It was on the website, myelin.org (now defunct), that I first learned about Sobetrirome and it's role in upregulating ABCD2.
I believe myelin.org was run by Lorenzo Odone's parents. It was there that I first read about Ampyra as well. But, I digress.
It was originally envisaged that sobetirome could be used to stimulate hepatic pathways that lower cholesterol without harmful side effects and might be used in conjunction with statins.
Obviously, they didn't bother with Sobetrirome, and just went with statins to lower cholesterol levels.
There is evidence for Lovastatin lowering VLCFA levels.
adrenoleukodystrophy.info/t....
Just not good enough.
But, how about Sob-AM2?
Sob-AM2 delivers a more potent and effective therapy for the most common type of ALD – adrenomyeloneuropathy (AMN) – than the original drug sobetirome.
Sob-AM2 is a chemical compound created from sobetirome.
blogs.ohsu.edu/researchnews...
ncbi.nlm.nih.gov/pmc/articl...
Lowers ABCD2 and promotes remyelination.
Here is a long copy/paste:
"Sobetirome and Sob-AM2 work because they activate or upregulate ABCD2, a gene which is classically thyroid hormone-regulated,” said Scanlan.
In their dose-response study, the team treated ABCD1-deficient mice with Sob-AM2, which activated ABCD2 to correct the ABCD1 defects. They measured the levels of VLCFAs in the brain and spinal cord, as well as blood and organs.
“We found that Sob-AM2 was more potent than sobetirome at reducing elevated VLCFA in the CNS, particularly in the spinal cord which is relevant for AMN,” said Scanlan. “Sob-AM2 also reduced elevated VLCFA in blood and peripheral tissue, but not as potently as sobetirome, which makes sense because Sob-AM2 targets the CNS and sobetirome targets the periphery.”
The findings established that Sob-AM2 provides significant and sustained reductions to the elevated levels of VLCFA that are responsible for the pathology in ALD. The next step is clinical trials in patients.
Looks very promising indeed.
Are carrier symptoms just "mild"
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