Sun Yat-sen University to Transfer Patent for Demyelinating Disease Drug at RMB 50,300

Recently, Sun Yat-sen University released a public notice on the transformation of scientific and technological achievements, proposing to transfer its jointly owned"Use of Compounds in the Preparation of Medicaments for Treating and/or Preventing Demyelinating Diseases"The patent right for invention is transferred through listing and transfer, with the transaction price being50,300 yuan. This patent focuses on the pharmaceutical and healthcare sector, with its core application in drug development for demyelinating diseases such as multiple sclerosis, Guillain-Barré syndrome, and neuromyelitis optica. It breaks through the limitations of existing treatments that merely suppress immune-mediated damage, promotes myelin regeneration, fills the technological gap in myelin repair, and provides new therapeutic options for patients, demonstrating both social value and market potential. The inventors of this patent are Deng Wenbin and his team.

In the human nervous system, myelin in the central nervous system is produced by oligodendrocytes, whereas myelin in the peripheral nervous system is synthesized by Schwann cells. Oligodendrocytes are generated through the differentiation and maturation of oligodendrocyte precursor cells.

The physiological functions of myelin are critically important. Composed of approximately 70% lipids by dry weight, it forms an “insulating protective sheath” structure that envelops the exterior of nerve axons. It not only protects against external damage and maintains axonal integrity but also enables saltatory conduction of nerve impulses. This characteristic increases signal transmission speed by tens of fold compared to unmyelinated axons, serving as the foundation for the normal operation of neurological functions such as motor control, sensation, and cognition.

However, various demyelinating diseases, such as multiple sclerosis, Guillain-Barré syndrome, and neuromyelitis optica, cause persistent damage to and loss of the myelin sheath surrounding nerve axons through mechanisms including immune attacks and inflammatory responses. This impairs nerve signal conduction and exposes axons to injury, leading to a series of neurological deficits such as limb numbness, blurred vision, muscle weakness, and balance disorders. In severe cases, the condition may progress to paralysis, urinary and fecal incontinence, and even cognitive decline.

At the clinical level, patients faceRefractory, Progressive, and Multidimensional BurdenThe Triple Dilemma.

First, most diseases presentChronic relapsing course, with a disease trajectory of “acute exacerbation – partial remission – slow progression” throughout.Each episode leads to the continuous accumulation of irreversible neurological damage. Patients initially present with symptoms such as limb numbness and blurred vision, which gradually progress to difficulty walking and slurred speech, ultimately potentially resulting in the loss of independence in activities of daily living and prolonged suffering from declining physical function.

Second,Diagnostic Delay Is a Prominent Issue. Early symptoms lack specificity and are easily confused with conditions such as cervical spondylosis, cerebrovascular disease, and optic neuritis, often resulting in missed opportunities for optimal intervention. This can lead to lesion expansion and exacerbated injury, significantly adversely affecting prognosis.

Third,Significant Dual Pressure on Body and Mind. The unpredictability and progressive nature of the disease often leave patients trapped in prolonged negative emotional states, such as anxiety and depression, with social isolation being a prevalent issue. Moreover, long-term rehabilitation training and daily care take a significant physical and psychological toll on patients.

Fourth,Heavy Economic BurdenPatients require long-term use of high-cost medications to manage their condition, and when combined with expenses for regular check-ups, rehabilitation therapy, and nursing care, this imposes a significant financial burden on ordinary families. Some patients are forced to interrupt treatment due to an inability to afford these costs.

More critically, existing treatment regimens suffer from core deficiencies that are difficult to overcome, and these limitations persist throughout the entire treatment process.

First, there are fundamental shortcomings in the treatment logic.Currently, the mainstream clinical therapies are based onTargeting the Immune Systemas the core, the drugs used include immunosuppressants, glucocorticoids, monoclonal antibodies, etc., only allowing for“Passive Defense”, that is, by suppressing the immune response to reduce new myelin damage, but it cannot reach"Remyelination"this core therapeutic target. Meanwhile, under pathological conditions, oligodendrocyte precursor cells (OPCs) stored in the adult brain struggle to spontaneously differentiate into mature oligodendrocytes. Conventional approaches fail to overcome this differentiation barrier, neither promoting new myelin synthesis nor repairing damaged axons, ultimately making it difficult to reverse neurological decline in patients and leading to a dilemma of “addressing symptoms rather than the root cause.”

Second, the risk of drug side effects is relatively high.Long-term use of immunosuppressants can compromise patients' immune function, increasing the risk of complications such as infections and hepatic or renal impairment; glucocorticoids may induce osteoporosis, hyperglycemia, and gastrointestinal ulcers. Some patients have to adjust their treatment regimens due to intolerance to these adverse effects.

Furthermore, the coverage of treatment is limited.Existing medications effectively control symptoms during the acute phase, but exhibit limited efficacy in intervening in the chronic progressive stage of the disease, making it difficult to halt ongoing demyelination and functional decline.

Furthermore, there are individual differences in response.Some patients are insensitive to existing medications or develop drug resistance, leading to suboptimal therapeutic outcomes. The lack of alternative treatment options further exacerbates the clinical dilemma.

Therefore, existing clinical regimens are ineffective in addressing the issue, highlighting an urgent need to develop new therapeutic agents and treatment protocols.

