A Belgian Mother and Son Travel to China for "Last Hope" Treatment, Reborn Months Later

In the winter of 2024, Alex, a 37-year-old Belgian man, was brought by his mother to Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology (hereinafter referred to as Tongji Hospital HUST).

Four years earlier, Alex was diagnosed with relapsing-remitting multiple sclerosis – an autoimmune disease of the nervous system. A year later, his condition worsened; he walked sideways like a crab, used a cane for short distances, relied on a wheelchair for longer ones, and was even afraid to hold his two-year-old child. After searching far and wide, Alex's mother learned that China was conducting the world's first clinical research on Chimeric Antigen Receptor T-cell (CAR-T) therapy for progressive multiple sclerosis.

Travelling thousands of miles, mother and son came to Wuhan with what was "perhaps their last hope." After a strict evaluation, Alex began treatment. Months later, he could stand, walk, hug his child, and teach again, as if reborn.

This research, completed by the team of Professor Wang Wei at Tongji Hospital HUST, provided the first global evidence that CAR-T cells can safely cross the blood-brain barrier and clear pathogenic B cells within the central nervous system, offering potential cures for "incurable" neuroimmune diseases.

Recently, the related research findings were published in Cell and were hailed by international peer experts as a "landmark exploration in autoimmune disease treatment."

Wang Wei (center) and his team discussing a case.

Breaching the "Forbidden Zone," Opening the Door to Central Immunity

Autoimmune diseases of the nervous system are like "hidden bombs" within the neural network, continuously attacking the immune balance of the brain and spinal cord, causing patients to gradually lose motor, sensory, and even cognitive functions. These diseases have long courses, relapse repeatedly, and are difficult to treat with traditional drugs due to the blood-brain barrier. Many patients ultimately exhaust their hope confined to wheelchairs and sickbeds, making this one of medicine's greatest challenges.

On the map of human immunotherapy, the central nervous system was once a "forbidden zone."

The blood-brain barrier, like a solid wall, kept almost all drugs out; inflammation in patients with progressive multiple sclerosis was trapped in an isolated "island" within the brain, leading to continuous neurological decline and uncontrollable relapse. For half a century, the global scientific community repeatedly tried to breach this defense, without success.

Wang Wei's team used CAR-T to open this door for the first time. They targeted B-cell maturation antigen (BCMA), bringing CAR-T – originally used for hematologic tumors – into the field of neuroimmune diseases. This was a high-risk attempt: Could CAR-T cross the blood-brain barrier? Could it accurately find and clear pathogenic B cells? Could it avoid causing fatal neurotoxicity? Internationally, there were no answers.

Wang Wei (right) engaging in academic exchange with international counterparts.

Wang Wei told China Science Daily: "We need to turn laboratory innovation into therapies that can truly change patients' destinies."

After multiple rounds of strict ethical review, the team initiated the research and obtained key evidence: CAR-T cells could safely proliferate in the central nervous system environment, precisely clear pathogenic B cells, and remodel the immune ecosystem.

Post-treatment, patients showed sustained neurological improvement; MRI scans showed inflammatory lesions gradually shrinking; autoantibodies in cerebrospinal fluid remained at low levels long-term; nerve conduction signals recovered, and the central immune environment tended towards balance. Some patients could stand and walk again.

This is the world's first scientific evidence proving CAR-T can not only "reach the brain" but also "reboot immunity." The research provides the first CAR-T treatment paradigm for progressive multiple sclerosis and, through single-cell multi-omics, reveals the mechanism of remodeling the central immune microenvironment, opening new directions for immunotherapy of chronic neuroinflammatory diseases.

Thus, the direction of immunotherapy has been redefined: it is no longer just about "suppressing immunity" but "rebuilding balance"; no longer confined to "peripheral regulation" but advancing towards "central intervention." This is a shift in treatment philosophy from "immune suppression" to "immune reset," holding promise for achieving long-term, deep remission in neuroimmune diseases, allowing neurons to play the music of life once more.

Achieving Success on Multiple "Fronts"

The starting point of this research was an attempt to find the way in "unmapped territory."

"We thought it was theoretically feasible," recalled team member Professor Qin Chuan of the Department of Neurology, Tongji Hospital HUST. "But the dosage, approach, and monitoring standards all had to be rebuilt."

