Editor's note: This is part of our continuing series of stories about Bruce Kramer, the former dean of the College of Education, Leadership and Counseling at the University of St. Thomas, as he copes with life after being diagnosed with ALS, also known as Lou Gehrig's Disease. You can read all the stories in the series by clicking here.
The news is filled with stories about the latest medical study, device or drug. Some of them turn out to be effective treatments, but many others don't pan out. That is the case of a drug that intrigued doctors who treat and patients who have ALS, or Lou Gehrig's disease.
Made by Biogen Idec of Massachusetts, Dexpramipexole in early tests appeared to slow the progression of the disease that makes a patient lose control of their bodies and ultimately their breathing. In April of 2011, company officials announced they were taking the drug into stage-three trials to determine whether it would be only the second drug ever approved to combat the terminal disease.
Phase 3 of the trial enrolled 943 people with ALS at 81 sites in 11 countries One of the patients in the study was Bruce Kramer of Minneapolis. We first talked to Bruce in June of 2011, shortly after he started taking the experimental drug.
"I still see the progression but I feel better," Kramer said at the time. "My head feels better. I feel clearer. I ask myself, am I getting better? Am I on the drug or is it the placebo effect? Well, viva la placebo!"
In many drug studies participants don't know if they're getting the drug or a placebo, but over the past 18 months, Kramer felt it was likely he was on Dexpramipexole. His skin became oily and his hair grew thicker.
"It would help me clear through the fatigue of ALS and I found that almost to be an immediate effect and it stayed pretty much all the way though," he said recently.
"I probably knew about the announcement within about 45 minutes of it happening," Kramer said, adding that it caught him off guard. "All of a sudden it popped up in one of these places that I monitor and you know, Dexpramipexole is a wash out."
Dr. Douglas Kerr, Biogen's lead medical director for the study and an ALS neurologist, called the trial a "very comprehensive, very large study which was designed to definitively answer the question as to whether this drug worked in ALS."
"This study showed definitively that the drug did not work and it is tremendously disappointing for the ALS community," Kerr said. "We wanted to look at how patients were functioning and whether we could preserve function - meaning turning over in bed, caring for themselves, walking, using their hands, swallowing. We also wanted to know if it prolonged survival and whether it enhanced their ability to breathe independently so we looked at all those measures and unfortunately, the drug showed no efficacy in any of those measures. "
When he got the news, Kramer said he was "really disappointed. "
"It didn't survive the scientific tests and yet I believe that there are, and I'm sure there are others that believe there are some effects that didn't get measured. I'm having breathing problems now that I didn't have five days ago. Is that a coincidence or is that it just happens to be the progression of ALS?" Kramer said.
Kerr says the results are definitive: The drug has no benefits for ALS patients.
“We need to really be much smarter about how we evaluate patients and their disease progression.”Dr. Douglas Kerr
"When we found that the drug didn't work for the overall study population, we then went and very carefully looked at small groups of patients to detect if there was any benefit in any of those small groups," he said. "If it had been, even that would have been a small victory. And we didn't find any efficacy in any of those small groups."
At the same time, Kerr said the trial did help in other ways.
"It is possible that we don't have the perfect way to evaluate disease progression in ALS and I think it's one of the major contributions of this study. Even though the drug did not work, we as a community are really understanding ALS better ," he said. "There is heterogeneity in ALS, in other words, not every patient is the same. We need to really be much smarter about how we evaluate patients and their disease progression."
Some analysts estimated Biogen spent $75-$100 million to conduct this late stage trial. Kerr said he wasn't privy to the amount of money spent, and added that the curtailing of the study was not a case of "cut our losses."
"The reason to terminate this study was because we felt ethically, deeply obligated, to provide this information to patients," he said. This was such an unequivocal finding and we didn't want to be ambiguous about this in any way."
"It hasn't slowed my progression. As you can tell, I'm still in a wheelchair. I haven't thrown down my crutches or anything," Kramer said. "It's disappointing because we still only have one drug. I think the community was really hopeful about Dexpramipexole. We thought it might be the second one. If you get a second one there's an opportunity to tweak it, to continue to work it and then maybe there's a third and they start combining. But that, of course, is out the window now."
"I am frustrated," Kramer said. "I'm not frustrated that they think it's a failure. That's not it. I'm frustrated that we have not, with these remarkable, creative brains we have as humans, have not figured out another way of approaching this that might give us more confidence in the effects of something. I'm positive that there are drugs that are probably sitting out there that would have in some way, shape or form that would mediate some of the effects of ALS. We don't know them we don't know the connection between them.
"I didn't go into the trial that I'm in, or was in, with the idea that there would be a cure," he added." You can't do that. Even if there is, it's too late for me. It's going to be something that's going to take years.
In the meantime, Kramer is applying to get into another drug trial and will know if he's accepted in February.