks; that risk-taking is the essence of science. Since becoming Director of NIH, you have consistently supported embryonic and adult stem cell research to develop cell-based therapies for heart disease, stroke, and countless other devastating conditions. What's been the progress?

Dr. Zerhouni: Biomedical research over the past 30 years has markedly reduced the mortality due to heart disease and stroke, for instance. But understanding the software of DNA, of how things play out, is the challenge of 21st century biomedical research. That is why NIH scientists are working hard to understand how an infant cell—a childhood neuron—can create new circuits and regenerate them, whereas an adult neuron is much less capable

Dr. Zerhouni

Photo courtesy of NIH

Klose: How difficult is the challenge?

Dr. Zerhouni: It is extraordinarily complex. Picture the embryonic cell (and its DNA) as a very complex keyboard, with thousands of keys. With the human genome, we've identified the size of the keyboard, the number of keys, and the notes each plays. What we haven't figured out is how they end up playing Bach and Beethoven, so to speak—how becoming a neuron is one symphony, a skin cell another.

There are billions of cells, all with their own music. As the brain develops, it turns out they're pretty harmonious—an orchestra of keyboards. So signaling among them becomes important. But who is the conductor? How is an adult cell that only plays neuron music conducted, why does it stop, and how can you make it play heart or skin music? And for the embryonic stem cell—a keyboard that has never played—how is it directed to play neuron music and not muscle or liver music, even though all of our cells contain the same instruction books?

This is what stem cell science is all about.

Klose: All in search of a conductor?

Dr. Zerhouni: Yes. We've made great progress, but people still suffer. With stroke, as with all conditions, we must be able to predict them earlier so we can act at the personal level—through changes in lifestyle, diet, exercise, etc.—to preempt it from striking, if possible. Fundamentally, however, we need to understand how things are organized.

Therefore, all avenues of research need to be pursued.

"Good science is good ethics."

Klose: Despite the controversy over stem cells?

Dr. Zerhouni: Yes. Unfortunately, the scientific foundation of stem cell research is sometimes lost in the societal, moral, and ethical battle between hype and hope. But our job at NIH is to push the science forward to serve our patients.

Klose: When can we expect results?

Dr. Zerhouni: The research is advancing at an incredible pace, but as we move forward we're finding more complexity. When I became director of the NIH we only knew of one gene that had anything to do with diabetes. Then, last June, we discovered 10 more that are clearly associated; we must accelerate our research into how these notes play.

Klose: And that's the issue of supporting basic research, isn't it?

Dr. Zerhouni: That's right. We must continue the research at all levels, or there will be no progress. We need to understand these fundamental facts of life: how and why the young brain adapts, why the old does not. If we can know how a disease occurs, we can prevent it.

So I think it's a multi-pronged attack, both from the point of basic understanding and continually improving what we do. Finally, it's important to emphasize that science evolves with strong ethics.

Good science is good ethics.

Summer 2007 Issue: Volume 2 Number 3 Pages 2 - 3