Tag: trial

The 2020 Linked Clinical Trials meeting

~ Simon ~ 11 Comments

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Each year in September, The Cure Parkinson’s Trust and Van Andel Institute hold the international Linked Clinical Trials (iLCT) meeting.

This is a drug-repurposing initiative focused on disease modification in Parkinson’s. For two days the iLCT committee discuss and debate the virtues of 20+ molecules to decide which should be prioritised for clinical evaluation. 

Due to the current COVID-19 situation, the 2020 iLCT meeting was held virtually.

In today’s post, we will discuss what the iLCT program is and provide an overview of what happened at the 2020 meeting.

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The top line results of the PD-STAT clinical trial evaluating the cholesterol-reducing drug simvastatin in Parkinson’s were recently announced (Click here to read more about this). Preclinical data had suggested that this agent displayed neuroprotective properties in models of Parkinson’s, and given its long history of clinical use and agreeable safety profile, simvastatin seemed like an ideal candidate for repurposing to Parkinson’s.

A large Phase II clinical trial was set up and conducted across nation-wide network of 23 hospitals in the UK. It recruited over 230 brave individuals to be treated with the drug for 2 years and undergo regular clinical assessments.

The results of the study found that the treatment has had no impact on slowing the progression of Parkinson’s (Click here to read more about this).

That’s disappointing. What happens next?

Disappointing as the result is, the findings of the study provide us with a definitive answer, allowing us to move forward with testing other drugs of interest.

Simvastatin was a drug that was prioritised by the international Linked Clinical Trials programme, and while this agent might not have shown any beneficial efforts in Parkinson’s the good news is that there are lots of other drugs that have been prioritised by the international Linked Clinical Trials programme and they are now being clinically tested.

What is the international Linked Clinical Trials programme?

Continue reading “The 2020 Linked Clinical Trials meeting” →

Monthly Research Review – September 2020

~ Simon ~ 2 Comments

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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 September 2020.

The post is divided into seven parts based on the type of research:

  • Basic biology
  • Disease mechanism
  • Clinical research
  • New clinical trials
  • Clinical trial news
  • Other news
  • Upcoming conferences/lectures
  • Review articles/videos

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So, what happened during September 2020?

In world news:

September 2nd – Researchers found a way of floating toy boats under a levitating liquid (Click here to read more about this and click here to read a press summary)

September 3rd – The skeletons of 200 mammoths and 30 other animals are unearthed at a construction site for the Mexico City Santa Lucía Airport. It is the largest find of mammoth bones to date, surpassing the Mammoth Site in the U.S. which had 61 skeletons (Click here to read more about this).

September 14th – The Royal Astronomical Society announces the detection of phosphine in Venus’ atmosphere, which is suggested to be a strong predictor for the presence of microbial life (Click here to read more about this).

September 19th – A 1634 edition of The Two Noble Kinsmen, the last play by English playwright William Shakespeare, is discovered at the Royal Scots College’s library in Salamanca, Spain. It is believed to be one of the oldest copies of any of his works.

September 25th – Researchers report the development of rectangular, magnetically controlled robots (300 micrometers long) that can coax nerve cells to grow new connections (Click here to read more about this and click here to read a press summary)

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

In September 2020, there were 1,021 research articles added to the Pubmed website with the tag word “Parkinson’s” attached (7,969 for all of 2020 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 Parkinson’s news

Continue reading “Monthly Research Review – September 2020” →

Problems with PARKIN in PARIS

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PARKIN is a protein that is associated with a young-onset form of Parkinson’s. Individuals carrying tiny genetic variants in the region of DNA producing this protein have a higher risk of developing Parkinson’s before the age of 40 than non-carriers.

The PARKIN protein is believed to play an important role in the disposal of old/damaged mitochondria (the power stations of cells). But recent research points towards another protein – that interacts with PARKIN – which may also be implicated in the health and well being of mitochondria.

That other protein is called PARIS.

In today’s post, we will discuss what PARKIN does, explore how PARIS could be involved, and reflect on what this could mean for future therapies targeting PARKIN-associated Parkinson’s.

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paris

No label required. A magnificent city. Source: HathawaysofHaworth

Paris is not my favourite city (Hanoi takes that spot), but it is probably in the top 10.

