Contact: Jill Byrd, (210) 450-5550

Carolyn Boone’s cancer has stabilized as a result of her participation in a clinical trial testing a new drug at The Cancer Therapy & Research Center at The University of Texas Health Science Center at San Antonio.
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SAN ANTONIO (May 19, 2008) — Five years ago, she was diagnosed with cancer and became the first person in the world to take an experimental therapeutic drug being tested at The Cancer Therapy & Research Center at The University of Texas Health Science Center at San Antonio. This week, with her cancer stabilized, Carolyn Boone celebrates five years of survival and, ultimately, her life.

Diagnosed in 2003 with mullerian sarcoma, Boone’s oncologist told her traditional therapy would not help and that she had approximately one year to live. The cancer started in her uterus and had already spread to her liver and lungs. Having received no chemotherapy, she was enrolled at the CTRC in a Phase I research study evaluating the anti-cancer agent AP23573 (brand name deforolimus).

Boone becomes first patient in new study
“The experimental study my doctor actually sent me to CTRC for was already full, but there was a brand-new drug study available for my type of cancer. I was told I would be the first person in the world to enroll in the study and take the drug,” Boone, 66, said. “I remember feeling a little panicky and anxious, but I was willing to try anything to stay alive.”

Cancer responds to new treatment
She began traveling from her home in Dallas every other week for the five days of intravenous treatment. Within 10 months, the cancer on her liver had disappeared. As Boone continued on the therapy, tests showed that the sarcoma and the cancer on her lungs had stabilized.

“I continue to live life one day at a time,” Boone said. “I sometimes grow tired of traveling, but I do what I have to do to stay alive. I’m scared not to.”

Monica Mita, M.D., will soon present initial results for an oral form of version of the drug.

CTRC’s Monica Mita, M.D., is principal investigator
The CTRC physician and principal investigator on the study, Monica Mita, M.D., is delighted that the drug has successfully treated Boone’s sarcoma. “Carolyn’s story is truly unique in the sense that not only was she the first patient in the world treated with this active drug, but she also experienced significant tumor reduction that is stable for five years now,” Dr. Mita said. “Her story is an inspiration for other patients and care providers alike, and is really what we would like to see for all patients with advanced cancer — significant and durable response with a normal quality of life.”

Background and progress of deforolimus testing
Deforolimus is a drug that inhibits the activity of mammalian Target of Tapamycin (mTOR), an enzyme that plays a key role in the growth and proliferation of cells. The Phase I trial for the drug began in 2003. A Phase II trial for sarcoma patients was opened in 2005. The drug is now being evaluated in a Phase III trial as an oral medication for patients with sarcoma.

According to study results, the drug is well tolerated by patients. The most common side effect is mouth sores. Other patients with sarcoma in the initial Phase I study showed good results with the treatment, as well as patients with other types of tumors, such as non-small cell lung cancer.

Dr. Mita to report results of oral formulation at ASCO annual meeting
Dr. Mita is scheduled to report the results from the group of patients in the Phase I study who took the oral form of deforolimus. She will make her presentation on May 31 at the American Society of Clinical Oncology annual meeting in Chicago.

“These results present us with important information about deforolimus’ oral formulation, providing an alternative route of administration that has the potential to offer greater patient convenience and treatment flexibility than the intravenous form,” Dr. Mita said. “I also am very pleased to see that the antitumor activity and safety profile appear consistent with our previous clinical experience with the intravenous formulation.”

Understanding study phases
According to the National Cancer Institute, a Phase I study is the first time a drug has been given to humans. Phase I trials evaluate how a drug should be administered, including how often it should be given and what dose is safe. A Phase II study continues to test the safety of the drug and begins to evaluate how well the drug works, usually focusing on a particular type of cancer. A Phase III study tests the drug, in combination with other drugs or other procedures, in comparison to the current medical standard.

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The Cancer Therapy & Research Center (CTRC) at The University of Texas Health Science Center at San Antonio is one of the nation’s leading academic research and treatment centers, serving more than 4.4 million people in the high-growth corridor of Central and South Texas including Austin, San Antonio, Laredo and the Rio Grande Valley. CTRC is one of a few elite cancer centers in the country to be named a National Cancer Institute (NCI) Designated Cancer Center, and is one of only three in Texas. CTRC handles more than 120,000 patient visits each year and is a world leader in developing new drugs to treat cancer. The CTRC Institute for Drug Development (IDD) is internationally recognized for conducting the largest oncology Phase I clinical drug trials program in the world, and participated in the clinical and/or preclinical development of many of the cancer drugs approved by the U.S. Food & Drug Administration. For more information, visit www.ctrc.uthscsa.edu.