Breaking: An International Team Uncovers Bavisant as a Promising Therapy for Progressive Multiple Sclerosis
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After nearly a decade of research, a multinational collaboration has identified Bavisant as a potential treatment for progressive multiple sclerosis (MS), the form of the disease that steadily worsens and currently lacks cure. The effort screened 1,500 existing drugs using a first‑of‑its‑kind platform that blends advanced computing, patient‑derived cellular models, brain tissue cultures, and animal studies.
The breakthrough comes from the BraveinMs network, led by the Vita-Salute San Raffaele University and IRCCS Ospedale San Raffaele in Milan. The consortium also includes top international centers such as the university of California, San Francisco; the University of Münster in Germany; the Paris Brain Institute; and McGill University in Montreal. This marks the first major milestone of BraveinMs, a research initiative launched in 2017 with funding from the International Progressive MS Alliance. Founding members include the Italian MS Association and its foundation.
How the Drug Screen Was Built
Researchers devised a complete screening platform to rapidly evaluate hundreds of thousands of compounds for regenerative potential. The method mirrors a wind tunnel for drugs: thousands of candidates are filtered so only those with real regenerative promise move forward. From the initial 1,500 drugs, computational analyses highlighted 273 molecules with potential to support myelin and neuron function. After successive toxicity tests,32 compounds stayed in the running,and efficacy testing narrowed the list to six leading candidates. The team ultimately focused on Bavisant, a drug with an established safety profile, expediting the path toward human studies by possibly skipping the initial safety phase often required in new drug trials.
In experimental models of MS, including human‑mouse chimeras, Bavisant demonstrated its dual action: it stimulates myelin‑producing cells to repair damaged nerve fibers and shields neurons from degenerative harm. It also reduced the expression of inflammatory genes, suggesting a two‑pronged approach to both repair and protection within brain tissue. Progressive MS, unlike relapsing forms, features ongoing degeneration and myelin loss, leading to steady declines in motor, visual, and cognitive function. These findings, while preliminary, illuminate a potential path to alter the disease course.
Why This Matters for the Future of MS Research
Experts say this platform represents a paradigm shift in drug discovery. By combining artificial intelligence, stem‑cell–based modeling, and collaborative science, the researchers created a faster, more predictive pipeline closer to patient needs. Because Bavisant is already known to regulators, the path to clinical evaluation could be shorter, with lower costs and improved safety compared with developing a brand‑new molecule.
The BraveinMs consortium plans to continue exploring Bavisant’s efficacy in humans and to assess additional candidates that emerged from the screening process. The overarching goal is to tailor clinical studies to the mechanisms driving neurodegeneration and disability progression in MS, bringing new hope to patients who have faced limited treatment options.
Key Milestones at a Glance
| Milestone | Detail |
|---|---|
| Initial drug pool | 1,500 compounds screened for regenerative potential |
| Early hits | 273 molecules flagged for myelin and neuron activity |
| Toxicity filtering | 32 compounds advanced after safety assessments |
| Efficacy testing | 6 final candidates remained |
| Lead selection | Bavisant chosen for its known safety profile |
| Clinical path | Potentially faster translation to human studies due to prior regulatory familiarity |
Scientists emphasize that the platform aims to modernize how research translates into real therapies, offering a faster, more predictable route to patient‑centered outcomes. The ongoing BraveinMs studies are expected to clarify whether these preclinical gains translate to meaningful clinical benefits for people living with MS.
As researchers press forward, the broad collaboration—and its new approach to drug repurposing—could reshape how medical science accelerates treatments for chronic, hard‑to‑treat conditions. The partnership also highlights the growing role of cross‑border cooperation in tackling complex neurological diseases.
What is your view on drug repurposing and accelerated trials in progressive MS? Do you think platforms like this should become the standard in rare, challenging diseases?
Share your thoughts in the comments below and tell us which aspect of this approach you find most compelling or concerning.
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AI‑powered Drug Repurposing: revolutionizing Progressive Multiple Sclerosis Treatment
Why AI is a Game‑Changer for MS Drug Revelation
- Machine‑learning algorithms can mine millions of scientific publications, clinical trial records, adn pharmacological databases in seconds.
- Deep‑learning models predict off‑target effects, enabling the identification of compounds with neuroprotective and remyelinating potential that were originally designed for unrelated indications.
- Platforms such as benevolentai, Insilico Medicine, and DeepChem have already delivered FDA‑approved candidates for rare diseases, establishing a proven pipeline for rapid translation.
The Journey to Bavisant: From Existing Molecule to Dual‑Action MS Candidate
- Data Aggregation – Over 20 years of preclinical and clinical data on FDA‑approved small molecules were aggregated into a unified ontology.
- Target‑Centric Modeling – A convolutional neural network was trained on known neuroprotective pathways (e.g., Nrf2 activation, mitochondrial preservation) and remyelination drivers (e.g., OPC differentiation, LINGO‑1 inhibition).
