Crohn's disease is a lifelong gastrointestinal illness that usually shows up when people are in their 20s or 30s. It is often debilitating and current therapies do not help everyone. Now, a new approach to therapy may offer relief to those who are not helped by standard treatments.
With every stroke, Kelly Perkowski falls deeper into her comfort zone.
"I get lost in it. I feel like I could never really finish something," Perkowski said.
Her comfort zone isn't always easy to get to. Perkowski has had Crohn's disease for 20 years.
"You're so fatigued. You just can't even do your daily activities, or you have abdominal pain and it's all consuming," said Perkowski.
All consuming to the point where she was at the end of the line. There were no other therapies to try. Nothing was working.
Then she met Brian Dieckgraefe, M.D., Ph.D, a gastroenterologist at Washington University in St. Louis. He offered Perkowski a new treatment. According to him, it's polar opposite to what other drugs are doing.
Instead of suppressing the immune system, this drug, dubbed GM-CSF, which stands for granulocyte-macrophage colony stimulating factor, also known as Leukine stimulates it.
"At first blush, this looks like we're pouring oil on the fire," said Dr. Dieckgraefe.
However, the drug keeps the disease in check for many patients. Eighty percent of people had a reduction in symptoms.
"We also saw 53 percent of the individuals we treated go into complete remission," said Dr. Dieckgraefe.
Perkowski was one of them. "All of a sudden, I would realize, 'Wow, it's another good day. Hey, I'm feeling good today. I haven't gone to the bathroom 10 times,'" she says.
The downside? Since this phase of the study ended, Perkowski can no longer get the drug.
"I haven't had it for, I guess, a year and a half since the study was over, and I've been sick ever since then," Perkowski said.
Of course she hopes the drug will soon be available.
"I was thoroughly enjoying life. So I'm anxious to get that back again," said Perkowski.
GM-CSF is given by self-injection once a day. It is already FDA approved for other uses, but not for Crohn's disease. A larger phase two study is currently underway at about 30 centers across the country.
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St. Louis, MO 63108