Creutzfeldt-Jakob Disease (CJD)
Creutzfeldt-Jakob disease (CJD) remains without effective treatment, representing a critical gap in neurological care. Protheragen has assembled a dedicated team whose members have decades of cumulative experience in CJD research. Leveraging this deep knowledge, the group is wholly engaged in translating scientific insight into innovative therapies specifically designed to halt the disease process.
Creutzfeldt-Jakob Disease (CJD)
Creutzfeldt-Jakob disease (CJD) is an uncommon and relentlessly advancing brain disorder characterized by the build-up of abnormally folded prion proteins. As a member of the transmissible spongiform encephalopathies (TSEs), it presents with symptoms including progressive dementia, myoclonus, ataxia, and behavioral changes, culminating in death, usually within twelve months of the first signs. The disease appears in different variants: the sporadic type accounts for about 85 percent of instances, while the familial, variant, and iatrogenic forms complete the spectrum.
Fig.1 Pathophysiology of Creutzfeldt-Jakob disease (CJD). (Dar K B, et al., 2020)
Pathogenesis of Creutzfeldt-Jakob Disease (CJD)
Creutzfeldt-Jakob disease (CJD) originates from the abnormal folding of the normal cellular prion protein (PrPC) into a misfolded, protease-resistant form (PrPSc), driving a pathogenic cascade through a self-propagating process. The newly converted PrPSc accumulates within the brain, giving rise to extensive amyloid deposit formation. Its accumulation disturbs synaptic integrity, heightens oxidative stress, and precipitates programmed neuronal death. The ensuing neuropathological hallmark—spongiform change, evident as vacuolation and gliosis—culminates in a rapid and relentless loss of neurological function.
Fig.2 Variant Creutzfeldt-Jakob disease (vCJD) is a distinct human prion strain. (Wadsworth J D F, et al., 2010)
Therapeutic Development for Creutzfeldt-Jakob Disease (CJD)
Currently, there is no cure for Creutzfeldt-Jakob disease (CJD), and no treatments exist to slow its progression. However, medications can help manage some symptoms and improve the patient's comfort.
| Drug Names | Mechanism of Action | Targets | Research Phase |
| Clonazepam | Positive allosteric modulator of GABAA receptors, enhancing inhibitory neurotransmission | GABAA receptors | Approved |
| Diphenylhydantoin | Sodium channel blocker, stabilizes neuronal membranes and reduces hyperexcitability | Voltage-gated sodium channels | Approved |
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
As a leading provider of preclinical research services, Protheragen is committed to expediting advances against Creutzfeldt-Jakob disease (CJD). We deliver comprehensive services that span diagnostic development, innovative therapeutic development, highly reproducible disease modeling, and stringent preclinical validation. Our proprietary in vitro blood-brain barrier model permits quantitative evaluation of compound CNS permeability, enabling prediction of brain exposure while safeguarding against systemic toxicity.
Therapeutic Development Services
By Mechanism of Action
Disease Model Development Services
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Protheragen is steadfastly dedicated to meticulously validating and optimizing therapies for Creutzfeldt-Jakob disease (CJD) through a thorough series of pharmacodynamics (PD), pharmacokinetics (PK) and toxicology studies. If you are interested in our services, please feel free to contact usfor more details and quotation information of related services.
References
- Dar K B, Bhat A H, Amin S, et al. Elucidating critical proteinopathic mechanisms and potential drug targets in neurodegeneration[J]. Cellular and Molecular Neurobiology, 2020, 40: 313-345.
- Wadsworth J D F, Asante E A, Collinge J. Contribution of transgenic models to understanding human prion disease[J]. Neuropathology and applied neurobiology, 2010, 36(7): 576-597.