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New hope is emerging for the millions of people affected by multiple sclerosis (MS). A recent scientific breakthrough could reverse the symptoms of this neurodegenerative disease by regenerating myelin, the protective sheath around nerves that is often damaged in MS patients. That’s according to researchers at the University of California, San Francisco (UCSF), who have developed a promising drug that can repair the damage caused by the disease.
While multiple sclerosis has no curative treatment, a study has revived the great hope of being able to regenerate the myelin sheath damaged in this disease. Zoom in on a great hope for millions of patients.
A significant advance in the treatment of MS
Multiple sclerosis, which affects about 2.8 million people worldwide, is an autoimmune disease in which the immune system attacks myelin, a substance that surrounds and protects nerve fibers in the brain and spinal cord. Without this protection, nerve signals slow down or are completely blocked, causing symptoms ranging from fatigue to paralysis.
Current treatments focus on slowing the progression of the disease and managing symptoms. However, this new approach could go much further. According to the results published by UCSF, the new drug could not only stop the progression of MS but also regenerate damaged myelin, thereby reversing symptoms.
The revolutionary mode of action of the new drug
Patients with multiple sclerosis (MS) experience a progressive loss of oligodendrocytes, the cells responsible for producing myelin. However, a new therapeutic approach, called PIPE-307 and derived from clemastine, a drug that has already been shown to be effective against the disease, aims to activate a specific receptor (M1R) present on certain inactive cells in the brain. By blocking this receptor, the therapy encourages these cells to transform into oligodendrocytes, capable of wrapping around axons to regenerate the myelin sheath.
Surprising fact noted in the story of their discovery on the UCSF websitethe exact localization of the M1R receptor in the brain was made possible by the MT7 toxin, which is extracted from the venom of the green mamba snake.
Tests in animal models have shown promising results. Not only was myelin regenerated, but the animals’ motor skills were also restored. These findings raise the possibility of developing a treatment that could repair the damage in human patients, a prospect long considered impossible.
Clinical trials in humans underway
Despite these advances, there is still a long way to go before this drug can be available to patients. In 2021, PIPE-307 successfully completed two Phase I clinical trials, demonstrating its safety. It is currently being tested in Phase II in patients with MS. If clinical trials confirm these initial observations, we could be witnessing a true revolution in the treatment of MS.
The implications of such a treatment are immense. For the millions of people battling MS, the hope of regaining lost function could soon become a reality. This treatment could also pave the way for similar approaches for other neurodegenerative diseases, marking a major turning point in regenerative medicine.