From Biology To New Therapies
While seven new Parkinsons drugs have come to market since 2014 a heartening expansion of treatment options scientists continue working aggressively on many fronts toward next-generation therapies to greatly improve the treatment and management of motor and non-motor symptoms. These projects require, in tandem, funding, objective biological measures, infrastructure to support recruitment, and coordination with regulators and payers.
Several novel formulations of levodopa are currently anticipated to receive regulatory approval in the near future. These include inhaled and transdermal formulations backed and “de-risked” by The Michael J. Fox Foundation in early development. An improved delivery of levodopa has been a primary field goal for years, as scientists have searched for a more consistent way to get the drug into the brain. When taken orally, absorption of the traditional oral formulation into the blood and brain can be inconsistent, coming in peaks and valleys, which is believed to lead to motor fluctuations, including disabling off periods.
While no longer considered the “silver bullet” scientists once hoped, stem cell approaches continue advancing toward therapeutic relevance. Induced pluripotent stem cells are expected to one day treat motor symptoms by replacing damaged dopamine cells.
What Is The Prognosis
The average life expectancy of a person with PD is generally the same as for people who do not have the disease. Fortunately, there are many treatment options available for people with PD. However, in the late stages, PD may no longer respond to medications and can become associated with serious complications such as choking, pneumonia, and falls.
PD is a slowly progressive disorder. It is not possible to predict what course the disease will take for an individual person.
One commonly used scale neurologists use for describing how the symptoms of PD have progressed in a patient is the Hoehn and Yahr scale.
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.
Repurposing
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.
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Assembling Your Care Team
Assembling a team that will provide you with physical and emotional support and adapt to your needs over time is one of the best ways to remain healthy. Parkinsons disease is complex and requires an interdisciplinary approach to care. The care team may include, but is not limited to:
- Movement disorder specialist
- Rehabilitation specialists including physical, occupational, and speech therapists
- Nurse
Key Programs And Resources
The Parkinsons Disease Biomarkers Programs , a major NINDS initiative, is aimed at discovering ways to identify individuals at risk for developing PD and Lewy Body Dementia and to track the progression of the disease. It funds research and collects human biological samples and clinical data to identify biomarkers that will speed the development of novel therapeutics for PD. Goals are improving clinical trials and earlier diagnosis and treatment. Projects are actively recruiting volunteers at sites across the U.S. NINDS also collaborates with the Michael J. Fox Foundation for Parkinsons Research on BioFIND, a project collecting biological samples and clinical data from healthy volunteers and those with PD. For more information about the PDBP and how you can get involved, please visit the PDBP website.
The NINDS Morris K. Udall Centers of Excellence for Parkinsons Disease Research program supports research centers across the country that work collaboratively to study PD disease mechanisms, the genetic contributions to PD, and potential therapeutic targets and treatment strategies.
The NINDS Intramural Research Program conducts clinical studies to better understand PD mechanisms and develop novel and improve treatments.
The NINDS Biospecimens Repositories store and distribute DNA, cells, blood samples, cerebrospinal fluid, and autopsy tissue to PD researchers around the world.
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What Genes Are Linked To Parkinsons Disease
Several genes have been definitively linked to PD:
- SNCA. This gene, which makes the protein alpha-synuclein, was the first gene identified to be associated with Parkinsons. Research findings by the National Institutes of Health and other institutions prompted studies of the role of alpha-synuclein in PD, which led to the discovery that Lewy bodies seen in all cases of PD contain clumps of alpha-synuclein. This discovery revealed the link between hereditary and sporadic forms of the disease.
- LRRK2. Mutations in LRRK2 were originally identified in several English and Basque families as a cause of a late-onset PD. Subsequent studies have identified mutations of this gene in other families with PD as well as in a small percentage of people with apparently sporadic PD. LRRK2 mutations are a major cause of PD in North Africa and the Middle East.
- DJ-1. This gene normally helps regulate gene activity and protect cells from oxidative stress and can cause rare, early forms of PD.
- PRKN . The parkin gene is translated into a protein that normally helps cells break down and recycle proteins.
- PINK1. PINK1 codes for a protein active in mitochondria. Mutations in this gene appear to increase susceptibility to cellular stress. PINK1 has been linked to early forms of PD.
- GBA . Mutations in GBA cause Gaucher disease , but different changes in this gene are associated with an increased risk for Parkinsons disease as well.
