It’s rare that we highlight medicine 2.0, but we couldn’t help but highlight this story. Researchers at OHSU ( Oregon Health and Science University ) have shown regeneration of the myelin sheath after experimenting with a 20 year old drug that has seen very little use.
The drug is sobetirome, originally invented as a means of lowering cholesterol. The original drug was superseded by blockbuster pharmaceuticals like Lipitor, and consequently left on the shelf; until in 2013, the idea that it might be useful in the treatment of Multiple Sclerosis came about. Early lab tests showed some success in mice – but there was concern that the drug would damage the bones, muscle, and heart.
So what changed? Researchers at OSHU added a chemical tag to the sobetirome molecule which eliminated it’s negative electrical charge. This allows the drug to pass-through the brain-blood barrier much more effectively, meaning that the effective dose for Multiple Sclerosis could be 10 times lower – vastly reducing the concern about side effects. In a new test with the improved drug, mice showed substantial motor improvements, showing close to a full recovery.
The researchers behind the therapy – Scanlan and Bourdette – are confident that the drug will work on humans as well. The University has licensed the technology to Llama Therapeutics Inc., who is now putting the drug through human trials.
For more information, please see OHSU’s news story here: https://news.ohsu.edu/2019/04/18/study-shows-promise-in-repairing-damaged-myelin