The relentless focus on getting a clearer picture…

This is my latest post on MND Research. I hope you find it interesting.

I have now written a few quarterly updates since being diagnosed, each with my opinions. The aim of these posts was to provide a solid base for all those diagnosed, recently or for some time, to help cut through the mire of information.

The core principles for my pieces has been, “trust the doctors, neurologists and professionals”. Part of this was seeded by observing statements made by sufferers, fuelled by their obvious frustrations, but sadly often misguided. Seeing people waste money and time on false hopes, and at the same time losing faith in scientists etc, I felt compelled to create a simple resource for the community. Dare I say, “fake news” was, and still is, deeply pervasive in the MND Community as well as in all walks of life.

So in preparation for this update, I have reviewed and made slight adjustments to the entire Research section of my blog.

If you are new to my musings, why not have a quick refresh before reading this new post. You can see how some things have progressed, or not! In addition some of the articles discuss items such as environmental factors and personalised medicine.

On with my update.

Is there any significant new research news since I last wrote on the subject?

Earlier this year I listed a number of drug trials that were in some focus at the time. Here is the table that was included that post back in March 2018.

Here is MY updated opinion chart…

There has been no move on Edaravone here in the UK. This was only the second drug in history approved for MND, but only in the USA, over a year ago. In my view this drug will most probably never be approved in the UK. The situation is still the same as earlier this year, and a marketing application would have to be made by the owning pharmaceutical company. None has been made. In addition a further trial would be required to satisfy our proof levels. I have heard the phrase “Patients are not missing out on much really” made by a leading UK professor at a conference. This says it all to me.

The same appears to apply to Masitinib, a drug that has apparently shown some efficacy, but only in a study that is NOT considered by the scientific community as proven. Again, the owning company, would need to run an additional trial for which there appears to be zero enthusiasm currently. So in my opinion, this is also off the radar.

However, don’t let this information get you depressed.

These were what I call “small effect” drugs, at best, a subject which I wrote about at length in This ain’t no technological breakdown… 

There are 3 major therapy focus research channels that currently keep my attention and I recommend you watching. These are: gene therapy, reducing neuro-inflammation, and anti-viral drug investigations.

We might see some first results in 2019 on the first attempts of gene therapy in humans. No news yet, but we just have to keep a close eye on developments.

Mirocals is a major trial that is targeting neuro-inflammation (ie an over active immune response). This is going to take at least another 2 years to return results.

The Lighthouse Project is proposing trialing a drug currently used in HIV treatment. As far as I can see, this is still pre-funding and pre-trial planning stage.

And that’s it! But……

We are moving firmly forward and I release this post just days ahead of the annual ALS/MND Symposium.  This year being held in Glasgow, UK. This conference is THE gathering of experts worldwide, organised by the UK’s MND Association.

To help with getting the best information to patients and the community, the MND Association has released a little Periodic Table tool to help navigate this year’s conference in a unique way. The Association will report on social media, and all presentation abstracts are available on the Internet. Although I won’t be attending, I can get a real understanding of what is being spoken about through such useful tools as this periodic table. Just click on the image below to take you to the live MND Association website.

Image provided courtesy of the MND Association

If you are the sort of person who appreciates more detail, you can even download all the abstracts in one single handy document from the symposium just by clicking – download abstracts.

One session is being live streamed.  The Ask the Experts Session on Wednesday 5th December at 2:30pm. is being shared on Facebook Live.  It’s well worth a watch, and you can ask questions online. This session sets the scene for the whole conference over the following days.

After the symposium I will write a further opinion update after having time to assimilate all the news and chat.

I leave you this week with a lesson from the world of air crash investigation that reflects some aspects of human disease research and shows how just how relentless, persistent, unwavering focus, and thorough analysis will bring results sooner or later.

Clear to Take off

Over 40 years ago, in 1977, what still remains the most lethal air disaster in history happened on the island of Tenerife. Two Boeing 747s collided on a runway killing 583 people!

The investigation was different to most accident analysis. The disaster happened on an extremely foggy night, the collision involving one plane taking off and taxiing into the path of another that had strayed onto the runway. Early on after the tragedy it was apparent that technical failure of either plane was probably not the cause but the result of external factors. But which ones?

On the day, to add the extreme weather, a terrorist incident at the Island’s main airport had forced aircraft to land at a smaller regional airport, including the KLM and Pan Am jets that were to be involved in the later accident. There was increased air traffic and the controllers were under extreme pressure.

I won’t detail the entire investigation, which you can read about here on Wikipedia, but I will summarise one of the final safety improvements that was recommended after the investigation was completed.

From the outside, a simplistic view would have just been to put the accident down to the weather. In fact, had it not been for the weather, the disaster would have been most likely avoided visually. The conclusion, however, was that it was most likely caused by misunderstandings between the air traffic controllers and the KLM Pilot. It was hypothesised that he might have believed that permission to take off had been granted when it hadn’t. It was most likely language mis-hearing. The resulting report delivered the greatest change in communications standards ever made in aviation history. One of the changes was the use of the specific words

“Take off”.

They are now only ever spoken when clearance is finally given. Up until clearance is given, words such as departure MUST be used.

A simple change, but significantly increasing safety. But it may never had been identified if not for this incident!

We don’t yet have any such change that humans could implement that would reduce the chance of developing a disease like MND. But, perhaps we will someday…. Intriguing? I might write about this in my follow-up post.

Until my next research update. That’s all readers!

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