This week a new clinical trial was registered which caught our attention here at the SoPD HQ. It is being sponsored by a small biotech called Neuraly and involves a drug called NLY01.
NLY01 is a GLP-1R agonist – that is a molecule that binds to the Glucagon-like peptide-1 receptor and activates it. Other GLP-1R agonists include Exenatide (also called Bydureon) which is also also about to start a Phase III clinical trial in Parkinson’s (Click here to read a previous SoPD post about this).
There is a lot of activity in the Parkinson’s research world on GLP-1R agonists at the moment.
In today’s post, we will discuss what a GLP-1R agonist is, what we know about NLY01, and what the new clinical trial involves.
Every week there are new clinical studies being announced for Parkinson’s.
This week one particular newly registered clinical trial stood out. It involves a small biotech company Neuraly (which is owned by parent company D&D PharmaTech).
What is a GLP-1R agonist?
In August 2017, the results of a Phase II double-blind, placebo controlled clinical trial investigating whether the diabetes drug Exenatide (aka Bydureon) can be repurposed for the treatment of Parkinson’s were published.
Despite the fact that the study did not meet most of its end points, the Parkinson’s community got very excited about one of the results: The exenatide treated group demonstrated a stabilisation of their motor features over the 48 week trial, while the control group continued to worsen.
Over night, for many in the community, the hypothetical (a “disease-halting medication”) suddenly become a possibility. After such a long trail of negative clinical trial results, it was a very human and natural response for everyone to get excited. But with the news this month, that the Phase III exenatide clinical trial is about to start, the community needs to curb that excitement in order for a proper evaluation of the drug to take place.
In today’s post, we will look at the details of the new Phase III clinical trial for Exenatide and discuss why it is important to manage expections.
Here on the SoPD website we are often discussing novel potentially disease modifying therapies for Parkinson’s. And it is rather staggering the number and range of different approaches currently being tested on Parkinson’s.
And I am often asked, “Simon, if you were a betting man, which one I would put my money on? Which one are you expecting to work?”
Now, before we go on dear reader, please understand that my answer to this question will problably disappoint you.
You see, I do not expect any of these experimental treatments being clinically tested to work.
Now, before you turn off, please let me explain – because this is important (it is not click-bait).
Ok, I’m listening. Why don’t you expect any of these treatments to work?!?
Things were a bit quiet on the SoPD over the summer, but for good reasons. There was a short hiatus for a family break, but the rest of the time I was rather occupied with the day job. Tremendous efforts were being made at the Cure Parkinson’s Trust, as we were gearing up for our main event of the year: the Linked Clinical Trials (LCT) meeting.
This is an annual meeting at which 20 Parkinson’s experts from around the world, gather for a two day face-to-face pow-wow. They evaluate dossiers which contain everything we know about 20+ compounds which have exhibited potential for disease modification in Parkinson’s. The goal of the committee is to decide which of them is ready for clinical evaluation.
The writing of those LCT dossiers is a year long exercise, which inevitably becomes a bit of a panic in June and July (hence the lack of activity here at SoPD HQ during that period). It is a mammoth, marathon task, but as you shall see it is one that I rather like.
In today’s post, we will discuss what the Linked Clinical Trials initiative is, the process behind the project, and some of the progress being made by the programme.
Archimedes. Source: Lecturesbureau
Archimedes of Syracuse (287 BC – 212 BC) the ancient Greek mathematician, once said that the “shortest distance between two points is a straight line“.
My dad (who is not a regular readers of this blog, but is possibly on par with Archie – just in case he does ever read this) has often been heard saying “Just get to the point Simon“.
Millennia apart, but their collective wisdom is same: Ignore everything else, and get straight to the heart of the matter as quickly as you can.
And this is one of the aspect I really like about the Linked Clinical Trials initiative.
It is all about getting to potentially disease modifying treatments for Parkinson’s to the community as quickly as possible.
What is the Linked Clinical Trials programme?
Today’s post is a recap of Day 3 – the final day – at the World Parkinson’s Congress meeting in Kyoto, Japan.
I will highlight some of the presentations I was able to catch and try to reflect on what was an amazing meeting.
The final day of the WPC meeting for me started with Parkinson’s advocate Heather Kennedy‘s presentation on “Your radical new life: Creative ways to overcome our challenges”. In her talk, she spoke of the mindset that is required for tackling Parkinson’s and provided some advice on what-to-do and what-not-to-do.
