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A Parkinson’s-focused biotech company called Enterin has had a very busy start to the new year, with publication of some interesting preclinical research and the announcement of Phase II clinical trial results.
The clinical trial results met both the primary and secondary endpoints (the pre-determined measures of whether the treatment is effective) indicating a successful study, and the preclinical result provides new potential insights into the functions of the Parkinson’s-associated protein, alpha synuclein.
In today’s post, we will discuss both the clinical trial results and the preclinical work, and consider what this means for our understanding of Parkinson’s.
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In scientific nomenclature, they are referred to as Squalus acanthias.
Many people call them ‘Spurdogs’. Or ‘Mud sharks’. Or even ‘Piked dogfish’.
But they are more commonly known as spiny dogfish.
Source: X-ray Mag
Fun facts about spiny dogfish:
- They live in the shallow saltwater habitats of the North Pacific and the North Atlantic oceans
- The females are longer (49 inches or 124 cm) than the males (39 inches or 99 cm)
- They have two dorsal fins, both with venomous spines (hence the name)
- A pregnant females will have an average litter of 6 pups
- They have very long gestation periods – up to 24 months!
- The average lifespan ranges between 20 and 24 years
- Spiny dogfish are very fast swimmers – able to swim at about 6.2 feet/s (1.9 m/s)
- They have a special organ called the ‘Ampullae of Lorenzini‘ which they use to detect the electric field generated by their prey.
- They have a very keen sense of smell and two-thirds of their brain is involved in their sense of smell.
Oh, and they are extremely robust when it comes to infection.
Seriously, they never get sick, which is fascinating given that they have a relatively “primitive” immune system (Click here to read more on this).
Very interesting. But what does any of this have to do with Parkinson’s?
Continue reading “Are we Enterin a new age?”
Researchers at Cambridge University published a new report this week that extends on a very interesting line of Parkinson’s research. The studies focus on a compound (and derivatives of that compound) that has been derived from the dogfish shark.
The protein – called Squalamine – has an amazing ability to prevent the Parkinson’s-associated protein alpha synuclein from clustering (or aggregating) together. The aggregation of alpha synuclein is considered to be a key component of the biology underlying Parkinson’s, and thus any compound that block/reduce this aggregation is viewed with therapeutic applications in mind.
Unfortunately there is a problem with squalamine: it does not cross the blood brain barrier (the protective membrane surrounding the brain).
But a derivative of squalamine – called Trodusquemine – does!
In today’s post, we will look at what Squalamine and Trodusquemine are, we will review the new research, and look at current clinical research efforts involving these compounds.
The effects of aggregated Alpha Synuclein protein in a neuron. Source: R&D
We often talk about one particular protein on this website. It is called alpha synuclein. It is one of the most common proteins in the human brain, and it appears to be centrally involved with Parkinson’s.
In the Parkinsonian brain, alpha synuclein clumps (or aggregates) together, which is believed to lead to the appearance of Lewy bodies.
What are Lewy bodies?
Continue reading “Squalamine begets Trodusquemine”
There has been a lot of discussion on this site (and elsewhere on the web) regarding the need for more objective systems of measuring Parkinson’s – particularly in the setting of clinical trials.
Yes, subjective reports of patient experience are important, but they can easily be biased by ‘placebo responses’.
Thus, measures that are beyond the clinical trial participants conscious control – and focused on biological outcomes – are needed.
In today’s post, we will consider one possible approach: Smart pills. We will discuss what they are, how they work, and how they could be applied to Parkinson’s research.
In order to encourage a growing discussion regarding objective measures of Parkinson’s (and to follow up on previous rants – Click here and here for examples), I have decided to regularly (once a month) highlight new technologies that could provide the sort of unbiased methods of data collection that are required for assessing whether a treatment is having an impact on Parkinson’s.
Today, we will look at smart pills.
What is a smart pill?
Continue reading “Objective measures: Getting smart about pills”
In addition to looking at current Parkinson’s disease research on this website, I like to look at where technological advances are taking us with regards to future therapies.
In July of this year, I wrote about a new class of engineered viruses that could potentially allow us to treat conditions like Parkinson’s disease using a non-invasive, gene therapy approach (Click here to read that post). At the time I considered this technology way off at some point in the distant future. Blue sky research. “Let’s wait and see” – sort of thing.
So imagine my surprise when an Italian research group last weekend published a new research report in which they used this futurist technology to correct a mouse model of Parkinson’s disease. Suddenly the distant future is feeling not so ‘distant’.
In today’s post we will review and discuss the results, and look at what happens next.
Technological progress – looking inside the brain. Source: Digitial Trends
I have said several times in the past that the pace of Parkinson’s disease research at the moment is overwhelming.
So much is happening so quickly that it is quite simply difficult to keep up. Not just here on the blog, but also with regards to the ever increasing number of research articles in the “need to read” pile on my desk. It’s mad. It’s crazy. Just as I manage to digest something new from one area of research, two or three other publications pop up in different areas.
But it is the shear speed with which things are moving now in the field of Parkinson’s research that is really mind boggling!
Take for example the case of Squalamine.
In February of this year, researchers published an article outlining how a drug derived from the spiny dogfish could completely suppress the toxic effect of the Parkinson’s associated protein Alpha Synuclein (Click here to read that post).
The humble dogfish. Source: Discovery
And then in May (JUST 3 MONTHS LATER!!!), a biotech company called Enterin Inc. announced that they had just enrolled their first patient in the RASMET study: a Phase 1/2a randomised, controlled, multi-center clinical study evaluating a synthetic version of squalamine (called MSI-1436) in people with Parkinson’s disease. The study will enrol 50 patients over a 9-to-12-month period (Click here for the press release).
Wow! That is fast.
Yeah, I thought so too, but then this last weekend a group in Italy published new research that completely changed my ideas on the meaning of the word ‘fast’. Regular readers will recall that in July I discussed amazing new technology that may one day allow us to inject a virus into a person’s arm and then that virus will make it’s way up to the brain and only infect the cells that we want to have a treatment delivered to. This represents non-invasive (as no surgery is required), gene therapy (correcting a medical condition with the delivery of DNA rather than medication). This new study used the same virus we discussed in July.
Continue reading “AAV-PHP.B: The future is apparently now”