The Promise of Precision Medicine

By Catherine
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By Catherine Bolgar

Medicine is moving away from a one-size-fits-all model.

Precision medicine, sometimes called personalized medicine, holds so much promise that the U.S., China, and France have announced massive investments in this field over the past year.

“Precision medicine, we contend, has the potential to result in systemic savings,” says Christopher J. Wells, communications director at the Personalized Medicine Coalition (PMC), a Washington nonprofit organization representing scientists, patients, providers and insurers. “Now, medicine is by trial and error. You try one treatment and if doesn’t work, you try another.

The power of precision medicine is that if you get it right the first time, everybody benefits.”

IV solution in a patient hand and IVS machinePrecision medicine has made the biggest strides in oncology, where time is of the essence and chemotherapy drugs have strong side effects. For example, some breast cancers may be resistant to treatments such as trastuzumab. But that chemotherapy drug is very effective for breast cancers caused by the HER2 mutation.

No two cancers are the same,” says Susanne Haga, associate professor of medicine at Duke University in Durham, N.C. “They may be the same with respect to their tissue origin. But each person has a unique set of mutations that give rise to uncontrollable cell-division cycle, or cancer. With that in mind, each person would have some commonalities with other patients but also some unique qualities.”

However, the majority of patients still don’t receive personalized care, notes Mr. Wells. The PMC notes that a previous study has demonstrated chemotherapy use would drop 34% in women with breast cancer if they had a genetic test of their tumor before treatment.

With cystic fibrosis, “we thought it was one disease,” says Euan Ashley, associate professor of medicine at Stanford University in Stanford, California. “But as we dig deeper with genetic sequencing, we find it’s many diseases. If you can subcategorize patients, you can treat a disease much more effectively.”

Dr. Ashley’s specialty, cardiovascular disease, is the leading cause of death globally. “So we tended to do very large population studies, giving the same drug to everyone,” he says. “But if you study closely afterward, you see that a small number of people drive the effect—they get a significant benefit. Many get no benefit. And a few get significant harm.”

Precision medicine is prompting different ways of thinking about populations and individuals. “The answer has to be to measure people in as high a resolution as we can and work out who is responding and why,” he says.

With the cost of developing a prescription drug at about $2.6 billion, pharmaceutical companies have a big interest in seeing that the drugs they make get tested on the right segment of patients. A drug could appear to have no effect, when in fact it’s highly effective but only for a smaller number of patients.

Scientist woman“Drug companies now are building precision medicine into their research-and-development strategies,” says Mr. Wells. Personalized medicines accounted for 28% of novel new drugs approved by the U.S. Food and Drug Administration in 2015, and for 35% of novel new oncology drugs. Novel new drugs go beyond improved formulations or new dosages to deliver truly innovative advances.

Meanwhile, pharmacogenomics looks at how different people metabolize drugs. “There are a number of genes that are particularly active in the liver,” says Dr. Haga of Duke, adding that there are many variations of these genes among people. “We can test whether a patient metabolizes fast or slow and, if necessary, can prescribe a different drug that goes through different pathways so the affected genes aren’t involved.”

This test is valid for life, because one’s genes don’t change. Some institutions are trying to incorporate the information into electronic medical records, so all the different doctors and specialists one might see—as well as pharmacists—would be more knowledgeable, for prescribing drugs.

Because the cost of genetic sequencing has fallen dramatically, to about $1,000 today for a genome from $100 million in 2001, some are asking why everybody doesn’t get tested. It could speed up treatment for cancer patients or could allow for early intervention to arrest development of certain other diseases.

The U.S. National Institutes of Health devoted $25 million to four projects of genetic sequencing in newborns over five years with the goal of diagnosing conditions at the start of life. China just launched a project to do genetic tests on 100,000 newborns over the next five years, to improve treatment strategies and patients’ quality of life.

“The technology is going to continue to improve,” Mr. Wells says. “But already we’re at a point where the scientific advances are incredible.”

 

 

Catherine Bolgar is a former managing editor of The Wall Street Journal Europe, now working as a freelance writer and editor with WSJ. Custom Studios in EMEA. For more from Catherine Bolgar, along with other industry experts, join the Future Realities discussion on LinkedIn.

Photos courtesy of iStock




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