And Heather was speaking from personal experience. Having been diagnosed in 2012, she has become an active advocate, supporter of Davis Phinney and Michael J Fox Foundations, and an administrator on several online sites. And she regularly speaks about different methods for overcoming the challenges of Parkinson’s:
“It is not ‘why is this happening to me?’, it is ‘what is this teaching me?”
Here is a presenation she gave at the recent Parkinson’s Eve meeting in the UK earlier this year:
Key among her pieces of advice is the need to make connections:
Today’s post is a recap of Day 2 at the World Parkinson’s Congress meeting in Kyoto, Japan.
I will highlight some of the presentations I was able to catch and discuss some of my key take-aways from the day’s activities.
Early meetings meant that I arrived late to the morning session of presentations on the Day 2 (6th June) of the WPC meeting. But luckily I was in time to catch Benjamin Stecher giving his talk in the main hall.
Diagnosed at 29 years of age with young onset Parkinson’s, Ben has spent the last couple of years touring the world requesting meetings with Parkinson’s researchers, to learn more about what they do and what still needs to be done. This quest has give him a unique perspective on the state of Parkinson’s research, and has helped him in his role as an advocate.
I was looking forward to hearing him speak on the main hall stage,…
…and, like everyone else in the room, I was surprised by what he did during his talk.
Recent analysis of blood samples collected during the Phase II clinical trial of Exenatide in Parkinson’s has uncovered a very interesting finding that could have major implications for not only Parkinson’s, but for many different neurological conditions.
Exenatide is a treatment that helps to control glucose levels in people with diabetes. More recently, however, it has been suggested that this drug may also have beneficial effects in Parkinson’s. A collection of clinical trials in Parkinson’s are currently unway to test this idea.
The researchers who conducted a Phase II clinical trial of Exenatide in Parkinson’s have analysed ‘exosomes‘ collected from the blood of participants, and they found something rather remarkable.
In today’s post we will discuss what exosomes are, what the researchers found, and why their discovery could have major implications for all of neurological research.
This week, however, researchers involved in the study reported yet another really interesting finding from the trial. And this one could have profound consequences for how we study not only Parkinson’s, but many other neurological conditions.
What did they find?
Last week this report was published:
Title: Utility of Neuronal-Derived Exosomes to Examine Molecular Mechanisms That Affect Motor Function in Patients With Parkinson Disease: A Secondary Analysis of the Exenatide-PD Trial.
Authors: Athauda D, Gulyani S, Karnati H, Li Y, Tweedie D, Mustapic M, Chawla S, Chowdhury K, Skene SS, Greig NH, Kapogiannis D, Foltynie T.
Journal: JAMA Neurol. 2019 Jan 14. doi: 10.1001/jamaneurol.2018.4304. [Epub ahead of print]
In the Exenatide Phase II clinical trial, 60 people with moderate Parkinson’s were randomly assigned to receive either 2mg of Exenatide or placebo once weekly for 48 weeks followed by a 12-week washout (no treatment) period. The results suggested a stablisation of motor features over the 48 weeks of the study in the treated group (while the condition in the placebo group continued to progress).
During the study (which was conducted between June 2014 – June 2016), blood samples were collected at each assessement.
From those blood samples, serum was collected and analysed.
Remind me again, what is serum?
In a recent post we discussed the results of the Exenatide clinical trial from last year, and looked at some further analysis of the data, which hinted at the possibility that the drug may be having additional benefits (Click here to read that post).
The researchers behind the Exenatide study have now published the results of a second deep dive into the data and found something potentially very interesting and useful: they may have identified certain characteristics of those participants in the study who responded the best to the drug.
The researchers are quick to point out that this type of post hoc analysis is only conducted for the purpose of generating hypotheses, but it will be interesting to determine if this is finding is validated in further clinical investigations of Exenatide.
In today’s post, we will review the new finding and discuss what they could potentially mean.
Tom Isaacs. Source: GrannyButtons
For most of the Parkinson’s community, Tom Isaacs requires no introduction.
In 1996 – at just 27-years of age – the London-based surveyor was diagnosed with Parkinson’s. After dealing with the initial shock of it all, Tom embraced his situation and became a committed, (utterly) tireless activist. He first walked the entire coastline of the UK to raise money and awareness for Parkinson’s. His book, “Shake well before use“, discusses that trip and adapting to life with Parkinson’s. It is a fantastic read.
And upon returning from his epic walk, he (along with three others) founded and set up the Cure Parkinson’s Trust.
It is pretty safe to say that beyond Michael J Fox and Muhammad Ali, Tom was one of the most impactful members of the Parkinson’s community on marshaling scientific research efforts to find a cure for Parkinson’s.
