Dr. Moser had published an article[1] that suggested that if pre-symptomatic boys received Lorenzo’s Oil, they had a reduced chance of developing the bad stuff associated with adrenoleukodystrophy. Problem: this stuff was hard to come by, in the U.S., anyway. I wondered, could this deficiency actually be a novel angle?
Expectant and new parents, with good reason, may well be the most anxious lot ever. I recognized that the pleas of a few men struggling with AMN, were drowned out by the urgent screams of hundreds of nervous moms and dads who just learned that their child had a deadly disease with no cure. Maybe this reality might benefit everyone? Sort of a rising tide lifting all ships sort of thing?
Well, it was fine in theory, but I knew that without a proper sponsor to push for the registration of the Oil as an approved drug, it was a steep hill. And the money thing…
Anyway, as it was, there were only a handful of childhood diagnoses each year. From what I remember, bone marrow transplants were not as common in those days as the are now- the Oil seemed to be the main hope for plenty of boys. And it seemed to work out: from what I understood, Kennedy-Krieger was enrolling many of these kids in their trial, providing them with the Oil. The supply was doing a fine job of meeting the demand.
That status quo persisted until sometime in 2010. I learned that NIH, which was funding much of this work, was tightening its belt. As a result, many of their grants were reduced or eliminated. The upshot of this was that Kennedy-Krieger would discontinue nearly 200 boys from its trial. While I had thought that an army of anxious new parents could turn the tide, when faced with this reality, I got sad, then mad.
Somehow, the selfishness of my own survival finally shifted. The thought of these young boys, deprived of their lifesaving therapy could not be allowed. The age-old question: “what to do?” I was just one person. I did not know anyone rich or famous. So far as raising money: no idea. Still, I guess it was my unrealistic optimism: if I could get it started, others would jump on board.
My first idea took root while laying awake, jet lagged in Spain. I picked out my pitch, two fingers at a time, on a BlackBerry, to my dear friend Trisha Doyle, a documentary film producer that I had known for years. I finally went to sleep and quickly forgot about my crazy thinkings.
I guess I must have said the right things, because sometime later, Trisha got back to me. Somehow, she had gotten funding to include me in a rare disease documentary project.
Was this the break? I was not Brad Pitt, that I was sure of. Nonetheless, she was making a film. This would be the platform I needed to tell my story. This would certainly attract the interest and money needed to properly develop the Oil.
[1] Moser HW, Raymond RV, Lu SE, et al. Follow-up of 89 asymptomatic patients with adrenoleukodystrophy treated with lorenzo’s oil. Arch Neurol. 2005;62:1073-80
Walking the Rails 
Reblogged this on Survivors Blog Here Mental Health Collaborative .
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