Like London and New York, La Ville Lumière is an incredible place to be fortunate enough to visit.

If you ever find yourself in Paris, bored of all the art, culture, food, etc, and you feel like something more scientific, make your way up the Seine river to the 5th arrondissement, and try to find the Muséum national d’histoire naturelle. Once you get there, ask for the “Galerie de Paléontologie et d’Anatomie comparée” (Paleontology and comparative Anatomy Gallery):

This is the realm of Georges Cuvier – the French paleontologist who researched fossils and in 1796 laid out the first ideas for extinction theory. It is a hall of scientific wonder.

As I say, it is worth a visit if ever you are bored in Paris. But be warned that parking is an issue at the Jardin des plantes where the gallery is located.

In fact, parking is an issue everywhere in Paris.

It seems like Parking and Paris do not mix.

I’m sorry, but what does this have to do with Parkinson’s?

Continue reading “Problems with PARKIN in PARIS” →

$161 million over three years

~ Simon ~ 6 Comments

 

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The Aligning Science Across Parkinson’s (or ASAP) initiative is a major new source of funding for Parkinson’s research. And I mean MAJOR!

It is a global basic research initiative focused on fostering collaboration and resources to better understand the underlying causes of Parkinson’s. A return to basics in order to get a better grip on the biology of the disease.

Recently, the initiative announced their first round of grant awardees – handing out US$161 million for 3 year projects. This is one of the largest single rounds of research funding for Parkinson’s research ever!

In today’s post, we will look at what ASAP is, what the awarded projects will be investigating, and what this means for Parkinson’s research.

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NIH Parkinson’s research funding. Source: NIH

In 2016, the US National Institutes of Health (NIH – the world’s largest funder of medical research) allocated $161 million to Parkinson’s research.

It was a small fraction of the $30+ billion spent by the NIH on medical research that year, but it was still a much needed amount of money invested into research on this neurodegenerative condition.

This week, a major new Parkinson’s research program – called Aligning Science Across Parkinson’s (or ASAP – click here to read a previous SoPD post on this initiative) – announced the rewarding of $161 million in research funding to 21 projects involving 96 research leaders from 60 institutions across 11 countries (and 31 of the research leaders are female). Importantly, all of them are seeking to “accelerate targeted basic research and move us toward more meaningful advancements for Parkinson’s” (Click here to read the annoucement).

Think about that for a second:

ASAP has basically just allocated the same amount of funding to Parkinson’s research as the entire US Government did in 2016.

Wow!

Continue reading “$161 million over three years” →

The PASADENA study announcement (part 2)

~ Simon ~ 6 Comments

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In April of this year it was announced that the closely watched Phase II PASADENA clinical trial had not to met its primary objective. This was a large clinical evaluation of an immunotherapy approach (called prasinezumab) for disease modification in Parkinson’s. 

At the time of the announcement, it was indicated that the researchers who conducted the study had seen “signals of efficacy” in the data.

This week the results of the study were presented at an international conference and it was reported that prasinezumab “significantly reduced decline in motor function by 35% (pooled dose levels) vs. placebo after one year of treatment“.

In today’s post, we will discuss what the PASADENA study was, review the results that have been released, and discuss what might happen next.

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At 7am (just prior to the opening of the Swiss Stock Exchange) on Wednesday 22nd April 2020, the pharmaceutical company Roche published its sales results for the 1st Quarter. The financial report looked good, particularly considering the current COVID-19 economic climate, but there was one sentence on page 133 of the results (highlighted below) that grabbed a lot of attention:

From page 133. Source: Roche

For those of you (like myself) who struggle with fine print, the sentence reads:

“Study did not meet its primary objective, but showed signals of efficacy“

This was how the Parkinson’s community found out about the top line result of the closely followed Phase II PASADENA study evaluating the immunotherapy treatment prasinezumab in individuals recently diagnosed with Parkinson’s.

Many within the Parkinson’s community were basically:

Yet another negative clinical trial result.

But then, later that same day, the biotech firm Prothena – which developed prasinezumab and is partnered with Roche in the clinical testing – kindly provided a press release.

And in that document, the company repeated that prasinezumab “showed signals of efficacy” , but importantly: “These signals were observed on multiple prespecified secondary and exploratory clinical endpoints“.

