News

8.17.2011

Out of Options, First Patient in Bladder Cancer Trial Now Has Hope

In May, when Robert Chambers became the first patient to receive a potential new bladder cancer therapy developed by Miller School Nobel laureate Andrew V. Schally, Ph.D., M.D.h.c., D.Sc.h.c., he was out of treatment options and hope. But the West Palm Beach resident figured he might help other patients by enrolling in the phase 1 clinical trial for AEZS-108 at Sylvester Comprehensive Cancer Center.

Now, after three months and six doses of the hybrid drug that pairs the chemo drug doxorubicin with a peptide that, like a guided missile, delivers the well-known cytotoxic agent directly to receptors on cancerous cells, the tumors that originated in Chamber’s bladder and spread to his liver and lymph nodes have shrunk by more than 70 percent. He’s also pain-free and has an appetite again.

“After the second infusion I was feeling a lot better,’’ the retired casino entertainment executive and father of two said Tuesday, as he was prepped to receive the final dose in the protocol. “I had a tumor in a lymph node gland in my neck that I could feel and, after the second treatment, it was gone. It’s a pretty amazing thing. It’s almost hard to describe. You go from being… realistic about dying and then you get this gift.’’

Nearly two decades in the making, the gift was made possible by Schally, Distinguished Medical Research Scientist in the Department of Veterans Affairs, Distinguished Professor of Pathology at the Miller School and professor of medicine in the Division of Hematology-Oncology at Sylvester, who was awarded the Nobel Prize in Medicine in 1977 for his work on hypothalamic hormones.

Schally’s pioneering work led to the discovery of luteinizing hormone-releasing hormone (LHRH), and its use as a guided missile to deliver targeted chemotherapy directly to cancerous cells, sparing the rest of the body the deleterious side effects.

Produced in the hypothalamus, LHRH directs the pituitary gland to produce lutenizing hormone, which in turn directs the production of estrogen and testosterone. But, as it turns out, almost all cancer cells have receptors for LHRH, and Schally knew the peptide could make an ideal vehicle to deliver targeted chemotherapy.

Except he faced one huge hurdle: LHRH disintegrates rapidly after its release in the body, negating its usefulness. So Schally developed a synthetic version of LHRH capable of lasting for hours; then he figured out how to combine it with doxorubicin.

“It took him eight years to connect the two pieces,’’ said Norman Block, M.D., professor of pathology, urology, and biomedical engineering and clinical director of Schally’s Endocrine, Polypeptide and Cancer Institute, who was instrumental in recruiting Schally to UM. “LHRH normally disintegrates in seconds, so Dr. Schally designed and built a synthetic version, which lasts for minutes or hours, so it has time to reach the cancer cells, attach to the receptors and release the chemotherapy. It’s like the receptor is a target and the LHRH is a bomb designed to hit the target.’’

Away on vacation, Schally was unable to meet Chambers, but Block said the Nobel laureate is elated about the patient’s progress. So, too, is Gustavo Fernandez, M.D., assistant professor of clinical medicine and the principal investigator of the trial, which is designed for 64 patients with bladder cancer who have failed other chemotherapies.

“When he came here he already failed two or three rounds of chemotherapy,’’ Fernandez said. “Literally, he was dying. Now he’s pain-free, his quality of life has changed dramatically and the cancer is shrinking … It’s very hard to put a clinical trial together; it involves a lot of people and a lot of time, but this makes it all worth it.’’

The clinical trial also received extensive TV coverage and a section front display story in The Miami Herald.

In the coming months, Sylvester is expected to launch AEZS-108 trials for pancreatic and breast cancer as well.

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