Rare Neuroinflammatory Diseases
Rare neuroinflammatory diseases represent a diverse spectrum of uncommon disorders characterized by inflammation within the nervous system. At Protheragen, we focus on developing novel therapeutics and building accurate animal models to accelerate preclinical studies of potential therapies for rare neuroinflammatory diseases. Our expertise ensures that your research receives the most reliable and relevant support, accelerating your drug development journey.
Overview of Rare Neuroinflammatory Diseases
Rare neuroinflammatory diseases encompass a group of immune-mediated disorders characterized by chronic or recurrent inflammation of the central nervous system (CNS), often leading to progressive neurological damage. These diseases include neuromyelitis optica spectrum disorder (NMOSD), autoimmune encephalitis (AE), and Aicardi-Gutierrez syndrome (AGS), which present with unique pathological features. Clinically, these diseases often are misdiagnosed because their symptoms overlap with more common neurological diseases.
Fig.1 Role of inflammation in Parkinson's disease (PD) pathogenesis. (Giri P M, et al., 2024)
Pathogenesis of Rare Neuroinflammatory Diseases
The pathogenesis of rare neuroinflammatory diseases involves a dysregulated immune response against components of the central nervous system (CNS), with mechanisms varying from disease to disease. Common pathogenic mechanisms include disruption of immune privilege at the blood-brain barrier, aberrant B cell/plasma cell activity producing autoantibodies, and maladaptive innate immune responses involving microglia and astrocytes. Although the triggers vary, these common pathways ultimately lead to disruption of neural networks through excitotoxicity, oxidative stress, and impaired glial homeostasis.
Fig.2 Inflammatory pathways in Alzheimer's disease (AD). (Zhang W, et al., 2023)
Therapeutic Development for Rare Neuroinflammatory Diseases
| Drug Name | Indications | Mechanism of Action | NCT Number | Research Phase |
| Bortezomib | Autoimmune Encephalitis (AE) | Proteasome inhibitor inducing plasma cell apoptosis | NCT03993262 | Phase II |
| Ravulizumab | Neuromyelitis Optica | Anti-C5 monoclonal antibody preventing complement-mediated cytotoxicity | NCT06398158 | Approved |
| Ianalumab | Sjogren's Syndrome | Anti-BAFF-R monoclonal antibody inhibiting B-cell survival | NCT05350072 | Phase III |
| JAK Inhibitor | Aicardi-Goutières Syndrome | JAK1/2 inhibitor blocking type I interferon signaling (e.g., baricitinib) | NCT06898372 | Phase II |
Disclaimer: Protheragen focuses on providing preclinical research services. This table is for information exchange purposes only. This table is not a treatment plan recommendation. For guidance on treatment options, please visit a regular hospital.
Our Services
At the forefront of neuroimmunology research, Protheragen provides precision diagnostic and therapeutic development solutions for rare neuroinflammatory diseases. We identify disease-specific biomarkers to support the development of in vitro diagnostic (IVD) kits to enable early diagnosis. Our therapeutic development pipeline targets the root causes of disease and utilizes cutting-edge therapies such as antibodies, CAR-T and JAK inhibitors. All CNS-targeting drug candidates are evaluated in our proprietary blood-brain barrier (BBB) model to ensure optimal brain penetration.
Types of Rare Neuroinflammatory Diseases
| A-C | |
| Acute Disseminated Encephalomyelitis Autoimmune Encephalitis (AE) Aicardi-Goutières Syndrome (AGS) Acute Hemorrhagic Leukoencephalitis |
Behçet's Disease Chronic Inflammatory Demyelinating Polyneuropathy Cluster Headache Complex Regional Pain Syndrome |
| D-Z | |
| Mog Antibody Disease Multiple Sclerosis (MS) Neuromyelitis Optica Neurosarcoidosis Primary Angiitis of the Central Nervous System |
Schilder's Disease Sjogren Syndrome Susac Syndrome Suspected Autoimmune Encephalitis Vogt-Koyanagi-Harada Disease |
Therapeutic Development Services
By Mechanism of Action
Disease Model Development Services
Protheragen takes pride in offering comprehensive preclinical research services for rare neuroinflammatory diseases using advanced disease models. These services encompass various aspects of drug research, including pharmacodynamics (PD), pharmacokinetics (PK), and safety evaluations, ensuring a holistic approach towards drug development in this challenging medical area. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.
References
- Giri P M, Banerjee A, Ghosal A, et al. Neuroinflammation in neurodegenerative disorders: current knowledge and therapeutic implications[J]. International Journal of Molecular Sciences, 2024, 25(7): 3995.
- Zhang W, Xiao D, Mao Q, et al. Role of neuroinflammation in neurodegeneration development[J]. Signal transduction and targeted therapy, 2023, 8(1): 267.