Scientists Are Close To Finding A Cure For Parkinsons Disease
Researchers studied mutant fruit flies to uncover how the disease affects the brain
SCIENTISTS are on the brink of finding a cure for Parkinsons disease after discovering what causes the illness and how it affects brain cells.
Experts previously thought people who fell ill at a younger age than most who develop Parkinsons suffer from a poorly functioning mitochondria.
These are the powerhouses of cells and without reliable sources of energy, neurons wither and die.
But Medical Research Council researchers at University of Leicester found this may not be the complete picture of what is happening within the brain cells affected by Parkinsons.
Now it is thought neuro deterioration occurs in response to stress on the endoplasmic reticulum part of the outer skin of the cell rather than failure of the mitochondria as previously thought.
During the study fruit flies were injected with chemicals to prevent the effects of ER stress and as a result, it prevented the death of neurons which are commonly associated with the disease.
Dr Miguel Martins said: This research challenges the current held belief that Parkinsons disease is a result of malfunctioning mitochondria.
By identifying and preventing ER stress in a model of the disease it was possible for us to prevent neurodegeneration.
Lab experiments, like this, allow us to see what effect ER stress has on Parkinsons disease.
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Advanced And Future Treatments For Parkinsons
While theres no cure for Parkinsons disease, recent research has led to improved treatments.
Scientists and doctors are working together to find a treatment or prevention technique. Research is also seeking to understand who is more likely to develop the disease. In addition, scientists are studying the genetic and environmental factors that increase the chance of a diagnosis.
Here are the latest treatments for this progressive neurological disorder.
In 2002, the FDA approved deep brain stimulation as a treatment for Parkinsons disease. But advances in DBS were limited because only one company was approved to make the device used for the treatment.
In June 2015, the FDA approved the
Can Exercise Help Patients Gain Ground On Parkinsons Disease
In medicines ongoing battle with disease, technology plays a major, ever-evolving role. Advances abound in the form of new drugs, medical devices and gene therapies. But a decidedly low-tech treatment strategy for at least one disease simply requires putting one foot in front of the other literally.
The target is Parkinsons disease, a progressive movement disorder that affects around 1 million people in the United States and 10 million worldwide. While there is no cure, there are drugs to treat the symptoms of Parkinsons, including tremors, rigidity, and impairment of fine motor movements. But a growing body of evidence suggests that a powerful counter to this movement disorder may be, well, movement.
A new nationwide trial that includes the University of Colorado is putting that idea to the test. Study in Parkinson Disease of Exercise is a randomized clinical trial investigating whether regular, moderate and high-intensity exercise can slow the progression of symptoms in patients in the early stages of Parkinsons disease who have not yet begun drug treatment.
Groundwork previously laid
The study, which is underway at 29 sites in North America, builds on the findings of SPARX2. That trial concluded in 2016, with results published in 2018 in JAMA Neurology. SPARX2 was led at CU by Dr. Margaret Schenkman, then director of the Physical Therapy Program and a pioneering investigator in using physical therapy to treat Parkinsons disease.
The SPARX3 Team:
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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
Susanna Lindvall
Lead image credit: Parkinsons UK Picturing Parkinsons Flickr album
I See A Much Greater Role For Informed Engaged Patients
Professor Bastiaan Bloem, MD, PhD, medical director, Parkinson Center Nijmegen
Breakthroughs in the area of Parkinsons disease will come from two main directions: the cure and the care. My own research and innovation focus is exclusively on care issues. The major breakthrough that I foresee here is a much greater role for fully informed and engaged patients as equal partners in healthcare, who have easy access to integrated healthcare networks of well-trained professionals who work according to a value-based healthcare concept.
Care will be delivered largely outside institutions and within the patients own home environment, and increasingly via telemedicine approaches. This and other technological developments will help to drive these aforementioned innovations. Implementing this into the western world will take at least 10 more years, and unfortunately, much longer in less developed countries.
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The History Of Parkinsons
Parkinsons disease was first defined as a shaking palsy in 1817 by James Parkinson. Half a century later, in 1872, the Parisian neurologist Jean-Martin Charcot coined the term Parkinsons disease.
Though Parkinson was the first to describe the disease in modern medicine, Charcot and his colleagues revolutionised treatments in the mid-19th century.. Parkinson was a proponent of blood-letting from the neck, in a bid to siphon off inflammatory pathogens and prevent them from reaching the brain. But Charcot and his colleagues favoured pharmaceutical approaches centred around anticholinergic drugs, which block the action of a neurotransmitter called acetylcholine. Anticholinergics are still in use today.