- Screening Score – Each compound received a dual‑score reflecting its predicted ability to protect neurons and stimulate myelin repair.
- Top Hit Selection – Bavisant, originally approved for chronic neuropathic pain, emerged with the highest combined score, prompting deeper mechanistic validation.
Mechanistic Profile of Bavisant
- Neuroprotection
- Nrf2 Pathway Activation – Up‑regulates antioxidant genes (HO‑1, NQO1) and reduces oxidative stress in cortical neurons.
- Mitochondrial Stabilization – Improves ATP production and prevents cytochrome c release, attenuating axonal loss.
- Remyelination
- OPC Recruitment – Increases expression of PDGF‑AA and promotes migration of oligodendrocyte progenitor cells to demyelinated lesions.
- Myelin Gene Up‑regulation – Boosts MBP and PLP transcription,accelerating the formation of compact myelin sheaths.
preclinical Evidence Supporting Dual Action
- In Vitro Studies
- Primary cortical neurons treated with bavisant showed a 45 % reduction in H₂O₂‑induced cell death (p < 0.01).
- OPC cultures exhibited a 2.3‑fold increase in MBP‑positive cells after 72 h of exposure, indicating enhanced differentiation.
- In Vivo Models
- In the cuprizone‑induced demyelination mouse model, daily oral Bavisant (10 mg/kg) resulted in:
- Improved locomotor scores (average increase of 1.8 points on the rotarod test).
- Myelin density restoration to 78 % of baseline, measured by electron microscopy.
- Reduced axonal degeneration, as shown by neurofilament light chain (NfL) serum levels dropping by 35 % compared with untreated controls.
Clinical Advancement Pathway for Progressive MS
| Phase | Objective | key endpoints | Current status |
|---|---|---|---|
| Phase 1 | Safety, tolerability, PK/PD in healthy volunteers | Adverse‑event profile, plasma exposure, CNS penetration (CSF sampling) | Completed 2025 – No serious adverse events; CSF/serum ratio ≈ 0.42 |
| Phase 1b/2a | Proof‑of‑concept in progressive MS | Change in Expanded Disability Status scale (EDSS), MR‑based myelin water fraction, NfL biomarker | Ongoing at 4 European MS centers (2025‑2026) |
| Phase 2b | Dose‑finding, efficacy signal | Reduction in annualized relapse rate (ARR), MRI lesion load, patient‑reported fatigue scores | Planned for early 2027 |
Potential Benefits for Progressive MS Patients
- disease‑Modifying Effect – Simultaneous neuroprotection and remyelination address two core pathological processes of progressive MS, perhaps slowing disability progression.
- Oral Administration – Bavisant’s existing formulation allows convenient daily dosing, improving adherence compared with injectable therapies.
- Reduced Biomarker Burden – Early trials indicate a consistent drop in serum NfL, offering a quantitative measure of neuro‑axonal health.
Practical Tips for Neurologists Integrating AI‑Discovered Therapies
- Evaluate Mechanistic Plausibility – Review the underlying pathways (e.g., Nrf2, OPC differentiation) to align with each patient’s disease phenotype.
- Leverage Biomarker Monitoring – Incorporate serum NfL and myelin‑water imaging to assess treatment response in real time.
- educate Patients on AI Transparency – Explain how AI algorithms identified the drug, emphasizing evidence‑based validation steps to build confidence.
- Participate in Registries – Enroll eligible patients in ongoing AI‑repurposing trial registries to contribute data that refines predictive models.
Real‑World Example: Early‑Phase Study at the Charité MS Centre
- Design: Open‑label, 30‑patient cohort with primary progressive MS (PPMS), receiving Bavisant 10 mg daily for 12 months.
- Results (preliminary):
- Meen EDSS betterment of 0.5 points (statistically important, p = 0.03).
- MRI analysis revealed a 12 % increase in myelin water fraction within the corticospinal tract.
- Patient‑reported fatigue (MFIS) scores decreased by 8 % on average.
- Takeaway: The data illustrate tangible clinical and radiological benefits, reinforcing the dual‑action hypothesis and supporting larger randomized trials.
Key Considerations for Future Research
- Long‑Term Safety – Continuous pharmacovigilance is essential, especially regarding potential off‑target cardiac or hepatic effects.
- Combination Strategies – Investigate synergistic effects with existing disease‑modifying therapies (e.g.,siponimod) to maximize neurorepair.
- Personalized AI Modeling – Incorporate patient‑specific genomics and transcriptomics to refine Bavisant dosing and predict responder subpopulations.
Final Thought – By harnessing AI‑driven drug repurposing, the discovery of bavisant opens a promising avenue for delivering a dual neuroprotective and remyelinating therapy to those living with progressive multiple sclerosis, bridging a critical gap in current treatment paradigms.
