Materiały źródłowe

• #1 Creutzfeldt-Jakob disease – Diagnosis & treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/creutzfeldt-jakob-disease/diagnosis-treatment/drc-20371230

  No effective treatment exists for Creutzfeldt-Jakob disease or any of its variants. Many medicines have been tested and haven’t shown benefits. Health care providers focus on relieving pain and other symptoms and on making people with these diseases as comfortable as possible.

• #2 Creutzfeldt-Jakob Disease (CJD) | Symptoms & Treatments | alz.org

  https://www.alz.org/alzheimers-dementia/what-is-dementia/types-of-dementia/creutzfeldt-jakob-disease

  Creutzfeldt-Jakob disease (CJD) is the most common human form of a group of rare, fatal brain disorders known as prion diseases. […] There is no treatment that can slow or stop the underlying brain cell destruction caused by Creutzfeldt-Jakob disease and other prion diseases. Various drugs have been tested but have not shown any benefit. Clinical studies of potential Creutzfeldt-Jakob disease treatments are complicated by the rarity of the disease and its rapid progression. […] Current therapies focus on treating symptoms and on supporting individuals and families coping with Creutzfeldt-Jakob disease. Doctors may prescribe painkillers such as opiates to treat pain if it occurs. Muscle stiffness and twitching may be treated with muscle-relaxing medications or antiseizure drugs. In the later stages of the disease, individuals with Creutzfeldt-Jakob disease become completely dependent on others for their daily needs and comfort.

• #3

  https://www.nhs.uk/conditions/creutzfeldt-jakob-disease-cjd/treatment/

  There’s no proven cure for Creutzfeldt-Jakob disease (CJD), but clinical studies are under way at the National Prion Clinic to investigate possible treatments. […] At present, treatment involves trying to keep the person as comfortable as possible and reducing symptoms with medicines. […] For example, psychological symptoms of CJD, such as anxiety and depression, can be treated with sedatives and antidepressants, and muscle jerks or tremors can be treated with medicines like clonazepam and sodium valproate. […] Any pain experienced can be relieved using powerful opiate-based painkillers. […] For more information about how some of the specific symptoms of CJD may be treated, see: treating ataxia (loss of physical co-ordination), treating urinary incontinence (loss of bladder control), bowel incontinence (loss of bowel control), treating dysphagia (swallowing difficulties), dystonia (muscle spasms and stiffness), blindness or vision loss.

• #4 Symptomatic treatment | National Prion Clinic – UCL – University College London

  https://www.ucl.ac.uk/national-prion-clinic/symptomatic-treatment

  Although there is no cure for CJD, treatments are available for a variety of symptoms that occur in patients with CJD. […] It is often necessary to change therapy in an attempt to control the breakthrough symptoms and use combinations of different treatments even though some combinations appear to be duplication of therapy. […] Cortical myoclonus is often reduced by gabanergic drugs of which sodium valproate, an agent that increases cortical gamma amino butyric acid (gaba), is the most frequently prescribed. […] Clonazepam can be dramatically effective in a small dose but many patients only respond to large doses (up to 15mg) which can result in excessive drowsiness. […] Subcortical myoclonus also occurs in CJD. […] Clonazepam is the drug of choice and ethyl alcohol is sometimes beneficial.

• #5

  https://www.nhs.uk/conditions/creutzfeldt-jakob-disease-cjd/treatment/

  There’s no proven cure for Creutzfeldt-Jakob disease (CJD), but clinical studies are under way at the National Prion Clinic to investigate possible treatments. […] At present, treatment involves trying to keep the person as comfortable as possible and reducing symptoms with medicines. […] For example, psychological symptoms of CJD, such as anxiety and depression, can be treated with sedatives and antidepressants, and muscle jerks or tremors can be treated with medicines like clonazepam and sodium valproate. […] Any pain experienced can be relieved using powerful opiate-based painkillers. […] For more information about how some of the specific symptoms of CJD may be treated, see: treating ataxia (loss of physical co-ordination), treating urinary incontinence (loss of bladder control), bowel incontinence (loss of bowel control), treating dysphagia (swallowing difficulties), dystonia (muscle spasms and stiffness), blindness or vision loss.

• #6 Variant Creutzfeldt-Jakob Disease and Bovine Spongiform Encephalopathy Treatment & Management: Approach Considerations, Experimental Treatments

  https://emedicine.medscape.com/article/1169688-treatment

  Although there is no cure for Creutzfeldt-Jakob disease (CJD), interleukins and other drugs may help slow the progression of disease. Medications to help treat the symptoms of CJD include antiepileptics to manage seizures (eg,diphenylhydantoin, carbamazepine, and gabapentin) and, occasionally, clonazepam to treat myoclonus. Antiepileptics are also used to treat violent outbursts. […] Dementia in patients with CJD can be treated with donepezil, galantamine, rivastigmine, and memantine. If a person develops disruptive behavior, antipsychotics can be used. Antidepressants may help patients come to terms with the prognosis. […] Some general principles for the treatment of patients with variant CJD include the following: Discontinue any medication that could impair cognition or cause confusion; Many patients need psychiatric care, including antidepressants, which may provide temporary relief; Some patients need treatment to ameliorate sensory symptoms in the limbs; All patients have increasing need for supportive care, including palliative and terminal care; Family members need significant support in coping with both emotional and care needs; they also need reassurance and information regarding the nature of disease transmission.

• #7

  https://www.nhs.uk/conditions/creutzfeldt-jakob-disease-cjd/treatment/

  There’s no proven cure for Creutzfeldt-Jakob disease (CJD), but clinical studies are under way at the National Prion Clinic to investigate possible treatments. […] At present, treatment involves trying to keep the person as comfortable as possible and reducing symptoms with medicines. […] For example, psychological symptoms of CJD, such as anxiety and depression, can be treated with sedatives and antidepressants, and muscle jerks or tremors can be treated with medicines like clonazepam and sodium valproate. […] Any pain experienced can be relieved using powerful opiate-based painkillers. […] For more information about how some of the specific symptoms of CJD may be treated, see: treating ataxia (loss of physical co-ordination), treating urinary incontinence (loss of bladder control), bowel incontinence (loss of bowel control), treating dysphagia (swallowing difficulties), dystonia (muscle spasms and stiffness), blindness or vision loss.

