Whether they investigate how medicines work, study the life cycle

of bacteria or crunch numbers in a computer, scientists across the

globe are united by a compelling desire: to better understand how life works.

Many of these basic biomedical scientists are funded by the National Institute

of General Medical Sciences (NIGMS) at the National Institutes of Health.

Rather than focusing on a specific disease, these researchers seek to answer

important biological questions like how cells talk to each other, how biological machines fold into their active shapes and how genes are regulated.

These studies may not have an immediate impact on our health. Yet such

“untargeted” research often leads to new medicines, technologies and

research tools. Examples of advances that grew out of basic research include:

• Countless drugs to treat diseases ranging from cancer to AIDS;

• Magnetic resonance imaging (MRI), which provides clear

pictures of the body’s organs and tissues; and

• The polymerase chain reaction, a laboratory technique that

is the basis of “DNA fingerprinting,” which revolutionized

criminal forensics.

Curiosity

Creates Cures

The Value

and Impact of

Basic Research

U.S. DEPARTMENT OF

HEALTH AND HUMAN SERVICES

National Institutes of Health

National Institute of General Medical Sciences

“Even if it brings no

immediate benefits,

scientific research

that advances the

frontiers of knowledge is necessary

and should be

supported.”

— 80 percent of

respondents to a 2003

poll conducted by

Research!America
             

                           

                       

                   

                     

                         

                   

                         

               

                   

                       

                         

                   

                       

                     

                       

                     

                     

                       

                           

                   

                         

                         

             

                 

                     

                       

                             

                     

                     

                   

                     

                       

                   

                           

   

     

       

   

   

   

r sea ch

Re

ic

as B no

st hgilt opS

UNTANGLING ALZHEIMER’S. The leading form of dementia in older people,

Alzheimer’s disease is caused in part when a protein, called tau, loses its normal

shape. Tau forms knotty tangles that skew communication between nerve cells in

the brain. A basic researcher studying cell division identified an enzyme, dubbed

Pin1, that restores tau’s normal shape in laboratory experiments. The discovery

helps researchers in the quest for new therapies to treat Alzheimer’s disease and

other neurodegenerative conditions.

CURIOSITY YIELDS CANCER DRUG. A scientist wondered why the body sometimes

destroys its own proteins and why muscles waste away when they’re not used. His

research group discovered the culprit: cellular garbage­disposal­like structures called

proteasomes. While creating compounds to clog proteasomes, the scientists noticed

that one of their substances had anticancer properties. Under the name Velcade®

, this

drug is now used to treat multiple myeloma, the second most common blood cancer.

DESIGNER DRUGS FOR BLOOD CLOTS. While studying the structures of complex

sugars, scientists developed prototypes of new drugs to help control blood clots,

which can cause heart attacks and strokes, during surgery. Using “molecular scissors,”

the scientists tailored heparin, a commonly used blood thinner, into a much more

potent form. They also developed a molecular tool that can selectively deactivate

heparin after surgery, enabling doctors to control bleeding with minimal side effects.

“Research is formalized

curiosity. It is poking

and prying with a

purpose.”

— Zora Neale Hurston

American author,

folklorist, playwright

and anthropologist

GOT YOUR FLU SHOT? WILL IT WORK? To create effective flu vaccines, scientists

must predict months in advance which strains of influenza are going to be most

troublesome. A multidisciplinary research team used a computer­based approach to

analyze a database containing DNA sequences of 560 flu viruses from 16 flu seasons.

The team discovered patterns of genetic changes in the viruses that could help create

more effective vaccines, saving many lives each year.

WHAT MAKES ANTHRAX SO DEADLY? Scientists recently revealed an answer by

determining the structure of an anthrax protein. The protein, called edema factor,

causes potentially fatal swelling and fluid buildup in the body. Its structure revealed

that when it binds to another molecule in the body, edema factor changes shape to

create a deep pocket in which deadly chemical reactions occur. This discovery—based

on basic research into cell communication—may enable researchers to design drugs

that clog the protein pocket and help disarm the lethal microbe.

HANG ON, BABY! Scientists studying carbohydrates on the surfaces of cells learned

that these molecules are involved in the implantation of embryos in a woman’s

uterus. The research holds promise for understanding and treating infertility, because

the failure of an embryo to implant properly is a common reason for problems in

early pregnancy.
This famous quote is attributed to Mary Lasker, a

philanthropist and advocate for medical research.

Her words acknowledge that by providing a solid

understanding of the cellular and molecular changes

that cause disease, basic research may help prevent disease or cure it in its

early stages, yielding tremendous savings of both money and misery. The

quote is clearly borne out by the numbers. Regardless of what measure is

used—reduced health care costs or increased productivity due to longer

life and decreased illness—studies show returns on investment ranging

from $10 to more than $80 for every dollar spent on basic research. Not

surprisingly, the longer a medical advance is available, the greater the

benefits. Basic biomedical research also benefits the economy in more

direct ways. Many nonbiomedical industries have been either created or

enhanced by biomedical discoveries. Together, the following industrial

applications contribute tens of billions of dollars to the U.S. economy

every year:

• Freeze­drying, which was developed to concentrate and preserve

laboratory samples, is now widely used in the food industry.

• Basic studies of digestive enzymes led to improvements including

meat tenderizers; bread dough conditioners; milk coagulants for

cheese production; stain­removal additives in laundry detergent;

and preservatives for beer, wine and juice.