During that period, the team repeatedly refined the plan – there was no template, no reference, everything started from zero. Project initiation, ethical review, registration – they advanced step by step. At that time, there were only three similar studies globally, and China had only this one. Finally, the team successfully registered the study on ClinicalTrials.gov.

Wang Wei diagnosing a patient.

During the clinical stage, they faced another round of tests. "CAR-T therapy can potentially cause cytokine release syndrome and neurotoxicity reactions, which progress extremely rapidly," Qin Chuan explained. To address this, the team established a rapid response system involving neurology, hematology, ICU, radiology, and immunology, using real-time monitoring and biomarkers to safeguard patient safety throughout.

The first patient to receive treatment suffered from primary progressive multiple sclerosis, with frequent relapses and near paralysis. After months of communication and preparation, he finally underwent treatment. Weeks later, the patient stood up again.

This was not an isolated case. On other "fronts" involving refractory immune diseases, the team continued to achieve remarkable success – they were the first globally to use CAR-T therapy for neuromyelitis optica spectrum disorder, immune-mediated necrotizing myopathy, myasthenia gravis, and chronic inflammatory demyelinating polyneuropathy, among other neuroimmune diseases. This resulted in 83% of refractory patients achieving relapse-free status for over two years, reaching a state of long-term clinical remission without the need for medication maintenance.

The research team was the first to use CAR-T for treating relapsing refractory immune-mediated necrotizing myopathy, enabling Xiao Deng, a 25-year-old young man paralyzed and bedridden, to regain his ability to move. Today, he is a medical postgraduate student pursuing his dreams. After successfully using BCMA-targeted CAR-T (CT103A) to treat refractory myasthenia gravis for the first time, patient Ms. Xie not only broke free from long-term drug dependence but also miraculously gave birth to a healthy baby. These two achievements were published in PNAS and EMBO Journal, respectively.

A team expert with a successfully treated patient who regained mobility and is now a medical postgraduate student with dreams. Photo provided by the interviewee.

The team's series of achievements were included in the annual cell therapy advances by Nature Reviews Rheumatology, evaluated by the international Guthy-Jackson Charitable Foundation NMO Project Group as "one of the highly potential treatment strategies," and contributed to updates in the latest international diagnosis and treatment guidelines for the disease. They were also incorporated into the clinical practice for innovative cell therapies in autoimmune diseases by the European Society for Blood and Marrow Transplantation.

In April 2025, the Expert Consensus on Chimeric Antigen Receptor T-Cell Therapy for Refractory Autoimmune Diseases of the Nervous System (2025 Edition), led by Wang Wei and co-authored by several domestic neurology experts, was officially released. This provides new standardized guidance for the clinical treatment of autoimmune nervous system diseases in China, marking a solid step forward for the country in this cutting-edge therapeutic field.

From Rejection to Top Journal

In 2022, the team submitted their early results to a top-tier medical journal. The external reviewers unanimously considered the research "significant." However, at the final review stage, the editor-in-chief rejected the manuscript, stating it was "not yet suitable for publication."

To let the world see the exploration by Chinese scientists, the team decided to first make their voice heard "closer to home." In January 2023, the results were published in Signal Transduction and Targeted Therapy. This was the world's first paper systematically reporting the use of CAR-T for neuroimmune diseases. After publication, it attracted widespread attention domestically and internationally. Peers from Germany, Finland, Italy, Spain, Japan, and other countries sent invitations. During that period, they were almost constantly traveling – giving reports, attending conferences, answering questions, repeatedly discussing data and mechanisms face-to-face with international counterparts.

Meanwhile, scientific research continued. In October 2024, the team formally submitted their manuscript to Cell. This time, they were better prepared. "We are one of the first research centers globally to conduct CAR-T therapy for immune diseases, with over two years of follow-up data. Among the currently published data, none have such long-term efficacy observation and such in-depth molecular characterization analysis as ours," said team member Professor Tian Daishi of the Department of Neurology, Tongji Hospital HUST.

Soon, they received a response from the editor, and the acceptance process was unusually smooth, passing with only one round of revisions.

"We were lucky, but the exploration of CAR-T therapy is far from over," Tian Daishi explained. Currently, the team is conducting more in-depth stratified research, hoping to identify patient subgroups that did not fully benefit and develop more precise targets and treatment plans for different pathogenic B-cell subsets.