What’s the Big Deal About Precision Medicine? - Promise
What's the Big Deal About Precision Medicine? | Promise: Powered by Nemours Children's Health System

What’s the Big Deal About Precision Medicine?

Better health outcomes for all of us, at a lower cost and with fewer side effects.

It’s no secret that each of us is different – we’re born with different genetic backgrounds, grow up in different environments, and lead different lifestyles. But until now, much of the medical care we receive has been delivered in a one-size-fits-all way.

Precision medicine, a newer approach for disease treatment and prevention, takes into account all the things that make us different – and uses those factors to create more effective treatments with fewer side effects.

In the same way that a doctor might take your child’s medical history (think allergies, blood type, past surgeries or illnesses) into consideration before recommending a course of treatment, precision medicine can guide health care professionals in the same way.

But precision medicine is more than a fancy technique. Instead, it’s a movement for understanding, diagnosing, managing and treating pediatric disease – throughout a child’s early life, and into adulthood.

How it works:

Patients’ blood (and sometimes, other tissues) are collected and detailed in what’s called a biobank. The data from those tissues are shared privately with other hospitals and organizations. Then, with the help of advanced bioinformatic analytical tools, the data is analyzed and interpreted to help make predictions and recommendations about patients’ health and some available treatment options. These specific recommendations can lower the costs of treatment, and eliminate the trial-and-error process that comes with effectively managing conditions like asthma.

Precision medicine is especially important when it comes to rare diseases, because it improves health care providers’ ability to reach an accurate diagnosis. Doctors caring for one Nemours patient, a baby with acute kidney failure, relied on precision medicine to diagnose just what type of kidney disease she had. Since the genetic testing her case required was not available in the U.S., her blood was sent to the United Kingdom for analysis. There, doctors were able to determine that the child would need a new kidney right away – and that she was at risk for kidney cancer in the future. In the end, this young patient survived because of precision medicine. Knowing what type of kidney disease she was facing was crucial to finding a care plan, and saved her life. The information gleaned from precision medicine also spared her from steroid treatment, which has numerous side effects.

Treatment for other diseases, like pediatric cancer, can also be greatly improved with precision medicine. Physicians can more accurately predict which cancers in which children are likely to resurface, and which will go into remission. It’s all based on what kind of tumor or cancer each child has, along with their unique genetic makeup.

The road ahead:

Precision medicine already has a great deal of momentum behind it, but so far, one crucial part of the population is missing from research efforts: children. The National Institutes of Health has launched the Precision Medicine Initiative, which includes the All of Us Research Program – a mission to gather genetic data from more than one million Americans. And while children stand to gain so much from precision medicine, the All of Us program is not yet collecting data from a meaningful number of pediatric patients. That’s where Nemours comes in.

Nemours Children’s Health System is one of the few organizations in the U.S. with the capability to collect genetic data from some children, and then use that data to guide treatment decisions. We believe that a personalized approach to treatment through precision medicine truly embodies our mission: involving the patient and the family in their care, for better health outcomes. To that end, we’re seeking out the support of the federal government, private foundations, laboratories and insurance companies to move this groundbreaking work forward.

Additionally, we are partnering with other leading pediatric researchers to increase awareness of the importance of pediatric research on Capitol Hill.Children are truly our future, and they should be the ones who benefit most from the medical breakthroughs in our country.

 

Vicky Funanage, PhD

About Vicky Funanage, PhD

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Dr. Funanage serves as the Operational Vice President of Research at Nemours. She joined Nemours as a molecular geneticist in July 1981, and holds faculty positions at Jefferson Medical College/Thomas Jefferson University and the University of Delaware. Dr. Funanage was appointed to the position of Director of Biomedical Research for Nemours in June 2000, and OVP of Research in 2014. She also serves as the Director of the CLIA-certified Molecular Diagnostic Laboratory and Head of the Musculoskeletal Inherited Disease Laboratory at Nemours/AIDHC.