Overview: A hopeful step toward remyelination in MS
Multiple sclerosis (MS) is a chronic autoimmune disease characterized by damage to the myelin sheath that insulates nerve fibers. This protective layer is essential for fast and efficient nerve signaling between the brain and body. In MS, the immune system often attacks myelin, leading to a range of symptoms such as numbness, vision changes, and mobility challenges. While current therapies can reduce inflammation and new flare-ups, there are no approved treatments that reliably protect neurons or restore damaged myelin. A recent line of research, supported by the National MS Society, has identified two drug candidates with the potential to promote remyelination and improve nerve function.
The discovery: Two lead candidates for remyelination
The study, published in Scientific Reports, was led by Seema Tiwari-Woodruff, a professor of biomedical sciences at the University of California, Riverside, and John Katzenellenbogen, a chemist at the University of Illinois Urbana-Champaign. The work built on earlier findings around indazole chloride, a compound known to promote remyelination in mouse models but lacking the pharmacological properties for clinical development. By screening more than 60 analogs of indazole chloride in collaboration with UIUC chemists Sung Hoon Kim and John Katzenellenbogen, the team identified two lead candidates: K102 and K110.
Of the two, K102 emerged as the lead compound. Researchers found that K102 not only promotes remyelination but also modulates immune function—an important balance for MS therapies. In tests with human cells, K102 supported remyelination in oligodendrocyte precursor cells differentiating into myelin-producing oligodendrocytes, suggesting potential translatability from animal models to human disease.
Why remyelination matters in MS
Oligodendrocyte precursor cells normally mature into myelin-producing oligodendrocytes to repair damaged myelin. In MS, this regenerative process often falters, leading to lasting nerve damage and disability. A therapy that enhances remyelination could accelerate nerve signal conduction and potentially reduce long-term disability for people living with MS.
Dual potential: K102 and K110
K110 is described as a strong candidate with slightly different central nervous system (CNS) effects, possibly making it suitable for other conditions such as spinal cord injury or traumatic brain injury. Keeping both compounds in the pipeline aligns with a strategy to tailor remyelination therapies to diverse CNS injuries and diseases while advancing toward clinical evaluation.
From academia to industry: Accelerating translation
The National MS Society’s Fast Forward program played a pivotal role by speeding the transition from discovery to development. The program supports academic-industry partnerships and provided the data necessary to license the technology to Cadenza Bio, Inc. The exclusive, worldwide license has positioned Cadenza Bio to advance K102 and K110 into non-clinical studies required to support first-in-human trials. The collaboration benefited from traditional investigator-initiated grants as well as the Fast Forward accelerator, illustrating how sustained funding can move promising science toward patient impact.
Building a path to clinical trials
With financial backing and a clear regulatory pathway, K102 is advancing through non-clinical studies aimed at establishing safety, pharmacokinetics, and dosing strategies. Researchers hope to begin clinical evaluation soon, transforming remyelination science into a first-in-class therapy for MS. While the immediate focus is MS, the team believes the same approach could apply to other neurological diseases involving demyelination and axon damage, broadening the potential impact of this work.
Broader context and future directions
Grants from the National Institutes of Health and ongoing investment in the program have underwritten the collaboration among the University of California, Riverside; the University of Illinois Urbana-Champaign; and Cadenza Bio. The partnership highlights a shift in neurodegenerative research—from slowing disease progression to repairing damaged neural tissue. If clinical trials demonstrate safety and efficacy, K102 could become a first-in-class remyelination therapy that helps restore nerve conduction and improve quality of life for people with MS. Beyond MS, the approach may inform treatments for stroke and other neurodegenerative conditions where myelin is compromised.
Conclusion: A long journey toward real-world impact
After more than a decade of collaboration, the teams are closer than ever to translating bench science into tangible patient benefits. As researchers prepare for non-clinical studies and eventual human trials, the MS community remains hopeful that repairing myelin could redefine the standard of care for MS and related CNS injuries.
