Derailed

The plan was a 4-year study, but 3.5 years in, I got a letter ordering me to stop taking the oil immediately, no explanation. I later learned that the IRB shut it down because enrolment was slow, inadequate patient retention and a safety issue. Turns out the safety thing was in a placebo patient, but the other problems were dealbreakers. Ugh. Aggravation. The smartest, most dedicated people with the best chance to find a cure could not do their work. Maybe it would be ok, but I was concerned that the sample size was too small. As I do, I began to troubleshoot. The investigators were topnotch, nope, not that. I thought about Dr. Moser begging NIH for money, and the penny pinching that went on when they were setting the trial up. Of course, it was like any business; If underfunded, it would fail.

My greatest fear had always been the single-site situation. I had worked on enough trials to know that enrolling 200 subjects at one study center would be a challenge. And it was. Enrolment seemed to peak at around 50 per year. This was plenty crazy, with a skeleton crew managing this influx of new patients, while still taking care of us returning for follow up visits.

The reasons did not really matter- they study was over. It had failed to execute. So, this was not going to be the way to test the Lorenzo’s Oil hypothesis. It was an unfair fight from the start. I went from sad to mad.

I guess if you think about it, it was sort of ridiculous of me to think that Dr Moser, relying on government grants, could hope to get a drug approved. Now, it was clear, that the only way that this was going to get done was to get big money interested in AMN.

At the time, the Orphan Drug act had been around for a while, but I had seen only a few projects reach fruition. Even then, an economic demon remained. No matter the indication, drug development is not cheap. And, with a limited pool of patients to recover their investment, if something did get approved, the sponsor would have no choice but to sell the drug at a cost that would be out of reach for many. This distracted me further, to contemplate the unfairness of a wealthy country with such great inequities.

Taken in composite, plenty to explode my mind. It left me a bit dazed, without a way to figure my way out of this mess. Either way, it occurred to me that if left to pure capitalism, I was not going to get what I wanted.

But maybe I was overreacting? They had managed to collect bunches of information. Maybe, just maybe, if the effect was large enough, even the truncated study might show something. Even a hint of activity could do it. I pushed myself, hard, to get optimistic. Though it was not easy for me. I had spent 15 years dreaming of this study, and the rug had been yanked out from under me. This outcome made me feel sabotaged, bushwhacked, hijacked, I don’t know, but not a good thing.

Published by bradleygillespie

I am just a guy with a disease called adrenomyeloneuropathy. I want other guys with the disease to see the good parts of disability. Not the gloom. Not the doom. Make sense?

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