Tag: UPDRS

Monitoring an Apple in motion

~ Simon ~ 3 Comments

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Wearable technology offers the potential to more accurately monitor the symptoms of Parkinson’s in real time. Such information could allow for better and more precise management of the condition, as well as providing objective measures for clinical trials exploring novel therapies.

Assessing some of the features of Parkinson’s, however, is not easy. Differentiating jerky involuntary movements like tremor or dyskinesias from planned movements like typing or shaking someone’s hand has proven difficult

Recently, researchers at the tech giant Apple have been applying some focus to this problem and they are now sharing their results with the Parkinson’s community.

In today’s post, we will review a research report presenting the results of the Apple study and discuss other recent events in wearable tech for Parkinson’s.

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Source: Tes

I used to be an Apple fan back in the day (mid-late 2000s). Wonderful user interface, superb design, lovely innovative products.

But I have to admit: gradually over time I became disenchanted with them.

Why?

The products became too expensive, the “Walled garden” mentality around the operating system frustrated me, and there has been a lack of serious innovation (a new iteration on a phone or tablet every year just doesn’t cut it… and now they are thinking of getting into the crowded space of electric cars… yippee, inspiring stuff).

Maybe we came to expect too much from them, but (personal opinion here) I think they lost their fanatical drive in the absence of Steve.

Source: Dansilvestre

[Positive way to start a post on, huh? It gets better. Stay with me]

All of that said, Apple published a research report earlier this year that deserves the Parkinson’s community’s attention and respect.

What did they report?

Continue reading “Monitoring an Apple in motion” →

We need to talk about the UPDRS

~ Simon ~ 11 Comments

 

“The measure of who we are is what we do with what we have” – Vince Lombardi

The measuring of Parkinson’s is complicated. There is such enormous variability between individual cases that the task of assessing people is very difficult.

The primary method that is used in clinics around the world is the Unified Parkinson’s Disease Rating Scale (or UPDRS). It is by no means perfect, and recently (in the wake of several unsuccessful clinical trials) there has been heated debate as to whether it is really up to the task.

Does it accurately reflect the condition? Does it really capture the lived experience? Can it pick up subtle changes associated with potentially disease modifying therapies in clinical trials? Or is it simply a “we’ve always done it this way” kind of tool?

In today’s post, we will look at what the UPDRS is, discuss some of the criticisms associated with it, and consider what solutions to those issues could look like.

 

Source: Wired

This is Andy Grove and his story is rather remarkable.

Born in 1936 to a Jewish family in Budapest, he managed to survive the Nazis, and then fleed Hungary when Soviet tanks started rolling in. Arriving in the US with absolutely nothing, he taught himself English, before going to City College of New York and later the University of California (Berkeley) where he received a PhD in chemical engineering.

And that was just the start of his amazing tale.

After completing his PhD (and publishing a textbook on semiconductors), Grove joined the seminal Silicon Valley company – Fairchild Semiconductor – in 1963. He worked his way up from researcher to assistant director of development, before becoming the first person that Robert Noyce and Gordon Moore (of Moore’s Law fame) hired after they departed Fairchild to start their own little company in 1968.

The name of that company was Intel.

Source: Wikipedia

Grove also worked his way up the ladder at Intel – from director of engineering to CEO – and he is credited with transforming the company from a struggling memory chip maker into the processor powerhouse it is today. He was Time’s ‘Man of the Year’ in 1997 and he was a widely revered figure in Silicon valley.

Source: Time

But the path to success was not easy.

Having survived prostate cancer in 1995, Grove was diagnosed with Parkinson’s in 2000. Viewing the situation as a problem solving exercise, he poured tens of millions of his own money into researching Parkinson’s.

Andy & Michael J Fox. Source: MJFF

But coming from the world of ‘Moore’s Law’, Grove became frustrated by a.) the slow speed of progress in the world of biomedical research and b.) the tools used to assess it.

In particular, he disliked the UPDRS, which he referred to as a “piece of crap” (Source – you should read the linked article).

What is the UPDRS?

