Promising New Compounds Show Potential for Remyelination in Multiple Sclerosis Treatment

Multiple sclerosis (MS) is a chronic autoimmune disorder affecting over 2.9 million people worldwide. It occurs when the immune system mistakenly attacks the myelin sheath—the protective covering of nerve fibers—disrupting nerve signaling between the brain and the body. This leads to a variety of symptoms, including numbness, vision problems, tingling sensations, and even paralysis.

Current therapies primarily aim to reduce inflammation and manage symptoms, but they do not effectively protect nerve cells or repair existing damage to the myelin sheath. Recent research offers hope with the discovery of two experimental compounds that could promote remyelination, potentially restoring nerve function and slowing disease progression.

Published in Scientific Reports, the study was led by Seema Tiwari-Woodruff of the University of California, Riverside, and John Katzenellenbogen from the University of Illinois Urbana-Champaign. The research team focused on developing new drug candidates capable of repairing damage caused by MS. After years of collaboration, they identified two promising compounds, K102 and K110, which demonstrated improved safety, efficacy, and drug-like properties in both animal models and human cells.

K102, in particular, has shown remarkable potential to enhance remyelination by promoting the maturation of oligodendrocyte precursor cells into myelin-producing oligodendrocytes. Additionally, it modulates immune responses, which is essential in balancing immune activity in MS. Human oligodendrocytes derived from pluripotent stem cells responded positively to K102, suggesting strong potential for translation into human therapies.

The development of these compounds builds on earlier research involving indazole chloride, a molecule known for promoting remyelination and immune modulation. However, indazole chloride lacked the necessary properties for clinical development, prompting the team to synthesize and screen over 60 analogs. This resulted in the identification of K102 and K110 as leading candidates.

K110 differs slightly in its effects within the central nervous system and could be more suitable for conditions involving spinal cord injuries or traumatic brain injuries, highlighting its versatility.

This research represents over a decade of dedicated effort, with the potential to significantly impact not only MS treatment but also other neurological conditions involving nerve damage, such as stroke and neurodegenerative diseases. The team at Cadenza Bio is now moving K102 into further preclinical studies necessary for first-in-human clinical trials. The hope is that these compounds will eventually provide a groundbreaking, disease-modifying therapy for MS patients.

This breakthrough exemplifies translational science—transforming laboratory discoveries into practical medical solutions that could improve the lives of millions affected by neurological diseases.

[source: https://medicalxpress.com/news/2025-10-experimental-compounds-benefit-people-multiple.html]