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New Institute Aims to Match Medical Treatment to Genes

By translating genetic discoveries into new ways to diagnose and treat disease, a new research institute being launched at Carolina aims to make drugs safer and more effective and speed laboratory discoveries to physicians and patients.

The Institute for Pharmacogenomics and Individualized Therapy (IPIT), based at the UNC School of Pharmacy, is bringing together researchers and clinicians across Carolina to create therapies and treatments for patients suffering from a wide variety of conditions.

“The promise of the science of pharmacogenomics is that each of us will get exactly the right medicine at the right time in just the right amount,” said Chancellor James Moeser at an event in early May held to inaugurate the institute. “As this new institute creates the tools to achieve this goal and expand the power of individualized therapy, we will save time, save money and, most importantly, save lives.”

The institute initially will partner with the UNC Lineberger Comprehensive Cancer Center to focus on cancer therapy, with planned expansion into cardiovascular disease, psychiatric disorders and global health. The results are expected to have both economic and health benefits.

“While extensive clinical trials often tell us how effective a drug is in a large population, those trials tell us little about any single person’s reactions,” said Howard McLeod, director of the new institute and the Fred Eshelman Distinguished Professor of pharmacy. “There are dramatic differences among people in their reaction to a particular medicine. We will look at the entire health care process to examine the potential of genes to guide medical therapy, allowing physicians to optimize drug selection and drug dosing based according to variations in each patient’s genes,” McLeod said.

Pharmacogenomics is a new field exploring how information in our genes influences our response to drugs. It involves integrating pharmacology with modern advances in genome analysis. The institute’s goal is to fully integrate personalized medicine into medical practice by providing tools and tests for physicians to identify patients at risk for adverse reactions or those who are likely to benefit from a particular treatment. Institute researchers also will identify drug targets, such as genetic markers in tumor cells, to guide development of new drugs.

McLeod has helped identify specific genetic variants that predispose patients to risk of severe side effects or inadequate benefit from drugs for diseases including colorectal cancer and childhood leukemia. His research also has had an impact on the Food and Drug Administration’s guidelines for warfarin, a blood thinner prescribed to more than 2 million people in the U.S.

“Warfarin is a textbook example of a drug with a narrow therapeutic index,” McLeod said. “Give a little bit too much and people bleed. Give too little and they clot. You have to get is just right.” Based on research by McLeod and others, the FDA issued new dosing guidelines because of two genes that affect how warfarin acts in patients.

IPIT will house one of 10 research centers that form the National Institutes of Health’s Pharmacogenomics Research Network. The institute’s researchers also lead the Pharmacogenomics for Every Nation Initiative, a global effort to help countries make better informed public health decisions using genetic information.

Bob Blouin, dean of the School of Pharmacy, said he expects the institute’s work to generate both health and economic benefits. “Fewer side effects and adverse reactions, more effective treatment and quicker recovery, and less wasted time and money are all obvious benefits to patients. But just think of the economic and social benefits of getting all these people back on their feet sooner and living productive lives. It could amount to billions of dollars.”


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