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Experimental stem cell treatment passes road test

A motorcyclist with Crohn's disease rides in comfort using a drug that may offer hope for others like him.

October 29, 2006|Tricia Bishop | The Baltimore Sun

For 13 days this summer -- one day for each year he'd lived with the chronic pain and fatigue of Crohn's disease -- Robert Gagne gleefully rode his pearl-white Yamaha Road Star nearly 5,000 miles through 17 states, sometimes logging 12 hours on the motorcycle.

He saw Mt. Rushmore and Colorado's Estes Park. He woke up one July morning in Rapid City, S.D., and went to bed 693 miles away in La Crosse, Wis. And on his many stops, he didn't once turn to the local Yellow Pages to look up the nearest emergency room, as he was so used to doing for so many years, just in case.

"It's one of the best experiences of my life; I would put it right up there in the top five or six, being able to do something like that," said the 58-year-old Virginian, who eight months ago didn't have the stamina to make the trip.

The change, he said, began sometime in May, about eight weeks after he'd received an experimental stem cell treatment developed by Baltimore's Osiris Therapeutics Inc., which on Oct. 18 announced results of the 10-person clinical trial Gagne took part in.

The drug, which analysts say is close to becoming the first pure stem cell product on the market, is already under investigation as a treatment for a rare condition associated with bone marrow transplants. But if it also gains approval as a therapy for Crohn's disease -- and possibly its cousin irritable bowel syndrome -- the drug Prochymal could be the first stem cell product with a large application, as well as a big breadwinner for the newly public Osiris.

Combined, the two conditions affect about a million Americans.

"We think it's about a billion-dollar market opportunity," said Chief Executive C. Randal Mills. Osiris' stock has risen about 37% since debuting at $11 on the Nasdaq in August.

Preliminary data show that all 10 patients in the midstage clinical trial saw a reduction in symptoms after receiving intravenous infusions of the adult stem cell treatment, and three of them went into temporary remission.

"That's a tremendous response," said Dr. Michael Pandak, who ran part of the trial from the Hunter Holmes McGuire VA Medical Center in Richmond, Va., about 45 minutes north of Gagne's Prince George County home. "We had pretty sick patients who were not doing well with the standard medications."

Eight of the 10 had already undergone surgery to control the disease, characterized by chronic intestinal inflammation, and tried out multiple medications. Still, Pandak tempered his enthusiasm with reminders that the trial was relatively tiny and that no one was cured. The data do, however, bode well for further trials and exploration, he said.

"We like to get really excited about new things like this, and we should, but we also have a lot of work to do," Pandak said of those in the medical field.

According to the National Institutes of Health, Crohn's symptoms typically include abdominal pain and frequent diarrhea, though rectal bleeding, intestinal ulcers and arthritis are not uncommon.

Gagne said his pain had gotten so bad over the last five or so years that he'd been to one emergency room or another eight times. And Gagne had reached a point where he couldn't even run a push mower across his lawn for more than 30 minutes without having to rest.

Between 25% and 50% of those with Crohn's will need surgery to control the disease, and about half of all patients are resistant to current medical treatments, such as antibiotics, steroids and immune system suppressants, said Dr. Raymond Cross Jr., chief of gastroenterology at the Baltimore VA Medical Center and director of the inflammatory bowel disease program at the University of Maryland School of Medicine.

"It can have a profound negative impact on the quality of a patient's life; in fact, some patients even become disabled because of the symptoms," Cross said. Although he was unfamiliar with the study, he said a cursory review showed the limited available data appeared encouraging.

"It's a very interesting concept," Cross said. "This is a very refractory group of patients with very few treatment options. If this is a positive drug for this group of patients, it really is very hopeful."

The purpose of the trial was to find out whether the anti-inflammatory Prochymal would have any effect on Crohn's. Clinicians and the company were simply looking for some kind of correlation. Now that they have one, CEO Mills said Osiris had begun talking with the Food and Drug Administration about designing late-stage, more efficient trials that could best serve those patients who had exhausted most other options.

In the meantime, Gagne, who retired three years ago from the federal government, is cautiously enjoying his renewed energy.

"I have hopes, but I'll be very candid: I've been on other treatments as long as 13, 14, 15 months that worked reasonably well. The vote is still out on this," he said. "Right now, it's probably the best treatment that I've ever had based on the results that I've achieved."

But he added that he was waiting to see whether it worked as well "for, let's say, 15, 16, 24 months."

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