Breakthrough Compounds Show Promise in Repairing Nerve Damage in Multiple Sclerosis
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A new generation of therapies targeting myelin repair is on the horizon, offering hope for the over 2.9 million people worldwide living with multiple sclerosis (MS). Scientists have identified two compounds, K102 and K110, capable of promoting remyelination – the process of rebuilding the protective coating around nerve fibers damaged by the autoimmune disease.
Multiple sclerosis is a chronic, debilitating condition where the immune system mistakenly attacks the myelin sheath, leading to communication disruptions between the brain and body. Symptoms range from numbness and tingling to vision problems and paralysis. While current treatments focus on managing inflammation, a true repair mechanism has remained elusive – until now.
A decade of Collaboration Yields Promising Results
The research, published in Scientific Reports, is the culmination of over a decade of work led by Seema Tiwari-Woodruff, a professor of biomedical sciences at the University of California, Riverside, School of Medicine, and John Katzenellenbogen, a professor of chemistry at the University of Illinois urbana-Champaign (UIUC). The project received crucial funding from the National multiple Sclerosis Society, including support from its Fast Forward program, designed to accelerate the commercialization of promising research.
“Our work represents more than a decade of collaboration, with the last four years focused on identifying and optimizing new drug candidates that show strong potential to treat MS and possibly other neurological diseases involving demyelination,” Tiwari-Woodruff stated.
From Indazole Chloride to K102 and K110
The team’s success builds upon earlier studies involving a compound called indazole chloride, which demonstrated initial promise in promoting myelin repair in mouse models. Though, indazole chloride lacked the necessary chemical properties and patent potential for clinical progress.
Working with UIUC chemists Sung Hoon Kim and Katzenellenbogen, Tiwari-Woodruff’s team, spearheaded by recent UC Riverside graduate Micah Feri, screened over 60 analogs of indazole chloride. This rigorous process identified K102 and K110 as particularly promising candidates. These compounds exhibited improved potency, selectivity, and drug-like properties compared to their predecessor.
Securing Investment and Moving Towards Clinical Trials
Recognizing the potential of K102 and K110, the universities filed for patent protection, and actively promoted the technology to investors. According to Grace Yee, assistant director of technology commercialization at UCR, the combined efforts of the universities and the National MS Society were instrumental in attracting investment and ultimately securing the licensing agreement with Cadenza Bio.
Elaine Hamm, chief operating officer at Cadenza Bio, expressed excitement about the potential to move beyond slowing axon damage to actively repairing it. “This is the future we want to build,” Hamm said.”It is why we licensed the technology, and why we are excited to move it forward to patients in need.”
A Legacy of Collaboration and a Hopeful Future
Tiwari-Woodruff and Katzenellenbogen have collaborated for over 12 years, with Tiwari-Woodruff’s move to UCR in 2014 proving to be a turning point. She emphasized the extraordinary support received from UCR, stating, “None of this would’ve been possible without that backing. Funding for academic labs like mine and John’s is crucial. This is selfless work, driven by a deep love of science and commitment to human health.”
while the initial focus remains on MS, the team envisions potential applications for K102 and K110 in other neurological conditions, including stroke and neurodegeneration.
Cadenza Bio is currently conducting the necessary non-clinical studies to support first-in-human clinical trials. “We’re hopeful that clinical trials can begin soon,” said Tiwari-Woodruff. “It’s been a long journey – but this is what translational science is all about: turning discovery into real-world impact.”
