What precision medicine is, and how it might save your life some day

January 30, 2015
Graduate student Katie Bates studies a slice of rodent Parkinsonian brain tissue slices in the Nanomedicine Lab at UCL's School of Pharmacy in London

A researcher examines a slide. REUTERS/Suzanne Plunkett

The term “precision medicine’’ is one that most Americans had likely never heard before President Barack Obama’s State of the Union address, but it is an area of exciting promise in biomedical research that will eventually improve health outcomes and potentially reduce healthcare costs.

The concept revolves around understanding the specific genetic changes that underlie such diseases as cancer, diabetes and cystic fibrosis. In general, patients with these types of diseases fall into different subgroups based on the genetic changes that can cause the diseases. Drugs tailored to address the changes are likely to be more effective. A good example is the cancer drug Gleevec, which inhibits an enzyme that is mutated in a subtype of leukemia and has had remarkable success with few side effects.

Scientists have been sequencing the DNA of many types of cancers to identify the specific patterns of mutations that can lead to disease and to try to find new drugs to treat them. While significant advances have been made recently in our understanding of these diseases, new investment is needed to turn discoveries made in the laboratory into drugs that can be tested in patients.

Some have argued that developing drugs for small groups of patients will increase the costs of drugs because developmental costs will be borne by fewer patients taking them. This is most likely not the case, for two reasons. First, skeptics need only look to the success of Gleevec to realize that these drugs are sometimes effective in subpopulations other than those for whom they were developed. Second, using precision medicine to target therapies to patients will greatly improve the odds that a drug will succeed in clinical trials and gain FDA approval; the failure rate and the associated costs of drug development will decline. Of course, there will be additional tests to determine the genetic profile of patients, but the costs of DNA sequencing have dropped dramatically in the past decade, making these types of tests much more affordable.

Others have argued that genomic information is less important than patient behavior in predicting and preventing disease. Precision medicine is not about predicting the probability that someone will develop a disease. It is the improved understanding of the underlying causes of a disease already present, and how best to treat and respond to the genetic changes.

Besides helping develop more effective drugs, precision medicine will help doctors prescribe the right drug for the right patient. Patients won’t get drugs that won’t work; they will be spared the wasted time and side effects that an ineffective drug might produce. Getting patients treated more quickly with the best drug for them based on their genetic profiles will result in better health outcomes, and overall treatment costs will fall.

Of course, challenges remain. Identifying the sets of biomarkers that will best predict which drug will work for which patient is essential and will require time and investment in the president’s Precision Medicine Initiative. And in diseases such as cancer, the tumors can mutate to develop resistance to targeted therapies, which is why combinations of drugs are often needed for the most advanced cancers.

Whether the president’s proposal will result in additional funding for the National Institutes of Health is unclear, but new investment in research is sorely needed. Since 2003, funding at the medical research agency has been essentially flat, with its overall budget purchasing power eroding by more than 10 percent. This despite the fact that the return on investment in overall economic growth spurred by agency spending is as high as 30 percent to 100 percent, and every dollar invested in research produces more than two dollars in new state business activity.

While the Precision Medicine Initiative is an exciting prospect, we should not be robbing Peter to pay Paul. We should increase investment in scientific biomedical research that can create new jobs, improve medical outcomes, reduce healthcare costs and save lives.

11 comments

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Re: Saving Lives.

Peace Saves Millions Lives.

Adapting to rapid accelerated climate change Will Save Billions of Lives.

Also if the corporate world would stop the practice of poisoning Our World food supply (chemicals and GM crops) and water (for their lust of greed) then investment into “genetic correction” wouldn’t be a necessity.

Poisoning the public – To cure them; is a big industry and a big political lobby.

God gave you The Model of the Theory of Everything to solve “All” that ails the World today.

With Faith, Hope and Charity.

Implement it Now!

For The Sake of Life Eternal; It is Written.

Peace be with you All.

Love
Omega

Posted by Lovetwo | Report as abusive

Cures don’t make money. Illness makes money. I doubt any hospital wants this.

Posted by brotherkenny4 | Report as abusive

Funding will probably be used for creating genetic weapon .

