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What’s in an Ipilimumab?

A medical experiment strikes gold as doctors manage to eliminate two prostate tumors on what had been inoperable cancers

Ipilimumab. You might not know how to pronounce it, but this antibody might just cure prostate cancer, claim doctors at the Mayo Clinic, Rochester, Minn.

Two men with advanced, inoperable prostate tumors are now cancer free thanks to a combination of standard and experimental treatments. The men underwent a common hormone therapy known as androgen ablation then participated in a trial of ipilimumab, an immunotherapeutic drug that blocks the activity of CTLA-4, thus sustaining immune responses to cancer cells.

In a few weeks’ time, both men had experienced enough tumor shrinkage that they could undergo surgery, where the researchers encountered something unexpected.

“The tumors had shrunk dramatically,” says Dr. Michael Blute, a Mayo urologist who operated on both men. “I had never seen anything like this before. I had a hard time finding the cancer.”

Dr. Blute and Dr. Eugene Kwon, the study’s leader, believe the treatment worked because ipilimumab builds upon the anti-tumor action of the androgen ablation hormones and creates a massive immune response, promoting cancer cell death. The trial was not meant to isolate ipilimumab’s effects but to learn if it could advance current treatment for the disease.

Doubters admit the results are notable but too small a sample for the disease to be claimed cured, warning against the potential for autoimmune diseases like colitis brought on by ipilimumab. Mayo has not released the number or results of other patients in the study, compounding nonbelievers’ reticence.

Dr. Derek Raghavan of the Cleveland Clinic’s Taussig Cancer Institute, Cleveland, Ohio, told WebMD.com that “it is way too early to get excited” because ipilimumab’s effects have not been isolated.

“We and others have shown remarkable responses after initial androgen ablation in this group of patients,” he says. “In this small, phase II study it is impossible to dissect out the impact of this … antibody.”

But Dr. Kwon says his team’s discovery is a landmark in cancer treatment.

“This is one of the holy grails in prostate cancer research,” he says. “We’ve been looking for this for years.”

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