New experimental compounds show potential to repair nerve damage in multiple sclerosis

Researchers in the United States have identified two experimental compounds that may help repair the nerve damage caused by multiple sclerosis (MS), a chronic autoimmune disease that affects millions of people worldwide. If the early findings translate to humans, the treatment approach could go beyond slowing inflammation and actually help restore the protective coating around nerves.

The compounds, known as K102 and K110, were developed after more than a decade of collaborative work and are now being prepared for clinical testing. Scientists hope they could become the first therapies specifically designed to rebuild damaged myelin, the insulating layer that is attacked in MS.

How multiple sclerosis damages nerves

MS is an autoimmune condition in which the immune system mistakenly targets the myelin sheath, a fatty protective layer that surrounds nerve fibers in the brain and spinal cord. When myelin is damaged, electrical signals no longer travel efficiently along nerves, disrupting communication between the brain and the rest of the body.

This disruption can lead to a wide range of symptoms, including numbness, tingling, vision problems, muscle weakness, difficulty with coordination, and in severe cases, paralysis. More than 2.9 million people worldwide are estimated to be living with MS.

Many current MS medications focus on reducing inflammation and moderating immune activity, which can help slow disease progression and reduce relapses. However, there are still no approved treatments that reliably protect nerve cells or actively repair the damaged myelin that surrounds them.

Discovering K102 and K110

The newly reported work, published in the journal Scientific Reports, was led by Seema Tiwari-Woodruff of the University of California, Riverside School of Medicine, and chemist John Katzenellenbogen of the University of Illinois Urbana-Champaign. Their teams received support from the National Multiple Sclerosis Society through both a traditional research grant and the organization’s Fast Forward program, which aims to speed up the development of promising therapies.

The study grew out of earlier research on a molecule called indazole chloride. In animal models that mimic MS, indazole chloride had shown an ability to support myelin repair and influence immune responses. Despite this, it did not have the right properties to move forward as a practical drug candidate or to be protected effectively by patents.

To overcome these limitations, chemists at the University of Illinois Urbana-Champaign, including Katzenellenbogen and Sung Hoon Kim, designed more than 60 structural variations of indazole chloride. A team led by UC Riverside researcher Micah Feri then tested these analogs in laboratory and animal experiments.

From this large set of candidates, two compounds stood out: K102 and K110. Both showed improved safety profiles, stronger effects, and better “drug-like” characteristics compared with the original molecule when tested in mice and in human cells.

K102: a leading candidate for myelin repair

Among the two promising molecules, K102 emerged as the primary candidate for further development. In preclinical studies, K102 appeared to promote remyelination, the biological process in which damaged myelin is rebuilt around nerve fibers.

The compound also showed an important additional effect: it helped regulate immune activity. Because MS involves both immune dysfunction and damage to myelin, a therapy that addresses both aspects of the disease could be particularly valuable.

K102 was tested not only in animal models but also in human oligodendrocytes derived from induced pluripotent stem cells. Oligodendrocytes are the cells in the central nervous system that produce myelin. Under normal conditions, precursor cells mature into fully functioning oligodendrocytes that restore myelin after injury. In MS, this repair process frequently fails, leaving nerves exposed and vulnerable to lasting damage.

By enhancing the function or maturation of these myelin-producing cells, a compound like K102 could help improve signal conduction along nerves and potentially reduce long-term disability. The positive results in human-derived cells suggest that the findings from animal studies may be more likely to apply to human disease, although this still needs to be tested in clinical trials.

K110 and possible uses beyond MS

K110 also showed strong potential in preclinical testing. According to the researchers, it has somewhat different effects in the central nervous system compared with K102. Because of these characteristics, K110 may be more suitable for other conditions in which myelin or nerve fibers are damaged, such as spinal cord injury or traumatic brain injury.

For now, K110 remains in development as part of a broader pipeline of possible therapies, while K102 is being prioritized for movement into human studies for MS.

From academic lab to biotechnology company

The transition from basic discovery to potential therapy has been supported by the National Multiple Sclerosis Society’s Fast Forward program, which encourages collaboration between university researchers and industry partners. Funding from this initiative allowed the team to generate the data needed to attract commercial interest.

The intellectual property related to K102 and K110 is jointly owned by the University of California, Riverside and the University of Illinois Urbana-Champaign. The universities have granted an exclusive worldwide license to Cadenza Bio, Inc., a biotechnology company that is now driving the next stages of development.

Technology transfer offices at both universities, including UC Riverside’s Office of Technology Partnerships, worked together to secure patent protection and present the opportunity to potential investors. Entrepreneurs-in-residence advised the project team on how to frame the scientific progress in terms of its clinical and commercial potential, highlighting the lack of existing therapies that can repair axon and myelin damage in MS.

Executives at Cadenza Bio were particularly interested in the possibility of shifting from simply slowing nerve damage to actively repairing it. With investor backing, the company is now performing the non-clinical studies needed before first-in-human trials can begin.

A long-term collaboration aimed at real-world impact

The development of K102 and K110 is the result of more than 12 years of collaboration between Tiwari-Woodruff and Katzenellenbogen. Tiwari-Woodruff’s move from UCLA to UC Riverside in 2014 played a key role in advancing the work, as she has credited the institutional support and research infrastructure at UC Riverside with helping the project grow.

The researchers emphasize that sustained funding for academic laboratories is essential for this type of long-term, translational project. Their goal has been to take a basic scientific insight—how to influence myelin repair and immune function—and carry it all the way toward a therapy that might eventually help people living with MS.

Although the initial target is MS, the team believes that the same strategy could potentially be adapted to other neurological conditions marked by demyelination or nerve damage, including stroke and certain neurodegenerative diseases. Those possibilities will depend on the outcomes of future studies.

What happens next

Cadenza Bio is currently advancing K102 through the regulatory and safety testing required before it can be given to human volunteers in formal clinical trials. These non-clinical studies are designed to examine factors such as dosing, potential side effects, and how the compound behaves in the body.

If the necessary safety and regulatory milestones are met, early-phase trials in people with MS could begin, focusing first on safety and then on signals of effectiveness. Only through these stepwise clinical stages will researchers learn whether the encouraging results seen in animal models and human cell systems translate into meaningful benefits for patients.

The research has also received support from the U.S. National Institutes of Health and from Cadenza Bio. The study brought together scientists from UC Riverside, the University of Illinois Urbana-Champaign, The Scripps Research Institute in Florida, and Cadenza Bio in Oklahoma, reflecting the highly collaborative nature of modern drug development.

While much work remains, the progress of K102 and K110 marks an important step toward therapies that not only slow MS, but may eventually help repair the damage it leaves behind.