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Scientists discovered how multiple sclerosis damages neurons: inflammation in the brain is linked to the breaking of gray matter DNA

Find out what new research from UCSF, the University of Cambridge, and Cedars-Sinai shows about multiple sclerosis. We bring an overview of the discovery according to which chronic inflammation damages not only myelin but also gray matter neurons, which may help explain cognitive difficulties and disease progression.

Scientists discovered how multiple sclerosis damages neurons: inflammation in the brain is linked to the breaking of gray matter DNA
Photo by: Domagoj Skledar - illustration/ arhiva (vlastita)

New phase of multiple sclerosis research: scientists have for the first time described in detail how inflammation in the brain leads to the death of neurons important for thinking and memory

For decades, most multiple sclerosis research has focused on myelin, the protective sheath around nerve fibers that is damaged and deteriorates in this disease. Lesions in the brain’s white matter, visible on magnetic resonance imaging, have long been the main sign by which doctors recognize disease activity. But alongside this process, another loss equally important for patients is occurring in the brain: the gradual death of neurons in the cerebral cortex, the area that is crucial for higher cognitive functions such as attention, planning, information processing, and memory.

Now an international team of scientists from the University of California, San Francisco, the University of Cambridge, and Cedars-Sinai Medical Center has published two related studies that offer the most concrete explanation of this process to date. According to the papers published on April 1, 2026, in the journal Nature, neurons that are particularly important for higher brain functions show increased sensitivity to DNA damage when inflammation associated with multiple sclerosis gets out of control. In other words, the researchers claim that the disease does not affect only the “insulation” of the nervous system, but can also directly disrupt the genetic stability of the neurons themselves, which ultimately leads to their death.

Why gray matter is becoming increasingly important in understanding the disease

Multiple sclerosis is a chronic inflammatory disease of the central nervous system in which the immune system mistakenly attacks the body’s own nerve tissues. In clinical practice, damage to white matter is most often discussed because such changes are easier to detect on brain scans and were long the main diagnostic reference point. However, in recent years there has been increasing evidence that the disease also affects gray matter, that is, the areas where neuron cell bodies are located. These changes are often associated with cognitive difficulties, slower information processing, and the gradual accumulation of disability, especially in progressive forms of the disease.

Official data from the U.S. National Institute of Neurological Disorders and Stroke describe multiple sclerosis as a chronic neurological disorder in which the immune system attacks healthy cells in the brain and spinal cord. The National MS Society emphasizes that changes in cognition can occur in any course of the disease, but are more common in progressive forms, where the number and location of brain lesions and the degree of brain atrophy are also important. The new research does not change this picture, but it provides it with a more precise biological mechanism: the inflammatory environment can create such oxidative and chemical pressure that certain neurons can no longer repair damage to their own DNA.

Two studies, one mechanism

The published papers do not examine the problem from just one angle. The first study deals with brain development and the question of how neurons carrying the CUX2 marker are formed. This is a special group of neurons located in the upper layers of the cerebral cortex, in areas important for complex cortical connections and higher functions. In mouse brain models, the researchers showed that these neurons pass through a period of pronounced stress during development: they multiply rapidly, migrate to target areas of the cortex, and create a broad network of connections. To survive such developmental pressure, they need a very efficient DNA repair system.

At the center of that system is the ATF4 gene. According to the paper in Nature, ATF4 acts as a key regulator of the response to DNA damage and activates a series of mechanisms that help preserve chromosome stability. When the scientists experimentally removed the function of ATF4, the consequences were dramatic: in neuron precursors there was an accumulation of DNA damage, disturbances in the repair of double-strand breaks in genetic material, and cell death, while the development of the front parts of the cerebral cortex was severely impaired. This made it clear that CUX2 neurons are not only anatomically special, but also biologically vulnerable.

The second study transfers that developmental insight into disease in the adult brain. By analyzing tissue from people with multiple sclerosis, the researchers found signs of increased DNA damage specifically in CUX2 neurons in gray matter lesions. A similar pattern was also recorded in mouse models of neuroinflammation and demyelination. In these experiments, inflammation stimulated the production of reactive oxygen compounds and other chemical processes that damage DNA. The system that had protected these neurons from stress during development could no longer keep up with the intensity of the damage, so selective neuron death and additional damage to the cerebral cortex followed.

What exactly are CUX2 neurons and why are they important

At first glance, this is a highly specialized detail from neurobiology, but its significance for understanding the disease may be substantial. CUX2 neurons are located in the superficial layers of the cortex and are connected with cortico-cortical networks, that is, the system of connections among different parts of the cerebral cortex. These layers are important for complex information processing, data integration, and functions that in everyday life are recognized as concentration, judgment, planning, or flexible thinking.

Scientists therefore assume that their sensitivity may help explain why some people with multiple sclerosis over time develop more pronounced cognitive difficulties, even when classic white matter lesions cannot fully explain the clinical picture. Researchers from Cambridge described CUX2 neurons as a kind of “canary in the coal mine” for the brain affected by multiple sclerosis: if it becomes possible to protect this particularly vulnerable population of cells, it may be possible to slow the spread of damage before the disease enters a deeper, more progressive phase.

