Tag: clinical trial

EJS-ACT PD

~ Simon ~ 9 Comments

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This week an announcement was made regarding The Edmond J. Safra Accelerating Clinical Treatments for Parkinson’s Disease (EJS-ACT PD) Initiative.

It is hoping to revolutionise the way clinical trials for potentially disease-modifying drugs for Parkinson’s are conducted.

The project is focused on the setting up a multi-arm, multi-stage (MAMS) platform for evaluating new therapies for PD.

In today’s post, we will discuss what MAMS trials involve and the current details of the EJS-ACT PD initiative.

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Source: Motionarray

This week I boarded a train for the first time in 16 months and made my way down to London. It felt a wee bit surreal.

I arrived at Liverpool street station and was immediately shocked by the lack of crowds, the lack of face masks (seriously?!? I’ve had my two jabs as well, but I’m still wearing my mask – you are nuts if you don’t!), and the large number of empty shops. How the world has changed.

In the early morning light, I walked across central London towards St Pancras station – the weather was spectacular and it was an incredible pleasure to stroll through some old stomping grounds.

Source: Parksandgardens

At St Pancras station, I made my way to the enormous Francis Crick institute, where a group of Parkinson’s researchers and advocates were gathering for a really intriguing meeting.

Source: Timeshighereducation

What was the meeting about?

Continue reading “EJS-ACT PD” →

I’ll have the fish please

~ Simon ~ 16 Comments

We have previously discussed the importance of the right foods for people with Parkinson’s on this blog – Click here for a good example.

Recently, new data from researchers in Sweden points towards the benefits of a specific component of fish in particular.

It is a protein called β-parvalbumin, which has some very interesting properties.

In today’s post, we discuss what beta-parvalbumin is, review the new research findings, and consider how this new information could be applied to Parkinson’s.

A very old jaw bone. Source: Phys

In 2003, researchers found 34 bone fragments belonging to a single individual in a cave near Tianyuan, close to Beijing (China).

But it was not the beginning of a potential murder investigation.

No, no.

This was the start of something far more interesting.

Naming the individual “Tianyuan man”, the researchers have subsequently found that “many present-day Asians and Native Americans” are genetically related to this individual. His bones represented one of the oldest set of modern human remains ever found in the eastern Eurasia region.

Tianyuan caves. Source: Sciencemag

But beyond the enormous family tree, when researchers further explored specific details about his jaw bone (or lower mandible as it is called) they found something else that was very interesting about Tianyuan man:


Title: Stable isotope dietary analysis of the Tianyuan 1 early modern human.
Authors: Hu Y, Shang H, Tong H, Nehlich O, Liu W, Zhao C, Yu J, Wang C, Trinkaus E, Richards MP.
Journal: Proc Natl Acad Sci U S A. 2009 Jul 7;106(27):10971-4.
PMID: 19581579                     (This research article is OPEN ACCESS if you would like to read it)

In this study, the investigators analysed the carbon and nitrogen isotopes found within bone collagen samples taken from the jaw bone of Tianyuan man. In humans, the carbon and nitrogen isotope values indicate the sources of dietary protein over many years of life.

The researchers found that a substantial portion of Tianyuan man’s diet 40,000 years ago came from freshwater fish.

Interesting preamble, but what does this have to do with Parkinson’s?

Continue reading “I’ll have the fish please” →

BIIB054: An immunotherapy update

~ Simon ~ 13 Comments

Immunotherapy is an experimental treatment that is being tested in Parkinson’s in the hope that it will be able to slow down the progression of the condition.

This week the Pharmaceutical company Biogen provided an update regarding their immunotherapy program for Parkinson’s.

It involves a drug called BIIB054.

In today’s post we will look at what BIIB054 is, how it works, and review the results of Biogen’s first clinical trial with this treatment.

This week the 2018 American Academy of Neurology ANN Annual Meeting is being held in Los Angeles (California). The meeting is an opportunity each year for researchers to meet and share new discoveries. A lot of neuroscience-focused biotech companies use the meeting to release new clinical trial results.