• #8 Creutzfeldt-Jakob Disease (CJD) – Brain, Spinal Cord, and Nerve Disorders – Merck Manual Consumer Version

  https://www.merckmanuals.com/home/brain-spinal-cord-and-nerve-disorders/prion-diseases/creutzfeldt-jakob-disease-cjd

  Currently, CJD cannot be cured, and its progress cannot be slowed. The disease is fatal, usually within months or a few years. However, certain medications may be given to relieve symptoms. For example, the antiseizure medication valproate and the antianxiety clonazepam may reduce muscle jerking. Sedative or antipsychotic medications can sometimes help calm people who are anxious. […] General support and care for the person and family members are important. Hospice care, respite care, and long-term care may be useful. The CJD Foundation provides support and information.

• #9

  https://www.nhs.uk/conditions/creutzfeldt-jakob-disease-cjd/treatment/

  There’s no proven cure for Creutzfeldt-Jakob disease (CJD), but clinical studies are under way at the National Prion Clinic to investigate possible treatments. […] At present, treatment involves trying to keep the person as comfortable as possible and reducing symptoms with medicines. […] For example, psychological symptoms of CJD, such as anxiety and depression, can be treated with sedatives and antidepressants, and muscle jerks or tremors can be treated with medicines like clonazepam and sodium valproate. […] Any pain experienced can be relieved using powerful opiate-based painkillers. […] For more information about how some of the specific symptoms of CJD may be treated, see: treating ataxia (loss of physical co-ordination), treating urinary incontinence (loss of bladder control), bowel incontinence (loss of bowel control), treating dysphagia (swallowing difficulties), dystonia (muscle spasms and stiffness), blindness or vision loss.

• #10 Creutzfeldt-Jakob Disease | Conditions | UCSF Health

  https://www.ucsfhealth.org/conditions/creutzfeldt-jakob-disease

  As international leaders in the field of dementia disorders, UCSF specialists are working to find new treatments and a cure for Creutzfeldt-Jakob disease. We offer the full scope of diagnostic tests and therapies to control symptoms and keep patients as comfortable as possible. […] Currently, there is no cure for Creutzfeldt-Jakob disease (CJD). Researchers have tested many drugs, including acyclovir, amantidine, antibiotics, antiviral agents, interferon and steroids. None has shown consistent benefit. […] Treatment is aimed at alleviating symptoms and making the patient as comfortable as possible. Drugs can help relieve pain if it occurs. The drugs clonazepam and sodium valproate may help relieve myoclonus or irregular, jerking movements. […] Researchers at the UCSF Memory and Aging Center are trying to identify compounds for treatment or a cure for CJD and other diseases caused by the infectious particles called prions.

• #11 Symptomatic treatment | National Prion Clinic – UCL – University College London

  https://www.ucl.ac.uk/national-prion-clinic/symptomatic-treatment

  A variety of antipsychotic drugs are used to control them but there are no firm data on efficacy. […] Quetiapine, a drug that has marked 5HT1A and 2 blocking activity, together with some activity against D1 and 2 and less against alpha adrenergic and H1 receptors, is most frequently prescribed. […] Because of the beneficial effect of centrally acting anticholinergic drugs upon visual hallucinations in cortical Lewy body disease it is reasonable to prescribe donepezil or rivastigmine to CJD patients with disturbing hallucinations and the former does seem to be effective in some patients with intractable visual hallucinations. […] Benzodiazepines such as diazepam, which act on benzodiazepine receptors associated with gaba receptors, are effective anxiolytic drugs and can promote sleep.

• #12 Symptomatic treatment | National Prion Clinic – UCL – University College London

  https://www.ucl.ac.uk/national-prion-clinic/symptomatic-treatment

  A variety of antipsychotic drugs are used to control them but there are no firm data on efficacy. […] Quetiapine, a drug that has marked 5HT1A and 2 blocking activity, together with some activity against D1 and 2 and less against alpha adrenergic and H1 receptors, is most frequently prescribed. […] Because of the beneficial effect of centrally acting anticholinergic drugs upon visual hallucinations in cortical Lewy body disease it is reasonable to prescribe donepezil or rivastigmine to CJD patients with disturbing hallucinations and the former does seem to be effective in some patients with intractable visual hallucinations. […] Benzodiazepines such as diazepam, which act on benzodiazepine receptors associated with gaba receptors, are effective anxiolytic drugs and can promote sleep.

• #13 Symptomatic treatment | National Prion Clinic – UCL – University College London

  https://www.ucl.ac.uk/national-prion-clinic/symptomatic-treatment

  Olanzapine, which has its maximum receptor blocking activity against 5HT2 receptors, can be helpful although care is needed in the elderly because of an increased risk of stroke and diabetes. […] Sleep disturbance is characteristic of the fatal insomnias and some of the thalamic variants of CJD; it is also found in other forms of CJD and is often associated with anxiety. […] Depression occurs in some patients with CJD, particularly those with a family history, early in the evolution of the disorder. […] Gabapentin and amitriptyline are often effective and can be given in combination. […] The pain sometimes resolves as the disease progresses. […] Levetiracetam and sodium valproate are useful for generalised tonic clonic seizures and is effective for seizures with myoclonus. […] Increased tone is generally extrapyramidal.

• #14 Symptomatic treatment | National Prion Clinic – UCL – University College London

  https://www.ucl.ac.uk/national-prion-clinic/symptomatic-treatment

  Olanzapine, which has its maximum receptor blocking activity against 5HT2 receptors, can be helpful although care is needed in the elderly because of an increased risk of stroke and diabetes. […] Sleep disturbance is characteristic of the fatal insomnias and some of the thalamic variants of CJD; it is also found in other forms of CJD and is often associated with anxiety. […] Depression occurs in some patients with CJD, particularly those with a family history, early in the evolution of the disorder. […] Gabapentin and amitriptyline are often effective and can be given in combination. […] The pain sometimes resolves as the disease progresses. […] Levetiracetam and sodium valproate are useful for generalised tonic clonic seizures and is effective for seizures with myoclonus. […] Increased tone is generally extrapyramidal.