• Fundamental research on the role of immune factors in controlling herpes led to a vaccine for a deadly disease in chickens.

“Basic research...pays

off in terms of key discoveries almost twice

as handsomely as

other types of research

and development

combined.”

— a major conclusion

of a landmark study that

revealed that the most

important advances in

cardiovascular medicine

grew out of the work of

basic scientists.*

*Comroe JH, Dripps RD.

Scientific basis for support of

biomedical science. Science 1976;

192:105­11.

Off

ys Pa

ch

ar

se

e R

ic

saB

“If you think research is   

expensive, try disease.”
             

               

       

           

           

               

                     

           

                 

   

                 

                     

                 

                     

                 

                   

                   

                   

                   

                     

                 

                     

               

             

                   

                       

                     

               

     

         

       

           

       

   

     

? e Res arch

od Go

es ka M

t ah W

In a familiar science fiction scenario, a scientist

knocks over a test tube and creates a medicine—

or weapon—with incredible power. Although a

number of important advances have occurred by

chance, most scientific advances are not accidental. Neither are they made by lone scientists.

“It is hard to predict

how science is going

to turn out, and if it

is really good science,

it is impossible

to predict.”

— Lewis Thomas

late biologist and writer

Advances result from years of intensive labor by teams of researchers that

include many young scientists in training. Increasingly, interdisciplinary

groups of scientists are working together to tackle problems that transcend

their individual expertise.

In many cases, basic research has unexpected applications. For example,

studies on how viruses infect bacteria led to the discovery of restriction

enzymes, which are a cornerstone of the biotechnology industry. Research

on how electric fields affect bacteria led to an important cancer medication, cisplatin. And curiosity about substances that break down bacterial

walls led to the identification of penicillin as an antibiotic drug.

Basic research often relies on studies in “model organisms” such as mice,

fruit flies or bacteria. Because human cells contain the same molecular

building blocks and pathways as those of most other living things,

researchers can learn much about the way our cells work by studying

these simpler organisms. These creatures allow scientists to design and

control their experiments tightly and to select the type of organism best

suited for examining a specific problem or process.

Ingredients of good research include stimulating scientific collaboration,

adequate funds and the ability to shift direction to pursue promising

leads. Sometimes, all it takes is having the right scientists in the right

place at the right time. Whether it comes as blockbuster discoveries or

incremental advances, history shows that over time, untargeted basic

research yields inestimable rewards.
NAME NOBEL PRIZE OFFICIAL CITATION

Ei­ichi Negishi Chemistry 2010 For palladium­catalyzed cross

couplings in organic synthesis

Venkatraman

Ramakrishnan,

Thomas A. Steitz

& Ada E. Yonath

Chemistry 2009 For studies of the structure

and function of the ribosome

Elizabeth H.

Blackburn,

Carol W. Greider

& Jack W. Szostak

Physiology or

Medicine 2009

For the discovery of how

chromosomes are protected

by telomeres and the enzyme

telomerase

Osamu Shimomura,

Martin Chalfie

& Roger Y. Tsien

Chemistry 2008 For the discovery and development of the green fluorescent

protein, GFP

Mario R. Capecchi

& Oliver Smithies

Physiology or

Medicine 2007

For the discovery of principles

for introducing specific gene

modifications in mice by the

use of embryonic stem cells

Roger D. Kornberg Chemistry 2006 For his studies of the molecular

basis of eukaryotic transcription

Andrew Z. Fire

& Craig C. Mello

Physiology or

Medicine 2006

For the discovery of RNA

interference—gene silencing

by double­stranded RNA

Avram Hershko

& Irwin Rose

Chemistry 2004 For the discovery of ubiquitinmediated protein degradation

[a vital cellular pathway]

The Nobel Prizes, the highest honors bestowed in

science, are frequently awarded to basic researchers.

As instructed in Alfred Nobel’s will, the prizes recognize those who, in specific fields, “have conferred

the greatest benefit on mankind.”

n a i m nk d

Hu

to

s it

ef

ne B

t aer G

“The pursuit of

curiosity about the

basic facts of nature

has proven, with

few exceptions

throughout the

history of medical

science, to be the

route by which the

successful drugs

and devices of

modern medicine

were discovered.”

— Arthur Kornberg

1959 Nobel Laureate in

physiology or medicine

To learn more about the basic research and training that NIGMS supports and the

opportunities for funding it offers, go to http://www.nigms.nih.gov

Recent Nobel Laureates Supported by NIGMS

For a complete list, go to http://www.nigms.nih.gov/GMNobelists.htm
             

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For photo credits and figure legends, go to

http://publications.nigms.nih.gov/curiosity/photo credits.html

For descriptions of other free science education publications and an order form, go to

http://publications.nigms.nih.gov/order

This publication can be made available in formats that are more accessible to people with

disabilities. To request this material in a different format or to order additional copies, contact

the NIGMS Office of Communications and Public Liaison at 301 496 7301, send e mail to

info@nigms.nih.gov or write to the office at the following address: 45 Center Drive MSC 6200,

Bethesda, MD 20892 6200. If you have questions about this publication, you can use the same

contact information to reach the writer, Alisa Zapp Machalek.

NIH Publication No. 05 5493

Revised November 2010

http://publications.nigms.nih.gov/curiosity

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