Overview: A hopeful step toward remyelination in MS
Multiple sclerosis (MS) is a chronic autoimmune disease that disrupts nerve signals when the immune system damages the myelin sheath, the protective insulation around nerve fibers. More than 2.9 million people worldwide live with MS, and symptoms can range from numbness and vision loss to paralysis. While existing therapies reduce inflammation, there has been a long-standing need for treatments that protect neurons and actively repair myelin. Recent research supported by the National MS Society marks a pivotal advance: two drug candidates showing potential to promote remyelination and modulate immune responses are moving toward clinical evaluation.
From discovery to development
The project builds on prior work with indazole chloride, a compound known to foster remyelination and influence immune function in mouse models. However, indazole chloride lacked the pharmacological properties and patentability needed for clinical development. In a collaboration between UC Riverside’s (UCR) Seema Tiwari-Woodruff and UIUC chemist John Katzenellenbogen, as well as Sung Hoon Kim, researchers synthesized more than 60 analogs and identified two lead candidates: K102 and K110. These compounds demonstrated improved safety, efficacy, and “drug-like” properties in both mouse models and human cells.
Among the two, K102 emerged as the lead candidate. It not only promotes remyelination but also modulates immune function—an important balance for MS therapies. Importantly, human oligodendrocytes derived from induced pluripotent stem cells showed promising responses to K102, suggesting better translation 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 repair process often stalls, leading to lasting nerve damage and disability. A successful remyelinating treatment could restore faster nerve conduction, potentially reducing long-term disability and improving quality of life for people with MS.
In addition to K102, the second candidate, K110, has distinct central nervous system effects that may make it suitable for other conditions—such as spinal cord injury or traumatic brain injury—while still representing a strong therapeutic option within the same program.
From bench to biotech
The National MS Society’s Fast Forward program played a crucial role in accelerating this work, enabling two grants that funded essential data generation and collaboration. This support helped license the program to Cadenza Bio, which continues to develop K102 and K110 and advance toward non-clinical studies and first-in-human trials. The patents are jointly held by UCR and UIUC, with exclusive worldwide licensing to Cadenza Bio.
Researchers emphasize that this progress reflects more than a decade of collaboration. The team’s goal is to translate discovery into a real-world therapy for MS and possibly other neurological diseases involving demyelination. As trials approach, there is cautious optimism that K102 and K110 could become first-in-class therapies for people living with MS.
Looking ahead
Current work suggests K102 is the strongest lead, yet both candidates represent meaningful advances in remyelination research. The compounds’ ability to promote myelin repair in human-derived cells signals promise for future clinical testing, where safety, efficacy, and real-world impact will be the defining hurdles. If successful, this line of therapy could complement existing anti-inflammatory treatments by addressing the underlying damage to nerve insulation.
Beyond MS, researchers anticipate potential applications in other neurological conditions characterized by neuronal loss or demyelination, including stroke and general neurodegeneration. The ongoing partnership among academia and industry—supported by the National MS Society and invested by Cadenza Bio—illustrates how translational science can move discoveries from the lab toward tangible patient benefits.
As the team awaits non-clinical data and regulatory milestones, the forward path remains focused on responsible development and patient-centered outcomes. The collaboration between UCR, UIUC, and industry partners exemplifies a model for turning scientific insights into therapies that could one day restore myelin and improve lives for those affected by MS.