The core innovation of this patent from Sun Yat-sen University lies in breaking through the limitations of traditional treatments, with“Active Myelin Repair”As the core objective, a clear and verifiable therapeutic pathway has been established.

First, the mechanism of action precisely targets the core pathology.This patent focuses on demyelinating diseases“Impaired Differentiation of Oligodendrocyte Precursor Cells”To address this critical challenge, five compounds with specific structures, designated as Formula I through Formula V, were screened. By inhibiting the activity of the TM7SF2 protein in vivo, these compounds specifically enhance the accumulation of follicular fluid-meiosis-activating sterol (FF-MAS) secreted by cumulus cells within the cholesterol metabolic pathway. FF-MAS directly promotes the differentiation of oligodendrocyte precursor cells into oligodendrocytes, thereby addressing the root cause of impaired autonomous myelin regeneration. This approach achieves a dual effect of “inhibiting damage and promoting repair,” which is fundamentally distinct from existing therapeutic strategies that target only the immune system.

Second, its activity and safety have been fully validated through experiments.Differentiation assays using primary cultured oligodendrocyte precursor cells confirmed that the compound significantly increases the proportion of MBP (myelin basic protein, a core marker of oligodendrocyte maturation)-positive cells at specific concentrations. Furthermore, Western blot analysis revealed that MBP expression was markedly higher than in the negative control group, further corroborating the compound’s specific activity in promoting myelin regeneration and providing robust experimental data to support its role in this process.

Third, drug development is highly flexible and adaptable.The compounds protected by this patent can be used either as the sole active ingredient or in combination with other ingredients to form pharmaceutical compositions, with content levels ranging from0.1 to 99 wt%(preferably 1 to 90 wt%), capable of meeting diverse administration requirements. Meanwhile, derivatives of the compound include various forms such as pharmaceutically acceptable salts, esters, hydrates, and solvates, which can be formulated into dozens of common dosage forms including decoctions, tablets, capsules, injections, and liposomal preparations. This versatility not only satisfies the needs of different clinical administration routes, such as oral and injectable, but also lowers the threshold for technological translation.

From targeted drugs to non-invasive stimulation, and covering both central and peripheral lesions, a series of studies on the treatment of demyelinating diseases have emerged successively. These advancements not only address the pain points of traditional therapies—such as “heavy suppression, light repair” and “significant side effects”—but also expand the applicable scenarios and technical pathways for treatment, bringing new hope to patients worldwide.

The Second Hospital of Hebei Medical University and The Second Affiliated Hospital of University of South ChinaThe teamTherapeutic Potential of IDH1 Inhibitors in Central Nervous System Demyelinating DiseasesExploration has been conducted, revealing four core functions: first, directly inhibiting myelin destruction, ameliorating demyelination, and preserving the structural integrity of nerve axons; second, attenuating central nervous system inflammation by suppressing the activation of macrophage inflammasomes and the production of microglial inflammatory cytokines (such as IL-1β); third, improving neurological and motor dysfunction in patients, thereby alleviating symptoms such as limb weakness and balance disorders; and fourth, slowing disease-related weight loss to maintain patients’ physical condition. Currently, only animal experiments in EAE mice have been completed.

Liu Yan's Team at Nantong UniversityR&DLong-acting glucose-dependent insulinotropic polypeptide receptor agonist(D-Ala²-GIP), which aims to achieve dual repair of central and peripheral demyelinating injuries. This technology not only inhibits the apoptosis of myelin-related cells such as oligodendrocytes and Schwann cells, enhances the expression of key myelin synthesis proteins including MBP, P0, and MPZ, and promotes myelin regeneration in the spinal cord, brain, and sciatic nerve; but also significantly suppresses inflammatory responses and oxidative stress in demyelinated lesions, improves the local microenvironment, maintains the structural integrity of nerve axons, and enhances the efficiency of neurological functional recovery.

Massachusetts Institute of Technology（MIT）Li-Huei Tsai's TeamIn-depth Exploration of40 Hz Audiovisual Stimulation Non-Pharmacological Therapy. The core mechanism of this therapy lies in achieving multifaceted effects through synchronized audiovisual stimulation at specific frequencies. On one hand, it protects cerebral white matter by directly promoting remyelination and repair, thereby improving neural conduction deficits caused by demyelination. On the other hand, it accelerates cerebrospinal fluid flow, facilitating the clearance of pathogenic protein deposits in the brain, reducing further damage to myelin sheaths caused by protein accumulation, and simultaneously protecting cerebral gray matter, thus realizing the dual benefits of “neuroprotection + metabolic regulation.”

Currently, this therapy has entered Phase II clinical trials. It requires no pharmacological intervention, offering the advantages of being non-invasive and free from side effects. Furthermore, it can be used in combination with other treatment modalities and is suitable for patients with demyelinating diseases of varying severity.

These studies not only encompass diverse technological pathways ranging from pharmacological to non-pharmacological interventions and from small molecules to gene vectors, but have also achieved critical breakthroughs in enhancing efficacy, optimizing safety, and expanding therapeutic indications. As some candidate drugs enter clinical trials and non-invasive therapies continue to advance, a treatment paradigm characterized by “precision targeting plus multi-pathway synergy” is expected to emerge. This will provide patients with safer and more effective personalized treatment regimens, fundamentally transforming the current landscape of diagnosis and treatment for demyelinating diseases.