World MS Day 2026: emerging regenerative medicines
Multiple sclerosis (MS) is one of the most common diseases of the central nervous system, affecting an estimated 2.9 million people worldwide. World MS Day, coordinated by the MS International Federation, takes place annually on 30th May to raise awareness, bring together the global MS community and campaign for better services, care and support for those with MS. Here, we review some recent advancements in MS research and examine current and emerging regenerative approaches that hold promise for enhancing treatment options.
Multiple sclerosis
MS is a chronic, immune-mediated disorder affecting the central nervous system. It occurs when the immune system mistakenly attacks myelin, the protective sheath surrounding nerve fibers. Damage to myelin disrupts nerve signal transmission, leading to a wide range of symptoms, including numbness, paralysis, vision problems, and cognitive impairments. The exact cause of MS remains unknown, but it is thought to arise from a combination of genetic predisposition and environmental factors [1].
The disease manifests in different forms, including relapsing-remitting MS (RRMS), secondary progressive MS (SPMS), and primary progressive MS (PPMS). Symptoms vary widely among individuals and may include fatigue, muscle weakness, coordination difficulties, and memory issues. While there is currently no cure for MS, available treatments aim to manage symptoms, reduce relapses, and slow disease progression [1].
Recent advances in regenerative medicine, including cell and gene therapies, are paving the way for innovative approaches to repair damage caused by MS and potentially alter the course of the disease.
Stem cell therapies
Over the past decade, stem cell therapies have emerged as one of the most promising areas of research in MS treatment. These therapies aim to repair the damage caused by the disease by promoting remyelination and protecting nerve cells from further degeneration. By harnessing the regenerative potential of stem cells, researchers hope to develop treatments that not only slow disease progression but also restore lost function. Below, we explore two of the most advanced stem cell therapies currently under investigation.
NG01
NG-01 is an investigational autologous bone marrow-derived stromal cell product delivered directly to the CNS via intrathecal injection. Once in the CNS, NG-01 cells secrete neurotrophic, anti-inflammatory and remyelinating factors, aiming to protect nerve cells and repair the damaged myelin sheath.
In March 2026, the US Food and Drug Administration (FDA; MD, USA) granted Regenerative Medicine Advanced Therapy (RMAT) designation for NG01 for the treatment of secondary progressive MS. This decision was based on Phase II trial results (NCT06961383), which demonstrated that intrathecal administration of NG-01 may slow disease progression, reduce disability, and improve motor functions such as walking and hand dexterity. The study also reported reductions in neurofilament light chain (NfL) and glial fibrillary acidic protein (GFAP), two key biomarkers of neurodegeneration.
Autologous Hematopoietic Stem Cell Transplantation (AHSCT)
AHSCT is one of the most extensively studied stem cell therapies for MS. This approach aims to reset the immune system, preventing it from attacking myelin and causing inflammation in the CNS. The process involves harvesting stem cells from the bone marrow, administering chemotherapy to eliminate the existing immune system, and then reintroducing the harvested stem cells into the bloodstream, where they differentiate into new immune cells and rebuild a new, less autoreactive immune system. AHSCT has shown promise in halting disease progression, particularly in patients with highly active relapsing-remitting MS [2].
CAR-T therapy
CAR-T therapy is a groundbreaking approach in MS research that leverages the body’s own immune cells to target and eliminate the underlying drivers of the disease. By engineering T cells to recognize and attack specific immune cells involved in the autoimmune response, CAR-T therapy offers a highly targeted and potentially transformative treatment option. Below, we explore one of the most promising CAR-T therapies currently under investigation for MS.
Obe-cel
Obe-cel, also known as AUCATZYL, is an autologous CAR-T therapy approved by the FDA for relapsed or refractory B-cell acute lymphoblastic leukemia. It targets CD19, a protein expressed on the surface of B cells. Since B cells play a significant role in driving the autoimmune response in MS by producing autoantibodies and activating other immune cells, targeting B cells could help modulate the disease. By eliminating CD19+ B cells, obe-cel may reduce the population of autoreactive B cells that contribute to the immune attack on the CNS.
The therapy is currently being investigated in a Phase I clinical trial for progressive forms of MS (NCT07139743), with the first patient dosed in October 2025. If successful, obe-cel could offer a one-time treatment option for progressive MS, potentially transforming outcomes for patients.
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Drug repurposing
Drug repurposing has become an increasingly attractive strategy in MS research, offering a faster, more cost-effective path to new treatments. By identifying existing drugs with potential therapeutic effects for MS, researchers can bypass many of the time-consuming and expensive steps involved in developing entirely new compounds. Below, we explore a promising example of drug repurposing in MS treatment.
Bavisant
Bavisant, a histamine H3 receptor antagonist previously studied for sleep and wake disorders, has emerged as a promising therapeutic candidate for MS. This discovery was made by the BRAVEinMS consortium, an international research network established in 2017 with support from the International Progressive MS Alliance.
Using an innovative drug-screening platform that integrates artificial intelligence, stem cell–based human models, and experimental MS models, researchers analyzed over 1,500 molecules. Bavisant was identified as a top candidate due to its ability to stimulate myelin repair, protect neurons, and reduce inflammation in preclinical MS models. Repurposing existing drugs like bavisant offers significant advantages, including shorter development timelines, reduced costs, and improved safety compared to developing entirely new treatments. The BRAVEinMS consortium is now preparing for Phase II clinical trials to further evaluate bavisant’s potential.
The Future of MS Research
The field of MS research is advancing rapidly, with regenerative medicine and innovative therapeutic approaches offering new hope for patients and expanding the therapeutic landscape. While challenges remain, the integration of technologies such as artificial intelligence and humanized models is accelerating the pace of discovery and bringing us closer to more effective, personalized treatments for MS.
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