Continue reading “We need to talk about the UPDRS” →

Spine-tingling research

~ Simon ~ 4 Comments

Deep brain stimulation (DBS) has now become a standard treatment option for people with Parkinson’s (Click here to read more about DBS), but recently researchers have been investigating a whole new form of stimulation to further help alleviate the symptom of the condition.

Spinal cord stimulation – the electrical modulation of the spinal cord – has been tested in models of Parkinson’s in laboratories for the last decade, and this week we saw the publication of the results of a pilot clinical study testing this approach in humans with Parkinson’s.

In today’s post we will discuss what spinal cord stimulation is, review the results of this pilot study, and discuss what could happen next for this new treatment approach.

Source: Videoblocks

2017 was the 200th anniversary of the first report of Parkinson’s by one James Parkinson in 1817 (Click here to read a previous post on this), and the 20th anniversary of the discovery of the first genetic mutation associated with Parkinson’s (Click here to read more about this).

It was also the 50th anniversary of the first use of a technique called spinal cord stimulation.

What is spinal cord stimulation?

Spinal cord stimulation is a form of implantable neuromodulation. Similar to deep brain stimulation (or DBS), it involves using electrical signals to modulate neural activity. But rather than electrodes being placed into the brain (in the case of DBA), spinal cord stimulation involves – as the label on the can suggests – specific areas of the spinal cord being stimulated for the treatment of certain types of pain.

The treatment involves a column of stimulating electrodes that is surgically implanted in the epidural space of the spine. And before you ask: the epidural space is the area between the outer protective skin of the spinal cord (called the dura mater) and the surrounding vertebrae. So the device lies against the spinal cord, and is protected by the bones that make up the spine (as shown in the image below).

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The stimulating electrodes within the epidural space. Source: SpineOne

An electrical pulse generator is implanted in the lower abdomen and conducting wires are connected between the electrodes to the generator. Much like deep brain stimulation, the system is entirely enclosed in the body and operated with a remote control.

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An x-ray of the spine with a stimulator implanted (towards the top of the image, and cords leading off to the bottom left). Source:Wikipedia

How does spinal cord stimulation work?

Continue reading “Spine-tingling research” →

Voyager Therapeutics: phase Ib clinical trial results

~ Simon ~ 15 Comments

 

This week a biotech company called Voyager Therapeutics announced the results of their ongoing phase Ib clinical trial. The trial is investigating a gene therapy approach for people with severe Parkinson’s disease.

Gene therapy is a technique that involves inserting new DNA into a cell using a virus. The DNA can help the cell to produce beneficial proteins that go on help to alleviate the motor features of Parkinson’s disease.

In today’s post we will discuss gene therapy, review the new results and consider what they mean for the Parkinson’s community.

Source: Joshworth

On 25th August 2012, the Voyager 1 space craft became the first human-made object to exit our solar system.

After 35 years and 11 billion miles of travel, this explorer has finally left the heliosphere (which encompasses our solar system) and it has crossed into the a region of space called the heliosheath – the boundary area that separates our solar system from interstellar space. Next stop on the journey of Voyager 1 will be the Oort cloud, which it will reach in approximately 300 years and it will take the tiny craft about 30,000 years to pass through it.

Where is Voyager 1? Source: Tampabay

Where is Voyager actually going? Well, eventually it will pass within 1 light year of a star called AC +79 3888 (also known as Gliese 445), which lies 17.6 light-years from Earth. It will achieve this goal on a Tuesday afternoon in 40,000 years time.

Gliese 445 (circled). Source: Wikipedia

Remarkably, the Gliese 445 star itself is actually coming towards us. Rather rapidly as well. It is approaching with a current velocity of 119 km/sec – nearly 7 times as fast as Voyager 1 is travelling towards it (the current speed of the craft is 38,000 mph (61,000 km/h).

Interesting, but what does any of that have to do with Parkinson’s disease?

Well closer to home, another ‘Voyager’ is also ‘going boldly where no man has gone before’ (sort of).

