$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!

Set up by the Brin family foundation, ASAP 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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Google co-founder Sergey Brin. Source: Emaze

Two years ago, key Parkinson’s researchers and advocates from around the world were invited to the Milken Institute Center in for a large meeting that was organised to map out the foundations of a major new Parkinson’s research program that was to be called Aligning Science Across Parkinson’s (or ASAP – click here to read a previous SoPD post on this initiative).

The event was organised by Google co-founder Sergey Brin and his family foundation.

The Brin family has been affected by Parkinson’s – Sergey’s mother and aunt have both been  diagnosed with the condition, and Sergey has a genetic risk factor that increases his risk of developing Parkinson’s.

The Brin Family – Sergey and his mother on the right. Source: CS

Sergey and his mother both carry a genetic variation (G2019S) in a region of DNA called PARK8. It is also known as Leucine-rich repeat kinase 2 (or simply LRRK2 – pronounced ‘lark 2’). The variant increases the risk of developing a young-onset, slow progressing form of Parkinson’s (Click here to read a previous SoPD post on LRRK2).

Sergey may never actually develop the condition, but he has decided not to take any chances. He has taken out an “insurance policy” by investing hundreds of millions of dollars into Parkinson’s research.

Part of that insurance policy is ASAP.

And this week, the organisation made a major announcement.

What did they announce?

Continue reading “$161 million over three years” →

The PASADENA study announcement (part 2)

~ Simon ~ 2 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 ~ 5 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” →

Billion dollar bets: Denali+Biogen

~ Simon ~ 2 Comments

 

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This week the biotech firm Denali Therapeutics made two major announcements regarding the development of their LRRK2 inhibitor program for Parkinson’s.

First, the company revealed that they have signed an agreement with the pharmaceutical company Biogen to co-develop and co-commercialise small molecule inhibitors of LRRK2 for Parkinson’s.

Second, Denali also announced that they have a green light from the US FDA to start the next phase of clinical testing of their LRRK2 inhibitor DNL151.

In today’s post, we will discuss what is meant by LRRK2 inhibitor, what the details of the announcements are, and what all of this means for the Parkinson’s community.

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Denali. Source: Wikipedia

Peaking at 20,310 feet (or 6,190 m) above sea level, Denali (Koyukon for “the high one”; also known as Mount McKinley) is the highest mountain in North America. The first verified ascent of this Alaskan mountain occurred on June 7, 1913, when four climbers (Hudson Stuck, Harry Karstens, Walter Harper, and Robert Tatum) conquered it.

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 to the mountaineering kind.

Founded in 2013 by a group of former Genentech executives, San Francisco-based Denali Therapeutics is a biotech company which is focused on developing novel therapies for people suffering from neurodegenerative diseases. Although they have product development programs for other condition (such as Amyotrophic Lateral Sclerosis and Alzheimer’s disease), Parkinson’s is definitely their primary indication of interest.

And this week, the company made two major announcements with regards to their Parkinson’s research program.

The first announcement was that Denali have signed an agreement with the pharmaceutical company Biogen to co-develop and co-commercialise small molecule inhibitors of LRRK2 for Parkinson’s (Click here to read the press release).

What is LRRK2?

Continue reading “Billion dollar bets: Denali+Biogen” →

Monthy research review – July 2020

~ Simon ~ 4 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 July 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
  • Review articles/videos

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What happened in July 2020?

In world news:

July 1st – With the introduction of the new national security law, academics in Hong Kong expressed concern about being able to recruit talented researchers and to import critical high tech equipment (due to international sanctions – click here to read more about this).

July 16th – Six year old Bridger Walker gained social media fame after saving his sister from being attacked by a charging dog. Bridger was bitten several times on the head and face, requiring 90 stitches. Celebrities praised his brave actions:

 

July 17th – Capt Sir Tom Moore was knighted – what a legend! (Click here to read more about this).

July 25th – A record high temperature of 21.7°C (71.1°F) is recorded on the Arctic archipelago of Svalbard, according to the Norwegian Meteorological Institute.

July 31st – Researchers reported that human sperm do not swim the way we thought they did (rather rolling and lopsided tail flicks keep the cells moving in a straight line – click here to read more about this).

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

In July 2020, there were 753 research articles added to the Pubmed website with the tag word “Parkinson’s” attached (6058 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 “Monthy research review – July 2020” →

Smarter than the average björn

~ Simon ~ 3 Comments

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Certain life-style and environmental factors may render individuals more vulnerable to Parkinson’s, and a better understanding of them could aid in effects to reduce the incidence of the condition.

Some of these risk factors (such as smoking and drinking coffee – PLEASE DO NOT START SMOKING!) can reduce the risk of developing Parkinson’s, while others such as exposure to pesticides can increase the chances of PD.

Recently, researcher in Sweden have made a rather curious association: High IQ is associated with Parkinson’s

In today’s post, we will discuss the results of this new study and we will speculate a little with regards to what they could mean for Parkinson’s.

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Lund. Source: Copenhagen-travel

When I was younger and less beautiful, I spent half a decade of my life enjoying the charms of a small Swedish city called Lund.

I was there to do a PhD (the chance of a life time for a simple country boy from the back-waters of third world New Zealand), but I absolutely fell in love with the place (a quiet, cobble-stoned, academic town) and the local customs.

Source: Beaps

Life in Sweden was really good. They had a fantastic quality of life, but more importantly they encouraged a very strong sense of social consciousness, inclusion, and justice.

But there was one aspect of ‘svenska livet’ that surprised me.

Despite a strong military history, Sweden chose to adopt a policy of neutrality in 1815 and it has not taken part in any armed warfare since that time (with the exception of peacekeeping). And yet, the country has maintained mandatory military service (in Swedish: värnplikt) for men since 1901 (the practice was paused between 2010 and 2017, but has subsequently been restarted on a reduced level).

Interesting, but what does mandatory military service have to do with Parkinson’s?

Continue reading “Smarter than the average björn” →