Compassionate Use Rule Allows Patient to Benefit from Clinical Trial Treatment

Clinical trial participant Sharon Kollman with the UW Carbone Cancer Center's Dr. Noelle LoConte.Sharon Kollman's newest career involves pre-planning funerals for other people.


"Every once in a while, someone will tell me they want to donate their body to science," Kollman says, "and I think to myself – I already did that!"


The Only Patient in the World


Kollman has been part of a clinical trial for a rare type of liver cancer for the past five years. She is the only patient in the world on her drug, Brivanib, which worked for her but did not work for enough patients for it to win FDA approval as a standard treatment.


"It worked very well for me," she says. "I'm still alive, after all."


Kollman, who lives in the Fond du Lac area, was an insurance saleswoman for most of her career. She's says it's almost a fluke that she was diagnosed with a type of liver cancer in February 2011.


Even though she was not an alcohol drinker, she had developed cirrhosis, and her doctor decided to do an ultrasound. The image showed tumors throughout her liver of a rare type of cancer called hepatocellular carcinoma. Her doctor gave her the shocking news that she may have only 18 months to live.


"Why not do a trial?"


She came to UW Carbone Cancer Center for radiation treatments. But because there were so many tumors, she couldn't have enough radiation to cure the cancer, so her doctors suggested she consider a clinical trial.


"I figured why not do a trial?" she says. "I didn't expect it to work for me."


When she started on the double-blind national trial in June 2011, there were about 1,300 people enrolled. By the time it ended, just 22 remained. The drug did not work for the majority, but in Kollman's case, it stopped the tumors from growing.


"She is the only patient in the world currently taking this medication, as Sharon was the only patient who was known to derive benefit from the study drug," says Andrew Tatar, who coordinates clinical trials for UW Carbone Cancer Center.


Tatar explains that Kollman continues to receive the drug under a "compassionate use" trial. The drug company, Bristol Meyers Squibb, has had to round it up from the study sites around the world in order to keep getting her the study drug.


She continues seeing GI cancer oncologist Dr. Noelle LoConte every few months for blood tests and scans that show that her cancer remains stable.


Kollman says she takes her pill very early in the morning, then goes back to sleep since it makes her feel "woozy." But she says that's a small price to pay for a cancer that had a poor prognosis.


"I never thought I'd be back working, but here I am," she says.