Investigational Cancer Agents now Being Tested at
Mayo Clinic's Campus in Florida

Wednesday, July 22, 2009

JACKSONVILLE, Fla. – With the recent opening of its fourth phase I study, the Mayo Clinic campus in Jacksonville has quickly expanded a program designed to offer cancer patients in Florida and the Southeast access to investigational therapies through rigorously monitored clinical trials.

The most recent study, testing an experimental agent that targets insulin-like growth factors 1 and 2 (IGF1, IGF2) – components of a cellular pathway increasingly recognized as important to a variety of cancers – joins three others that are evaluating other targeted agents. These are drugs designed to affect cell mechanisms critical to the growth and survival of tumors. The best control of cancer will likely come from agents that use different ways to shut down multiple cellular pathways activated during cancer development and progression, and this program is designed to test such novel therapies, says Michael Menefee, M.D., who heads the Phase I Clinical Trial Program in Oncology at Mayo Clinic, Jacksonville.

"The cancer field is rapidly moving to use of these new agents as a way to improve upon the success we have seen with more traditional therapies, such as chemotherapy and radiation," says Dr. Menefee who is an oncologist.

"While current chemotherapeutic drugs are effective and will continue to be important in the treatment of patients with cancer for the foreseeable future, we are always trying to identify drugs that are more effective and have less side effects, as well as agents that can be used when other treatments fail."

The program is one of only 14 nationwide that investigates experimental therapies developed by the NCI's Cancer Therapy Evaluation Program (CTEP). It can also conduct other "investigator-initiated" phase I studies supported by pharmaceutical industry funding, or translational studies developed based on discoveries in the laboratory of Mayo Clinic basic scientists.

Only a few phase I programs of this kind exist in the southeast region of the United States, Dr. Menefee says.

"We are pleased to be able to offer these phase I studies in Northeast Florida and the Southeast because we believe the agents we are testing represent the future of cancer care," he says. "These clinical trials are very resource intensive, which is why few of these programs exist regionally. We monitor our patients enrolled in phase I clinical trials very closely to ensure safety and minimize any risk associated with the experimental therapy, whether the agent being tested is a novel investigational drug or an FDA-approved drug that is being combined with another chemotherapy."

Phase I testing is integral to a long process of careful evaluation of potential therapies, says Dr. Menefee. "Almost every chemotherapeutic drug that is currently being used for patients with cancer was once an experimental therapy that underwent testing as part of a phase I clinical trial," he says.

After a drug has been evaluated in the laboratory and in animal models, it then goes into testing in humans if it is deemed to be promising. A phase I clinical trial is the first stage of testing a treatment in humans, usually in a small group of patients. "These studies are designed to assess the safety of an agent, the effects of the drug on various organ systems in the body, such as the liver and kidneys, and the effects of the body on the drug – for example, how the drug is metabolized," Dr. Menefee says. This process applies to newly designed drugs as well as drugs that have already been approved for other uses that are now being combined with other chemotherapeutics, he says.

These studies can also reveal early evidence of drug activity, such as whether or not the medication may slow down growth of tumors, although the studies are not explicitly designed to test effectiveness, Dr. Menefee adds. "If an agent is safe and appears promising, follow-up Phase II and Phase III studies can be conducted where a more accurate assessment of how effective a drug works in a particular tumor type is determined.

Many of the studies will be testing combinations of agents. "For many cancers, it will likely require the targeting of more than one pathway in the cancer cell to effectively kill it; this will often require the use of multiple drugs," says Dr. Menefee.

"Combinations of targeted therapies are actively being investigated, but many of the older chemotherapy drugs will remain important, as well."

Researchers at Mayo Clinic's campus in Florida often collaborate with existing phase I clinical trials programs at the Mayo Clinic campuses in Minnesota and Arizona. All three sites are part of the Mayo Clinic Cancer Center.

The program has only been running studies for nine months, says Dr. Menefee. He adds that he expects the number of studies to double by the end of 2009. Phase I clinical trials currently under way at Mayo Clinic in Florida include:

• Testing a combination of two targeted agents to treat solid tumors. The agents, dasatinib and lapatinib, are already approved for use in chronic myelogenous leukemia (CML) and breast cancer, respectively. This study, the first to test these two agents together, is industry supported and has completed its enrollment.

• Studying a combination of bortezomib, a drug approved to treat multiple myeloma, and an experimental agent known as 17-AAG. Patients with solid tumors are still being enrolled in the study, which is NCI-sponsored.

• Testing another combination of targeted agents, this time bortezomib paired with sorafenib, a drug approved to treat cancers of the kidney and liver. The NCI-sponsored study is currently only open to patients with multiple myeloma and chronic lymphocytic leukemia.

• The newest study, testing Medi-573, the agent designed to target IGF1 and IGF-2. This industry-sponsored study is still open to patients with solid tumors.

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About Mayo Clinic

Mayo Clinic is the first and largest integrated, not-for-profit group practice in the world. Doctors from every medical specialty work together to care for patients, joined by common systems and a philosophy of "the needs of the patient come first." More than 3,700 physicians, scientists and researchers, and 50,100 allied health staff work at Mayo Clinic, which has campuses in Rochester, Minn; Jacksonville, Fla; and Scottsdale/Phoenix, Ariz.; and community-based providers in more than 70 locations in southern Minnesota., western Wisconsin and northeast Iowa. These locations treat more than half a million people each year. To obtain the latest news releases from Mayo Clinic, go to www.mayoclinic.org/news. For information about research and education, visit www.mayo.edu. MayoClinic.com (www.mayoclinic.com) is available as a resource for your health stories.