How Close Are We To A Cure For Parkinson’s

If Replacing Dopamine In The Brain Via Carbidopa/levodopa Helps Reduce And Sometimes Completely Eliminate Parkinsons Symptoms Why Arent We Closer To Finding A Cure Is Parkinsons Truly Caused By A Lack Of Dopamine

Here’s what he said:

In 1817, James Parkinson wrote the article that first described the disease, called An Essay on the Shaking Palsy.  This essay, and our understanding of the disease from the earliest days until the 1970s, focused on the major clinical symptoms of Parkinson’s that emerge from how the disease impacts the dopamine system, notably the dopamine-producing neurons of the part of the brain called the substantia nigra, which basically means, “black stuff,” named in the days when anatomists just cut up corpses and named what they saw with little insight.

In the late 1960s and 1970s, clinical researchers figured out that they could treat Parkinson’s with dopamine replacement therapy. Parkinson’s was thus a disease of dopamine and therefore, we could focus on the dopamine system in treatments of Parkinson’s. End of story, right?

Wrong.

Focusing on dopamine for Parkinson’s is like saying that global warming is a problem of temperature. Cooling the air wouldn’t solve the problem of climate change and replacing dopamine doesn’t cure Parkinson’s.  Parkinson’s is largely a disease of neurons, and to stop/fix/cure Parkinson’s, we need to stop that disease from making neurons sick.

The neurons are being made sick by being polluted with too much of a protein called alpha-synuclein. This protein seems to principally affect dopamine neurons in Parkinson’s, but as the disease advances, it can harm other neurons, too.

Why?

A Cure Means Different Things To Different People But What We Can Be Certain Of Is That We Dont Yet Have One For Parkinsons Dec 3, 2018

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 Parkinson’s.

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 Parkinson’s 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 weren’t clear cut, GDNF has shown promise to restore damaged cells in people with Parkinson’s.

However, the GDNF protein requires complex robot-assisted surgery to deliver the treatment to the brain because it’s a large molecule that can’t 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.”

Related Article

How Close Do You Think The Scientific Community Is To Finding A Cure For Parkinsons Disease

Whenever I say the word ‘cure’, I kind of put it in quotes. I think it’s important to remember that a cure can mean different things to different people. If you’re able to improve the symptom management of the disease to an extent where having the disease has very little impact on your day to day life, that could be considered a cure. If you’re able to slow or halt the disease progression so that the moment you get diagnosed, it never progresses beyond that point but you’re still taking a pill every day for the rest of your life, that could be a cure.

There’s a recognition now that Parkinson’s disease is not one disease experience. It is a disease experience that is very variable, so we have to be open minded because a cure for one person could be very different than a cure for another person.

I think the fact that this is now accepted in the research community is a good thing for patients. It’s not a one size fit all approach. We have finally understood that we need lots of different cures to fit the different patient experiences under the name ‘Parkinson’s disease’.

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 Parkinson’s disease research. We’re still in the early days of it, but we’re trying to go that route. Right now, it’s very much focused on genetics underpinning Parkinson’s disease, and there are quite a few clinical trials looking at therapies targeted for particular groups who have a genetic mutation. That’s 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 we’re 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 Alzheimer’s community has been doing, is that there’s 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 Parkinson’s 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 ‘it’s about preventing Parkinson’s disease from ever beginning’.

In The Next 10 Years I Hope We Will Have Effective Methods For Early Diagnosis

Susanna Lindvall, vice-president of the European Parkinson’s Disease Association

Until now, Parkinson’s 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 Parkinson’s 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 Parkinson’s disease. Researchers have discovered an interaction in neurons that contributes to Parkinson’s 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 Parkinson’s – 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: Parkinson’s UK Picturing Parkinson’s Flickr album

New Treatment May Have The Potential To Slow Stop Or Reverse Parkinson Disease Wallace Stephens

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 Parkinson’s 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.

Reference

Which Parkinson’s Symptoms Do Patients Most Want To See Improved By Treatment

A survey by Parkinson’s UK identifies which aspects of the condition matter most to patients. These findings have implications for improving quality of life and directing future research, reports the Journal of Parkinson’s Disease

Peer-Reviewed Publication

IOS Press

image: Symptoms or side effects reported in response to the question "what aspect of Parkinson’s do you most wish to see improvement in?" presented by priority. Percentages show the relative frequency of symptoms or side effects reported within primary responses , secondary responses , and tertiary responses .view more 

Amsterdam, April 26, 2021 – Individuals with Parkinson’s disease face a wide range of symptoms and challenges. A team from Parkinson’s UK, including several Patient and Public Involvement contributors, surveyed patients to find out which PD symptoms troubled them most and how priorities may change with condition duration. Their goal was to identify where improved treatments and strategies are most needed to help maintain independence and quality of life. They report their findings in the Journal of Parkinson’s Disease.

