Tagged: immunotherapy

The hunt for a vaccine

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This week, the biotech firm AFFiRiS published the long awaited results of their Phase 1 clinical trial evaluating a vaccine for Parkinson’s. The vaccine – called PD01A – targets a protein that clumps/aggregates together in certain neurons in the brains of people with Parkinson’s.

The multi-year study suggests that the treatment is safe and tolerated. In addition, it causes the immune system to generate antibodies that target the aggregated form of alpha synuclein.

And while it must be remembered that this is a small, open-label study, there are some intriguing statements made in the report.

In today’s post, we will discuss what PD01A is, review the results of the clinical study, and explore what happens next.

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Source: NHI

As the world awaits the development of a vaccine that will combat COVID-19, the neurodegenerative research community has quietly been watching a biotech company in Austria that has been developing a vaccine of a different sort: A vaccine for Parkinson’s.

The company is called AFFiRiS:

Source: Twitter

And this week they published the results of their Phase 1 safety/tolerability clinical trial of their immunotherapy treatment (PD01A) that they are testing in people with recently diagnosed Parkinson’s.

What is immunotherapy?

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The Pasadena study announcement

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This week the outcome of an ongoing Parkinson’s clinical trial was announced.

Data collected during Part 1 of the ongoing Phase 2 PASADENA alpha synuclein immunotherapy study for Parkinson’s apparently suggests that the treatment – called prasinezumab – has not achieved it’s primary endpoint (the pre-determined measure of whether the agent has an effect in slowing Parkinson’s progression – in this case the UPDRS clinical rating scale).

But, intriguingly, the announcement did suggest ‘signals of efficacy‘ in secondary and exploratory measures.

In today’s post, we will discuss what immunotherapy is, what we know about the PASADENA study, and why no one should be over reacting to this announcement.

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At 7am on Wednesday, April 22nd, 2020, the pharmaceutical company Roche published its sales results for the 1st Quarter. This was just prior to the opening of the Swiss Stock Exchange. The financial report looked very good, particularly considering the current COVID-19 economic climate.

There was, however, one sentence on page 133 of the results that grabbed some attention:

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 pharmaceutical giant announced the top line result of the ongoing Phase II PASADENA study evaluating the immunotherapy treatment prasinezumab in recently diagnosed individuals with Parkinson’s (listed on the Clinicaltrials.gov as NCT03100149).

At the time of publishing this SoPD post, Roche are yet to provide any further information (press release, announcement, memo, tweet, etc) regarding the results of the study.

Thankfully, a smaller biotech firm called Prothena – which is also involved in the development of the agent being tested in the Pasadena study – has kindly provided a few more details regarding these results.

I usually don’t like discussing clinical trial results on the SoPD until the final report is published, but in this circumstance I will make an exception.

In today’s post we will discuss what details have been shared in the Prothena press release regarding the Prasinezumab clinical trial in Parkinson’s (Click here to read the press release).

What is Prasinezumab?

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That time APOE met Alpha Syn

  

Recently two independent research groups published scientific papers providing evidence that a genetic variation associated with Alzheimer’s may also be affecting the severity of pathology in Parkinson’s.

The genetic variation associated with Alzheimer’s occurs in a gene (a functional region of DNA) called ApoE, and the Parkinson’s pathology involves the clustering of a protein called alpha synuclein.

Specifically, both researchers reported that a genetic variation called ApoE4 is associated with higher levels of alpha synuclein clustering. And ApoE4 is also associated with worse cognitive issues in people carrying it.

In today’s post, we will discuss what ApoE is, what is known about ApoE4, what these new studies found, and what it could mean for the future treatment of Parkinson’s and associated conditions.

 


A mutant. Source: Screenrant

When I say the word ‘mutant’, what do you think of?

Perhaps your imagination drifts towards comic book superheroes or characters in movies who have acquired amazing new super powers resulting from their bodies being zapped with toxic gamma-rays or such like.

Alternatively, maybe you think of certain negative connotation associated with the word ‘mutant’. You might associate the word with terms like ‘weirdo’ or ‘oddity’, and think of the ‘freak show’ performers who used to be put on display at the travelling carnivals.

Circus freak show (photo bombing giraffe). Source: Bretlittlehales

In biology, however, the word ‘mutant’ means something utterly different.

What does ‘mutant’ mean in biology?

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Prothena: Phase I results published

This week, biotech firm Prothena published the results of their Phase I safety and tolerance clinical trial of their immunotherapy treatment called PRX002 (also known as RG7935).