Continue reading “Voyager Therapeutics: phase Ib clinical trial results” →

Exenatide: An editorial

~ Simon ~ 25 Comments

editorial

In my previous post, we briefly reviewed the results of the phase II double-blind, randomised clinical trial of Exenatide in Parkinson’s disease. The study indicates a statistically significant effect on motor symptom scores after being treated with the drug.

Over the last few days, there have been many discussions about the results, what they mean for the Parkinson’s community, and where things go from here, which have led to further questions.

In this post I would like to address several matters that have arisen which I did not discuss in the previous post, but that I believe are important.

bydureon

I found out about the Exenatide announcement – via whispers online – on the afternoon of the release. And it was in a mad rush when I got home that night that I wrote up the post explaining what Exenatide is. I published the post the following evening however because I could not access the research report from home (seriously guys, biggest finding in a long time and it’s not OPEN ACCESS?!?!?) and I had to wait until I got to work the next day to actually view the publication.

I was not really happy with the rushed effort though and decided to follow up that post. In addition, there has been A LOT of discussion about the results over the weekend and I thought it might be good to bring aspects of those different discussion together here. The individual topics are listed below, in no particular order of importance:

1. Size of the effect

There are two considerations here.

Firstly, there have been many comments about the actual size of the effect in the results of the study itself. When people have taken a deeper look at the findings, they have come back with questions regarding those findings.

And second, there have also been some comments about the size of the effect that this result has already had on the Parkinson’s community, which has been considerable (and possibly disproportionate to the actual result).

The size of the effect in the results

The results of the study suggested that Exenatide had a positive effect on the motor-related symptoms of Parkinson’s over the course of the 60 week trial. This is what the published report says, it is also what all of the media headlines have said, and it sounds really great right?

The main point folks keep raising, however, is that the actual size of the positive effect is limited to just the motor features of Parkinson’s disease. If one ignores the Unified Parkinson’s Disease Rating Scale (UPDRS) motor scores and focuses on the secondary measures, there isn’t much to talk about. In fact, there were no statistically significant differences in any of the secondary outcome measures. These included:

Continue reading “Exenatide: An editorial” →

Exenatide: One step closer to joblessness!

~ Simon ~ 39 Comments

bydureon

The title of today’s post is written in jest – my job as a researcher scientist is to find a cure for Parkinson’s disease…which will ultimately make my job redundant! But all joking aside, today was a REALLY good day for the Parkinson’s community.

Last night (3rd August) at 23:30, a research report outlining the results of the Exenatide Phase II clinical trial for Parkinson’s disease was published on the Lancet website.

And the results of the study are good:while the motor symptoms of Parkinson’s disease subject taking the placebo drug proceeded to get worse over the study, the Exenatide treated individuals did not.

The study represents an important step forward for Parkinson’s disease research. In today’s post we will discuss what Exenatide is, what the results of the trial actually say, and where things go from here.

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Last night, the results of the Phase II clinical trial of Exenatide in Parkinson’s disease were published on the Lancet website. In the study, 62 people with Parkinson’s disease (average time since diagnosis was approximately 6 years) were randomly assigned to one of two groups, Exenatide or placebo (32 and 30 people, respectively). The participants were given their treatment once per week for 48 weeks (in addition to their usual medication) and then followed for another 12-weeks without Exenatide (or placebo) in what is called a ‘washout period’. Neither the participants nor the researchers knew who was receiving which treatment.

At the trial was completed (60 weeks post baseline), the off-medication motor scores (as measured by MDS-UPDRS) had improved by 1·0 points in the Exenatide group and worsened by 2·1 points in the placebo group, providing a statistically significant result (p=0·0318). As you can see in the graph below, placebo group increased their UPDRS motor score over time (indicating a worsening of motor symptoms), while Exenatide group (the blue bar) demonstrated improvements (or a lowering of motor score).

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Reduction in motor scores in Exenatide group. Source: Lancet

This is a tremendous result for Prof Thomas Foltynie and his team at University College London Institute of Neurology, and for the Michael J Fox Foundation for Parkinson’s Research who funded the trial. Not only do the results lay down the foundations for a novel range of future treatments for Parkinson’s disease, but they also validate the repurposing of clinically available drug for this condition.

