Parkinson’s Awareness Month

M3 Global Research is currently recruiting patients with Parkinson’s to participate in usability testing for a new device used in conjunction with a smart phone (both provided) in the United States and in Germany. We’re offering generous compensation to the patients taking part and also the physicians referring them. If you are a member of the M3 Global Research panel, contact M3_US_support@eu.m3.com to find out if you qualify. If you are a patient who is not a member and wish to participate, please register by clicking here if you are in the USA and here if you are in Germany. You will then receive an invite by email.

Around seven to 10 million people worldwide live with Parkinson’s disease, a condition that currently has no cure. For this year’s WordParkinson’s Day, Parkinson’s UK, a charity that works towards finding a cure and improving life for everyone affected by the condition, has launched the campaign #UniteForParkinsons. It aims to give voice and platform to the Parkinson’s community by featuring their experiences in a world-wide campaign video and encouraging others to do the same. Watch it here:


Complexity of Parkinson’s ‘massively underestimated’ in the UK

In a survey to mark World Parkinson’s Day (Wednesday 11 April), Parkinson’s UK has discovered that 78% of the public massively underestimate how many symptoms of Parkinson’s there are. Although most people are aware of visible symptoms like tremor, Parkinson’s can also come with more than 40 less well-known symptoms such as sleep issues, anxiety and hallucinations. Shockingly, more than a third (37%) thought there were fewer than ten symptoms of Parkinson’s and more than 41% thought there were fewer than 30.

The charity is warning that this lack of awareness means that people with Parkinson’s often feel they need to hide their symptoms in public, or don’t want to go out at all due to being incorrectly judged or mocked. Previous findings from the charity have uncovered:

  • A quarter (25%) have had symptoms mistaken for drunkenness
  • 11% have been laughed at because of their symptoms
  • More than a third (34%) feel they would be judged if they were out in public
  • Almost a third (32%) don’t feel like their symptoms are socially acceptable

These symptoms are merely the tip of the iceberg, it warns, and do not reflect what people with Parkinson’s most want addressing. In a recent project carried out by the charity to identify priorities to focus on for improving everyday life, tremor came 26th on a list of what people with Parkinson’s want research to tackle.

Artificial intelligence to help develop new Parkinson’s treatments

Parkinson’s UK is actively involved in research, and recently one of its research proposals has won the BenevolentAI Award. The project demonstrated how AI technology could solve specific research challenges in Parkinson’s.

There have been no major breakthroughs in Parkinson’s treatments in the last 50 years. Current treatments revolve around medication that works by restoring the level of dopamine in the brain or mimicking its actions; deep brain stimulation (DBS), a type of surgery where electrodes are implanted deep inside specific parts of the brain, but which is not suitable for every patient; and physical therapies such as physiotherapy, speech and language therapy and occupational therapy, that are important in the management of the condition.

Parkinson’s UK’s proposal will use BenevolentAI platform’s capabilities to reason, deduce and suggest entirely new treatments for Parkinson’s. The aim is to identify at least three currently available medicines that can be repurposed to address Parkinson’s, and two brand-new ways to treat the condition with new drugs. Read more about the project: Artificial intelligence to help develop new Parkinson’s treatments.

*All rights belong to Parkinson’s UK. We would like to thank the charity for sharing this content with us.

Talking to… Dr. Lawrence Feldman

Dr. Feldman, a dermatologist specialising in the treatment of melanoma, practising for 30 years in the US, spoke to the M3 blog about prevention, new treatment developments and the changes in patients’ approaches to the condition.

M3 Global Research is about to launch a large patient outcome study about melanoma. If you are a dermatologist or an oncologist and are interested in participating, please contact blog@eu.m3.com. If you are not a member of our panel and are interested in participating, you can register for this study here.

What do you think about new developments such as optical biopsy, or tests that look at gene expression patterns and allow physicians to see if stages 1 and 2 are likely to spread, new types of immunotherapy and targeted therapies etc?

