Many of the estimated 1 million people in the U.S. with multiple sclerosis deal with eye and vision problems, as well as others with certain neurological conditions. A new drug may be able to restore vision for people battling these illnesses.
Researchers from the University of Colorado Anschutz Medical Campus published a study about the promising medication, LL-341070, in the Nature journal. This drug is described in the study as thyromimetic and a “highly potent remyelination therapeutic.”
Myelin is an insulating sheath that forms around nerves, made of a protein and fatty substances. It allows electrical impulse to transmit quickly and efficiently along nerve cells and if it is damaged the impulses can slow down, the National Library of Medicine explained. This in turn can cause disease such as MS and can result in vision loss.
On the other hand, remyelination “is the phenomenon by which new myelin sheaths are generated around axons in the adult central nervous system (CNS),” following the loss of myelin in diseases like MS, according to an article from the International Review of Neurobiology. Although remyelination occurs in many MS lesions, it “becomes increasingly incomplete/inadequate and eventually fails in the majority of lesions and patients,” said the article.
“Efforts to understand the causes for this failure of regeneration have fueled research into the biology of remyelination and the complex, interdependent cellular and molecular factors that regulate this process,” it added.
This recent study is one of these efforts. Study authors said their research has advanced understanding of therapeutic-induced remyelination in particular.
“We found that distinct drugs stimulate oligodendrocyte gain with different temporal dynamics they said, referring to the oligodendrocyte cells that form the myelin sheath around axons in the central nervous system. The researchers also added that: “Importantly, we demonstrated that the new thyromimetic LL-341070… accelerates oligodendrocyte regeneration and recovery of neuronal function.”
In this way, the drug “enhances the brain’s ability to repair damaged myelin,” a press release explained. Myelin loss is also associated with loss of motor skills and cognitive decline, but this research was focused on vision. It indicates that the brain is able to repair itself when myelin is damaged, but that the process becomes slow and inefficient, issues that were mitigated by LL-341070.
“Researchers observed that LL-341070 significantly accelerated the repair process and improved brain function related to vision in mice, even after severe damage,” said the press release.
Ethan Hughes, PhD, co-lead author and associate professor in the Department of Cell and Developmental Biology at the CU School of Medicine, said the research is a step towards an ultimate goal of allowing the brain to have the capacity to heal itself.
“By harnessing this potential, we hope to help people with diseases like MS by potentially reversing some of the damage, offering people the opportunity to regain their vision and cognitive function,” he said. He also said this study is “just the beginning.”
Daniel Denman, PhD, co-lead author of the study and assistant professor in the Department of Physiology and Biophysics at the CU School of Medicine, said the new drug “could be a game-changer.”
Furthermore, the research demonstrated the importance of intervention with severe injury, said the press release. Even a partial repair of myelin significantly improved vision-related brain functions, it explained.
Going forward, the researchers plan to test the drug in other areas of the brain.