And this year one result in particular has been rather encouraging.

At 3:30pm on 24th April, the pharmaceutical company Biogen made a presentation entitled “Randomized, Double-Blind, Placebo-Controlled, Single Ascending Dose Study of AntiAlpha-Synuclein Antibody BIIB054 in Patients with Parkinson’s Disease,” which provided some of the first insights into the companies immunotherapy program for Parkinson’s.

What is immunotherapy?

Continue reading “BIIB054: An immunotherapy update” →

Monthly Research Review – March 2018

~ Simon ~ 9 Comments

At the end of each month the SoPD writes a post which provides an overview of some of the major pieces of Parkinson’s-related research that were made available during March 2018.

The post is divided into four parts based on the type of research (Basic biology, disease mechanism, clinical research, and other news). 

So, what happened during March 2018?

In world news:

March 25th – Qantas launches direct non-stop Boeing 787 Dreamliner flights between Perth Airport and Heathrow Airport, making it the first commercially non-stop service between Australia and the United Kingdom (17 hours on a plane – strewth!).

Boeing 787 Dreamliner. Source: Deredactie

March 14th – Prof Stephen Hawking, English theoretical physicist and cosmologist, sadly passed away at age 76. Diagnosed with in a very rare early-onset, slow-progressing form of Amyotrophic lateral sclerosis (ALS; also known as motor neurone disease or MND) in 1963 at age 21, he was gradually left him wheel chair bound. An amazing mind and a sad loss.

p03dn27d

Prof Stephen Hawking. Source: BBC

A funeral for Prof Hawking was held in Cambridge. The bell at Great St. Mary’s tolled 76 times at the start of the service. His remains will be cremated and his ashes will be interred at London’s Westminster Abbey near the remains of Isaac Newton.

King’s Parade in Cambridge was absolutely packed with mourners. Source: News.rthk

March 19th – In other sad news, ‘Sudan’, the world’s last male northern white rhinoceros died in Kenya, making the subspecies ‘functionally extinct’. Poachers had reduced the population from 2000 in the 1960s to just 15 1980s, and efforts to keep the species alive .

Sudan, the last surviving male northern white rhino. Source: PBS

March 24 – In over 800 cities internationally, people participated in student-led demonstrations against gun violence and mass shootings, calling for stronger gun control in the ‘March for our lives‘.

Source: Marchforourlives

And finally, on the 17th March, a driver in Milton Keynes (UK) got into big trouble with the law when he was pulled over and presented a police officer with an obvious fake drivers license (Source: Sky News):

Fake news Mr Trump? Clearly a fake. Everyone knows Homer lives at 742 Evergreen Terrace!

In the world of Parkinson’s research, a great deal of new research and news was reported:

Continue reading “Monthly Research Review – March 2018” →

Objective measures: Getting smart about pills

~ Simon ~ 4 Comments

There has been a lot of discussion on this site (and elsewhere on the web) regarding the need for more objective systems of measuring Parkinson’s – particularly in the setting of clinical trials.

Yes, subjective reports of patient experience are important, but they can easily be biased by ‘placebo responses’.

Thus, measures that are beyond the clinical trial participants conscious control – and focused on biological outcomes – are needed. 

In today’s post, we will consider one possible approach: Smart pills. We will discuss what they are, how they work, and how they could be applied to Parkinson’s research.

Source: Chicagotribune

In order to encourage a growing discussion regarding objective measures of Parkinson’s (and to follow up on previous rants – Click here and here for examples), I have decided to regularly (once a month) highlight new technologies that could provide the sort of unbiased methods of data collection that are required for assessing whether a treatment is having an impact on Parkinson’s.

Today, we will look at smart pills.

What is a smart pill?

Continue reading “Objective measures: Getting smart about pills” →

The dilemma of success

~ Simon ~ 17 Comments

Late last year, I wrote a post for Parkinson’s UK‘s excellent blog on Medium.