As PD progresses, the number and severity of symptoms increase, and the amount of medication required to manage symptoms also increases, leading to a greater risk of side effects and fluctuations contributing to increased disability.

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DOI

10.3233/JPD-202346

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What Area Of Parkinsons Research Are You Are Excited To Find Out More About

Particularly in this COVID world that we’re living in, one of the things that is really exciting is we’re increasingly seeing the role that technology can play in helping to track the disease experience of patients.

In this unprecedented year, we’ve seen such a such an emphasis on sheltering in place, particularly among our vulnerable populations. We have an online clinical study called Fox Insight and we’ve really seen an increase in registrations because it shows the potential of being able to participate in research from the safety of your own home, and still contribute to sharing knowledge about the disease.

In the US, we’ve also seen the embrace of telemedicine as a way to continue to think about managing care – but not necessarily having to go into a doctor’s office. I think all of these experiences, coupled with ongoing advances in sensor technologies or smartphone apps, really show us the way technology can ease the burden, both of managing the disease and participating in research. I think that might be a little bit of a silver lining in a world where there aren’t very many silver linings at the moment.

Is There A Cure For Parkinsons Disease How Close Is A Cure For Parkinson’s

As of now, there is no known cure for this disease. However, treatments likedrug therapy or brain surgery can help reduce the symptoms and make life easier for patients with Parkinson’s Disease.

Although a cure is nowhere near yet, scientists claim that they will find better treatment in years to come, which is expected to happen within the decade. The past two years could have been the years of advancements for new drugs for PD, but the pandemic pushed some plans back.

As part of their advancements in finding the cure, Parkinson’s research has launched a virtual biotech for developing and testing new treatments.

Also, they are collaborating with international scientists to share thoughts, scientific discoveries to make testings more efficient and less costly.

Lastly, the research is also searching for other unlooked conditions that may pave the way for PD to develop in people. Searching for other possible options for why the loss of nerve cells is happening.

New Trial Platform Could Accelerate Finding A Cure For Parkinson’s Disease

Despite 30 years of research, not a single therapy has been found to successfully delay or stop the progression of Parkinson’s disease . In the Journal of Parkinson’s Disease scientists report on the possibility of using a multi-arm, multi-stage trial platform to evaluate several potential therapies at once, using lessons learned from other diseases.

Many potential disease-modifying therapies have been identified as suitable for clinical evaluation in PD. Each potential cure for PD has to go through three clinical trial phases to test its safety, whether it shows signs of improving PD, and whether there is any meaningful benefit to people with PD. Running a clinical trial is a huge logistical, costly, and time-consuming undertaking. For a single new therapy this process can take the best part of a decade. Currently, phase II and phase III clinical trials in PD are set up in isolation from each other, a process that is lengthy, costly, and inefficient.

In this review, scientists introduce the concept of a multi-arm, multi-stage PD trial platform. MAMS trials test many potential therapies in parallel , transitioning seamlessly through various phases , i.e., from a phase II safety and efficacy study to a phase III trial. Early analyses allow unsuccessful therapies to be replaced. At the interim checkpoint, ineffective arms can be dropped and replaced by new treatment arms, thereby allowing for the continuous evaluation of interventions.

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What Are Scientists Aiming To Achieve In The Near Future Through Research

This is an unbelievably robust time for Parkinson’s research in that drug development and the therapeutic development pipeline is incredibly varied. I think that’s what’s really exciting.

At the Michael J Fox Foundation, we always say that the more shots on goal that you have, the greater the likelihood that something is going to get into that goal. What’s particularly interesting now is that all of those shots on goal are not the same approach and they’re not the same type of therapy.

The experience patients have with the disease is so variable. So the fact that we have all of these different approaches to trying to develop therapies gives a greater likelihood that we are going to come up with interventions to tackle these different parts of patient experiences. It’s incredibly exciting to see not just researchers, but the biopharma sector and the investor community put resources into exploring all of these different approaches. It’s not like everybody has their eggs in one basket ­– we’re seeing a lot of different baskets, and that’s potentially really beneficial for the patient community.

