Phillips awarded $5 Million NCI grant to study personalized medicine

The wider world use of medical tests and treatments based on individual genetic differences is the focus of a new, US$5 million research program funded by the National Cancer Institute (NCI) and led by UCSF School of Pharmacy health economist Kathryn Phillips, PhD. The grant was awarded on September 16, 2008 and will be paid over three years.

This is the first major NCI grant focused on health policy issues related to personalized medicine and pharmacogenomics, says Phillips. The grant includes several research projects. Grant participants include scientists at leading US and international universities, and experts from the health insurance industry and government regulatory agencies.

The NCI grant, along with foundation grants, form the nexus of the newly established Center for Translational and Policy Research on Personalized Medicine based in the UCSF School of Pharmacy's Department of Clinical Pharmacy.

Gauging Drug Response

Drugs act differently in different people; this is the basis of pharmacogenomics and much of personalized medicine. Researchers aim to identify genetic differences that underlie these varying responses. Diagnostic tests can be developed to quickly identify important differences, and physicians may use the results to select and dose drugs.

Unfortunately, efforts to translate personalized medicine into actual clinical use and to develop appropriate health policies can be challenging, Phillips says.

"Some physicians may not even be aware that a diagnostic test exists that is applicable to their patients. In other cases, patients may be receiving costly medications without being tested to see if they would be likely to benefit, or even when they tested negative for the biological target of the drug."

"Even when physicians know when pharmacogenomics tests are meant to be used, they may not be familiar with the best way to use the information obtained in a particular case," adds Joseph Guglielmo, PharmD, chair of the UCSF School of Pharmacy's Department of Clinical Pharmacy where Phillips holds her faculty appointment.

"In some medical scenarios, the best application of the already available tools in pharmacogenomics remains an open research question," he says. "There are a whole lot of things that go into the pot and that ultimately result in a drug response, and pharmacogenomics is just one of them."

In recognition of the difficulties in sorting out this information, faculty pharmacists in the UCSF School of Pharmacy are available to advise UCSF Medical Center physicians on pharmacogenomics issues related to the care of individual patients.

The Impact of Diagnostics

"The NCI grant will allow us to explore data on large numbers of patients," says Phillips. "With this data, we'll learn more about how already available diagnostics are being used, and how their use influences treatment choices." Phillips believes a key thrust of research should be to investigate whether economic status, health insurance coverage, ethnicity and medical practice settings affect access and use of these new diagnostics, and how their use influences drug choices and health outcomes for patients.

"What we learn may affect how we decide we want to provide these technologies," she adds.

The Impact of Targeted Therapies

In evaluating new drugs, pharmaceutical companies are not required to study how genetic differences affect treatment outcomes. In some cases, however, companies have intentionally developed drugs they know are likely to benefit only a certain set of patients with a distinct genetic characteristic.

One of the first such targeted therapies is the breast cancer drug Herceptin. Patients with breast tumors that test positive for a genetic abnormality that elevates production of a protein called HER2 are likely to get the drug, which targets the protein. However, the genetic abnormality is present only in about 20 percent of breast cancers. Phillips is continuing her research on Herceptin use as part of the new NCI grant.

NCI Grant and New Center Build on School Expertise

The grant and the center in which it will live are the latest among several launched by UCSF researchers in pharmacogenomics and personalized medicine.

For several years, Kathy M. Giacomini, PhD, chair of the UCSF School of Pharmacy's Department of Biopharmaceutical Sciences, has led a large, multi-institutional study focused on identifying genetic variations among ethnically diverse populations. The study focuses on genes that affect how a wide range of drugs act in the body. The genes are the blueprints for proteins called membrane transporters, because they determine how efficiently drugs and other molecules are transported into or out of cells.

With its emphasis on health policy and medical practice, the new collaborative research led by Phillips further bolsters the UCSF School of Pharmacy's already strong leadership in pharmacogenomics research and its application in the practice of personalized medicine.


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