My piece was called the Dilemma of Success, and it explored a hypothetical situation that we may very well face in the not-so-distant future.

Optimistic as I am about the future of Parkinson’s research, I think this is a very serious issue – one which the Parkinson’s community needs to discuss and start planning for. I am re-posting it here today as I am keen for some thoughts/discussion on this matter.

 
BP

Lord Robert Baden-Powell. Source: Utahscouts

My scout master looked around the horse shoe, making eye contact with each of us, before asking a simple question:

“When did Noah build the ark?”

My fellow scouts and I looked at each other. Some of us were wondering if the guy had completely lost the plot and somehow thought that it was Sunday morning and he was doing the sermon. Others seriously looked like they were trying to calculate an exact date.

He waited a moment for one of us to offer up some idiotic attempt at an answer, before he solemnly said:

“Before the rain”

It’s one of those childhood moments that didn’t make sense at the time, but comes back to haunt you whenever you can foresee certain troubles coming over the hill towards you.

The dilemma of success

It will be nice to have this problem, but it will still be a problem.
And we need to plan for it

Continue reading “The dilemma of success” →

Monthly Research Review – February 2018

~ Simon ~ 13 Comments

At the end of each month, the Science of Parkinson’s writes a post which provides an overview of some of the major pieces of Parkinson’s-related research that were made available.

In this post we review some of the research from February 2018.

The post is divided into four parts based on the type of research (Basic biology, disease mechanism, clinical research, and other news). 

Seeing shadows: Punxsutawney Phil. Source: Wordonfire

In major world event news: On the 2nd February of 2018, Punxsutawney Phil – the groundhog who resides at Gobbler’s Knob of Punxsutawney, Pennsylvania – scurried out of his little hole and saw his shadow. This omen indicates that we have a long winter. Given how hard and bitter this particular winter has been, Americans naturally rejoiced.

On the 6th February, SpaceX successfully launched a Tesla sports car into space – see the video below for the highlights (and if you don’t have time to watch it all, at the very least jump forward to 3:45 and watch the two boosters land simultaneously – surely they didn’t plan for it to be that perfect!)

In other news, on the 1st February, the Centers for Disease Control and Prevention announced that it was dramatically downsizing its epidemic prevention activities in 39 out of 49 countries, due to concerns about funding.

And of course we had the 2018 Winter Olympics – where New Zealand came in 27th on the medals board:

Source: Madison365

In the world of Parkinson’s research, a great deal of new research and news was reported.

In February 2018, there were 698 research articles added to the Pubmed website with the tag word “Parkinson’s” attached (1577 for all of 2018 so far). In addition, there was a wave to news reports regarding various other bits of Parkinson’s research activity (clinical trials, etc).

The top 5 pieces of PD news

Continue reading “Monthly Research Review – February 2018” →

Reduce your RAGE as you AGE

~ Simon ~ 7 Comments

An Advanced Glycation Endproduct (or AGE) is a protein or lipid that has become glycated.

Glycation is a haphazard process that impairs the normal functioning of molecules. It occurs as a result of exposure to high amounts of sugar. These AGEs are present at above average levels in people with diabetes and various ageing-related disorders, including neurodegenerative conditions. AGEs have been shown to trigger signalling pathways within cells that are associated with both oxidative stress and inflammation, but also cell death.

RAGE (or receptor of AGEs) is a molecule in a cell membrane that becomes activated when it interacts with various AGEs. And this interaction mediates AGE-associated toxicity issues. Recently researchers found that that neurons carrying the Parkinson’s associated LRRK2 G2019S genetic variant are more sensitive to AGEs than neurons without the genetic variant. 

In today’s post we will look at what AGE and RAGE are, review the new LRRK2 research, and discuss how blocking RAGE could represent a future therapeutic approach for treating Parkinson’s.

