Tagged: trial

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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Is Radotinib ABL to beat Nilotinib?

 

At the Society for Neuroscience annual meeting in 2015, the results of a small phase I clinical trial were presented and the Parkinson’s community got really excited by what they saw.

The study had investigated the use of a cancer drug called ‘Nilotinib’ (also known as Tasigna) on Parkinson’s and the initial results were rather interesting.

Two larger phase II clinical trials of Nilotinib in Parkinson’s are currently being conducted, but this week preclinical research of a new drug (called Radotinib) was published. And these new findings suggest that Nilotinib may have some impressive competition.

In today’s post, we will look at what Nilotinib and Radotinib actually do, we will review the new research, and we will discuss what the findings could mean for the Parkinson’s community.


Lots of research. Source: Thedaily

Earlier this week I wrote a post highlighting research involving a new drug (NLY01; a GLP1 receptor agonist) being developed for Parkinson’s (Click here to read that post). It was an amazing amount of work and a very impressive achievement for the research group that conducted the work.

It must have taken a long time to perform the experiments, and I figured that the researchers behind the study would probably take a well earned break.

You will understand that I was a little surprised the day after publishing the post, that I woke up to find that that same research group had published another rather remarkable amount of research… on a completely different novel drug (called Radotinib) which is also being developed for Parkinson’s!!!

Basically sums my reaction. Source: Canacopegdl

The words ‘You have to be kidding me‘ actually passed across my lips as I downloaded the new research report.

And the new drug is really interesting.

It is very similar to Nilotinib.

What is Nilotinib?

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Blessed are the suppressed

Recently a study was published in which the researchers had used a large dataset from the the United States Medicare system. The dataset held medical prescriptions for beneficiaries aged 60–90 years.

In their analysis, the researchers found that several diagnoses commonly treated with immunosuppressant medications were inversely associated with Parkinson’s – suggesting that perhaps the immunosuppressants may be reducing the risk of developing PD.

When they looked closer at the immunosuppressants, the investigators found that of the six categories of immunosuppressants, two were clearly associated with a lower risk of PD.

In today’s post, we will discuss what immunosuppression means, we will review the data, and  we will consider some of the issues associated with immunosuppressants.


George Hitchings and Gertrude Elion. Source: Achievement

After her grandfather died of stomach cancer and her fiance died of inflammation of the heart, Gertrude Elion dedicated herself to a future in medical research.

But despite a passionate love for laboratory research and having an excellent academic record, she was unable to get a graduate fellowship (or even an assistantship) due to the gender discrimination that existed at the time.

In the late 1930s, she enrolled in secretarial school with the goal of saving enough money to continue her education and achieve her goal.

After a year and a half of temporary secretarial and teaching positions, having saved up enough money, Elion enrolled as Master’s student in chemistry at New York University. She worked part-time as a receptionist and later as a substitute teacher to pay for her expenses. And she spent nights and weekends in the laboratory doing her research. She completed her degree in 1941.

George & Gertrude in action. Source: Wikimedia

In 1944, Gertrude was hired by George Hitchings who was working at what is now the pharmaceutical company GlaxsoSmithKline. It was the beginning of an amazing collaboration! And even though she never achieved a PhD, the work that she and Hitchings did together – research that won them the 1988 Nobel Prize in Physiology or Medicine – not only changed the way we design new drugs, but also gave the world its first drugs for immunosuppression.

What is immunosuppression?

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What do you do with a problem like Exenatide?

 

 

At 23:30 on the 3rd August 2017, the results of a phase II clinical trial investigating the use of a Glucagon-like peptide-1 receptor (GLP-1R) agonist called Exenatide (Bydureon) in Parkinson’s were published the Lancet journal website.

The findings of the study were very interesting.

And after years of failed trials, the Parkinson’s community finally had a drug that appeared to be ‘doing something’. Naturally these results got many in the Parkinson’s community very excited.

Over the last couple of weeks, further research related to this topic has been published. In today’s post we will review some of this new research and ask some important questions regarding how to move forward with these results.


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In 2012, the Golden Goose Award was awarded to Dr John Eng, an endocrinologist from the Bronx VA Hospital.

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Dr John Eng. Source: Health.USnews

The Award was created in 2012 to celebrate researchers whose seemingly odd or obscure federally funded research turned out to have a significant and positive impact on society.