Immunotherapy is a method of artificially boosting the body’s immune system to better fight a particular disease. 

PRX002 is a treatment that targets a toxic form of a protein called alpha synuclein – which is believed by many to be one of the main villains in Parkinson’s. 

In today’s post, we will discuss what immunotherapy is, review the results of the clinical trial, and consider what immunotherapy could mean for the Parkinson’s community.


Source: uib

I have previously mentioned on this website that any ‘cure for Parkinson’s’ is going to require three components:

  1. A disease halting mechanism
  2. A neuroprotective agent
  3. Some form of cell replacement therapy

This week we got some interesting clinical news regarding the one of these components: A disease halting mechanism.

The Phase I results of a clinical trial being conducted by a company called Prothena suggest that a new immunotherapy approach in people with Parkinson’s is both safe and well tolerated over long periods of time.

The good folks at Prothena Therapeutics. Source: Prothena

What is immunotherapy?

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A vaccine for Parkinson’s – the AFFiRiS update

This week Austrian biotech firm, AFFiRiS AG, made an announcement regarding their experimental immunotherapy/’vaccine’ approach for Parkinson’s.

In their press release, the company provided the results of a long-term Phase I clinical trial testing the tolerability and safety of their treatment AFFITOPE® PD01A.

The treatment was found to be safe and well-tolerated in people with Parkinson’s. But there was one sentence which was particularly intriguing in the press release regarding clinical symptoms.

In today’s post, we will discuss what is meant by ‘immunotherapy’, outline what this particular clinical trial involved, review the results, and explore what this could mean for the Parkinson’s community.


Source: uib

I have previously mentioned on this website that any ‘cure for Parkinson’s’ is going to require three components:

  1. A disease halting mechanism
  2. A neuroprotective agent
  3. Some form of cell replacement therapy

This week we got some interesting clinical news regarding the one of these components: A disease halting mechanism

Clinical trial results from Austria suggest that a new immunotherapy approach in people with Parkinson’s is both safe and well tolerated over long periods of time.

What is immunotherapy?

Continue reading

When SERCA goes berserker

In a recent SoPD post, we discussed the importance of calcium and looked at how it interacts with the Parkinson’s-associated protein alpha synuclein, affecting the function and clustering of that protein.

During the writing of that post, another interesting research report was published on the same topic of calcium and alpha synuclein. It involved a different aspect of biology in the cell – a structure called the endoplasmic reticulum – but the findings of that study could also explain some aspects of Parkinson’s.

In today’s post, we will review the new research report, consider the biology behind the findings and how it could relate to Parkinson’s, and discuss how this new information could be used.


The original berserker. Source: Wikipedia

I can remember my father often saying “If you kids don’t be quiet, I’ll go berserk!”

Growing up, I never questioned the meaning of the word ‘berserk‘.

I simply took it as defining the state of mindless madness that my dad could potentially enter if we – his off-spring – pushed him a wee bit too far (and for the record, Dad actually ‘going berserk’ was a very rare event).

My father. But only on the odd occasion. Source: Screenrant

But now as I find myself repeating these same words to my own off-spring, I am left wondering what on Earth it actually means?

What is ‘berserk‘?

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BIIB054: An immunotherapy update

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?

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The road ahead: Parkinson’s research in 2018

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.

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2017 – Year in Review: A good vintage

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

The Llama-nation of Parkinson’s disease

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The clustering of a protein called alpha synuclein is one of the cardinal features of the brain of a person with Parkinson’s disease.

Recently published research has demonstrated that tiny antibodies (called nanobodies) derived from llamas (yes, llamas) are very effective at reducing this clustering of alpha synuclein in cell culture models of Parkinson’s disease. 

In today’s post, we will discuss the science, review the research and consider what it could all mean for Parkinson’s disease.


other-spit-long-farm-llama-animals-alpacas-alpaca-neck-animal-soft-furry-llamas-happy-picture-water-1366x768

Llama. Source: Imagesanimals

Ok, I confess: This post has been partly written purely because I really like llamas. And I’m not ashamed to admit it either.

I mean, look at them! They are fantastic:

llamas-and-haircuts-prince-harry1

Source: Vogue

Very cute. But what does this have to do with Parkinson’s disease?

Indeed. Let’s get down to business.

This post has also been written because llamas have a very interesting biological characteristic that is now being exploited in many areas of medical research, including for Parkinson’s disease.

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