Thank God it’s Friday, Friday, Friday! I would ‘sing’ the lyrics to you but I just looked them up and they are really rather uninspired. Good memories with the music, though. In 1978, I was a disco queen! Anyone else who had a totally enjoyable, misspent youth in the clubs? Those were the salad days.
Anyway, I am really packing tight three days a week at the counseling office. Still not enough clients for four. That means I will drive Lin crazy with many, many pages. Pray I get more work so she gets a break! [Lin/Linda: Please pray she gets more work! Pleasssseeee!]
Derek Daniel has Leber Hereditary Optic Neuropathy (LHON) and, true to form, when I learned about something, suddenly it is like Underdog. It’s everywhere. It’s everywhere!
Healio reported GenSight just announced more information on their REVERSE phase 3 clinical trial results. It turns out this clinical trial assessing the efficacy of a treatment for LHON did not meet primary endpoint goals but did meet several secondary endpoint goals.
What does that mean? eSearch tells us primary endpoints are results that will answer our most important questions for the research. Secondary endpoints are about other questions. They are not necessarily what we were trying to find out but the answers are generally kinda cool and good to know.
For example, contrast sensitivity almost doubled after treatment… Hey! We have problems with that! Maybe this treatment for LHON will have some crossover potential. Their secondary outcome measure may prove to be a boon for us.
LHON is a mitochondrial disorder with a maternal inheritance. In other words, it is a disorder of the powerhouse of the cells. (Mitochondria turn energy from food into a form that cells can use). Since the mitochondrial DNA comes to us through the maternal line only, it stands to reason the inheritance for LHON is through the maternal line.
Doesn’t sound like us; right? Stay with me. I do have something in mind.
The Healio page sent me to a 2012 journal article on Leber hereditary optic neuropathy and oxidative stress. After several paragraphs about how they produced mutant, transgender, blind mice (don’t ask me) to study the disorder, they got to what is, for me, the crux of the matter. Specifically, they are thinking miscoding in the mitochondria leads to this-then-that and then to super oxidation and then all hell breaks loose. In other words, they are thinking oxidative stress is a huge causative factor in yet one more eye condition! There is a pattern forming here.
They are reported to be experimenting with ways of reducing oxidative stress in those with LHON. There is evidence that should slow the progress of their condition.
Of course, as of 2012, they were still talking leafy greens and AREDS2, but that does not mean there will not be other ways of doing this the researchers will discover something. They have to be pretty clever; right? After all, a bunch of folks who say “Hey, let’s make a mutant, transgender, blind mouse!” and then actually do it have to be pretty smart.
Just glad they are on our side.
Written June 17th, 2018
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