The Twilight Zone – What is behind the door? Doo-doo-Doo-doo, Doo-doo-Doo-doo….

Please note – this post was originally published on June 26th 2016! I have reposted as it has some current relevance for us with Motor Neurone Disease with the recent CU-ATSM Drug media story. But also it has some slight Brexit overtones. I have checked how I describe this rather divisive subject, and it is gentle readers, and I believe totally non controversial. These comments are 2 1/2 years old but could be said today. So let’s go back to June 2016…..

Well what a historical time here in the UK. Even Lord Lee Bragg’s intervention on the new topical after midnight TV chat show “I’m smashed”, could not save the remain camp’s campaign.

Lord Lee Bragg, when asked “What would leaving the EU mean to you?”

Lord Bragg – “Well it would **** my business”

Interviewer – “Can you elaborate more on that, Lord Lee?”

Lord Bragg – “Yes sure thing. My business would be completely utterly ******* beyond repair”

That was Lord Lee Bragg, CEO of Bragg’s EU flags, banners and underwear (105% polyester – please take care near sources of combustion)

Lord Bragg added “Yes I might have some trade for EU fetish parties, and perhaps a bit of bunting but I am currently looking at a whole warehouse of EU Flags wondering who I can palm them off on, sorry sell them to, if this goes pear-shaped”

I understand he has a stall booked at the local car boot sale tomorrow. Cash only!

In my view the EU referendum was simply too big a decision to have ever put to us, the general public. It was, and is, a serious matter and there was no way that we would understand the full detail. This is exactly what we pay our leaders for! Taking any one sound bite can always appeal to the heart. It is too early to know the real result of this decision and, of course, I hope we can move forward with as little damage as possible.

Lee, how is this relevant to MND/ALS? Come on and get to the reason why people read your blog!

Ok, let me change direction and compare the process with the search for a drug and/or treatment for MND/ALS. We have the same challenge with “getting the real facts” when a story/news/potential breakthrough appears. Sifting anecdote and misguided, but good intention from the truth is not easy, just like the EU. With MND and other diseases, without treatment, a large group of vulnerable and susceptible people is created, ie patients seemingly without hope. It is all too simplistic for me to say we need to be careful, but yes beware, as any ill thought out actions could actually delay the eventual arrival of the required treatment for mankind, or create damaging false hope.

I wanna tell you a story!

Drug development is highly regulated, for example in the USA by the FDA (Food and Drug Administration) an organisation which, like the EU, is often depicted as monolithic and bureaucratic. I can hear my US readers saying “too right Lee”. But does it really deserve this?

Every so often an apparent miracle treatment finds its way above the parapet, and into news, tv media, social media and it is pretty much always portrayed as fact. Don’t get me going on journalists and their lack of detail!

About 18 months ago (now in 2019, over 4 years ago), a drug, called gm604, jumped into MND/ALS community news. In summary, a new potential therapy being developed by a private company apparently showed that in 7 out of a total of 8 patients treated there was no disease progression in a 12 week trial (that’s the sound, bite guys).

This sparked a political and social media firestorm with over 500,000 signatories demanding the FDA approve the drug as soon as possible. The argument seemed simple, ie patients are going to die anyway, can we try it? Also at first sight it appeared to be the wonder drug. The FDA were barraged, and were portrayed as the bad guys when they turned down the request. In fact, this is still going on and there is much bad talk directed at the regulatory establishment. I could detail the story and how it actually has played out, but it’s far better coming from Dr Richard Bedlack, one of the most eminent and articulate MND researchers in the world. I have positioned the video at the right point to hear his answer to a question from the audience regarding gm604 (I hear you gasp with wonder at my amazing technical skill!). If it doesn’t play in the right place, please just jump to 1h and 5s.

