http://seattletimes.nwsource.com/html/businesstechnology/2001765321_leukine14.html By Luke Timmerman
Seattle Times business reporter
Leukine, the largely uncelebrated cancer drug created by Immunex a dozen years ago, may be finding new life as a treatment for Crohn's disease.
Berlex Laboratories, which bought Leukine a year ago, is showing off the drug's clinical results today at the annual gathering of gastrointestinal physicians in Baltimore.
The study involved patients with moderate to severe forms of Crohn's, a chronic inflammatory bowel disease. The cause of Crohn's is not fully understood, but symptoms include recurring diarrhea and painful stomach cramps. Standard treatment is with painkillers, steroids that suppress hyperactive immune systems or surgery to remove damaged parts of the intestine. Between 300,000 and 500,000 Americans are believed to have the condition.
In the Berlex-sponsored study of 124 patients, doctors found that 48 percent of those on daily injections of Leukine had significant relief from symptoms after eight weeks, compared with 26 percent on a placebo. It also showed that 40 percent of patients reached clinical remission after eight weeks, compared with 19 percent on a placebo.
A few patients had to drop out of the study when their disease worsened, but investigators said they didn't believe Leukine was the cause. Common side effects of the drug were swelling at injection sites and bone pain.
Based on the results, Berlex said it is moving ahead to the final stage of testing, possibly with a pair of Leukine trials in about 400 Crohn's patients.
If the company can confirm the results, the next trials could serve as the basis for approval from the Food and Drug Administration. Berlex plans to start the trials early next year.
Dr. Joshua Korzenik, a professor of gastroenterology at Washington University in St. Louis and principal investigator of the study, said patients had dramatic things to say.
"They've said, 'Thank you for giving me my life back,' " Korzenik said. "I've heard patients say, 'With other drugs, I feel better, but with Leukine, I feel normal again.' "
The results also offer some validation for a counterintuitive scientific approach to Crohn's. The disease is caused by a hyperactive immune system attacking the intestines and has been treated with immune-suppression drugs, such as steroids, which can cause nasty side effects and leave patients vulnerable to infections.
Leukine works the opposite way: It stimulates growth of white blood cells that make up the immune system.
Researchers believe Leukine might work based on a new theory of the cause of Crohn's — that its wild inflammation stems from a deficiency in the immune system's first line of defense. Korzenik said it is believed that if the immune system can be restored, bacterial invaders in the intestines can be gobbled up early, and the body will not trigger the massive inflammatory overreaction found in Crohn's.
The company and investigators had feared that if the theory were wrong, Leukine could rev up an already overcharged immune system and make patients worse. But they haven't found evidence of that.
Berlex is following up with patients over six months and examining tissue samples to see if the theory stands.
Dr. Mark Gilbert, vice president of medical affairs for Berlex in Seattle, said the company is talking with the FDA about how to craft its final Crohn's trials of Leukine. If all goes well, he said, the drug could be approved for treating the disease by 2007.
Crohn's represents a big potential market for Leukine, which has long been in the shadow of its chief competitor in the cancer field, Amgen's Neupogen.
Berlex said it is committed to maximizing its $390 million cash investment in Leukine by pushing for its approval in Europe and for approval in the United States to treat Crohn's.
The drug had $91 million in sales in 2002.
Berlex Laboratories
Berlex is the U.S. wing of Schering, a global drug company based in Germany.
Location: Seattle and Bothell
Employees: 180
Puget Sound-area history: Berlex bought Leukine from Immunex for $390 million in May 2002.
Leukine sales: $91 million in 2002