In this post we will review what we know thus far. And to do that, let’s start at the very beginning with the obvious question:

So what is Exenatide?

Continue reading “Exenatide: One step closer to joblessness!” →

Helicobacter pylori: Unwanted passengers?

~ Simon ~ 3 Comments

helicobacter-pylori


Whether we like it or not, we house a great deal of microbes.

Many of these tiny creatures aid us in our daily living by conducting important functions. Some of these microbes, however, may not be helping us, getting a free ride, and potentially causing trouble.

In today’s post we will review recent research regarding one particular family of bacteria, Helicobacter pylori, and what they might be doing in relations to Parkinson’s disease.

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Source: ScienceFriday

In his magnificent book, I contain multitudes, science writer/journalist Ed Yong writes that we – every single one of us – release approximately 37 million bacteria per hour. By talking, breathing, touching, or simply being present in the world, we are losing and also picking up the little passengers everywhere we go.

Reminds me of that Pascal Mercier book “Night Train to Lisbon” – We leave something of ourselves behind when we leave a place,… I’m not sure if this is what he was referring to though.

Yong also points out that: 80% of the bacteria on your right thumb are different to the bacteria on your left thumb.

It’s a fascinating book (and no, I am not receiving any royalties for saying that).

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Microbes. Source: NYmag

We have discussed microbes several times on this blog, particularly in the context of the gut and its connection to Parkinson’s disease (Click here, here and here to read some of those posts). Today we are going to re-visit one particular type of microbe that we have also discussed in a previous post: Helicobacter pylori.

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Helicobacter pylori. Source: Helico

Continue reading “Helicobacter pylori: Unwanted passengers?” →

Glutathione – Getting the k’NAC’k of Parkinson’s disease

~ Simon ~ 43 Comments

NAC

The image above presents a ‘before treatment’ (left) and ‘after treatment’ (right) brain scan image from a recent research report of a clinical study that looked at the use of Acetylcysteine (also known as N-acetylcysteine or simply NAC) in Parkinson’s disease.

DaTscan brain imaging technique allows us to look at the level of dopamine processing in an individual’s brain. Red areas representing a lot; blue areas – not so much. The image above represents a rather remarkable result and it certainly grabbed our attention here at the SoPD HQ (I have never seen anything like it!).

In today’s post, we will review the science behind this NAC and discuss what is happening with ongoing clinical trials.

shutterstock_brains

Source: The Register

Let me ask you a personal question:

Have you ever overdosed on Paracetamol?

Regardless of your answer to that question, one of the main treatments for Paracetamol overdose is administration of a drug called ‘Acetylcysteine’.

Why are you telling me this?

Because acetylcysteine is currently being assessed as a potential treatment for Parkinson’s disease.

Oh I see. Tell me more. What is acetylcysteine?

Acetylcysteine-2D-skeletalAcetylcysteine. Source: Wikimedia

Acetylcysteine (N-acetylcysteine or NAC – commercially named Mucomyst) is a prodrug – that is a compound that undergoes a transformation when ingested by the body and then begins exhibiting pharmacological effects. Acetylcysteine serves as a prodrug to a protein called L-cysteine, and – just as L-dopa is an intermediate in the production of dopamine – L-cysteine is an intermediate in the production of another protein called glutathione.

Take home message: Acetylcysteine allows for increased production of Glutathione.

What is glutathione?

Glutathione-from-xtal-3D-balls

Glutathione. Source: Wikipedia

Glutathione (pronounced “gloota-thigh-own”) is a tripeptide (a string of three amino acids connected by peptide bonds) containing the amino acids glycine, glutamic acid, and cysteine. It is produced naturally in nearly all cells. In the brain, glutathione is concentrated in the helper cells (called astrocytes) and also in the branches of neurons, but not in the actual cell body of the neuron.

It functions as a potent antioxidant.

Continue reading “Glutathione – Getting the k’NAC’k of Parkinson’s disease” →