Oh, it wasn’t as it seemed, was it! The moral is of course, spend at least some time researching to get behind the claims. The drug, gm604, is but one of many prospective therapies that are out there currently at the same position in development. Bottom line, how do you choose which ones to invest in, after all it’s tax payers money etc? Regulation, peer, and scientific review, that’s how. We must push emotion aside. Using the same logic that the firestorm created, should the FDA give everyone free and total access to the 100s of prospective drugs, and pay for them? Rational? Of course not.

The good news is that organisations like the MNDA can do this filtering when it comes to funding.

2019 update. GM604 is not even discussed in the community now! The company are not pursuing the drug!

Fast forward, the above video is now over a year old, but as far I can see, the drug has not yet moved to the level of trial required as indicated by Richard. Also the biotech company have not released data requested over a year ago for review by the FDA! It appears that a small number of patients worldwide are receiving the drug in some form of private clinical evaluation. It is also not clear how these are funded, but I guess they must have paid. I don’t believe the drug would have been supplied otherwise. Why should it? So at second sight, one could make the conclusion that this is one rule for the wealthy and one for the poor. Again, this would be the wrong conclusion, you must always return to the facts. If someone wants to spend on unproven, non peered reviewed treatments that is an individual’s choice, but that doesn’t mean others are having treatment withheld.

In the USA, there is also currently a strong “right to try” campaign ongoing as we speak. I have to ask what this campaign is trying to do? The FDA already have a process to allow anyone to take ANY drug at ANY stage of development with the agreement of the drug company to sell it to the requester. The same actually applies here in the UK. So unless this campaign forces companies to supply unproven drugs, free of charge, I cannot understand the real aim.

My core point is that we need regulation, and the FDA are the good guys, not the bad guys. We have to have regulation otherwise you might as well try Lord Lee Bragg’s special secret mix of herbs to cure everything (available in 200kg handy bags). And above all, who is going to pay and do you create false hope? Yes, emotive videos play on our heartstrings, but we have to prioritise and fully think out.

If you are fascinated by science, and have the time, watch the whole of Richard’s video. He is an excellent presenter. He shows where we really are with MND research and that we are making real progress. It’s not necessarily the headlines that drive true progress.

Many of you reading this article, will have no knowledge of research for MND, but most people will recognise the phrase “they are always coming up with new things, it’s going to happen soon”. This is true, but this will only happen if we can continue a significant funding stream. So please give what you can to my justgiving MND fund-raising account, which all goes to care and research.

Back to the EU referendum. I voted remain for so many reasons, and also, I could not think of one truly valid objective reason to leave. On a selfish subject, medical research is potentially going to suffer significantly now. Central European funds, such as Horizon2020 (an €80 billion fund) have been powerful tools to the whole community enabling leading scientists apply for grants for vital research. Grants have been allocated on comprehensively objective criteria, including removing duplication etc. What are we to now expect? It will be interesting to watch how it plays out. I hope we haven’t thrown the baby out with the bath water.

Right, aside the referendum, what else has happened this week?

I had a funny dream the other night. I am a gadget freak, and love technology, but I think I found a flaw in one idea. In my dream (real dream honest), I had just had a phone implanted into my hand. So when it rang, I just lifted my hand to my ear. I am not joking you! In my dream I received a call, and the caller said “Is Jean there? Can I talk to her?” So I spent the next hour holding my hand next to Jean’s ear!

On the MND side of my life, I have had the results from a specific genetic test. It was negative, which of course doesn’t help me, but it has huge, good implications for my family. I will tell you why I decided to have the test in next week’s post and give you a little more background to complex disease genetics.

Until next time fellow Europeans and citizens of the world!

It’s time for my breakfast consisting of Italian Expresso and French Croissant, and for Dinner German…., no perhaps not. Sorry my German friends, you design and build some of the greatest cars on the planet but on the cooking side I think some improvement is needed!

Oh my word, this guy has just appeared, it is Judge Bragg from the year 2525, who has come from the future to resolve some issues! How can he help us?

It’s a vacuum cleaner, Lord Bragg, or is it?

Doo-doo-Doo-doo, Doo-doo-Doo-doo…………….