Posted by Macedonian | Report as abusive

Another example of how the Republican sequestration is damaging investment in our nation.

Posted by Flash1022 | Report as abusive

It is helpful that doctors can use DNA to “tailor” cures for diseases, but it does not address immune responses to “cures” that can be seen as enemy invaders by human immune systems. They should consider adding the possibility of identifying receptors that protect against hostile human immune responses to the tailored cures. It is a little more “tailoring” for more dangerous cases.

Posted by carlmartel | Report as abusive

We simply cannot afford to keep subsidizing Big Pharma like this for two reasons:

1) U.S. Healthcare costs, over the past 50 years, have spiraled out of control and out of our ability to pay the costs. And, since Big Pharma charges U.S. consumers many times more than it charges consumers from other countries, Big Pharma is a Big part of the problem.

2) Spending more hundreds of thousands of dollars to treat diseases is like mopping the floor without shutting off the tap. The diseases these drugs propose to treat are 75% caused by unhealthy lifestyles: obesity related to unhealthy diets and sedentary lifestyles.

Treating these chronic diseases already eats 80% of our healthcare dollars. We need to start preventing them rather than treating them. We can afford no other course.

But, Big Pharma will spend Big Bucks to convince American politicians to subsidize their industry…

Posted by GeorgeBMac | Report as abusive

First the research establishment advises the president to launch a brain initiative, now it’s precision medicine.

In a time of ever shrinking funding for research, earmarking federal budgets takes precedence. Particular interests seem to carve up the pie. The idea of the day trumps the most original and promising idea regardless of subject.

The competition for federal funding has become so visceral, because federal agencies do not only underwrite the cost of the means needed for the research but also the salaries for principal investigators, research assistants, research associates and graduate students and the host universities’ indirect cost for infrastructure and administration. The annual budget of the National Institutes of Health alone roughly equals the endowment of Harvard University, the country’s largest. Without federal government support our premier research universities would need to close

Read more here:
http://brainmindinst.blogspot.com/2008/1 0/lost-treasures-of-mind.html

Posted by PeterMelzer | Report as abusive

Hopefully this will help more people

Posted by KiarasBigFan | Report as abusive

The relatively sudden and rapidly increasing availability of computers and software the average person can afford (and utilize) over the last quarter of the 20th Century has quite literally changed everything. This change is greater in both effect and potential far beyond that of spoken language, written language or printed language.

Each of these is but a means of documenting thought and passing worthy concepts on to any and all interested to utilize as they may. The efficiency of the computer and the internet to analyze, store and share experience ans/or knowledge is unprecedented and without limit.

Just about anyone with the mind and the motivation can contribute to virtually any field of research, a fact that is fueling an explosion of understanding and shared knowledge heretofore unthinkable. That which is known within virtually every discipline, from the medical to the intellectual, is expanding at a rate approaching infinity

One visionary has suggested that the first human to live a thousand years may have already been born. The potential of humans is, however, a two-edged sword. For every Schweitzer there seem to emerge from the loins of mankind ten wannabe Hitlers. To reach any manifest destiny, humanity must first survive the next few decades.

Posted by OneOfTheSheep | Report as abusive

First the research establishment advises the president to launch a brain initiative, now it’s precision medicine.

In a time of ever shrinking funding for research, earmarking federal budgets takes precedence. Particular interests seem to carve up the pie. The idea of the day trumps the most original and promising idea regardless of subject.

The competition for federal funding has become so visceral, because federal agencies do not only underwrite the cost of the means needed for the research but also the salaries for principal investigators, research assistants, research associates and graduate students and the host universities’ indirect cost for infrastructure and administration. The annual budget of the National Institutes of Health alone roughly equals the endowment of Harvard University, the country’s largest. Without federal government support our premier research universities would need to close down.

PS: the lady in the picture looks at sections cut through a rat brain and stained for cell bodies. Without a microscope, she won’t be able to discover much.

Posted by PeterMelzer | Report as abusive

Cures will definitely generate a better world after implication of the effects of the Precision Medicine.

Posted by Parinda | Report as abusive