• #15 Symptomatic treatment | National Prion Clinic – UCL – University College London

  https://www.ucl.ac.uk/national-prion-clinic/symptomatic-treatment

  Although there is no cure for CJD, treatments are available for a variety of symptoms that occur in patients with CJD. […] It is often necessary to change therapy in an attempt to control the breakthrough symptoms and use combinations of different treatments even though some combinations appear to be duplication of therapy. […] Cortical myoclonus is often reduced by gabanergic drugs of which sodium valproate, an agent that increases cortical gamma amino butyric acid (gaba), is the most frequently prescribed. […] Clonazepam can be dramatically effective in a small dose but many patients only respond to large doses (up to 15mg) which can result in excessive drowsiness. […] Subcortical myoclonus also occurs in CJD. […] Clonazepam is the drug of choice and ethyl alcohol is sometimes beneficial.

• #16 Symptomatic treatment | National Prion Clinic – UCL – University College London

  https://www.ucl.ac.uk/national-prion-clinic/symptomatic-treatment

  Although there is no cure for CJD, treatments are available for a variety of symptoms that occur in patients with CJD. […] It is often necessary to change therapy in an attempt to control the breakthrough symptoms and use combinations of different treatments even though some combinations appear to be duplication of therapy. […] Cortical myoclonus is often reduced by gabanergic drugs of which sodium valproate, an agent that increases cortical gamma amino butyric acid (gaba), is the most frequently prescribed. […] Clonazepam can be dramatically effective in a small dose but many patients only respond to large doses (up to 15mg) which can result in excessive drowsiness. […] Subcortical myoclonus also occurs in CJD. […] Clonazepam is the drug of choice and ethyl alcohol is sometimes beneficial.

• #17

  https://www.nhs.uk/conditions/creutzfeldt-jakob-disease-cjd/treatment/

  As CJD progresses, people with the condition will need significant nursing care and practical support. […] Many people will also have problems swallowing, so they may have to be given nutrition and fluids through a feeding tube. […] It may be possible to treat someone with CJD at home, depending on the severity and progression of their condition.

• #18

  https://www.nhs.uk/conditions/creutzfeldt-jakob-disease-cjd/treatment/

  As CJD progresses, people with the condition will need significant nursing care and practical support. […] Many people will also have problems swallowing, so they may have to be given nutrition and fluids through a feeding tube. […] It may be possible to treat someone with CJD at home, depending on the severity and progression of their condition.

• #19 Caregiver Support – CJD Foundation

  https://cjdfoundation.org/caregiver-support/

  Be bold in reaching out to the healthcare professional if you are not hearing a response. With prion diseases, the patients condition may change more quickly than the doctor is accustomed to. […] Working with the hospice or a medical care provider, the family can assess options for addressing incontinence and learn how to minimize movement when changing bedding and clothing. […] As prion disease progresses, the patient may have difficulty with appetite, chewing, focusing on a meal, and ultimately with swallowing. At early stages, ergonomic utensils may be helpful to enable self-feeding. As dysphagia progresses, pureed foods and thickener in fluids may become necessary. […] If the patient develops myoclonus, caregivers can help by minimizing touching and movement in caring for the patient. At times, gentle massage may help relax the muscles, and the family may ask the physician about medications to help reduce spasticity and tremors.

• #20 Creutzfeldt-Jakob disease (CJD): Symptoms, causes, and treatment

  https://www.medicalnewstoday.com/articles/185884

  There is no cure for CJD, and no medications can control it or slow its progression. Scientists are looking into several treatment options for future use. […] Currently, doctors aim to relieve symptoms and make the person as comfortable as possible. […] Opiate drugs can help relieve the pain. Also, clonazepam and sodium valproate may help relieve involuntary movements, such as muscle twitching. […] In the later stages, a carer will move the person frequently to help prevent bedsores. A catheter will drain the person’s urine, and they will receive nutrients intravenously. […] Scientists continue to investigate how CJD affects the brain, in an effort to develop effective treatments.

• #21 Caregiver Support – CJD Foundation

  https://cjdfoundation.org/caregiver-support/

  Be bold in reaching out to the healthcare professional if you are not hearing a response. With prion diseases, the patients condition may change more quickly than the doctor is accustomed to. […] Working with the hospice or a medical care provider, the family can assess options for addressing incontinence and learn how to minimize movement when changing bedding and clothing. […] As prion disease progresses, the patient may have difficulty with appetite, chewing, focusing on a meal, and ultimately with swallowing. At early stages, ergonomic utensils may be helpful to enable self-feeding. As dysphagia progresses, pureed foods and thickener in fluids may become necessary. […] If the patient develops myoclonus, caregivers can help by minimizing touching and movement in caring for the patient. At times, gentle massage may help relax the muscles, and the family may ask the physician about medications to help reduce spasticity and tremors.

• #22 Symptomatic treatment | National Prion Clinic – UCL – University College London

  https://www.ucl.ac.uk/national-prion-clinic/symptomatic-treatment

  Dantrolene acts directly upon the muscle membrane and can relieve rigidity even though the tone increase is centrally generated. […] Anticholinergic drugs such as tolterodine XL are helpful for frequency in some patients or if the bladder is irritable causing catheter extrusion. […] Occasionally patients who have severe swallowing problems require anticholinergic drugs such as atropine to reduce salivary flow. […] Glycopyrronium bromide is effective in excessive salivation and can be administered orally or subcutaneously. […] Initially pureed food may help but ultimately this fails to maintain an adequate intake. […] Patients should be on appropriate sedatives such as opiates.

