Ptbp1: “One and done”(?)

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Recently a lot of media attention has been focused on a new study that reported the replacement of lost dopamine neurons in a mouse model of Parkinson’s, which resulted in the correction the associated behavioural/motor issues.

The researchers involved achieved this amazing feat by simply reducing a single protein in a special type of helper cell in the brain, called astrocytes. By lowering the levels of the protein, they were able to transform the astrocytes into dopamine neurons.

Intriguingly, the study represented independent replication of a previous study that demonstrated a similar result – transformation of astrocytes inside a mouse brain into dopamine neurons by reducing a single protein.

The protein in both studies is called Ptbp1, and in today’s post we will discuss what this protein does, what the new study found, and what the implications of this work could be.

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Source: Howtogeek

Earlier this year, I stated in my 2020 wish list for Parkinson’s research (Click here to read that post) that one of the big themes I was hoping to see more of was further research on regenerative approaches for the condition.

We have discussed this a few times, but any “curative” treatment for Parkinson’s will require 3 components:

  1. A disease halting mechanism – to slow/stop the progression of the disease
  2. A neuroprotective agent – to protect the remaining cells & provide a nurturing environment for,
  3. Some form of restorative/regenerative therapy – replacing what has been lost

Now the encouraging news is that if you look at the SoPD “The Road Ahead: 2020” post, you will see that there is a great deal of research being conducted on all three of these components at the clinical stage (in addition to vast amounts of work on the preclinical level).

But it is fair to say that the bulk of the clinical research being conducted on restorative therapy for Parkinson’s is centred around the transplantation of stem cell-derived dopamine neurons to replace the cells that have been lost in Parkinson’s (click here to read a recent SoPD post on this topic).

Embryonic stem cells in a petridish. Source: Wikipedia

In my wish list for 2020, I was hoping to see regenerative approaches beyond the well trodden path of cell transplantation (growing cells in culture and then injecting them into the brain).

Dopamine neurons (green) in cell culture. Source: Axolbio

Rather, I was hoping to see more research on new regenerative approaches that target/manipulate endogenous pathways in the brain – forcing changes within the central nervous system itself.

I didn’t have high expectations in this department, but I have to admit that now I have been pleasantly surprised by the number of research reports that have been published thus far this year highlighting novel regenerative approaches. We have discussed several of them here on the SoPD already (Click here and here for examples), and today we are going to review another which was recently published in the prestigious scientific journal Nature.

This is what all the news papers have been talking about?

Indeed. There has been a lot of media attention focused on this research report.

So what does the new study report?

Continue reading “Ptbp1: “One and done”(?)”

The Neuraly trial

 

 

 

This week a new clinical trial was registered which caught our attention here at the SoPD HQ. It is being sponsored by a small biotech called Neuraly and involves a drug called NLY01.

NLY01 is a GLP-1R agonist – that is a molecule that binds to the Glucagon-like peptide-1 receptor and activates it. Other GLP-1R agonists include Exenatide (also called Bydureon) which is also also about to start a Phase III clinical trial in Parkinson’s (Click here to read a previous SoPD post about this).

There is a lot of activity in the Parkinson’s research world on GLP-1R agonists at the moment.

In today’s post, we will discuss what a GLP-1R agonist is, what we know about NLY01, and what the new clinical trial involves.

 


 

Every week there are new clinical studies being announced for Parkinson’s.

Many of them are registered on the Clinicaltrials.gov website. Here at the SoPD, we try to keep track of new trials being registered (the SoPD Twitter account highlights the more interesting trials).

This week one particular newly registered clinical trial stood out. It involves a small biotech company Neuraly (which is owned by parent company D&D PharmaTech).

And the drug being tested in the Neuraly clinical trial is a GLP-1R agonist.

What is a GLP-1R agonist?

Continue reading “The Neuraly trial”

When the zombies are all in your head

In your brain there are different types of cells.

Firstly there are the neurons (the prima donnas that we believe do most of the communication of information). Next there are the microglia cells, which act as the first and main line of active immune defence in the brain. There are also oligodendrocyte, that wrap protective sheets around the branches of the neurons and help them to pass signals.

And then there are astrocytes.

These are the ‘helper cells’ which maintain a comfortable environment for the neurons and aid them in their task. Recently, researchers in California reported an curious observation in the Parkinsonian brain: some astrocytes have entered an altered ‘zombie’-like state. And this might not be such a good thing.

In today’s post, we’ll review the research and discuss what it could mean – if independently replicated – for the Parkinson’s community.


Zombies. Source: wallpapersbrowse

I don’t understand the current fascination with zombies.

There are books, movies, television shows, video games. All dealing with the popular idea of dead bodies wandering the Earth terrifying people. But why the fascination? Why does this idea have such appeal to a wide portion of the populous?

I just don’t get it.

Even more of a mystery, however, is where the modern idea of the ‘zombie’ actually came from originally.

You see, no one really knows.

Huh? What do you mean?

Some people believe that the word ‘zombie’ is derived from West African languages – ndzumbi means ‘corpse’ in the Mitsogo language of Gabon, and nzambi means the ‘spirit of a dead person’ in the Kongo language. But how did a word from the African continent become embedded in our psyche?

Others associate the idea of a zombie with Haitian slaves in the 1700s who believed that dying would let them return back to lan guinée (African Guinea) in a kind of afterlife. But apparently that freedom did not apply to situations of suicide. Rather, those who took their own life would be condemned to walk the Hispaniola plantations for eternity as an undead slave. Perhaps this was the starting point for the ‘zombie’.

More recently the word ‘zonbi’ (not a typo) appeared in the Louisiana Creole and the Haitian Creole and represented a person who is killed and was then brought to life without speech or free will.

Delightful stuff for the start of a post on Parkinson’s research, huh?

But we’re going somewhere with this.

Continue reading “When the zombies are all in your head”