NIGMS -- Medicines for YOU

Medicines for YOU

National Institutes of Health 

The National Institutes of Health aims to improve 

the health of all Americans through medical 

research that solves mysteries about how the 

human body normally works—and how and why it 

doesn’t work, when disease occurs. One goal of 

this research is to help improve the good effects of 

medicines while preventing bad reactions.

Medicines for You 

Your lifestyle, the food you eat, and where you live 

and work can all affect how you respond to 

medicines. But another key factor is your DNA, 

which contains your genes. Scientists are trying to 

figure out how the make-up of your DNA can 

contribute to the way you respond to medicines, 

including pain-killers with codeine like Tylenol®#3, 

antidepressants like Prozac®, and many blood 

pressure and asthma medicines. Scientific 

discoveries made through this research will 

provide information to guide doctors in prescribing 

the right amount of the right medicine for you. 

Did You Know? 

● 

According to a recent study, 2 

million people were hospitalized in 

one year alone for reactions to 

properly prescribed medicines. 

 ...and 100,00 of these people died 

 ... making this the fourth to 

sixth leading cause of death in 

the 

 United States.

● A normally safe dose of a certain 

cancer treatment can kill a child 

who has an unusual gene type.

● In some people, normally effective 

amounts of pain relievers such as 

codeine offer no pain relief and can 

produce uncomfortable or even lifethreatening side effects.

● Certain allergy medicines work well 

foe some people but not for others.

● The National Institutes of Health is 

launching a research effort to 

understand why people can leave 

such different reactions to 

medicines.

One Size Doesn’t Fit All

In their journey through your body, medicines interact with many different molecules called 

proteins. Some of these proteins work to get rid of medicines, while others help medicines do 

their jobs. Some proteins actually “turn on” certain medicines, by switching them from an 

inactive form to an active one. For example, the painkiller codeine (in Tylenol#3) is a medicine 

that is “switched on” in this way by proteins. Your genes provide the manufacturing 

instructions for all of the proteins in your body. Small differences between your genes and 

those of your relative or neighbor can affect how you react—or don’t react—to a medicine. 

Partly because of these genetic differences, when it comes to medicines, one size clearly does 

not fit all.

What’s the Payoff? 

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NIGMS -- Medicines for YOU

Uncovering differences in people’s genetic make-up will help doctors prescribe the right 

medicine in the right amount for each person, making today’s medicines more effective for 

everyone. The payoff will be preventing unnecessary illness and saving lives lost from the 

“one-size-fits-all” medicine dosing that is common today. A bonus of this type of research will 

be an increased understanding of the genes that cause or contribute to diseases such as 

cancer, heart disease, diabetes, depression, and asthma. This research will also help 

scientists design new strategies for making the most effective and safe future medicines. 

A Library of Knowledge

In most cases, research to find normal variations in the genes for the proteins that handle 

medicines in the body will involve a simple test. In many cases, researchers will rub the inside 

of a volunteer’s cheek with a cotton swab and then examine the DNA in those cheek cells. 

There are no health risks associated with this type of test. Some studies may involve asking 

people to take certain medicines. People who volunteer to do so will be fully informed of any 

potential risks or benefits. After identifying the DNA in samples from study participants, 

scientists will store this information in a library of knowledge that will be shared by other 

researchers who study how people react differently to medicines. To protect participants’ 

privacy, names and other identifying information will not be stored in this library. 

Questions and Answers

Aren’t prescribed medicines already safe and effective? 

For the most part, yes. But medicines are not “one-size-fits-all.” While typical doses work pretty well 

for most people, some medicines don’t work at all in certain people or the medicines can cause 

annoying, or even life-threatening, side effects. 

Why would a friend or relative react differently than I do to the same dose or type of medicine?

As medicines move through the body, they interact with thousands of molecules called proteins. 

Because each person is genetically unique, we all have tiny differences in these proteins, which can 

affect the way medicines do their jobs.

Who’s paying for this research?

The National Institutes of Health is providing money to scientists at universities and medical centers 

who come up with the best plans for carrying out research on how people respond differently to 

medicines. 

Why should my tax dollars be spent on medical research that is not directly related to specific 

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NIGMS -- Medicines for YOU

diseases?

Curing and preventing disease is the National Institutes of Health’s highest priority. Research on how 

people respond differently to medicines will make current and future treatments for diseases such as 

asthma, diabetes, heart disease, depression, and cancer safer and more effective. A bonus of this 

type of research will be a better understanding of the role many different genes play in causing or 

contributing to these and other diseases. 

Who will participate in this research? 

National Institutes of Health-funded scientists at universities and medical centers across the country 

will recruit volunteers from a wide variety of groups. Research of this type relies upon studying many 

different people with a broad range of genetic make-ups to find the small, but normal, genetic 

differences among them.

Will participating in this kind of study have any effects on my health? 

Most of these research studies will involve simply rubbing the inside of a volunteer’s cheek with a 

cotton swab. Scientists will then pull out DNA from inside the cheek cells they have collected. There 

are no health risks associated with this type of test. 

What will happen to my DNA sample? 

Scientists will only use your DNA sample for the research that they explain to you before you agree to 

participate. Researchers will store the DNA information provided by your sample in a library of 

knowledge to be shared by scientists who do research in this field. 

Will the scientists keep my sample?

Only if you agree to this in advance. Researchers will not use or pass on your sample for other 

studies you don’t know about. 

What about my privacy?

The privacy of all people participating in this research will be protected. Scientists will not match your 

name or other information that could identify you with the genetic information that is recorded in the 

library of knowledge. 

I heard something about “pharmacogenetics” and “pharmacogenomics” on the news—are the 

research studies you’re talking about the same as these?

Yes. Scientists studying how genes affect the way people respond to medicines refer to this field of 

research as “pharmacogenetics” or “pharmacogenomics.” The goal of this type of research is to tailor 

doses of medicines to a person’s unique genetic make-up. 

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How soon can I expect to benefit from this research? 

The first benefits to patients could come as soon as a few years from now. From then on, the 

knowledge gained through this type of research is expected to help doctors tailor the medicines they 

prescribe to best suit each patient’s individual needs. 

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