• #23 Symptomatic treatment | National Prion Clinic – UCL – University College London

  https://www.ucl.ac.uk/national-prion-clinic/symptomatic-treatment

  Dantrolene acts directly upon the muscle membrane and can relieve rigidity even though the tone increase is centrally generated. […] Anticholinergic drugs such as tolterodine XL are helpful for frequency in some patients or if the bladder is irritable causing catheter extrusion. […] Occasionally patients who have severe swallowing problems require anticholinergic drugs such as atropine to reduce salivary flow. […] Glycopyrronium bromide is effective in excessive salivation and can be administered orally or subcutaneously. […] Initially pureed food may help but ultimately this fails to maintain an adequate intake. […] Patients should be on appropriate sedatives such as opiates.

• #24 Caregiver Support – CJD Foundation

  https://cjdfoundation.org/caregiver-support/

  Families who receive a prion disease diagnosis are often shocked, confused and overwhelmed. They must rapidly accept the news that their loved one who probably appeared quite healthy just weeks ago is suffering from a terminal disease that has no treatment. […] Families who receive a prion disease diagnosis should contact hospice as early as possible. While hospice care may not be immediately needed in the early stages, the patients needs are likely to evolve rapidly. Choosing a hospice provider and setting up care in advance will free the family to focus more on the patient later. Hospice providers bring knowledge and resources that are invaluable to a family that is confronting rapidly evolving care needs. […] As the disease progresses, you will want to contact the physician, hospice, or healthcare provider about any concerns, such as: New symptoms that are not easily managed, particularly hallucinations, and extremely fearful or violent behavior.

• #25

  https://pmc.ncbi.nlm.nih.gov/articles/PMC9703894/

  The Creutzfeldt-Jakob disease (CJD) is a spongiform encephalopathy that manifests as a rapidly progressive dementia syndrome. Currently, CJD has no cure, and many patients die within the first year, but some drugs are being studied as options for managing this condition. […] None of the drugs evaluated proved significantly effective in increasing survival in patients with CJD. Flupirtine appears to have a beneficial effect in reducing cognitive deterioration in patients with CJD. However, additional studies are needed to establish better evidence and therapeutic options for the management of patients with CJD. […] Several potential therapeutic interventions are under development over the last years, but no treatment has been proven effective for CJD. […] Studies have shown that the malaria treatment drug quinacrine and the antipsychotic chlorpromazine are effective in clearing prions in non-human models.

• #26 World-first treatment for CJD shows promising early results | NIHRopens in a new window

  https://www.nihr.ac.uk/news/world-first-treatment-cjd-shows-promising-early-results

  A first-in-human treatment designed specifically for Creutzfeldt–Jakob disease (CJD) has shown encouraging early results and may contribute to the development of new treatments for other neurodegenerative diseases. […] CJD is a rare and fatal disease that causes brain damage and for which there is currently no licensed treatment. […] Researchers at the NIHR UCLH Biomedical Research Centre (BRC) developed a monoclonal antibody, called PRN100, that was given to six patients with CJD between October 2018 and July 2019. […] The results, published in Lancet Neurology, show the treatment is safe and able to access the brain. […] In three patients, disease progression appeared to stabilise when dosing levels were in target range. […] None of the six patients experienced side effects while receiving the treatment but all sadly died as a result of their condition.

• #27 World-first treatment for CJD shows promising early results | NIHRopens in a new window

  https://www.nihr.ac.uk/news/world-first-treatment-cjd-shows-promising-early-results

  A first-in-human treatment designed specifically for Creutzfeldt–Jakob disease (CJD) has shown encouraging early results and may contribute to the development of new treatments for other neurodegenerative diseases. […] CJD is a rare and fatal disease that causes brain damage and for which there is currently no licensed treatment. […] Researchers at the NIHR UCLH Biomedical Research Centre (BRC) developed a monoclonal antibody, called PRN100, that was given to six patients with CJD between October 2018 and July 2019. […] The results, published in Lancet Neurology, show the treatment is safe and able to access the brain. […] In three patients, disease progression appeared to stabilise when dosing levels were in target range. […] None of the six patients experienced side effects while receiving the treatment but all sadly died as a result of their condition.

• #28 World-first treatment for CJD shows promising early results | NIHRopens in a new window

  https://www.nihr.ac.uk/news/world-first-treatment-cjd-shows-promising-early-results

  A first-in-human treatment designed specifically for Creutzfeldt–Jakob disease (CJD) has shown encouraging early results and may contribute to the development of new treatments for other neurodegenerative diseases. […] CJD is a rare and fatal disease that causes brain damage and for which there is currently no licensed treatment. […] Researchers at the NIHR UCLH Biomedical Research Centre (BRC) developed a monoclonal antibody, called PRN100, that was given to six patients with CJD between October 2018 and July 2019. […] The results, published in Lancet Neurology, show the treatment is safe and able to access the brain. […] In three patients, disease progression appeared to stabilise when dosing levels were in target range. […] None of the six patients experienced side effects while receiving the treatment but all sadly died as a result of their condition.

• #29 World-first CJD treatment shows promising early results | UCL News – UCL – University College London

  https://www.ucl.ac.uk/news/2022/mar/world-first-cjd-treatment-shows-promising-early-results

  A world-first treatment for Creutzfeldt-Jakob disease (CJD), developed by scientists at the Medical Research Council (MRC) Prion Unit at UCL, has shown very encouraging early results following its use in six patients at University College London Hospitals (UCLH) NHS Foundation. […] Researchers at the MRC Prion Unit at UCL have developed a monoclonal antibody, called PRN100, which was given to six UCLH patients with CJD between October 2018 and July 2019. […] The results, published in the Lancet Neurology, show the treatment is safe and able to access the brain. In three patients, disease progression appeared to stabilise when dosing levels were in target range. […] This is the first time in the world a drug specifically designed to treat CJD has been used in humans and the results are very encouraging.