And despite the name, it is a very serious award – past Nobel prize winners (such as Roger TsienDavid H. Hubel, and Torsten N. Wiesel) are among the awardees.

This week a research report was published in the journal Nature Medicine that expanded on the work of Dr Eng (some 25 years after his big discovery).

And it could be very important to the Parkinson’s community.

Sounds intriguing. What did Dr Eng do?

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Don’t get mad! Get NAD!

Recently researchers have provided very interesting evidence that a form of vitamin B3, called Nicotinamide Riboside, may have beneficial effects for Parkinson’s.

Their data suggests that nicotinamide riboside was able to rescue problems in mitochondria – the power stations of cells – in both fly and human cell-based models of Parkinson’s.

And the results also suggest that this treatment could prevent the neurodegeneration of dopamine producing neurons.

In today’s post, we will discuss what nicotinamide riboside is, what is does in the body, how it may be having its beneficial effect, and we will consider the pros and cons of taking it as a supplement.


My pile of research reports to read. Source: Reddit

We have a serious problem in biomedical research at the moment.

Serious for ‘planet research’ that is (Good for ‘planet patient’! – click here to understand this sentence).

The problem is very simple: there is too much research going on, and there is now too much information to be absorbed. 

There has been an incredible increase in the number of research reports for ‘Parkinson’s’:

For Parkinson’s research alone, every day there is about 20 new research reports (approximately 120 per week). It used to be the case that there was one big research report per year. Then progress got to the crazy point of one big finding per month. And now things are ‘completely kray kray’ (as my 5 year old likes to say), with one new major finding every week!

On top of this, everyday there are new methodology reports, new breakthroughs in other fields that could relate to what is happening in PD, new clinical trial results, etc… The image below perfectly represents how many researchers are currently feeling with regards to the information flow:

How I feel most days. Source: Lean

Don’t get me wrong.

These are very exciting times, big steps are being made in our understanding of conditions like Parkinson’s. It’s just that it is really hard keeping up with the amazing flow of new data.

And this is certainly apparent here on the SoPD website. Occasionally, a few days after I publish something on a particular topic on the SoPD website, a fascinating new research report on that same topic will be published. When I get a chance to read it, I will sometimes add an addendum to the bottom of a post highlighting the new research.

Every now and then, however, the new research deserves a post all of its own.

Which is the case today.

A week after I published the recent Vitamin B3/Niacin post, a new study was published that dealt with a different form of Vitamin B3, called Nicotinamide Riboside. And the results of that study were really interesting.

Wait a minute. Vitamin B3 comes in different forms?

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Keep an eye on mild TBI

Last week one of the most comprehensive analyses assessing the risk of developing Parkinson’s following mild traumatic brain injury (or TBI) was published.

Using data collected from US military veterans, the study concluded that mild TBI was associated with a 56% increased risk of developing Parkinson’s.

In today’s post, we will review the study, discuss what TBI means, and consider what implications this study could have for the Parkinson’s community.


Source: Jamesriverarmory

At a recent research conference, a young PhD student was looking at me from across the room.

Finally, she walked across to me and asked:

“Excuse me. Are you the goat guy?”

I smiled.

It had been a while since anyone had asked me this.

“Yes,” I replied, “I am the goat guy”

Let me explain:

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The Mannitol results

Last week the first results of an ambitious project are being shared with the Parkinson’s community.

Clinicrowd is a “crowd sourcing platform exploring disease treatments that Pharma companies have no interest to investigate or promote”. Their initial focus was Parkinson’s (though they now have additional projects for other medical conditions), and their first experimental treatment for Parkinson’s was the sweetener ‘mannitol’.

The results provide some interesting insights into the properties of mannitol and into crowd sourced projects.

In today’s post, we will discuss what mannitol is, why it is interesting, outline the Clinicrowd project, and review the results of the mannitol study.


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Mannitol. Source: Qualifirst

Without a shadow of doubt, one of the most popular topics searched for on this website is ‘mannitol’.

In 2017, the second most visited page on the site (behind only the main/home page) was a post called “Update – Mannitol and Parkinson’s“. And as if to put an exclamation point on the matter, the fourth most visited page was “Manna from heaven? Mannitol and Parkinson’s

Understand though, that both of these posts were actually written in 2016!