The wonder of ageing. Source: Club-cleo

NOTE: Be warned, the reading of this post may get a bit confusing. We are going to be discussing ageing (as in the body getting old) as well as AGEing (the haphazard process processing of glycation). For better clarification, lower caps ‘age’ will refer to getting old, while capitalised ‘AGE’ will deal with that glycation process. I hope this helps.

Ageing means different things to different people.

For some people ageing means more years to add to your life and less activity. For others it means more medication and less hair. More wrinkles and less independence; more arthritis and less dignity; More candles, and less respect from that unruly younger generation; More… what’s that word I’m thinking of? (forgetfulness)… and what were we actually talking about?

Wisdom is supposed to come with age, but as the comedian/entertainer George Carlin once said “Age is a hell of a price to pay for wisdom”. I have to say though, that if I had ever met Mr Carlin, I would have suggested to him that I’m feeling rather ripped off!

George Carlin. Source: Thethornycroftdiatribe

Whether we like it or not, from the moment you are born, ageing is an inevitable part of our life. But this has not stopped some adventurous scientific souls from trying to understand the process, and even try to alter it in an attempt to help humans live longer.

Regardless of whether you agree with the idea of humans living longer than their specified use-by-date, some of this ageing-related research could have tremendous benefits for neurodegenerative conditions, like Parkinson’s.

What do we know about the biology of ageing?

Continue reading “Reduce your RAGE as you AGE” →

2017 – Year in Review: A good vintage

~ Simon ~ 13 Comments

At the end of each year, it is a useful practise to review the triumphs (and failures) of the past 12 months. It is an exercise of putting everything into perspective. 

2017 has been an incredible year for Parkinson’s research.

And while I appreciate that statements like that will not bring much comfort to those living with the condition, it is still important to consider and appreciate what has been achieved over the last 12 months.

In this post, we will try to provide a summary of the Parkinson’s-related research that has taken place in 2017 (Be warned: this is a VERY long post!)

The number of research reports and clinical trial studies per year since 1817

As everyone in the Parkinson’s community is aware, in 2017 we were observing the 200th anniversary of the first description of the condition by James Parkinson (1817). But what a lot of people fail to appreciate is how little research was actually done on the condition during the first 180 years of that period.

The graphs above highlight the number of Parkinson’s-related research reports published (top graph) and the number of clinical study reports published (bottom graph) during each of the last 200 years (according to the online research search engine Pubmed – as determined by searching for the term “Parkinson’s“).

PLEASE NOTE, however, that of the approximately 97,000 “Parkinson’s“-related research reports published during the last 200 years, just under 74,000 of them have been published in the last 20 years.

That means that 3/4 of all the published research on Parkinson’s has been conducted in just the last 2 decades.

And a huge chunk of that (almost 10% – 7321 publications) has been done in 2017 only.

So what happened in 2017? Continue reading “2017 – Year in Review: A good vintage” →

Inhibiting LRRK2: The Denali Phase I results

~ Simon ~ 7 Comments

Denali

This week Denali Therapeutics released the results of a phase I clinical trial of their primary product, called DNL-201.

DNL-201 is a LRRK2 inhibitor that the company is attempting to take to the clinic for Parkinson’s disease. 

In today’s post we will look at what LRRK2 is, how an inhibitor might help in Parkinson’s, and what the results of the trial actually mean.

Wonder_Lake_and_Denali

Denali. Source: Wikipedia

Denali (Koyukon for “the high one”; also known as Mount McKinley) in Alaska is the highest mountain peak in North America, with a summit elevation of 20,310 feet (6,190 m) above sea level. The first verified ascent to Denali’s summit occurred on June 7, 1913, by four climbers Hudson Stuck, Harry Karstens, Walter Harper, and Robert Tatum.

Tatum (left), Karstens (middle), and Harper (right). Source: Gutenberg

Robert Tatum later commented, “The view from the top of Mount McKinley is like looking out the windows of Heaven!”

More recently another adventurous group associated with ‘Denali’ have been trying to scale lofty heights, but of a completely different sort from the mountaineering kind.

Continue reading “Inhibiting LRRK2: The Denali Phase I results” →