Multiple sclerosis (MS) affects millions worldwide, damaging the protective myelin sheath around nerve fibers. While myelin can naturally regrow through exposure to thyroid hormones, researchers have historically avoided thyroid hormone therapies due to severe side effects.
However, recent breakthroughs have reignited hope for MS patients. Scientists at Oregon Health & Science University have developed a compound called sobetirome that stimulates myelin repair without the adverse effects associated with thyroid hormone therapy.
Sobetirome promotes remyelination by:
• Targeting the central nervous system selectively
• Avoiding systemic side effects of thyroid hormone therapy
• Penetrating the blood-brain barrier efficiently
Researchers also created Sob-AM2, a derivative of sobetirome that increases infiltration to the central nervous system tenfold.
In mouse models of MS, sobetirome treatment led to:
• Significant myelin repair
• Substantial motor improvements
• Near full recovery in some cases
These findings suggest that sobetirome could potentially revolutionize MS treatment by addressing the root cause of the disease rather than just managing symptoms.
While the results are encouraging, it may take several years before sobetirome advances to human clinical trials. However, researchers are optimistic about its potential to translate from mice to people.
OHSU has licensed the technology to Llama Therapeutics Inc., which is working to advance these molecules toward human clinical trials for MS and other diseases.
If proven effective in humans, sobetirome could offer several advantages over current MS treatments:
• Direct myelin repair
• Minimal side effects
• Potential to halt or reverse disease progression
While a cure for MS remains elusive, this breakthrough provides renewed hope for patients and researchers alike in the quest for more effective treatments.
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