• #30 Finding a treatment for CJD – The Cure CJD Campaign

  https://curecjd.org/what-is-cjd/finding-a-treatment-for-cjd/

  World-leading research at the MRC Prion Unit at UCL developed an experimental antibody treatment called PRN100. This was designed to stop prions from malfunctioning in the brain. […] Positive results were shown in laboratory testing and a small number of patients were given PRN100 in an experimental treatment programme in 2018/2019. […] UCLH NHS Foundation Trust approved a Treatment Programme using the limited amount of PRN100 that was available. Six patients were treated in this way exhausting all the material available. Although all of these patients eventually died, the results of the Programme were encouraging, with valuable information (set out in the Lancet Neurology article) which will be helpful in developing the treatment further. […] Funding for more research is required to keep momentum going in the quest for a successful treatment. […] Professor John Collinge discusses a first-in-human treatment programme to give PRN100, an anti-prion-protein monoclonal antibody, to patients with CreutzfeldtJakob disease; the report is published in the April 2022 issue of The Lancet Neurology.

• #31 World-first CJD treatment shows promising early results | UCL News – UCL – University College London

  https://www.ucl.ac.uk/news/2022/mar/world-first-cjd-treatment-shows-promising-early-results

  While the number of patients we treated was too small to determine whether the drug altered the course of the disease, this is nevertheless an important step forward in targeting prion infections. […] We hope the drug may also have the potential to prevent the onset of symptoms in people at risk of prion disease due to genetic mutations or accidental prion exposure and may contribute to the development of therapies for more common dementias, such as Alzheimers disease. […] We are encouraged by these results which demonstrate the treatment is safe and there is some signal of benefit. The hope is that this could pave the way for new treatments for other neurodegenerative diseases.

• #32 World-first CJD treatment shows promising early results | UCL News – UCL – University College London

  https://www.ucl.ac.uk/news/2022/mar/world-first-cjd-treatment-shows-promising-early-results

  While the number of patients we treated was too small to determine whether the drug altered the course of the disease, this is nevertheless an important step forward in targeting prion infections. […] We hope the drug may also have the potential to prevent the onset of symptoms in people at risk of prion disease due to genetic mutations or accidental prion exposure and may contribute to the development of therapies for more common dementias, such as Alzheimers disease. […] We are encouraged by these results which demonstrate the treatment is safe and there is some signal of benefit. The hope is that this could pave the way for new treatments for other neurodegenerative diseases.

• #33

  https://pmc.ncbi.nlm.nih.gov/articles/PMC9703894/

  Flupirtine is a triaminopyridine derivative that acts as a non-opioid analgesic. It is a well-tolerated drug that acts on the CNS, and has demonstrated a high cytoprotective effect in vitro and in vivo on neurons induced to apoptosis. […] Doxycycline is an antibiotic from the tetracycline group, whose effect is directly aimed at inhibiting protein synthesis, which prevents the nutrition of bacteria. […] PPS is a drug with anticoagulant and fibrinolytic effects. Studies conducted have hypothesized that infusion of the drug could inhibit PrPSc formation. […] To date, there are no robust studies that provide support for pharmacological approaches with doxycycline, PPS, or quinacrine in increasing survival of CJD patients. However, one clinical trial has suggested success of flupirtine in partially reducing cognitive deterioration in this population, when compared with the control group.

• #34

  https://pmc.ncbi.nlm.nih.gov/articles/PMC9703894/

  Flupirtine is a triaminopyridine derivative that acts as a non-opioid analgesic. It is a well-tolerated drug that acts on the CNS, and has demonstrated a high cytoprotective effect in vitro and in vivo on neurons induced to apoptosis. […] Doxycycline is an antibiotic from the tetracycline group, whose effect is directly aimed at inhibiting protein synthesis, which prevents the nutrition of bacteria. […] PPS is a drug with anticoagulant and fibrinolytic effects. Studies conducted have hypothesized that infusion of the drug could inhibit PrPSc formation. […] To date, there are no robust studies that provide support for pharmacological approaches with doxycycline, PPS, or quinacrine in increasing survival of CJD patients. However, one clinical trial has suggested success of flupirtine in partially reducing cognitive deterioration in this population, when compared with the control group.

• #35 Rethinking of doxycycline therapy in Creutzfeldt-Jakob disease | Journal of Neurology, Neurosurgery & Psychiatry

  https://jnnp.bmj.com/content/86/7/705

  Compassionate use of doxycycline, a tetracycline antibiotic, in patients with Creutzfeldt-Jakob disease (CJD) revealed an increased survival of 47 months as compared with historical controls, a result not confirmed by a randomised, double blind, placebo-controlled trial. […] The report of Assar et al on a single patient with variably protease-sensitive prionopathy (VPSPr), a rare subtype form of sporadic CJD, who received 4-year treatment with doxycycline at a relatively early stage of disease, suggests it is not and encourages novel studies on the use of doxycycline in prion diseases.

• #36

  https://pmc.ncbi.nlm.nih.gov/articles/PMC9703894/

  Flupirtine is a triaminopyridine derivative that acts as a non-opioid analgesic. It is a well-tolerated drug that acts on the CNS, and has demonstrated a high cytoprotective effect in vitro and in vivo on neurons induced to apoptosis. […] Doxycycline is an antibiotic from the tetracycline group, whose effect is directly aimed at inhibiting protein synthesis, which prevents the nutrition of bacteria. […] PPS is a drug with anticoagulant and fibrinolytic effects. Studies conducted have hypothesized that infusion of the drug could inhibit PrPSc formation. […] To date, there are no robust studies that provide support for pharmacological approaches with doxycycline, PPS, or quinacrine in increasing survival of CJD patients. However, one clinical trial has suggested success of flupirtine in partially reducing cognitive deterioration in this population, when compared with the control group.

