Glucocerbrosidase Enhancing The Cells Lysosomal System
GBAis a gene that increases the risk of developing PD. The GBA protein works in the lysosome, the garbage disposal system of the cell, breaking down cellular products that can be harmful to the cell. Having two abnormal GBA genes causes Gauchers disease, which is characterized by the buildup of these cellular products. This results in fatigue, bone pain, easy bleeding and an enlarged spleen and liver. When a person inherits only one abnormal gene, he or she does not develop Gauchers disease however, they do incur a small increased risk of PD. Most people with one mutated GBA gene do not develop PD.
Enzyme replacement therapy, in which the GBA protein is given intravenously, is available as a treatment for Gauchers disease. This protein is too big to cross the blood-brain-barrier however, and so it does not enter the brain and does not treat any symptoms caused by the abnormal buildup of cellular components in the brain. The following strategies were developed in an attempt to compensate for the effects of the GBA mutation in the brain:
- Ambroxol, approved in Europe for respiratory illnesses, improves the function of GBA in neurons NCT02941822 and NCT02914366
- These small molecules can cross the blood-brain-barrier and help decrease the amount of accumulated cellular products in the brain:
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Exploring Seven Recently Approved Parkinsons Treatments
Remarkably, in the last five years, seven new medications have been approved for the treatment of the motor symptoms of Parkinsons disease , with two approved in 2020. Thats exciting progress! And while it is great to have so many choices, the various options can be confusing so today I will describe these new medications and their uses.
How Could Stem Cells Help People With Parkinson’s
Stem cells are the parent cells of all tissues in the body. This means they can turn into any type of cell. The hope is that they will eventually be able to make these cells into specific types of cells, like dopamine-producing neurons, that can be used to treat Parkinson’s disease. However, there are concerns that patients may have the same risk of increased involuntary movements as those who undergo fetal cell transplantation. And, like fetal cell transplantation, stem cell therapy is surrounded by moral and ethical controversy.
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Will New Treatments Really Help
According to multiple clinical studies, treatment with Inbrija led to significant improvement in motor function, with an onset of 10 minutes. A review of 900 patients showed a reduction in the Unified Parkinson’s Disease Rating Scale, 30 minutes post-dose, after 12 weeks of treatment. Overall, 75% of patients who took Inbrija experienced a decline of daily OFF times.
According to Robert A. Hauser, MD, professor of neurology and director of the Parkinsons Disease and Movement Disorders Center at the University of South Florida:
Inbrija helps address a significant unmet need for people with Parkinsons, and we look forward to adding this new treatment option to our armamentarium.
Parkinsons Disease: Major Drug Trial Results To Watch In 2022
Four clinical trials in Parkinsons disease with readouts the second half of 2022 target motor symptom fluctuations and cognitive impairment.
In a slate of major Parkinsons disease trial readouts, drug developers are taking aim at the diseases most common symptom: motor fluctuations.
Around 80% of people with PD have some motor symptom fluctuations, says Dr Jeffrey Kordower, founding director of Arizona State Universitys Banner Neurodegenerative Disease Research Center. The rationale behind going after these symptoms is really strong.
Four major trials have readouts expected in the next six months, three of which target motor symptoms, also known as dyskinesia. Two trials focus on dyskinesia resulting from sustained use of generic levodopa, which is widely considered the standard of care in PD. A third study tests a new formulation of levodopa that could reduce treatment side effects such as dyskinesia, while a fourth trial takes aim at cognitive impairment in PD.
Parkinsons disease is a progressive central nervous system disease associated with deficiencies in the neurotransmitter dopamine. There are no disease-altering treatments available, but levodopa can increase dopamine levels in the brain and reduce symptoms.
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Treatments In Phase 3 Clinical Trials
DynaCirc is a calcium channel blocker already in use to treat high blood pressure. The drug may block damage caused by certain chemicals that flow through special channels in the brain cells that make dopamine. The drugs effectiveness in early Parkinsons disease is being evaluated in a Phase 3 study .
Note: Parkinsons News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.
What Are The Side Effects Of Parkinson’s Drugs
The most common reactions include nausea, vomiting, dizziness , sleepiness and visual hallucinations.
In the last few years, levodopa and dopamine agonists in particular have been associated with the emergence of behavioral changes such as impulse control disorders. These are characterized by failure to resist an impulse to perform certain actions.
Impulse control disorders include a range of behaviors such as compulsive gambling or shopping, hypersexuality, binge eating, addiction to the Internet or to other recreational activities. These activities are often pleasant in the moment, but over time may become harmful to you or to others. If you are experiencing these behaviours, tell your neurologist/doctor. Often the medication can be adjusted which can reduce or control the behaviour.
Care partners can play an important role in helping to identify when these behaviours occur. If you are a care partner, tell the person if you have noticed a change in his/her behaviour or personality and encourage him/him/her to speak with the doctor immediately so medication can be adjusted.
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Latest News In Parkinson Disease: Treatment Progress Cognition Improvements And More
An overview of the latest news in Parkinson disease reported across MJH Life Sciences.
An overview of the latest news in Parkinson disease reported across MJH Life Sciences.
FDA Approves Investigational NDA for Ketamine in Levodopa-Induced Dykinesia
As the gold standard of treatment for PD, levodopa effectively reduces parkinsonian symptoms, although long-term use has been linked with several adverse events. Chief among these, frequency of OFF time and abnormal involuntary movements, known as levodopa-induced dyskinesia , have been shown to significantly impact quality of life and treatment efficacy.
There are no approved treatments to address LID however, an article by NeurologyLive® indicates there may be some progress in addressing this issue. Last week, the FDA approved PharmaTher Holdings investigational new drug application for ketamine, an N-methyl-D-aspartate receptor-modulating drug, in the treatment of LID in patients with PD.
A phase 2 clinical trial evaluating the safety, efficacy, and pharmacokinetics of ketamine compared with the active control treatment of midazolam is expected to begin patient enrollment in the third quarter of this year. Pending success, the manufacturer noted that it will seek an agreement with the FDA to proceed to a phase 3 clinical study next year.
Neurological Disease Link With COVID-19 Severity, Death
A New Wearable System Tracks Parkinson’s Disease Symptoms Remotely
Parkinson’s disease affects 10 million people worldwide and its symptoms include tremors in the fingers and hands, small handwriting, loss of smell, walking difficulties, dizziness, and others. As these symptoms worsen over time, monitoring and treating PD is crucial to preserve the patients’ autonomy and enhance their quality of life.
This is the goal of a group of Portuguese researchers. The project iHandUapp extended the iHandU project to the full cycle of the PD. It resorts to mobile and wearable proprietary technologies to monitor PD patients’ symptoms, with the possibility of sharing the data with their doctors in real-time, including quantitative automatic evaluation of the symptoms, medication or even disease-related events.
James Parkinson was an English surgeon, geologist and political activist born in 1755. After almost being arrested due to his political career, he directed his attention to Medicine, following the steps of his father. As a strong advocate of the underprivileged, Parkinson focused on improving the general health of the population. The peak of his career in Medicine was the publication of “An Essay on the Shaking Palsy,” in which he described six patients with similar symptoms of unintended shaking. The disease that today bears his name was coined by the French neurologist Jean-Martin Charcot about 60 years after.
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What Will A Cure For Parkinson’s Look Like
Parkinson’s varies so much from person to person. There are over 40 symptoms of Parkinsons. Tremor. Pain. Hallucinations. Everyones experience is different.
Because of this, there may not be a single ‘cure’.
Instead, we may need a range of different therapies to meet the needs of the individual and their specific form of the condition.
This mix may include treatments, therapies and strategies that can:
- slow or stop the progression of the condition
- replace or repair lost or damaged brain cells
- control and manage particular symptoms
- diagnose Parkinson’s at the earliest possible stage.
And this could involve medical treatments, such as drugs and surgical approaches, as well as lifestyle changes, for example to diet and exercise.
Glucagonlike Peptide 1 Receptor Agonists And Other Antidiabetic Agents
Biological processes involved in PD share common features with obesity and type 2 diabetes mellitus , including the dysregulation of insulin signaling in the brain. The term brain insulin resistance has been suggested to describe decreased sensitivity of CNS pathways to insulin, followed by disturbances in synaptic, metabolic and immune response functions . Strategies to normalize insulin sensitivity in neurons have thus been in the spotlight of clinical trials aiming to establish whether they may provide neuroprotective actions.
The neuroprotective effect of GLP-1 RAs is assumed to be mediated by improved brain insulin sensitivity however, human studies evaluating their biological effect in the CNS are limited. Functional MRI imaging studies have primarily focused on investigating brain networks involved in the anorectic effect of GLP-1 RAs , but sparse mechanistic data are available for understanding neuroprotective effects of these drugs. In a more recent trial of exenatide in PD, disease modifying effects measured by nigrostriatal dopamine transporter imaging were reported . Subsequently, brain insulin and Akt signaling pathways were also evaluated in neuronal-derived exosomes and it was shown that exenatide treatment, but not placebo, activated these pathways . This significant, secondary analysis of the trial increases understanding of the molecular mechanism underlying the treatment effect and provides a possible biomarker to measure target engagement.
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Designer Neurons Offer New Hope For Treatment Of Parkinson’s Disease
- Date:
- Arizona State University
- Summary:
- Scientists describe a process for converting non-neuronal cells into functioning neurons able to take up residence in the brain, send out their fibrous branches across neural tissue, form synapses, dispense dopamine and restore capacities undermined by Parkinson’s destruction of dopaminergic cells.
Neurodegenerative diseases damage and destroy neurons, ravaging both mental and physical health. Parkinson’s disease, which affects over 10 million people worldwide, is no exception. The most obvious symptoms of Parkinson’s disease arise after the illness damages a specific class of neuron located in the midbrain. The effect is to rob the brain of dopamine — a key neurotransmitter produced by the affected neurons.
In new research, Jeffrey Kordower and his colleagues describe a process for converting non-neuronal cells into functioning neurons able to take up residence in the brain, send out their fibrous branches across neural tissue, form synapses, dispense dopamine and restore capacities undermined by Parkinson’s destruction of dopaminergic cells.
The current proof-of-concept study reveals that one group of experimentally engineered cells performs optimally in terms of survival, growth, neural connectivity, and dopamine production, when implanted in the brains of rats. The study demonstrates that the result of such neural grafts is to effectively reverse motor symptoms due to Parkinson’s disease.
New perspectives on Parkinson’s disease
What Is Parkinsons Disease
Parkinsons disease is a progressive brain disorder that causes shaking and muscle stiffness, and slows movement. It develops when neurons in a particular part of the brain stop working properly and are lost over time. These neurons produce an important chemical called dopamine. Dopamine is used by the brain to send messages across brain areas to help control movement. Eventually, the brain cannot make enough dopamine to control the movement properly.1,2
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Important Points About The New Medications
With multiple new medications available for the treatment of PD, there is more hope than ever that Parkinsons symptoms can be successfully managed for many years. A few things to consider:
- For people whose symptoms are difficult to control, these new treatments are welcome additions to what was previously available and many people with PD have been using these new medications with significant benefit.
- On the other hand, many of the newly-approved medications have the same mechanisms of action as older medications so they are not breaking new ground in treating symptoms.
- In addition, for some people, the effect on symptoms may be mild or not substantial.
These caveats may mean that your physician has not suggested a medication change for you. It is also important to note that despite all the new medications, carbidopa/levodopa remains the most potent medication to treat the motor symptoms of PD.
If your doctor does choose to try one of the new options, there may be multiple paths that your doctor can take when contemplating a medication adjustment. Often trial and error is the only way to determine the best medication regimen for you, so you may need to practice some patience as you work together with your doctor to determine what works or doesnt work.
Causes Of Parkinson’s Disease
The causes of Parkinsons disease are still greatly unknown. Scientists who have studied this disorder estimate that 10-15% of cases come from genetics after seeing a series of genetic mutations that were common in Parkinsons patients.
Doctors suspect that environmental factors and lifestyle choices may have effects on the severity of Parkinsons disease symptoms. Exposure to chemicals like pesticides may increase the likelihood of developing Parkinsons disease. On the other hand, a good diet and regular exercise may decrease your chances.
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What Are Surgery Options For Parkinsons Disease
Depending upon your needs, medical history, health, and symptoms, one of the following procedures may be considered for Parkinsons disease:
There are many other procedures being researched. One of the most promising involves the transplantation of fetal dopamine neurons into the brains of people with Parkinsons disease. The hope is that these cells will be able to re-grow the damaged dopamine-producing nerve cells.
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What We Know So Far
- Weve uncovered clues to the causes and genetic involvement in Parkinsons.
- Were figuring out the chain of events that leads to the damage and loss of brain cells.
- Were working to advance new treatments and therapies.
- Were exploring repurposing drugs to help manage some of the more distressing symptoms, like hallucinations and falls.
- And we know that, although people with Parkinsons share symptoms, each persons experience of the condition and response to treatment is different.
Now, the science is ready for us to develop the new treatments and cure that people with Parkinsons so desperately need.
Research takes time but if you have Parkinsons, you need better treatments now. Thats why weve launched the Parkinsons Virtual Biotech to speed up the most promising potential treatments. The more we can invest, the sooner well get there.
Why Scientists Believe Theyve Made New Breakthrough In Parkinsons Disease Treatment By Building On Gdnf Research
The Finnish researchers are now working to improve the properties of BT13 to make it more effective as a potential treatment that could benefit many people living with the disease.
The study, which was published online yesterday in the journal Movement Disorders, builds on previous research on another molecule that targets the same receptors in the brain.
GDNF or glial cell line-derived neurotrophic factor is an experimental treatment for Parkinsons discovered in 1993 that has been shown to bring dying brain cells back to life and particularly effective in dopamine neurons.
It was the subject of a BBC documentary in February 2019 that followed a phase two trial in Bristol involving 42 patients. While the results werent clear cut, GDNF has shown promise to restore damaged cells in people with Parkinsons.
However, the GDNF protein requires complex robot-assisted surgery to deliver the treatment to the brain because its a large molecule that cant cross the blood-brain barrier a protective wall that prevents some drugs from getting into the brain.
BT13 is a smaller molecule that is able to cross the blood-brain barrier and therefore could be more easily administered as a treatment if shown to be beneficial in further clinical trials.
Dr Yulia Sidorova, lead researcher on the study, said: We are constantly working on improving the effectiveness of BT13.
Our ultimate goal is to progress these compounds to clinical trials in a few coming years.
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