And then the Parkinson’s community was like:

This week we found out more about those “signals of efficacy” and the results of the PASADENA study, and they look interesting.

What do the results show?

Continue reading “The PASADENA study announcement (part 2)” →

CPTX: Gluing the brain back together

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Current clinical efforts at restorative medicine for neurodegeneration are still largely focused on stem cell and neurotrophic factor-based methods. Novel techniques are being preclinically proposed however, and some of them employ some radically different approaches.

An international group of researchers have recently published a report describing a means of repairing the damaged central nervous system that involves ‘gluing’ neurons together via an artificial protein.

They called this new method CPTX.

In today’s post, we will explore what this artificial protein does, what was reported in the new study, and consider how this could potentially be used for Parkinson’s.

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Source: Howtogeek

Earlier in the year I wrote a post called the 2020 wish list, where I discussed some hopes for Parkinson’s research this year. Despite everything that 2020 (annus horribilis) has thrown at us, there have been significant developments regarding Parkinson’s research and some of those wishes.

One of those hopes was the announcement of new and innovative methods for restorative techniques for Parkinson’s. At present, all of the restorative approaches in clinical trial for Parkinson’s are focused on stem cell transplantation (Click here to read a recent SoPD post describing an example of this), and it would be good to broaden the range of approaches being tested.

As a result of this particular wish, a theme here on the SoPD this year has been to write posts highlighting new restorative research as it has been published (Click here, here and here to read some examples).

In today’s post, we are going to continue that theme with an extremely radical bit of research that utterly boggled my mind.

Me after reading this report. Source: 1zoom

Be warned, this is very futuristic, blue sky, “way out there on the horizon”-kind of stuff.

But when I read this report in August, I was left stunned… and rather excited by the potential possibilities.

Sounds interesting, what was the research report about?

Continue reading “CPTX: Gluing the brain back together” →

A tête-à-tête about TET2

~ Simon ~ 3 Comments

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Epigenetics is the study of how non-genetic factors affect genetics. It explores the influence of lifestyle or environmental factors on the activity surrounding our DNA.

Recently researchers have been investigating epigenetics within the context of Parkinson’s, and they have discovered a “master switch” that could represent an important target for future therapeutic treatments for the condition.

The research focuses on a protein called TET2.

In today’s post, we will discuss what epigenetics means, review the new research, and consider what the implications of these new findings could be.

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Not the same. Source: AutismBlog.

Identifical twin studies have been extremely useful to our understanding of conditions like Parkinson’s (Click here to read a previous SoPD post on twins research in Parkinson’s)

But the funny thing about identical twins is that in 100% of cases they aren’t identical.

Yeah sure, they initially share exactly the same DNA, but the further they get away from that magically moment of conception, the less alike they are.

Same DNA, subtle differences. Source: National Geographic

And we’re not talking about personality or likes/dislikes here.

As biological organisms twins diverge significantly after conception and as the age through life. But if they share the same DNA – the same genetic blueprint – how are these difference possible?

The answer to this question may lie in epigenetics.

What is epigenetics?

Continue reading “A tête-à-tête about TET2” →

Monthy research review – August 2020

~ Simon ~ 1 Comment

 

 

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 August 2020.

The post is divided into seven parts based on the type of research:

  • Basic biology
  • Disease mechanism
  • Clinical research
  • New clinical trials (Oooh, new section for 2019!)
  • Clinical trial news
  • Other news
  • Review articles/videos

 

So, what happened during August 2020?

In world news:

August 4th – Two explosions caused by unsafely stored ammonium nitrate killed over 220 people, injure thousands, and severely damage the port in Beirut, Lebanon.

August 16th – In Death Valley (California), the appropriately named Furnace Creek (population of 24) logged a day time high temperature of 130° Fahrenheit (54.4° Celsius).

Augst 20th – The US FDA holds on Emergency Use Authorization for convalescent plasma the treatment of COVID–19, with health regulators stating the “importance of robust data through randomized control trials” and “that a pandemic does not change that”. Apparently the emerging data on the treatment was “too weak”

August 23rd – The US FDA issued “Emergency Use Authorization for convalescent plasma as potential promising COVID–19 treatment, Another Achievement in Administration’s Fight Against Pandemic” (Source). What a difference 3 days and no data makes….

