Organ Transplant Drug Morphs Into Anti-Cancer Agent  

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Posted: 3/26/2004

COLUMBUS, Ohio - Scientists are experimenting with a cousin of a popular drug used in organ transplant to see if it can lead a double life as a potent tumor killer. The drug, CCI-779, is structurally similar to rapamycin, often used in kidney transplants to suppress the patient’s immune system so the new organ won’t be rejected.

Researchers are especially interested in how CCI-779 might work in patients with multiple myeloma. Multiple myeloma is a cancer of plasma cells, a critical part of the body’s immune system. Plasma cells create antibodies that can fight infection, but patients with myeloma produce too many of them, causing painful tumors in the bone and bone marrow.

Patients at Ohio State University’s Arthur G. James Cancer Hospital and Richard J. Solove Research Institute are the first in the country to get CCI-779 for multiple myeloma. Physicians are offering it to patients who have already had previous treatments, but whose disease has come back. The hope is that CCI-779’s unusual mechanism of action will shrink their tumors or at least hold the disease in check.

“Multiple myeloma is a very serious disease, and even though patients may be treated with chemotherapy or stem cell transplant, it invariably returns,” says Dr. Sherif Farag, an expert in hematological malignancies at The James. “We are intrigued with CCI-779 because it is a new approach to blocking cancer cells’ growth, and for the most part, patients can tolerate it very well.”

CCI-779 blocks tumor growth by interrupting the cell cycle – scrambling a key growth signal certain proteins create as the cell is getting ready to divide.

“With CCI-779, instead of growing, some of the cells just stop in their tracks and die,” says Farag.

CCI-779 has been tested in early trials in breast, kidney, melanoma and brain cancers, and for some patients, the drug was helpful in slowing tumor growth.

Patients who enroll in the trial at The James will get the drug by infusion on a weekly basis for eight weeks and patients who respond will continue on the drug until their disease progresses. Scientists will measure their response and will also be testing participants to see if they carry a mutation in a tumor suppressor gene called PTEN. Some studies have shown that people with these mutations respond better to the drug than others.

“This is a very exciting drug that could add to our armamentarium of agents against this terrible disease,” says Farag. “In addition, it may be appropriate for other forms of cancer, too.”

There are approximately 45,000 people in the U.S. living with multiple myeloma, and roughly 14,000 new cases will be diagnosed this year. Patients typically live only a few years after diagnosis, but a small minority may survive as long as 10 years or more.

The Arthur G. James Cancer Hospital and Richard J. Solove Research Institute, part of the Comprehensive Cancer Center at The Ohio State University, is the only freestanding cancer hospital in the Midwest. It is a national and international leader in translational research and clinical care, and one of the charter members of the National Comprehensive Cancer Network. U.S. News & World Report has consistently ranked The James as one of the nation’s best cancer hospitals.

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Michelle Gailiun
Medical Center Communications

Tags: Cancer; Clinical/Translational Research; James Cancer Hospital; Transplant

The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC – James) 460 W. 10th Avenue, Columbus, OH 43210 Phone: 1-800-293-5066 | Email: