A Busy Year Ahead For Parkinsons Disease
Parkinsons disease research has ended in numerous dead ends despite substantial efforts over many years. Recently, Biogen and Sanofi scrapped their Parkinsons candidates, cipanemab and venglustat respectively, owing to lack of efficacy, and a disease-modifying therapy has yet to materialise.
But the push to find drugs that help beyond reducing symptoms continues, and Evaluate Vantage has delved into the pipeline of projects in active late-stage clinical trials. This year is shaping up to be crucial for the field, with 10 studies expected to yield data or to complete in 2021.
One target that crops up multiple times is GLP-1 this approach, traditionally employed in type 2 diabetes, is also being tested in Alzheimers. Among other avenues of research, it is hoped that gene therapy could offer a one-time cure for Parkinsons.
Research has suggested that GLP-1 agonists have neuroprotective benefits, and several trials of marketed diabetes drugs, as well as new GLP-1-targeting projects, are under way in Parkinsons. Some of these studies are investigator sponsored, including the most advanced, a UCL-run phase III trial of Astrazenecas Bydureon called Exenatide-PD3.
In the meantime, data are expected from several phase II studies of GLP-1 agonists, including a trial of Novo Nordisk’s Victoza, being run by Cedars-Sinai Medical in collaboration with the Danish company and The Cure Parkinson’s Trust. That study is set to complete in September.
How Can Stem Cell Technology Help
Stem cell technologies show promise for treating Parkinson’s Disease and may play an increasing role in alleviating at least the motor symptoms, if not others, in the decades to come.
“We are in desperate need of a better way of helping people with Parkinson’s disease. It is on the increase worldwide. There is still no cure, and medications only go part way to fully treat incoordination and movement problems,” said Claire Henchcliffe, from Weill Cornell Medical College in the US.
“If successful, using stem cells as a source of transplantable dopamine-producing nerve cells could revolutionize care of the Parkinson’s disease patient in the future,” said Malin Parmar, from Lund University in Sweden.
“A single surgery could potentially provide a transplant that would last throughout a patient’s lifespan, reducing or altogether avoiding the need for dopamine-based medications,” said Parmar.
In the past, most transplantation studies in PD used human cells from aborted embryos. While these transplants could survive and function for many years, there were scientific and ethical issues — foetal cells are in limited supply, and they are highly variable and hard to quality control.
Some patients were treated, while another developed allergy with the graft.
This approach is now rapidly moving into initial testing in clinical trials, researchers said.
The first systematic clinical transplantation trials using pluripotent stem cells as donor tissue were initiated in Japan in 2018.
Complementary And Supportive Therapies
A wide variety of complementary and supportive therapies may be used for PD, including:
A healthy diet. At this time there are no specific vitamins, minerals, or other nutrients that have any proven therapeutic value in PD. The National Institute of Neurological Disorders and Stroke and other components of the National Institutes of Health are funding research to determine if caffeine, antioxidants, and other dietary factors may be beneficial for preventing or treating PD. A normal, healthy diet can promote overall well-being for people with PD just as it would for anyone else. Eating a fiber-rich diet and drinking plenty of fluids also can help alleviate constipation. A high protein diet, however, may limit levodopas absorption.
Exercise. Exercise can help people with PD improve their mobility, flexibility, and body strength. It also can improve well-being, balance, minimize gait problems, and strengthen certain muscles so that people can speak and swallow better. General physical activity, such as walking, gardening, swimming, calisthenics, and using exercise machines, can have other benefit. People with PD should always check with their doctors before beginning a new exercise program.
Alternative approaches that are used by some individuals with PD include:
The State Of The Field
Understanding of Parkinson’s has grown substantially over the past two decades. The Michael J. Fox Foundation is building on this momentum to explore prevention of the disease and transform diagnosis and treatments.
Years of work spent uncovering Parkinsons secrets defining the highly variable patient experience, shedding light on genetic origins of disease, mapping molecular pathways are now paying off in a tangible quickening tempo of scientific progress. Investigators are increasingly linking cellular pathology to outward clinical symptoms to identify new therapeutic and biomarker targets. This has positioned drug makers to make rapid inroads toward treatments that have the potential to slow or stop progression of Parkinson’s disease . The field also is closer than ever to arriving at therapies that can treat all the symptoms of PD, including the less well understood non-motor aspects, such as cognitive impairment and mood disorders, sleeping and digestive issues, and speech and swallowing difficulties.
While the Parkinson’s pipeline is more active than at any previous point in the modern era of drug development, much work remains to be done in the quest to better understand the connection between pathological “bad actors” and the daily lived experience of the disease and to translate understanding of basic Parkinson’s biology into new therapies.
Which Scientific Breakthroughs Could Have A Big Impact For The Parkinsons Community
I think the concept of precision medicine or personalised medicine has now been integrated in Parkinsons disease research. Were still in the early days of it, but were trying to go that route. Right now, its very much focused on genetics underpinning Parkinsons disease, and there are quite a few clinical trials looking at therapies targeted for particular groups who have a genetic mutation. Thats the first step. The second step is taking that data, and understanding what it could mean for a broader patient community who may not have that genetic mutation. I think the fact that were trying to target things and make sure that the right therapy is being tested in the right patient population is very exciting.
The other thing, which is very similar to what the Alzheimers community has been doing, is that theres an opportunity to start to think how do we prevent the onset of symptoms?. The Foundation is now thinking about how to begin to get a sense of the pre-symptom phase of the disease. We know that prior to an individual having symptoms of the disease and getting diagnosed with Parkinsons disease, they probably have had the disease for a while. So how do we get that information to be able to intervene that much earlier before the symptoms start? There may be a day where we can say its about preventing Parkinsons disease from ever beginning.
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What Are The Symptoms Of Parkinsons Disease
Symptoms of Parkinsons disease and the rate of decline vary widely from person to person. The most common symptoms include:
Other symptoms include:
- Speech/vocal changes: Speech may be quick, become slurred or be soft in tone. You may hesitate before speaking. The pitch of your voice may become unchanged .
- Handwriting changes: You handwriting may become smaller and more difficult to read.
- Depression and anxiety.
- Sleeping disturbances including disrupted sleep, acting out your dreams, and restless leg syndrome.
- Pain, lack of interest , fatigue, change in weight, vision changes.
- Low blood pressure.
Can Parkinsons Disease Be Prevented
Unfortunately, no. Parkinsons disease is long-term disease that worsens over time. Although there is no way to prevent or cure the disease , medications may significantly relieve your symptoms. In some patients especially those with later-stage disease, surgery to improve symptoms may be an option.
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New Treatment May Have The Potential To Slow Stop Or Reverse Parkinson Disease
Results from a recent study suggest that a revolutionary treatment may have the potential to slow, stop, or even reverse the progression of Parkinson disease.
Results from a February study of a revolutionary treatment suggest that it may be possible to slow, stop, or even reverse the progression of Parkinson disease, according to findings in the Journal of Parkinsons Disease.
The 3-part, experimental study investigated whether using a novel delivery system to increase levels of glial cell line-derived neurotrophic factor can regenerate dying dopamine brain cells in patients with Parkinson disease and even reverse their condition. GDNF is a naturally occurring protein that promotes the survival of many types of neurons.
I believe that this approach could be the first neuro-restorative treatment for people living with Parkinson’s, which is, of course, an extremely exciting prospect, Steven Gill, MB, MS, FRCS, who designed the infusion device used in the study, said in a statement.
Initially, 6 patients enrolled in a pilot study which evaluated the safety of the treatment approach. After the pilot study, 35 additional individuals participated in a subsequent 9-month double-blind trial. Half of the participants were randomly assigned to receive monthly infusions of GDNF while the other half received placebos.
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 We Know So Far
- We’ve uncovered clues to the causes and genetic involvement in Parkinson’s.
- We’re figuring out the chain of events that leads to the damage and loss of brain cells.
- We’re working to advance new treatments and therapies.
- We’re exploring repurposing drugs to help manage some of the more distressing symptoms, like hallucinations and falls.
- And we know that, although people with Parkinson’s share symptoms, each person’s 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 Parkinson’s so desperately need.
Research takes time. But we launched the Parkinson’s Virtual Biotech to speed up the most promising potential treatments. The more we can invest, the sooner we’ll get there.
A Cure Means Different Things To Different People But What We Can Be Certain Of Is That We Dont Yet Have One For Parkinsons
Charities these days are all promising their research will lead to a cure, but while humanity has eradicated, or is on the way towards eradicating, some infectious diseases such as small pox, measles and polio it is unlikely that we will ever see a day when humans do not get cancer, heart disease or neurological conditions like Parkinsons.
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What Causes The Disease
The precise cause of PD is unknown, although some cases of PD are hereditary and can be traced to specific genetic mutations. Most cases are sporadicthat is, the disease does not typically run in families. It is thought that PD likely results from a combination of genetics and exposure to one or more unknown environmental factors that trigger the disease.
The protein alpha-synuclein. The affected brain cells of people with PD contain Lewy bodiesdeposits of the protein alpha-synuclein. Researchers do not yet know why Lewy bodies form or what role they play in the disease. Some research suggests that the cells protein disposal system may fail in people with PD, causing proteins to build up to harmful levels and trigger cell death. Additional studies have found evidence that clumps of protein that develop inside brain cells of people with PD may contribute to the death of neurons.
Genetics. Several genetic mutations are associated with PD, including the alpha-synuclein gene, and many more genes have been tentatively linked to the disorder. The same genes and proteins that are altered in inherited cases may also be altered in sporadic cases by environmental toxins or other factors.
Environment. Exposure to certain toxins has caused parkinsonian symptoms in rare circumstances . Other still-unidentified environmental factors may also cause PD in genetically susceptible individuals.
What Are The Symptoms Of The Disease
The four primary symptoms of PD are:
- Tremor. Tremor often begins in a hand, although sometimes a foot or the jaw is affected first. The tremor associated with PD has a characteristic rhythmic back-and-forth motion that may involve the thumb and forefinger and appear as a pill rolling. It is most obvious when the hand is at rest or when a person is under stress. This tremor usually disappears during sleep or improves with a purposeful, intended movement.
- Rigidity. Rigidity , or a resistance to movement, affects most people with PD. The muscles remain constantly tense and contracted so that the person aches or feels stiff. The rigidity becomes obvious when another person tries to move the individuals arm, which will move only in ratchet-like or short, jerky movements known as cogwheel rigidity.
- Bradykinesia. This slowing down of spontaneous and automatic movement is particularly frustrating because it may make simple tasks difficult. The person cannot rapidly perform routine movements. Activities once performed quickly and easilysuch as washing or dressingmay take much longer. There is often a decrease in facial expressions.
- Postural instability. Impaired balance and changes in posture can increase the risk of falls.
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How Do I Find Out More
Anyone who is considering taking part in research should speak to the professionals managing the project for more in-depth information. Contact details can be found on the project page.
If you have any queries, qualms or concerns please do ask the researchers, they should make your research visit as easy and enjoyable as possible.
Reasons Parkinson’s Disease Can Occur
When a loved one is living with Parkinsons, its natural to wonder what caused the disease to take root. Although most cases of Parkison’s have an unknown cause, a very small percent of cases can be hereditary . Some studies have linked long-term exposure to pesticides, including one called paraquat, with an increased likelihood of the development of Alzheimers and Parkinsons disease.
The pesticide was first introduced in the United States in the 1950s and is still used commonly by licensed users today. If you think that your loved one who has been diagnosed with Parkinsons may have been exposed to paraquat, its important that you work with a lawyer to learn whether you may be eligible for compensation. Caring for a loved one with Parkinsons can be costly and time-consuming, especially in the later stages when your loved one requires around-the-clock care. Financial compensation can make it easier to provide your loved one with the care they deserve throughout the progression of the disease.
Were here to help you decide what to do next after you find out that paraquat exposure may be responsible for the development of Parkinsons disease in your loved one. Reach out to us today for a free case review.
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In The Next 10 Years I Hope We Will Have Effective Methods For Early Diagnosis
Susanna Lindvall, vice-president of the European Parkinsons Disease Association
Until now, Parkinsons treatments have focused on symptom management in terms of drug-based treatments and surgical treatments such as deep brain stimulation. Now, there are several drugs in the pipeline such as MSDC-0160, a drug originally created to treat Type-2 diabetes and the Transeuro trial of cell therapy .
In the coming 10 years I hope we will have effective methods for early diagnosis. Right now, researchers have developed a new test that is able to detect abnormal alpha-synuclein in the spinal fluid of people with Parkinsons with remarkable specificity and sensitivity, at an early stage of the disease. It is early days, but the test is promising. There is also a new neuro-protective strategy where LRRK2 kinase inhibitors are being trialled as therapies for Parkinsons disease. Researchers have discovered an interaction in neurons that contributes to Parkinsons disease, and they have shown that drugs currently in development may block the process. The hope is that these will bring major improvements in the quality of life for people with Parkinsons life expectancy will improve and the progression of the condition may be slowed or even stopped.
Michael Okun, MD
Professor Baastian Bloem
Lead image credit: Parkinsons UK Picturing Parkinsons Flickr album
What Are The Surgical Treatments For Parkinsons Disease
Most patients with Parkinsons disease can maintain a good quality of life with medications. However, as the disease worsens, medications may no longer be effective in some patients. In these patients, the effectiveness of medications becomes unpredictable reducing symptoms during on periods and no longer controlling symptoms during off periods, which usually occur when the medication is wearing off and just before the next dose is to be taken. Sometimes these variations can be managed with changes in medications. However, sometimes they cant. Based on the type and severity of your symptoms, the failure of adjustments in your medications, the decline in your quality of life and your overall health, your doctor may discuss some of the available surgical options.
When Will There Be A Cure For Parkinsons Disease
You can tell a lot about a culture from its Armageddon myths, as propagated through pop culture. We have created quite a few across the last few decades, from the dystopian futures of Mad Max and the Terminator to the zombie apocalypses that have come to populate many an end-of-the-world tale. The latter often entail some experiment gone horribly awry, leading to a pandemic far more lethal and gruesome than any ever caused, for example, by influenza. Earlier this year, scientists warned of a possible new pandemic that has nothing to do with diseased bat guano, genetically modified whatever, or ancient Egyptian curses. Parkinsons Disease is on the rise, and so far theres no cure for this rare neurodegenerative disease.