August 24th – A 33 year old man in Hong Kong became the first confirmed case of coronavirus reinfection (additional cases have been confirmed). Humans appear to have a short immunity to COVID-19 (2-3 months, similar to influenza).

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

In August 2020, there were 890 research articles added to the Pubmed website with the tag word “Parkinson’s” attached (6,948 for all of 2020 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 6 pieces of Parkinson’s news

Continue reading “Monthy research review – August 2020” →

Can sigma-1 get it done?

~ Simon ~ 6 Comments

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An Australian charity seeking to find disease modifying therapies for Parkinson’s – The Shake It Up Australia Foundation – has announced a commitment to partially fund a clinical trial with a biotech firm called Anavex Life Sciences.

The trial will focus on a drug called ANAVEX2-73 (also known as ‘blarcamesine’). This experimental treatment is a Sigma-1 receptor agonist

In today’s post, we will discuss what the Sigma-1 receptor does, we will review some of the Parkinson’s research that has been conducted on this protein, and we will discuss what we know about the potential trial.

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A man on a mission. Source: Goulburnpost

The gentleman in the photo above is named Clyde Campbell.

True blue Aussie, innovative robotics engineer, keen sportsman, and all round nice guy.

Clyde also has Parkinson’s, which was diagnosed in 2009 after he noticed a tremor in his left hand while holding notes at a business meeting. After dealing with the initial shock of this life changing event, he turned his attention to doing something about it.

Clyde simply could not sit still and not do anything about his situation, so he grabbed the bull by the horns and decided to shake things up.In 2011, Clyde (and his brother Greg) founded a charity called the Shake It Up Australia Foundation:

Since its founding, the foundation has supported 38 Parkinson’s research projects across 12 institutes, with over AUS$11.5 million in funding. In addition, the organisation is a key partner in the Australian Parkinson’s Mission (Click here to read a previous SoPD post on this project).

To learn more about Clyde, watch this video where he shares his story:

This week the Shake It Up Australia Foundation made a big announcement.

What did they say?

Continue reading “Can sigma-1 get it done?” →

The ibuprofen post

~ Simon ~ 2 Comments

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Ibuprofen is a nonsteroidal anti-inflammatory drug that is used for treating pain, fever, and inflammation.

Previous preclinical research has demonstrated that ibruproen has the ability to reduce the loss of neurons in models of Parkinson’s, and epidemiological data suggests that it may lower the risk of actually developing the condition.

Recently published research points towards a specific sub-set of individuals vulnerable to Parkinson’s that ibuprofen may be particularly useful for: LRRK2-genetic variant carriers.

In today’s post, we will discuss the origins of ibruprofen, review some of the previous research indicating neuroprotective properties, and do a deep dive into the new LRRK2 data.

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Source: BBC

This story starts in 1953.

And it begins with two chemists – Stewart Adams and John Nicholson (Stewart is the chap in the banner photo at the top of this post holding the container of pills) – who were both working for Boots, a health/beauty retailer and pharmacy chain in the UK.

Source: Boots

Stewart and John were on a mission: To produce a new drug for rheumatoid arthritis.

You see, in 1953 there were only two drugs available for treating inflammatory pain: a corticosteroid drug and high dose aspirin. And neither of them was ideal. The chemists started their quest by looking for the activity of variations of aspirin, hoping to find a powerful alternative.

Adams (left) & Nicholson (middle). Source: Boots

Their search was not easy – it took 16 years and they screened over 20,000 molecules – but output of that effort was a drug called ibuprofen (sold under ‘Brufen’).

Legend has it that Adams initially tested the drug on himself as treatment for a particularly bad hangover (Source).

Ibuprofen was launched on the 3rd February 1969 as a treatment for rheumatoid arthritis in the United Kingdom, and it was introduced in the United States in 1974. It went on to become one of the most prescribed drugs in history and it is still widely used. In fact in 2015, Boots UK sold an average of one pack of ibuprofen every 2.92 seconds and, across all UK retailers, the sales figures for the medicine reached over £150 million.

This is all very interesting, but what does it have to do with Parkinson’s?

Continue reading “The ibuprofen post” →