Around the same time, a host of other treatments were being explored at a hospital in Paris. Hyoscyamine, a plant-derived medication, was put in bread and fed to patients. Other medications, such as a derivative of quinine, were mixed with a syrup of orange rinds.
Charcot also claimed to see the symptoms of patients with Parkinsons improving when travelling by train and horse-carriage. He became a proponent of vibration therapy, where patients bodies and heads were shaken vigorously by a rigged motor.
What Are Your Hopes For The Future Of Parkinsons Research
At the Foundation, we really are incredibly hopeful that a lot of the research that were supporting is going to yield a tangible benefit for patients.
The fact that theres so much in the pipeline, from preclinical all the way to clinical testing, means theres more thats going to come out. Its so important to have a lot of different things out there, so that patients with their care team can figure out how to get the most out of what is available.
Theres such an important role for the patient community to play in getting engaged with the search. If you have, please continue to do so. And if you havent, consider it and learn about it. There are a lot of ways to get engaged and Im pretty sure that youll find one that is comfortable for you. Were there to help and be your partner in this, because we cant do it alone.
A cure may still be a few years out, or several years out or a decade. But theres so much we can do between then that will impact a person whos living with Parkinsons in a very positive way.
Need to know
Sohini Chowdhury is deputy CEO of the Michael J Fox Foundation, US, overseeing the Research Partnerships team. She also works with the board of directors and executive leadership to advance the organisations work as a stakeholder in drug development. Find out more about the Michael J Fox Foundation.
To find out more about the latest Parkinsons disease research, please visit the EPDA website.
Read more:
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What Diseases And Conditions Resemble Parkinsons Disease
PD is the most common form of parkinsonism, in which disorders of other causes produce features and symptoms that closely resemble Parkinsons disease. Many disorders can cause symptoms similar to those of PD, including:
Several diseases, including MSA, CBD, and PSP, are sometimes referred to as Parkinsons-plus diseases because they have the symptoms of PD plus additional features.
In very rare cases, parkinsonian symptoms may appear in people before the age of 20. This condition is called juvenile parkinsonism. It often begins with dystonia and bradykinesia, and the symptoms often improve with levodopa medication.
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.
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Parkinsons Disease: How Close Are We To A Cure
Neurological disorders are the leading source of disability globally, and Parkinsons is the fastest-growing neurological condition. It could be due to several factors ranging from an increase in geriatric population, longevity, improved diagnostics methodologies, a better understanding of the disease to the specific use of some pesticides, and industrialization. The number of individuals with Parkinsons disease increased by 118% to 6.2 million globally from 1990 to 2015. The total diagnosed prevalence is exponentially rising and as per DelveInsights epidemiological estimates, the total diagnosed prevalent population of Parkinsons disease in the 7MM and Japan) expected to reach 3,284,084 in 2030.
However, there is still no cure for Parkinsons. However, medications are at the place to improve the main symptoms of Parkinsons disease, such as shaking and movement problems. The present treatment options for Parkinsons disease consists of medication, surgery, complementary therapies, and supportive therapies . The approved Parkinsons disease therapies are categorized into seven groups that include Levodopa, Carbidopa-levodopa infusion , Dopamine agonists , MAO B inhibitors , Catechol O-methyltransferase inhibitors , Anticholinergics , and Amantadine.
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Parkinsons Disease: How Could Stem Cells Help
What do we know?
Tremors, muscle rigidity and other symptoms of Parkinsons disease are caused by the death of dopamine-producing neurons in the brain. Dopamine producing neurons throughout the brain are affected, but the substantia nigra is the primary brain region where neurons are lost.
People affected by PD often develop abnormal protein clumps in their brain called Lewy bodies. These clumps are made of a protein called alpha-synuclein.
Levodopa is the primary drug used to treat PD. Levodopa is converted into dopamine when in the body, which compensates for lost dopamine-producing neurons.
What are researchers investigating?
Approximately 5% of people with PD have inheritable gene mutations linked to PD. Researchers are investigating what causes PD in the other 95% of patients in clinical studies, animal models and cell models.
Transplantation of young brain cells from human foetuses into people with PD has shown promising results in previous clinical trials. The current TRANSEURO study is re-examining this treatment method with the aim of minimising side effects and measuring efficacy.
Scientists can now make dopamine-producing neurons from both human embryonic stem cells and human induced pluripotent stem cells . Neurons made from human ESCs and iPSCs mature into human dopamine-producing neurons, survive and function after transplantation into mouse, rat and monkey models of PD.
What are the challenges?
Replacing lost cells
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