• #37 Creutzfeldt–Jakob disease – Wikipedia

  https://en.wikipedia.org/wiki/Creutzfeldt%E2%80%93Jakob_disease

  As of 2025, there is no cure or effective treatment for CJD. Some of the symptoms like twitching can be managed, but otherwise treatment is palliative care. Psychiatric symptoms like anxiety and depression can be treated with sedatives and antidepressants. Myoclonic jerks can be handled with clonazepam or sodium valproate. Opiates can help in pain. Seizures are very uncommon but can nevertheless be treated with antiepileptic drugs. […] Pentosan polysulfate (PPS) may slow the progression of the disease, and may have contributed to the longer than expected survival of the seven people studied. The CJD Therapy Advisory Group to the UK Health Departments advises that data are not sufficient to support claims that pentosan polysulfate is an effective treatment and suggests that further research in animal models is appropriate. A 2007 review of the treatment of 26 people with PPS finds no proof of efficacy because of the lack of accepted objective criteria, but it was unclear to the authors whether that was caused by PPS itself. In 2012 it was claimed that the lack of significant benefits has likely been caused because of the drug being administered very late in the disease in many patients.

• #38

  https://pmc.ncbi.nlm.nih.gov/articles/PMC9703894/

  The Creutzfeldt-Jakob disease (CJD) is a spongiform encephalopathy that manifests as a rapidly progressive dementia syndrome. Currently, CJD has no cure, and many patients die within the first year, but some drugs are being studied as options for managing this condition. […] None of the drugs evaluated proved significantly effective in increasing survival in patients with CJD. Flupirtine appears to have a beneficial effect in reducing cognitive deterioration in patients with CJD. However, additional studies are needed to establish better evidence and therapeutic options for the management of patients with CJD. […] Several potential therapeutic interventions are under development over the last years, but no treatment has been proven effective for CJD. […] Studies have shown that the malaria treatment drug quinacrine and the antipsychotic chlorpromazine are effective in clearing prions in non-human models.

• #39 Variant Creutzfeldt-Jakob Disease and Bovine Spongiform Encephalopathy Treatment & Management: Approach Considerations, Experimental Treatments

  https://emedicine.medscape.com/article/1169688-treatment

  A number of experimental interventions are currently being studied. They include some of the conventional medications, such as the antimalarial quinacrine and the antipsychotic chlorpromazine, which prevent conversion of the normal prion protein (PrPc) to abnormal prion protein (PrPSc), according to in vitro studies. […] Pentosan polysulphate (PPS) also has effects on prion protein production, replication, and associated cell toxicity. Some experimental results showed that if PPS is given to animals at a time relatively close to the point of experimental infection, an increase in the incubation period of disease may occur; in some instances, animals appear to be completely protected from the development of disease. […] Flupirtine has shown some beneficial effects on cognitive function in patients with CJD but without any evidence of increased survival. […] One strategy involves designer compounds that interact with PrPSc structure, inhibiting the conformation change of PrPc associated with the disease. […] Another potential approach is immunologic, in which immunization with alpha s-helix peptides is used to reduce cerebral amyloid accumulation.

• #40 Neural cell engraftment therapy for sporadic Creutzfeldt-Jakob disease restores neuroelectrophysiological parameters in a cerebral organoid model | Stem Cell Research & Therapy | Full Text

  https://stemcellres.biomedcentral.com/articles/10.1186/s13287-023-03591-2

  Sporadic Creutzfeldt-Jakob disease (sCJD), the most common human prion disease, is a fatal neurodegenerative disease with currently no treatment options. […] Together with the previous animal studies, our results support that cell therapy may have some functional benefit for the treatment of human prion diseases. […] One potential avenue for recovering cognitive function is the use of neural progenitor cells (NPCs). […] NPC transplantation has previously been shown to extend survival time in murine prion diseases, and as NPCs have the ability to integrate into the tissue and form new neurons, we hypothesized that restoration of function might be possible following NPC augmentation of organoids in culture. […] Our findings show that while the beneficial effects of NPC addition were mild, they were significant, increasing the detection of neuronal markers in the organoids as well as improving certain neuroelectrophysiological parameters.

• #41 Neural cell engraftment therapy for sporadic Creutzfeldt-Jakob disease restores neuroelectrophysiological parameters in a cerebral organoid model | Stem Cell Research & Therapy | Full Text

  https://stemcellres.biomedcentral.com/articles/10.1186/s13287-023-03591-2

  Our results indicate that NPCs have the potential to restore some function in COs that have established prion disease. […] Improving patient quality of life is a greatly sought-after aim when developing therapeutics for neurological diseases; extending life with no functional recovery offers little benefit for patients or their caregivers. […] Herein, we show that NPC supplementation into COs with established prion infection can improve neuronal network function.

• #42 Hope for prion diseases | Drug Discovery News

  https://www.drugdiscoverynews.com/hope-for-prion-diseases-15814

  Investigating preventative measures requires leveraging genetic information to show who’s at risk ahead symptom onset. It also means a certain way of thinking about drug development, where we think about creative ways to test drugs in healthy people for their ability to prevent disease, said Vallabh. […] Research groups are exploring a variety of approaches to target this protein and its downstream effects on the brain; Vallabh and Minikel plan to design a therapy to reduce the amount of prion protein in the brain. There are strong proof-of-concept studies to support this as a promising strategy, said Vallabh. […] Vallabh and Minikel began speaking with Ionis Pharmaceuticals about a potential therapeutic strategy using antisense oligonucleotides (ASO) to reduce target RNA levels in the brain.

• #43 Hope for prion diseases | Drug Discovery News

  https://www.drugdiscoverynews.com/hope-for-prion-diseases-15814

  Vallabh and Minikel established a collaboration with the company in 2016. In 2019, the research team produced an ASO that ameliorates disease caused by the Rocky Mountain Laboratory strain of prions in mice. […] According to Vallabh, the efficacy of the ASO against several different strains is especially promising and is not necessarily true of many potential therapeutics. […] Now that researchers have established that ASO can lower prion protein RNA in mice, Vallabh said that the question now is one of therapeutic window: Can researchers develop an ASO that can reduce prion protein enough, in a large enough area of the human brain, while still being tolerated? […] In October 2018, the team initiated a first-in-human trial. Six patients with Creutzfeldt-Jakob prion disease received intravenous infusions of the new antibody therapy, which is called PRN100.