And he did it all with style and humour:
This was a video of Tom in 2009, talking about life with Parkinson’s:
His passing last year was a terrible loss to the community, and this month, the European Journal of Neuroscience has a special tribute edition dedicated to the memory of Tom.
One of the research reports in that issue involves a study that was very close to Tom’s heart: The Exenatide study.
And the report provides some very interesting new results based on re-analysis of the results of the clinical study.
What does the new report say?
At 23:30 on the 3rd August 2017, the results of a phase II clinical trial investigating the use of a Glucagon-like peptide-1 receptor (GLP-1R) agonist called Exenatide (Bydureon) in Parkinson’s were published the Lancet journal website.
The findings of the study were very interesting.
And after years of failed trials, the Parkinson’s community finally had a drug that appeared to be ‘doing something’. Naturally these results got many in the Parkinson’s community very excited.
Over the last couple of weeks, further research related to this topic has been published. In today’s post we will review some of this new research and ask some important questions regarding how to move forward with these results.
Dr John Eng. Source: Health.USnews
The Award was created in 2012 to celebrate researchers whose seemingly odd or obscure federally funded research turned out to have a significant and positive impact on society.
This week a research report was published in the journal Nature Medicine that expanded on the work of Dr Eng (some 25 years after his big discovery).
And it could be very important to the Parkinson’s community.
Sounds intriguing. What did Dr Eng do?
A reader recently asked for an explanation of some recent research regarding diabetes and Parkinson’s.
You see, a significant proportion of the Parkinson’s community have glucose intolerance issues and some live with the added burden of diabetes. That said, the vast majority of diabetics do not develop PD. Likewise, the vast majority of people with Parkinson’s do not have a diagnosis of diabetes.
There does appear to be a curious relationship between Parkinson’s and diabetes, with some recent research suggests that this association can be detrimental to the course of the condition.
In today’s post we will look at what what diabetes is, consider the associations with Parkinson’s, and we will discuss the new research findings.
Foreman and Ali. Source: Voanews
1974 was an amazing year.
On October 30th, the much-hyped heavyweight title match – the ‘Rumble in the Jungle’ – between George Foreman and Muhammad Ali took place in Kinshasa, Zaire (Democratic Republic of the Congo).
Stephen King. Source: VanityFair
A 26-year-old author named Stephen King published his debut novel, “Carrie” (April 5, with a first print-run of just 30,000 copies).
Lucy. Source: Youtube
The fossil remains of a 3.2 million years old hominid skeleton was discovered in Ethiopia (November 24th). It was named ‘Lucy’ – after the song “Lucy in the Sky with Diamonds” by The Beatles which was played repeatedly in the expedition camp the evening after the team’s first day of work on the site (Source).
And Richard Nixon becomes the first US president to resign from office (August 9th).
President Richard Nixon. Source: Fee
In addition to all of this, in December of 1974, a small study was published in the Journal of Chronic Diseases.
It dealt with Parkinson’s and it presented a rather startling set of findings:
The great ice hockey player Wayne Gretzky once said “A good hockey player plays where the puck is. A great hockey player plays where the puck is going to be” (the original quote actually came from his father, Walter).
At the start of each year, it is a useful practise to layout what is planned for the next 12 months. This can help us better anticipate where ‘the puck’ will be, and allow us to prepare for things further ahead.
2017 was an incredible year for Parkinson’s research, and there is a lot already in place to suggest that 2018 is going to be just as good (if not better).
In this post, we will lay out what we can expect over the next 12 months with regards to the Parkinson’s-related clinical trials research of new therapies.
Charlie Munger (left) and Warren Buffett. Source: Youtube
Many readers will be familiar with the name Warren Buffett.
The charming, folksy “Oracle of Omaha” is one of the wealthiest men in the world. And he is well known for his witticisms about investing, business and life in general.
Warren Buffett. Source: Quickmeme
He regularly provides great one liners like:
“We look for three things [in good business leaders]: intelligence, energy, and integrity. If they don’t have the latter, then you should hope they don’t have the first two either. If someone doesn’t have integrity, then you want them to be dumb and lazy”
“Work for an organisation of people you admire, because it will turn you on. I always worry about people who say, ‘I’m going to do this for ten years; and if I really don’t like it very much, then I’ll do something else….’ That’s a little like saving up sex for your old age. Not a very good idea”
“Choosing your heroes is very important. Associate well, marry up and hope you find someone who doesn’t mind marrying down. It was a huge help to me”
Mr Buffett is wise and a very likeable chap.
Few people, however, are familiar with his business partner, Charlie Munger. And Charlie is my favourite of the pair.