The field has definitely changed dramatically, more so than any other field as we have gone from a situation where there was really no therapy that was effective, to now seeing therapies that can induce prolonged remission and even overall survival rates. As far as the gene expression profile, it is a huge advance, perhaps even replacing biopsy in the future. And with optical biopsy and dermatoscope use, the clinical diagnosis of melanoma has gotten much better.

How long ago would you say was the breakthrough?

I would say it began about five years ago. It’s really when things started to change, I guess with the introduction of Zelboraf (vemurafenib) – that was the big turning point.

How do you personally keep updated with news in melanoma treatment?

I would say conferences, congresses, symposiums, journal articles, Tumor Board. We have a Tumor Board at the hospital on a pretty regular basis, so all these things help us to keep up to date.

From your experience, how is the level of treatment in the US compared to other countries?

I think the level in the United States is high, I’d say it’s quite advanced. We definitively have access to many of the newer therapies.

And how is access to treatment for patients? Do most insurances cover melanoma treatment now?

If you are following NCCN (National Comprehensive Cancer Network) guidelines, access is very good. It is harder to get access off-label.

What’s the part played by prevention in skin cancer?

I would say it’s the key. Prevention is better than treatment. So, especially if it’s about avoiding sunburn early on, that would be probably the most critical thing.

During the past 30 years have you noticed changes in patients’ attitudes and empowerment? Are patients more aware of conditions such as melanoma and therefore pay more attention to their own bodies, coming to you with questions?

I think people are more aware of it. Even younger people are more aware of the sun causing it, and even tanning salons, that are ‘a big thing’ in the United States and also a big campaign that has got more publicity recently.

How important is early detection for the treatment of melanoma?

It’s key. We talk about 3 things: prevention, early detection, and treatment. Prevention is the most important, so you don’t have the problem at all; early detection is vital because if you catch the melanoma before it’s reached a certain depth, then you don’t really need any other therapy; and then early treatment, for patients that are less fortunate.

Living with haemophilia: ‘KT’ from Orem, Utah, US

Name: KT

Age: 57.

City and country: Orem, Utah, USA.

What type of haemophilia do you suffer from? Type A.

What is the severity of your condition: severe, moderate or mild? About 3 on a scale of 10.

Is there anyone else in your family who suffers from haemophilia? 1 brother and 3 cousins.

When were you diagnosed? In the mid 70s, I can’t remember the exact date.

How would you say having haemophilia has affected your daily life? To what extend do you think that having haemophilia has stopped you from having a life style that you would consider better?

I have to be cautious of cuts and bruising even as mild as I am. I have joint problems based on bleeding which make some basic tasks difficult. Other than some mobility issues, haemophilia hasn’t really stopped me from doing the things I want to do. I just suffer the consequences more.

Are you part of any support groups? If yes, what are your main activities and how does that help you?

I am connected with the local haemophilia society but I don’t engage with them very much.

Do you think haemophilia is on the media enough? Are associations doing a good job raising awareness on the condition?

I don’t believe that I have ever seen any awareness campaigns. Word is mostly spread through health providers who specialize in the disorder. Even my personal physician is a bit out of the loop. He relies on my knowledge when we discuss treatment. 

What treatment are you on now?

I take Cogenate (sp) as needed.

How would you evaluate your relationship with your physician(s)? Are you in close contact with them? Have they been your doctor(s) for a long time? How important it is for you to have a good relationship with them?

I have been involved with my personal physician and haematologist for many years. We have a very good working relationship.

How is your access to healthcare? Do you think you have enough support from the government of the US in any way?

I have good access but it is very expensive even with good health insurance. The government has no involvement but I do receive consideration from the Cogeniate (sp) providers which make it more affordable. 

Do you think that in the US there is sufficient investment in researching new treatments? Have you ever for example heard of new treatments that are being developed in other countries and you would like to have access to?

I haven’t heard of any new treatments that would be useful for my type and level of severity.

Do you think the research and pharma industry invest enough in research for haemophilia?

No.

Are you happy with the progress in the treatments for haemophilia? Do you see any improvements within the last few years?