• #44 Research | A new trial hopes to use mRNA to halt the devastating progression of Creutzfeldt-Jakob Disease | Channels – McGill University

  https://www.mcgill.ca/channels/channels/news/research-new-trial-hopes-use-mrna-halt-devastating-progression-creutzfeldt-jakob-disease-361983

  There is no treatment to slow progression. Once individuals become symptomatic with typical CJD, the median survival is less than six months, confirms Dr. Pandolfo. […] A new study at the CRU hopes to lead to a very different outcome for these patients. […] The study underway at The Neuro is the first ever in Canada to evaluate whether a treatment using an antisense oligonucleotide (ASO) therapy would be effective at stopping the misfolding of the prion protein, stalling the progression of the disease. […] One of the things you can do with ASO is repress the expression of a gene. If your body stops expressing the prion protein, we should be able to slow or stop the process, explains Dr. Pandolfo. ASO therapy has already been used successfully to treat other neurological conditions, and data in animal models that confirm that lowering the expression of prion protein significantly delay and slow CJD.

• #45 Drug Development – CJD Foundation

  https://cjdfoundation.org/drug-development/

  Human prion diseases, including CreutzfeldtJakob disease (CJD), are rapidly progressive, invariably fatal neurodegenerative conditions with no effective therapies. Their pathogenesis involves the obligate recruitment of cellular prion protein (PrPC) into self-propagating multimeric assemblies or prions. Preclinical studies have firmly validated the targeting of PrPC as a therapeutic strategy. […] Learn about Prion protein monoclonal antibody (PRN100) therapy for CJD from this recording of the 2022 Virtual Conference Session presented by Prof John Collinge, Professor of Neurology, MRC Prion Unit at UCL, UCL Institute of Prion Diseases. […] Researchers evaluated a novel epigenetic regulation approach using Zinc Finger Repressors (ZFRs) to ablate PrP expression at the transcriptional level. When delivered using adeno-associated virus (AAV), ZFRs potently and specifically reduced prion mRNA expression by 95% in vitro and to near undetectable levels within single neurons in vivo. In wildtype mice, ZFRs stably lowered neuronal PrP expression throughout the central nervous system for at least 17 months. In mice inoculated with misfolded PrP, AAV-ZFRs given at either early or late disease stages profoundly extended lifespan, significantly reduced PrP in the brain, and improved an array of molecular, histological, biomarker, and behavioral readouts.

• #46 Azthena logo with the word Azthena

  https://www.news-medical.net/news/20240516/Repurposing-human-approved-drugs-for-prion-disease-treatment.aspx

  „From a clinical standpoint, we believe this research has uncovered anti-prion properties of drugs that have already been shown to be safe to use in humans. Because of this, especially considering the absence of any effective treatment for these diseases, these compounds could be re-purposed for treatment of prion diseases,” said corresponding author David A. Harris, MD, PhD, the Edgar Minas Housepian professor and chair of biochemistry cell biology at the school. […] „Excitingly, five of these molecules have a history of use in humans: rimcazole and haloperidol for neuropsychiatric conditions, (+)-pentazocine for neuropathic pain, and SA 4503 and ANAVEX2-73, which are in clinical trials for ischemic stroke and Alzheimer’s disease, respectively,” explained lead author Robert C.C. Mercer, PhD, an instructor of biochemistry and cell biology at the school.

• #47 Variant Creutzfeldt-Jakob Disease and Bovine Spongiform Encephalopathy Treatment & Management: Approach Considerations, Experimental Treatments

  https://emedicine.medscape.com/article/1169688-treatment

  A number of experimental interventions are currently being studied. They include some of the conventional medications, such as the antimalarial quinacrine and the antipsychotic chlorpromazine, which prevent conversion of the normal prion protein (PrPc) to abnormal prion protein (PrPSc), according to in vitro studies. […] Pentosan polysulphate (PPS) also has effects on prion protein production, replication, and associated cell toxicity. Some experimental results showed that if PPS is given to animals at a time relatively close to the point of experimental infection, an increase in the incubation period of disease may occur; in some instances, animals appear to be completely protected from the development of disease. […] Flupirtine has shown some beneficial effects on cognitive function in patients with CJD but without any evidence of increased survival. […] One strategy involves designer compounds that interact with PrPSc structure, inhibiting the conformation change of PrPc associated with the disease. […] Another potential approach is immunologic, in which immunization with alpha s-helix peptides is used to reduce cerebral amyloid accumulation.

• #48 Caregiver Support – CJD Foundation

  https://cjdfoundation.org/caregiver-support/

  Families who receive a prion disease diagnosis are often shocked, confused and overwhelmed. They must rapidly accept the news that their loved one who probably appeared quite healthy just weeks ago is suffering from a terminal disease that has no treatment. […] Families who receive a prion disease diagnosis should contact hospice as early as possible. While hospice care may not be immediately needed in the early stages, the patients needs are likely to evolve rapidly. Choosing a hospice provider and setting up care in advance will free the family to focus more on the patient later. Hospice providers bring knowledge and resources that are invaluable to a family that is confronting rapidly evolving care needs. […] As the disease progresses, you will want to contact the physician, hospice, or healthcare provider about any concerns, such as: New symptoms that are not easily managed, particularly hallucinations, and extremely fearful or violent behavior.

• #49 Creutzfeldt-Jakob Disease Symptoms, Doctors, Treatments, Advances & More | MediFind

  https://www.medifind.com/conditions/creutzfeldt-jakob-disease/5727

  There is no cure for this condition. Different medicines have been tried to slow the disease, but none works well. […] The goals of treatment include: Provide a safe environment, Control aggressive or agitated behavior, Make the person comfortable, Meet the person’s needs. […] This may require monitoring and assistance in the home or in a care facility. Family counseling may help the family cope with the changes needed for home care. […] People with this condition may need help controlling unacceptable or dangerous behaviors. This involves rewarding positive behaviors and ignoring negative behaviors (when it is safe). They may also need help getting oriented to their surroundings. Sometimes, medicines are needed to help control aggression. […] It is helpful for people with CJD and their family to seek legal advice early on. Advance directive, power of attorney, and other legal actions can make it easier to make decisions about the care of the person with CJD.

