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.
Flip on the TV, especially during the evening network news shows and you'll hear a slew of drug ads for myriad maladies. Meanwhile, an estimated 30 million Americans living with any of about 6,500 rare medical conditions wait for the development of drugs that could better treat, or even cure, their illnesses.
In order to help, 30 years ago, in 1983, President Ronald Reagan signed the Orphan Drug Act into law, in the hope that financial incentives would encourage more pharmaceutical companies to develop drugs for these lesser known illnesses, including ALS, commonly known as Lou Gehrig's Disease.
There is only one FDA-approved medication for ALS and it was approved back in 1996. Numerous other drugs have been tried without success.
Bruce Kramer has been in two drug trials since being diagnosed. The most recent trial was for a drug called Tirasemtiv developed by the firm Cytokinetics. It's a drug that's supposed to help the skeletal muscles better metabolize calcium and hopefully strengthen them.
"Obviously we're not talking about a cure for ALS here," Kramer says. "But what we are talking about is that if there is some way to keep whatever muscles you have stronger, that would certainly be helpful."
Kramer started noticing positive changes shortly after he started taking the drug.
"Right before I got into the trial, I had been at Mayo, and one of the things I asked them for was a prescription for a neck brace," he said. "The reason being, my neck had become quite weak and especially at night was having a hard time holding my head up. I got the neck brace a couple of days before the trial and used it for about three nights."
To Kramer's delight, shortly after he started the drug trial, he didn't need the neck brace. His neck became strong enough and it stayed that way. He also slept through the night, both things adding to his quality of life -- effects not measured in the drug trial. After just 13 weeks, his role in the drug trial was over.
"I went off the drug May 30. On May 31 I woke up with -- I felt like I had been taken out and pummeled," he said. "I was so sore, my arms my legs. I had a horrible headache. That headache has lasted over two weeks."
The headache is mostly gone, but now the neck brace is back.
"To me, clearly there was some effect from whatever was happening," Kramer said. But then he was "ripped off the drug, literally cold turkey off the drug" with no recourse. "I have given a great commitment, and the people in this trial and the people in the Dexapremipexole trial, or other trials, have given great commitment to the protocols and to the companies that are funding it, but we're the ones that are doing it. And to not feel any obligation to continue if somebody were to ask for the drug under study, or for whatever it was they were taking, strikes me as a little disingenuous."
He takes a realistic tack when asked whether it makes sense for pharmaceutical companies to end drug trials when they are not exactly sure of a drug's long-term effects.
"I have ALS. Come on," he said with a laugh. "Sure, that's an argument there. But you know, what possible problem do we have here with a person with a disease like mine? ALS is killing me. I don't think I'm going to be one of the people with ALS who lasts a long, long time, so why not make the drug available?"
Kramer recently delivered a lecture to first year medical students at the University of Minnesota, and as part of the presentation talked a little bit about being in medical trials. Dr. Ezgi Tiriyaki, his host, told the students how amazing it was that he and certain other patients had gone through the trials because it was so unusual and difficult.
"It's hard to get people with ALS to do them," Kramer said. "I personally feel -- and I know that people who are in the hard sciences disagree with what I'm about to say -- but I personally feel that our models, the way we go about testing drugs, particularly for diseases like ALS, are probably flawed."
"The problem is that with ALS you have so many things cascading down in so many different ways. It presents itself in so many different forms that what might work for me, probably wouldn't work for you, or wouldn't work the same way with you," he said. "But with something like ALS -- and when you put it into this gold standard, double blind placebo controlled way of looking at it -- it probably is not going to show the effect that it might show if you're able to set the study up in a different way."
At this point, Kramer doesn't think he's eligible for another drug study. In the most recent entry on his blog, The Dis Ease Diary, he posted a letter to officials with Cytokinetics, the developer of Tirasemtiv, with copies to various lawmakers about his experiences in this latest trial, suggesting they reconsider their course of action.
"I'm not saying I have the right answers here. I keep coming back to the fact that we have one drug," Kramer said. "In the 160 years since we've known about ALS, the scientific method continues to belly up to ALS and we continue to do it expecting different results and there are people who are really into doing it that way and they will tell you it's just because we haven't found the right substances. And my thought is, maybe it's because we're not going about it in the correct way. So I'm hoping it'll spark some dialogue."
At last word, Kramer says officials at Cytokinetics quickly responded to his letter and are taking his request for the drug under consideration.