A shift in focus: from remyelination toward direct neuron protection

Over the past several decades, the development of therapies for multiple sclerosis has made great progress, especially when it comes to reducing relapses and suppressing acute inflammation. At the same time, more and more research has focused on remyelination, that is, the restoration of myelin after damage. However, expert reviews published in recent years warn that chronic, smoldering inflammation and neuronal dysfunction still remain the main unresolved problem when it comes to disability progression. It is precisely in this space that the new discovery gains weight.

The authors’ message is not that existing treatment approaches are no longer important, but that they are insufficient if gray matter is neglected. If part of the neurodegeneration in the brain affected by multiple sclerosis is the consequence of unsuccessful DNA repair in particularly sensitive neurons, then an entirely new therapeutic question opens up: can drugs be developed, alongside inflammation control and myelin restoration, that protect neuronal DNA, strengthen repair mechanisms, or reduce oxidative stress in critical neuron populations.

This is especially important for progressive multiple sclerosis, in which patients often do not have a large number of new relapses, but still gradually lose functions. Such a course of the disease has long indicated that demyelination alone is not the whole story. The new research suggests that part of this “hidden” progression could be linked precisely to cumulative damage to neurons in the cerebral cortex, where the consequences are not always easily visible on routine scans, but are gradually reflected in a person’s everyday functioning.

What is new compared with previous knowledge

The link between inflammation, oxidative stress, and neurodegeneration in multiple sclerosis is not entirely new. What these two papers bring is more precise mapping of the most vulnerable type of neuron, identification of the development of these cells as a biological “weak point,” and a demonstration that the same pattern of vulnerability is seen later as well, in inflammatory disease of the adult brain. It is also important that the findings were obtained through a combination of several approaches: from developmental biology and genetic manipulations in mouse models to the analysis of human brain tissue and models of neuroinflammation.

Such a combination increases the weight of the conclusion, but it does not mean that a therapeutic solution is just around the corner. Discovering a mechanism is not the same as a drug. There is currently no confirmation that targeting only ATF4 or only one signaling pathway is sufficient to prevent neuron loss in humans. In addition, interventions in DNA repair systems must be developed very carefully, because these are fundamental cellular processes that have broad effects in different tissues. In translation, this is an important step in understanding the disease, but not yet a therapy ready for clinical use.

Possible consequences for diagnosis and patient monitoring

Still, the consequences of these papers could be felt even before new drugs arrive. If further research confirms that DNA damage in CUX2 neurons represents an early or particularly informative sign of progressive damage, this could influence the development of more precise disease biomarkers. Already now, intense work is underway in scientific circles on biomarkers that would better capture not only relapse activity, but also the silent neurodegeneration that accumulates between visible deteriorations. In that context, it is becoming increasingly important to understand what is happening in gray matter, and not only in classic white lesions.

In the long term, this could also mean changes in the way treatment success is assessed. If the goal of treatment is not only to reduce the number of new inflammatory lesions, but also to preserve the neuronal network responsible for cognitive functioning, then more attention may be given to more sophisticated cortex imaging, laboratory indicators of neurodegeneration, and neuropsychological monitoring of patients. Such an approach is already emerging in contemporary neurology, and the new research gives it additional biological grounding.

Caution and realistic expectations

For patients and their families, it is important to emphasize two things. First, the study does not claim that a single cause of cognitive changes in multiple sclerosis has been found, but rather a mechanism that can explain one important part of the disease, especially in progressive states and gray matter lesions. Second, although the results open a new research front, developing therapies that would directly protect neurons will require further confirmation in a larger number of models, patient samples, and eventually in clinical studies.

Despite this caution, the publication of two related papers in the same issue of Nature is a strong signal that the field is moving toward a more detailed understanding of what is happening in the brain beyond the classic picture of demyelination. For neurology and neuroimmunology, this is an important shift, because disease progression remains the greatest therapeutic challenge. And for patients and clinicians, the message is that increasing attention is being directed toward preserving the cerebral cortex itself and the neurons that enable thinking, planning, and everyday independent functioning.

That is precisely why this discovery goes beyond narrow laboratory interest. It does not offer a sensationalist promise of a quick cure, but it brings a more convincing answer to the question of why multiple sclerosis can over time also affect the very “processing core” of the brain. If future research builds on the current results, the fight against the disease may increasingly be less about only repairing the damaged insulation of nerve fibers, and more about preserving the neurons that allow the brain to remain functional even when inflammation lasts for years.

Sources:
  • Nature – study on selective loss of CUX2 neurons in neuroinflammation (link)
  • Nature – study on the role of ATF4 in DNA repair during the development of CUX2 neurons (link)
  • Cambridge Stem Cell Institute – official explanation of the findings and the role of CUX2 neurons in multiple sclerosis (link)
  • Cedars-Sinai – official release on gray matter research and the neurons most exposed to DNA damage (link)
  • National Institute of Neurological Disorders and Stroke – basic official information on multiple sclerosis as a chronic neurological disorder (link)
  • National MS Society – official information on cognitive changes and the impact of lesions and brain atrophy in multiple sclerosis (link)
  • Nature Reviews Neuroscience – review article on the neuropathobiology of multiple sclerosis and the role of chronic inflammation in disability progression (link)

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