• #50 Creutzfeldt-Jakob Disease Symptoms, Doctors, Treatments, Advances & More | MediFind

  https://www.medifind.com/conditions/creutzfeldt-jakob-disease/5727

  There is no cure for this condition. Different medicines have been tried to slow the disease, but none works well. […] The goals of treatment include: Provide a safe environment, Control aggressive or agitated behavior, Make the person comfortable, Meet the person’s needs. […] This may require monitoring and assistance in the home or in a care facility. Family counseling may help the family cope with the changes needed for home care. […] People with this condition may need help controlling unacceptable or dangerous behaviors. This involves rewarding positive behaviors and ignoring negative behaviors (when it is safe). They may also need help getting oriented to their surroundings. Sometimes, medicines are needed to help control aggression. […] It is helpful for people with CJD and their family to seek legal advice early on. Advance directive, power of attorney, and other legal actions can make it easier to make decisions about the care of the person with CJD.

• #51 Creutzfeldt–Jakob disease: From presentation to palliative care

  https://www1.racgp.org.au/ajgp/2024/october/creutzfeldt-jakob-disease

  Currently there are no treatments for CJD. Symptomatic and supportive care with palliative care involvement helps manage symptoms and support patients and families. […] A recent study from the UK using prion protein monoclonal antibody (PRN100) in humans showed that it appeared to be safe and reached encouraging CSF and brain tissue concentrations. Identifying suspected CJD diagnoses and specialist involvement may facilitate referral for consideration of early enrolment in future treatment or research trials if desired. […] There are no consensus guidelines for the palliative management of this rare disease. A holistic focus with a multidisciplinary team is essential. Patients can progress daily, with management strategies requiring daily review in many cases. […] Antipsychotic medications such as olanzapine, benzodiazepines like clonazepam or a combination may be required. Planning for a transition to subcutaneous medications when swallowing is impaired is vital. Palliative care support throughout is helpful, particularly with the transition to medications for symptoms at the end of life.

• #52 World-first treatment for CJD shows promising early results | NIHRopens in a new window

  https://www.nihr.ac.uk/news/world-first-treatment-cjd-shows-promising-early-results

  Given the small number of patients treated, researchers say the findings should be regarded as preliminary and further studies are needed to draw more comprehensive conclusions. […] Professor John Collinge, Director of the MRC Prion Unit at UCL who led the development of the PRN100 treatment, said: “This is the first time in the world a drug specifically designed to treat CJD has been used in humans and the results are very encouraging. […] While the number of patients we treated was too small to determine whether the drug altered the course of the disease, this is nevertheless an important step forward in targeting prion infections. […] It has been a huge challenge to reach this milestone and we still have a long way to go but we have learned a great deal and these results now justify developing a formal clinical trial in a larger number of patients.”

• #53 World-first treatment for CJD shows promising early results | NIHRopens in a new window

  https://www.nihr.ac.uk/news/world-first-treatment-cjd-shows-promising-early-results

  Looking further into the future, Professor Collinge added: “We hope the drug may also have the potential to prevent the onset of symptoms in people at risk of prion disease due to genetic mutations or accidental prion exposure and may contribute to the development of therapies for more common dementias, such as Alzheimer’s disease.” […] Professor Bryan Williams, director of the NIHR UCLH BRC, said: “Creutzfeldt-Jakob disease (CJD) is a rare and cruel disease which rapidly destroys the brain and for which there is currently no cure or licensed treatment. […] We are encouraged by these results which demonstrate the treatment is safe and there is some signal of benefit. The hope is that this could pave the way for new treatments for other neurodegenerative diseases.”

• #54 Creutzfeldt–Jakob disease: From presentation to palliative care

  https://www1.racgp.org.au/ajgp/2024/october/creutzfeldt-jakob-disease

  Currently there are no treatments for CJD. Symptomatic and supportive care with palliative care involvement helps manage symptoms and support patients and families. […] A recent study from the UK using prion protein monoclonal antibody (PRN100) in humans showed that it appeared to be safe and reached encouraging CSF and brain tissue concentrations. Identifying suspected CJD diagnoses and specialist involvement may facilitate referral for consideration of early enrolment in future treatment or research trials if desired. […] There are no consensus guidelines for the palliative management of this rare disease. A holistic focus with a multidisciplinary team is essential. Patients can progress daily, with management strategies requiring daily review in many cases. […] Antipsychotic medications such as olanzapine, benzodiazepines like clonazepam or a combination may be required. Planning for a transition to subcutaneous medications when swallowing is impaired is vital. Palliative care support throughout is helpful, particularly with the transition to medications for symptoms at the end of life.

• #55 Creutzfeldt-Jakob Disease | National Institute of Neurological Disorders and Stroke

  https://www.ninds.nih.gov/health-information/disorders/creutzfeldt-jakob-disease

  Currently theres no cure for CJD or any other prion disease, although researchers are testing some drugs to control CJD. Today’s treatments focus on making the person comfortable and easing symptoms. Medications may help relieve behavioral changes, seizures that may develop, and muscle jerks. […] During later stages of the disease, people may need IV (intravenous) fluids and machine feeding. But because CJD and other prion diseases are incurable, these approaches can have limited use. Toward the end of life, people with CJD may receive hospice services.

• #56 Caregiver Support – CJD Foundation

  https://cjdfoundation.org/caregiver-support/

  Families who receive a prion disease diagnosis are often shocked, confused and overwhelmed. They must rapidly accept the news that their loved one who probably appeared quite healthy just weeks ago is suffering from a terminal disease that has no treatment. […] Families who receive a prion disease diagnosis should contact hospice as early as possible. While hospice care may not be immediately needed in the early stages, the patients needs are likely to evolve rapidly. Choosing a hospice provider and setting up care in advance will free the family to focus more on the patient later. Hospice providers bring knowledge and resources that are invaluable to a family that is confronting rapidly evolving care needs. […] As the disease progresses, you will want to contact the physician, hospice, or healthcare provider about any concerns, such as: New symptoms that are not easily managed, particularly hallucinations, and extremely fearful or violent behavior.

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