For Crohn’s Disease, a Breakthrough Drug with Risks

hen the Food and Drug Administration gave early approval to the new drug Remicaid a little more than a year ago, it was good news for thousands of patients with the inflammatory gastrointestinal disorder, Crohn’s disease. The medication offered hope particularly to patients with the worst type of Crohn’s—those with ulcers, or fistulas, that tunnel through the bowel wall into nearby organs or through the surface of the skin. In one study, at least half of the fistulas closed in 68 percent of patients taking Remicaid. In another, 48 percent of patients showed complete remission.

Gastroenterologist Mary Harris calls Remicaid the first new treatment in years to help people suffering from an extraordinarily debilitating disease that really has no cure. “We’ve had some patients with stool growing out of their abdomen from the fistulas,” she says, “who have taken the drug and now say this is the best they’ve ever felt in their life.” More than 23,000 patients have been prescribed the drug in its first year of availability.

Yet, Remicaid has big risks. It is an immunosuppressive that works by binding to a substance in the body called tumor necrosis factor (TNF), which causes the inflammation that produces the symptoms of Crohn’s. Some immunosuppressives have been linked to lymphomas and other cancers. When the FDA ended the Remicaid trials after only three years, in order to get the drug to desperately ill patients, it closed the window on evaluating the drug’s long-term effects. “We need to study people who’ve taken the drug for five to 10 years to see if there’s an increased risk of certain cancers,” Harris says.

Harris, who currently is the only clinician at Johns Hopkins approved to prescribe the drug, is meticulously tracking the responses of her patients. And because the drug is so powerful, she makes clear that it should be used only for patients who haven’t responded well to conventional therapies. Side effects include itching and stinging skin, chills, difficulty breathing, headache, nausea, dizziness, fatigue and fever. And since patients receive the drug intravenously over two hours, there may also be negative reactions to the infusion, so emergency drugs must be available at the bedside.

unt is teaching nurses around the country how to administer Remicaid, since many hospitals have turned to Hopkins for advice on handling the drug. Explains Harris: “They’re anxious to know what parameters we’re following, our thoughts on length of treatment, the speed of the drug’s response, and adverse events down the road.

“What’s clear,” she stresses, “is that this is an amazing drug. But it must be used with utter discrimination.”

—- GL