Sohini Chowdhury is deputy CEO at the Michael J Fox Foundation.

Breakthrough Ideas And Therapies Stretch Far Beyond A Single Drug

Dr Michael Okun, Adelaide Lackner professor and chairman of neurology at the Univeristy of Florida

The point that I emphasise with my patients is that breakthrough ideas and therapies in Parkinson’s disease stretch far beyond a single drug or stem cell. There is, in fact, a broader and more exciting picture and portfolio of breakthroughs spanning drug, cell, vaccine, device, genetics, care, and behaviour. Patients and families with personal investments in Parkinson’s disease should be informed and updated about all of the potential breakthrough therapies.

Andy Siderowf and colleagues at the University of Pennsylvania’s National Parkinson Foundation Center of Excellence, took another approach to constructing an economic model. The Penn investigators were interested in the ‘economic consequences of slower rates of Parkinson’s progression’. They used a technique called a Markov model and predicted the potential savings associated with slowing Parkinson’s disease progression. Slowing progression by 20% saved the health care system US$60,657 per patient. Stopping disease progression saved US$442,429 per patient.

Its Not Just One Condition There Are Multiple Types Of Parkinsons

Like cancer, we are starting to understand the importance of subtyping Parkinson’s and developing tailored treatments that will be more successful than a one size fits all approach. Better understanding is coming from large scale studies that follow vast numbers of people with the condition over time. And we are starting to see how the subtypes of Parkinson’s have different symptoms, progression rates and even different responses to medication.

To effectively treat Parkinson’s, we probably need to tackle each of these subtypes differently, providing the right treatments and support to suit the individual and their form of Parkinson’s. And this starts with understanding more about how we classify and identify these different types.

You can read more about research into personalised treatments in our recent blog ‘Precision medicine for Parkinson’s, how close are we?’

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 Parkinson’s 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 Parkinson’s 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 Parkinson’s.

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.

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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 Parkinson’s 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 Parkinson’s disease.

Leicester’s MRC Toxicology unit in Leicester carried out the experiment.

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• Topics

Current Parkinsons Treatments Cant Slow Down Onset Of Disease

Parkinson’s is a long-term degenerative disorder of the central nervous system, which mainly the area of the brain that controls movement – leading to a slow onset of symptoms including tremors, rigidity and slow movement.

More than 10 million people worldwide are estimated to be living with Parkinson’s disease, according to the US-based Parkinson’s Foundation, with the Parkinson’s News Today website saying it affects 1,900 per 100,000 among those aged over 80,

Typically, by the time people are diagnosed with the condition, they have already lost between 70% and 80% of their dopamine-producing cells, which are involved in co-ordinating movement.

While current treatments mask the symptoms, there is nothing that can slow down its progression or prevent more brain cells from being lost.

As dopamine levels continue to fall, symptoms get worse and new symptoms can appear.

What Lifestyle Changes Can I Make To Ease Parkinsons Symptoms

Exercise: Exercise helps improve muscle strength, balance, coordination, flexibility, and tremor. It is also strongly believed to improve memory, thinking and reduce the risk of falls and decrease anxiety and depression. One study in persons with Parkinson’s disease showed that 2.5 hours of exercise per week resulted in improved ability to move and a slower decline in quality of life compared to those who didn’t exercise or didn’t start until later in the course of their disease. Some exercises to consider include strengthening or resistance training, stretching exercises or aerobics . All types of exercise are helpful.

Eat a healthy, balanced diet: This is not only good for your general health but can ease some of the non-movement related symptoms of Parkinson’s, such as constipation. Eating foods high in fiber in particular can relieve constipation. The Mediterranean diet is one example of a healthy diet.

Preventing falls and maintaining balance: Falls are a frequent complication of Parkinson’s. While you can do many things to reduce your risk of falling, the two most important are: 1) to work with your doctor to ensure that your treatments — whether medicines or deep brain stimulation — are optimal; and 2) to consult with a physical therapist who can assess your walking and balance. The physical therapist is the expert when it comes to recommending assistive devices or exercise to improve safety and preventing falls.

Improve the quality of your sleep.

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 we’re supporting is going to yield a tangible benefit for patients.

The fact that there’s so much in the pipeline, from preclinical all the way to clinical testing, means there’s more that’s going to come out. It’s 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.