Throughout 2017-18, not a week has gone by without someone contacting me to ask about mannitol and the ‘CliniCrowd‘ project.

Thus, it brings me great pleasure to sit down tonight and write this post.

What is mannitol?

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To B3 or not to B3, that is the question

The results of a recent clinical study for Parkinson’s conducted in Georgia (USA) has grabbed the attention of some readers.

The study involved Niacin (also known as nicotinic acid), which is a naturally occurring organic dietary compound and a form of vitamin B3.

The study was very small, but the researchers noticed something interesting in the blood of the participants: Niacin was apparently switching some of the immune cells from an inflammatory state to an anti-inflammatory state.

In today’s post, we will discuss what Niacin is, how it relates to Parkinson’s, and we will consider some of the issues with having too much niacin in your diet.


Source: Universal

It is one of the most common requests I get:

“Can you give an opinion on this supplement ____ or that vitamin ____ as a treatment for Parkinson’s?”

And I don’t like giving opinions, because (my standard disclosure) “I am not a clinician, just a research scientist. And even if i was a clinician, it would be unethical for me to comment as I am not familiar with each individual’s medical history. The best person to speak to is your personal doctor“.

But I also don’t like giving opinions because of a terrible fear that if I write anything remotely positive about anything remotely supplemental or vitamintal (is that a word?), a small portion of readers will rush off and gorge themselves on anything that sounds remotely similar to that supplement or vitamin.

So you will hopefully understand why I am hesitant to write this post.

But having said that, the recently published results of a small clinical study conducted in Augusta (Georgia, USA) are rather interesting.

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Two birds, one stone?

This week interesting research was published in the journal EMBO that looked at the Parkinson’s-associated protein Leucine-rich repeat kinase 2 (or LRRK2).

In their study, the researchers discovered that lowering levels of LRRK2 protein (in cells and animals) affected the ability of Mycobacterium tuberculosis – the bacteria that causes Tuberculosis – to replicate.

In today’s post, we will discuss what Tuberculosis is, how it relates to LRRK2 and Parkinson’s, and we will consider why this is potentially REALLY big news for Parkinson’s.


Daedalus and Icarus. Source: Skytamer

In Greek Mythology, there is the tale of Daedalus and Icarus.

Daedalus was a really smart guy, who designed the labyrinth on Crete, which housed the Minotaur (the ‘part man, part bull’ beast). For all his hard work, however, Daedalus was shut up in a tower and held captive by King Minos to stop the knowledge of his Labyrinth from spreading to the general public.

Source: Clansofhonor

But a mere tower was never going to stop Daedalus, and he set about fabricating wings for himself and his young son Icarus (who was also a captive).

Being stuck in the tower limited Daedalus’ access to feathers for making those wings, except of course for the large birds of prey that circled the tower awaiting the demise of Daedalus and his son. But he devised a clever way of throwing stones at the birds in such a way, that he is able to strike one bird and then the ricochet would hit a second bird.

And thus, the phase ‘killing two birds with one stone’ was born (or so it is said – there is also a Chinese origin for the phrase – Source).

Interesting. And this relates to Parkinson’s how?!?

Well, this week researchers in the UK have discovered that a protein associated with Parkinson’s is apparently also associated with another condition: Tuberculosis. And they also found that treatments being designed to target this protein in Parkinson’s, could also be used to fight Tuberculosis.

Two birds, one stone.

What is Tuberculosis?

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Making. It. Personal.

This is one of those posts (read: rants) where I want to put an idea out into the ether for someone to chew on. It starts with a very simple question:

Why is ‘the drug’ the focus of a clinical trial?

If our goal is to find beneficial therapies for people with Parkinson’s, then the way we currently clinically test drugs is utterly nonsensical.

And if we do not change our “we’ve always done it this way” mindset, then we are simply going to repeat the mistakes of the past. Others are changing, so why aren’t we?

In today’s post, we will consider one possible alternative approach.


I hope you know who Grace Hopper is – if not, click here. Source: Mentalfloss

Why is ‘the drug‘ the focus of a clinical trial?

The way we clinically test drugs makes absolutely no sense when you actually stop and think about it.

Other medical disciplines (such as oncology) have woken up to this fact, and it is time for the field of Parkinson’s research to do this same.

Let me explain:

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