How Does Basic Research Improve Human Health

Basic research leads to new knowledge. It provides scientific capital. It creates the fund from which the practical applications of knowledge must be drawn. New products and new processes do not appear full-grown. They are founded on new principles and new conceptions, which in turn are painstakingly developed by research in the purest realms of science.

- Vannevar Bush, Director of the Office of Scientific Research and Development,in a report to Pres. Franklin D. Roosevelt, July 1945.

Why is basic research important?

Basic research is the study of the fundamental molecular mechanisms that drive life. It covers every aspect of cellular function, from the way cells process nutrients, grow, and divide, to the way they die. Basic researchers use human cells and animal models to ask questions like: How do cells communicate with one another and their environments? How do they respond to stress? What role do chemical changes to protein structure play in these processes?

The answers to these questions help scientists establish a baseline for what's normal. Then, they can begin asking questions about what happens when genetic or environmental changes perturb a cell and alter its behavior.

It's difficult to know where breakthroughs will come from. Progress in one research area provides data for other areas. So basic scientists share their findings with other scientists by publishing papers in scientific journals. Their peers—and future generations—build upon these findings.

At Sanford Burnham Prebys Medical Discovery Institute, Dr. Gregg Duester is proud to be a basic researcher. Researchers in his lab study retinoic acid, an active form of vitamin A. They want to know how retinoic acid tells the right body parts to form in the right places at the right time in a developing embryo. To figure out retinoic acid's role, they compare mice with and without the ability to convert vitamin A to retinoic acid. In a paper published in the scientific journal PLoS Biology, Dr. Duester and his team made an important discovery about the part of the developing brain that makes retinoic acid and its impact on the production of inhibitory neurons—the type of neuron that keeps functions like memory and learning in check.

Using this knowledge, Dr. Duester's team developed a retinoic acid recipe that, when added to stem cells, generates the most common type of inhibitory brain cells.

In Dr. Duester's own words, "To us, this is a basic science story and that's what's most important. We just want to know how things normally work during development. But what we found here suggests that others could use retinoic acid to make inhibitory neurons to alleviate symptoms in Huntington's disease, autism, schizophrenia, epilepsy and bipolar disorder—diseases that are hard to treat, but are believed to be caused by a loss of inhibitory neuron function—just the way an embryo does it naturally."

How do basic findings translate into clinical treatments?

The Translational Research Institute for Metabolism and Diabetes define translational research as scientific discoveries translated into practical applications that improve human health. These discoveries typically begin with basic research at "the bench," then progress to the clinical level—the patient's "bedside."

The "bench-to-bedside" approach goes both ways. Basic scientists provide physicians with new patient care tools—diagnostics, prevention methods, and treatments—to assess through clinical trials. In turn, clinical researchers make new observations about the nature and progression of disease that often stimulates new questions in the lab, driving basic science in new directions.

Dr. John Reed, is an expert on a basic cellular process called apoptosis, a programmed mechanism for cellular suicide. He's spent most of his career defining this pathway, publishing nearly 800 scientific papers that explore the various proteins involved, their interactions, and how the process goes awry in cancer, allowing cells to continue growing and dividing beyond their normal lifespan.

But Dr. Reed and his team also hope to "translate" these discoveries into new cancer treatments. One apoptosis protein in particular has captured Dr. Reed's attention for decades: Bcl-2. Bcl-2 prevents apoptosis in healthy cells, so it makes sense that neutralizing it would encourage cancer cells to kill themselves. To this end, Dr. Reed and his colleagues created the first DNA-based cancer drug (Genasense™), which targets Bcl-2. In Phase II clinical trials (sponsored by Sanof-Aventis), Genasense demonstrates promising patient survival benefit.

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