There’s 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 haven’t, consider it and learn about it. There are a lot of ways to get engaged and I’m pretty sure that you’ll find one that is comfortable for you. We’re there to help and be your partner in this, because we can’t do it alone.

A cure may still be a few years out, or several years out or a decade. But there’s so much we can do between then that will impact a person who’s living with Parkinson’s 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 organisation’s work as a stakeholder in drug development. Find out more about the Michael J Fox Foundation.

To find out more about the latest Parkinson’s disease research, please visit the EPDA website.

Read more:

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 Parkinson’s disease. Many disorders can cause symptoms similar to those of PD, including:

Several diseases, including MSA, CBD, and PSP, are sometimes referred to as “Parkinson’s-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.

Study Kindles New Hope For A Cure For Parkinsons Disease RKD LiveTwitterMessengerWhatsApp

New Delhi: Efforts to find a cure for Parkinson’s Disease is expected to get a big boost with researchers at the Indian Institute of Technology-Madras finding that energy deficiency in certain cells in the human brain could be a major cause for the development of the  neurodegenerative disorder.

Parkinson’s Disease is the second most prominent neurodegenerative disease across the world after Alzheimer’s disease. More than 200 years after it was first described by Dr. James Parkinson as “shaking palsy,” the world is still searching for a cure. Currently, the medical intervention  is focused mainly on the management of the disease.

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It is known that Parkinson’s Disease is caused by the loss of a group of cells called dopaminergic cells in a portion of the midbrain called substantia nigra pars compacta . However, the decisive cause of this cell loss has not been elucidated before. The new study could fill the gap. The IIT Madras researchers developed a computational model that showed that energy deficiency might be a major reason for the loss of the specific cells in Parkinson’s Disease.

The computational model was developed by Dr. Vignayanandam Ravindernath Muddapu, who completed his Ph.D. recently at IIT Madras, under the guidance of Prof. V. Srinivasa Chakravarthy at the Department of Biotechnology in IIT Madras.

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 Parkinson’s 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 patient’s 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.

What Are The Surgical Treatments For Parkinsons Disease

Most patients with Parkinson’s 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 can’t. 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.

What Is The Outlook For Persons With Parkinsons Disease

Although there is no cure or absolute evidence of ways to prevent Parkinson’s disease, scientists are working hard to learn more about the disease and find innovative ways to better manage it, prevent it from progressing and ultimately curing it.

Currently, you and your healthcare team’s efforts are focused on medical management of your symptoms along with general health and lifestyle improvement recommendations . By identifying individual symptoms and adjusting the course of action based on changes in symptoms, most people with Parkinson’s disease can live fulfilling lives.

The future is hopeful. Some of the research underway includes:

• Using stem cells to produce new neurons, which would produce dopamine.
• Producing a dopamine-producing enzyme that is delivered to a gene in the brain that controls movement.
• Using a naturally occurring human protein – glial cell-line derived neurotrophic factor, GDNF – to protect dopamine-releasing nerve cells.

Many other investigations are underway too. Much has been learned, much progress has been made and additional discoveries are likely to come.

Do You Think There Will Be A Cure For Pd In The Next 5

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What Medications Are Used To Treat Parkinsons Disease

Medications are the main treatment method for patients with Parkinson’s disease. Your doctor will work closely with you to develop a treatment plan best suited for you based on the severity of your disease at the time of diagnosis, side effects of the drug class and success or failure of symptom control of the medications you try.

Medications combat Parkinson’s disease by:

• Helping nerve cells in the brain make dopamine.
• Mimicking the effects of dopamine in the brain.
• Blocking an enzyme that breaks down dopamine in the brain.
• Reducing some specific symptoms of Parkinson’s disease.

Levodopa: Levodopa is a main treatment for the slowness of movement, tremor, and stiffness symptoms of Parkinson’s disease. Nerve cells use levodopa to make dopamine, which replenishes the low amount found in the brain of persons with Parkinson’s disease. Levodopa is usually taken with carbidopa to allow more levodopa to reach the brain and to prevent or reduce the nausea and vomiting, low blood pressure and other side effects of levodopa. Sinemet® is available in an immediate release formula and a long-acting, controlled release formula. Rytary® is a newer version of levodopa/carbidopa that is a longer-acting capsule. The newest addition is Inbrija®, which is inhaled levodopa. It is used by people already taking regular carbidopa/levodopa for when they have off episodes .