Breakthrough in Multiple Sclerosis Research: Repurposed Drug “Bavisant” Shows Promise in Progressive Forms of the disease
A novel approach to drug finding,leveraging artificial intelligence and existing medications,has yielded a promising candidate for treating progressive multiple sclerosis (MS),a debilitating condition affecting over a million people worldwide. Researchers have identified “Bavisant,” a previously approved drug for sleep and attention disorders, as capable of not only protecting neurons but also repairing myelin – the protective sheath around nerve fibers – in experimental models.
A Decade-Long Pursuit Yields Hope
Nearly ten years of intensive research, utilizing advanced algorithms, innovative biological modeling, and an unprecedented drug screening platform, culminated in the identification of Bavisant. The extensive process involved sifting through 1,500 molecules to pinpoint a therapeutic candidate that could rapidly alter the clinical course of progressive MS, a especially severe form of the disease impacting an estimated 15-20,000 individuals in Italy alone.
The groundbreaking discovery, published in Science Translational Medicine, is the result of a collaborative effort led by the Università Vita-Salute San Raffaele and the Irccs Ospedale San Raffaele in Milan. The international network includes leading MS research centers such as the University of California San Francisco, the University of Münster, the Paris Brain Institute, and McGill University in Montreal.
Repurposing for Speed and efficiency
The project, known as “braveinms” and launched in 2017 with funding from the International Progressive MS Alliance, adopted an ambitious strategy: to repurpose drugs already approved for other conditions. “We asked ourselves what would be the fastest and most effective way to evaluate their action, starting from a pool of products already known to regulatory agencies,” explained Gianvito Martino, a neuroimmunologist and director of scientific affairs at San Raffaele. This form is characterized by continuous degeneration of nerve fibers and loss of myelin, leading to an irreversible decline in motor, visual, and cognitive functions. Current treatments offer limited efficacy in slowing this progression. This discovery has the potential to fundamentally change that.
“This platform doesn’t just identify a new cure, but builds a new way of doing research: faster, more predictive, and closer to patient expectations,” explained Paola Panina, a professor of Experimental Cell Biology at Università Vita-Salute and senior co-author of the study. The fact that bavisant is already known to regulatory agencies will translate into shorter timelines, lower costs, and increased safety compared to developing a novel molecule, scientists noted.
Looking ahead: Clinical Trials on the Horizon
The BraveinMs consortium is continuing its studies of Bavisant with the goal of initiating efficacy trials in humans in the near future. Brahim Nait-Oumesmar, a co-senior author and head of research at the Paris Brain Institute, emphasized that this achievement represents a crucial step “towards the development of clinical studies targeting the mechanisms of neurodegeneration and disability progression in MS.”
Other experts echoed this sentiment. Tanja Kuhlmann, a professor at the University of Münster in Germany, described the findings as “important progress.” Sergio Baranzini, a professor at the University of California San Francisco, highlighted the power of computational tools in accelerating drug discovery, adding that this work “offers renewed hope to patients.”
A Collaborative Triumph
Gianvito Martino, a neuroimmunologist and director of scientific affairs at San Raffaele, underscored the collaborative nature of the project. “We bet on an idea: uniting artificial intelligence,stem cell-based modeling,and collaborative science to accelerate the discovery of new therapies for the disease,” he said. “That bet has produced not only a real candidate,but also another 30 perhaps usable candidates.” Moreover, the team has validated a highly effective screening platform capable of evaluating the neuroprotective potential of any molecule.
Mario Alberto Battaglia, president of Fism and the International MS Federation, concluded that this research model “is capable of transforming scientific collaboration into concrete opportunities for new cures, even for those forms of disease that have had fewer responses so far.” He added that patients can now count on a new therapeutic arsenal.