There have been a number of improvements since the 70s. I hear rumours of genetic treatments in the works and think that that would be amazing.

What do you hope for yourself and others who have haemophilia? How do you see the future for haemophilia patients?

I would love to see an eradication of the disorder. I am very lucky with my severity. I have known a number of others who have suffered and perished due to haemophilia. I have hope for the future.

Living with Haemophilia

The M3 Blog team spoke to six people living with haemophilia in the UK and the US, two of them the mothers of young boys with the condition, to understand their challenges, fears, their relationship with healthcare professionals and the treatments they are experiencing. The stories they’ve shared show more than that, they are about acceptance and hope.

The interviewees represented a wide cross-section of different ages and different types of haemophilia and degree of severity, which means treatment and perception of the condition are also different. Almost all of them were diagnosed at birth or whilst they were still very young, and some have very early memories of it: “My earliest memory was when I was about three years old and had bleeding to the joint in my knee and was admitted to hospital”, shared ‘FK’, also 45, from Birmingham. He has severe haemophilia A.

To some, haemophilia has affected their lives significantly. ‘LM’, the mother of a 12-year-old boy with moderate haemophilia A and inhibitors, described how his condition has had a massive impact on him and on their family. Not only does he have to inject himself every morning before school, in the last six or seven months they have been to over 40 hospital appointments, not including A&E visits or admissions. ‘LM’ also shared interesting insights on being a mother of a boy with haemophilia: “There are so many different phases: when he was thrashing around in his cot I’d worry about him bruising his arms, then when he was starting to walk I was worried about him falling over. Now he walks to school on his own and you worry about that. Soon it will be the teenage years when he will be out and about on his own more. Every age brings its own challenges”.

For ‘DB’, ‘a 45-year-old man with severe haemophilia B from Hitchin, UK,  it was particularly difficult when at 19 he contracted hepatitis C through a blood transfusion, amidst what is now known as the contaminated blood scandal from 1970s and 80s. On the other hand, he said haemophilia hasn’t impacted his life negatively on the whole. As he couldn’t play rugby and football when he was younger, it meant that he concentrated on arts. He learnt to play the drums, guitar, sing and to act, skills that have shaped his social life ever since. “I don’t think that I would have acted or played music if I didn’t have haemophilia”, he reflected.

According to ‘KT’, a 57-year-old male from Utah, US, with mild haemophilia A, haemophilia hasn’t really stopped him from doing the things he wants to do, he just suffers the consequences more. “I have to be cautious of cuts and bruising even as mild as I am. I have joint problems based on bleeding which make some basic tasks difficult”, he explained.

In a similar way, ‘LOSP’, a 35-year-old with mild haemophilia A who lives in London, told us he feels haemophilia is part of who he is and that he tries to manage his condition by avoiding injury and looking after himself, taking injections only when he has to.

Everyone we spoke to said they have a very good relationship with their physicians and nurses. The ones living in the UK praised the NHS staff and the support from haemophilia centres in general.

‘A’, also from London, takes care of her 9-year-old son with severe haemophilia A. He’s been on a global trial for 6 months now, and according to her for her son the results have been great. “He’s had no bleeds at all. His factor level has moved from 1% to 7-8% which is a move from severe to mild, and so far the trial has been really good – the medication is a factor VIII substitute so is being trialled with patients with inhibitors. It’s helped his confidence so much so far”.

Gene therapy seems to be the hope for most of the people who have shared their stories with us. Despite different levels of severity and success of treatments, the possibility of leading a “normal” life is a prospect they all look forward to – for themselves and future generations.

To read each interview in full, please click on the links below:

‘DB’, 45-year-old man – Hitchin, UK

‘LM’, mother of a 12-year-old boy – London, UK

‘LOSP’, 35-year-old man – London, UK

‘KT’, 57-year-old-man – Orem, Utah, US

‘A’, mother of a 9-year-old-boy – London, UK

‘FK’, 45-year-old man – Birmingham, UK