Thursday, June 16, 2022
Thursday, June 16, 2022
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How Close Are We To Finding A Cure For Parkinson’s

How Can People Cope With Parkinson’s Disease

How can we cure Parkinson’s?

While PD usually progresses slowly, eventually daily routines may be affectedfrom socializing with friends to earning a living and taking care of a home. These changes can be difficult to accept. Support groups can help people cope with the diseases emotional impact. These groups also can provide valuable information, advice, and experience to help people with PD, their families, and their caregivers deal with a wide range of issues, including locating doctors familiar with the disease and coping with physical limitations. A list of national organizations that can help people locate support groups in their communities appears at the end of this information. Individual or family counseling may also help people find ways to cope with PD.

People with PD may also benefit from being proactive and finding out as much as possible about the disease in order to alleviate fear of the unknown and to take a positive role in maintaining their health. Many people with PD continue to work either full- or part-time, although they may need to adjust their schedule and working environment to accommodate their symptoms.

How We’re Speeding Up The Search For A Cure

We believe that new and better treatments are possible in years, not decades, and we have a clear strategy for making this happen. This includes:

Who Gets Parkinsons Disease

Risk factors for PD include:

  • Age. The average age of onset is about 70 years, and the incidence rises significantly with advancing age. However, a small percent of people with PD have early-onset disease that begins before the age of 50.
  • Sex. PD affects more men than women.
  • Heredity. People with one or more close relatives who have PD have an increased risk of developing the disease themselves. An estimated 15 to 25 percent of people with PD have a known relative with the disease. Some cases of the disease can be traced to specific genetic mutations.
  • Exposure to pesticides. Studies show an increased risk of PD in people who live in rural areas with increased pesticide use.

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

This is an unbelievably robust time for Parkinsons research in that drug development and the therapeutic development pipeline is incredibly varied. I think thats whats 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. Whats particularly interesting now is that all of those shots on goal are not the same approach and theyre 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. Its incredibly exciting to see not just researchers, but the biopharma sector and the investor community put resources into exploring all of these different approaches. Its not like everybody has their eggs in one basket were seeing a lot of different baskets, and thats potentially really beneficial for the patient community.

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

The Investigation In Mice

REVEALED: Scientists are close to finding a cure for ...

In their recent study paper, the scientists refer to research suggesting that neurotrophic factors molecules that help neurons survive and thrive could, in theory, restore the function of neurons that produce dopamine. However, the clinical benefit of these factors had yet to be proven.

The team focused on bone morphogenetic proteins 5 and 7 . They had previously shown that BMP5/7 has an important role in dopamine-producing neurons in mice.

In the latest study, the scientists wanted to see whether BMP5/7 could protect the neurons of mice against the damaging effects of misfolded alpha-synuclein proteins.

To do this, they injected one group of mice with a viral vector that caused misfolded alpha-synuclein proteins to form in their brains. They used other mice as a control group. The scientists then injected the mice with the BMP5/7 protein.

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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

What Is Parkinson’s Disease

Parkinsons disease is movement disorder of the nervous system that worsens over time. As nerve cells in parts of the brain weaken or are damaged or die, people may begin to notice problems with movement, tremor, stiffness in the limbs or the trunk of the body, or impaired balance. As these symptoms become more obvious, people may have difficulty walking, talking, or completing other simple tasks. Not everyone with one or more of these symptoms has PD, as the symptoms appear in other diseases as well.

No cure for PD exists today, but research is ongoing and medications or surgery can often provide substantial improvement with motor symptoms.

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How Close Are We To A Cure For Parkinsons

Saskia Mair of Parkinsons Lifeinterviews Sohini Chowdhury, Deputy CEO of the Michael J Fox Foundation.

How close do you think the scientific community is to finding a cure for Parkinsons?

Whenever I say the word cure, I kind of put it in quotes. I think its important to remember that a cure can mean different things to different people.

If youre 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 youre able to slow or halt the disease progression so that the moment you get diagnosed, it never progresses beyond that point but youre still taking a pill every day for the rest of your life, that could be a cure.

Theres a recognition now that Parkinsons 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. Its 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 Parkinsons disease.

In terms of how close we are is it tomorrow? Absolutely not. But theres so many resources, money, scientific knowledge, and brainpower across the world being put forth into this.

What Genes Are Linked To Parkinsons Disease

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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.

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Shocking Facts About Parkinson’s Disease:

1. In 2015, PD affected 6.2 million people and resulted in about 117,400 deaths globally.

2. There’s no known cause of PD.

3. Parkinson’s disease typically occurs in people over the age of 60, of which about one per cent are affected.

4. Males are more often affected than females at a ratio of around 3:2.

5. The disease is named after the English doctor James Parkinson, who published the first detailed description in An Essay on the Shaking Palsy, in 1817.

6. The average life expectancy following diagnosis is between 7 and 14 years.

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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Slice Titleover 40 Symptoms No Two People The Same

Matt can’t get his food into his mouth. “It’s been so long since I have been able to eat without throwing it on the floor. Being able to take something that could give me back control of my body would be a game-changer.

Hema had to stop doing the job she loved. Most people are diagnosed when they’re older and may be retired, but you can get Parkinson’s at any age. Suddenly you can’t work. Can’t provide. Can’t support your family.

Paula takes 13 pills every day. It’s a constant balancing act. Delaying her medication by even 15 minutes can derail her whole day. And Parkinson’s meds can have serious side effects, from involuntary movements to hallucinations.

For Omotola, it can feel like a constant battle against negative thoughts. “The worst thing is that Parkinson’s makes you feel like you’re not enough. As a wife, a mum and a friend. But I am determined to defy the limitations that Parkinson’s has placed on me.”

What We Know So Far

New Parkinson
  • 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.

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

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

In this unprecedented year, weve 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 weve 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, weve also seen the embrace of telemedicine as a way to continue to think about managing care but not necessarily having to go into a doctors 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 arent very many silver linings at the moment.

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

by IOS Press

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.

Explore further

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Why Arent We Closer To Finding A Cure For Parkinsons

Heres a question we and those in our community consider every day: With all of the money that is going toward finding a cure for Parkinsons, why arent we there yet? So, when we had the chance to sit down with Pete Schmidt, a member of our Board and the Vice Dean at the Brody School of Medicine and Associate Vice Chancellor for Health Care Regulatory Affairs at East Carolina University, we asked him this question:

How Can Stem Cell Technology Help

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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.

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