Neuren and Darwinism : Neurenism?

Well, this will make Rett syndrome parents’ blood boil-
A blog post about the “failed” Phase II Rett study with NNZ-2566 by someone who cannot grasp the significance of the findings. It’s like someone saying the world is flat and all those round world map believers are wrong because they (the author) can’t comprehend the data.

Here’s the response I wrote-
Let me blunt, this diatribe of nonsense isn’t worth the time you took to write it. Speaking as a parent whose child went through the Phase II Rett trial, I can assure you, that it was in no way a failure. Breakthrough Status is given to only 30% of applications, so it is not surprising that it was not received on the first try, disappointing, though. While TROFINETIDE, (NNZ-2566) is not a cure at the cellular level, and it appears only you find this astonishing, it is a possible “virtual” cure by addressing multiple life threatening and life shortening symptoms, not unlike “Lorenzo’s Oil”.

Neuren and Trofinitide represent the best and ONLY hope right now that children with Rett syndrome and Fragile X may have a chance, how dare you scoff at it. I can personally defend the improvements seen with Trofinitide. Also, Rett doctor after Rett doctor HAVE come out and publically spoken about their support of that trial, Neuren and Trofinitide. In addition, the US Army, which has funded Trofinitide and it’s use in TBI would not continue to fund it if they personally were not seeing positive results. What one sees with their eyes does not always translate well into statistics, but you should be more careful before you wish for the only hope Fragile X and Rett syndrome suffers have to die and fade away, especially when it works.

And, in reference to Darwinism, I think that “survival of the fittest and natural selection” will come into play soon enough, with Neuren on top and you proven so wrong as to become obsolete and made to fade a way.

This entry was posted in Fragile X, Neuren Pharmaceuticals, Rett Syndrome and tagged , , , , . Bookmark the permalink.

3 Responses to Neuren and Darwinism : Neurenism?

  1. cbehrenbruch says:

    I am sorry if I upset you – I think you mistake the purpose of my commentary. Your response actually makes me wonder if you read a different blog!

    Although I am very skeptical of the benefit of GPE, there is no doubt that at a higher-level IGF-1 is an interesting pathway for possibly addressing Rett Syndrome and there is some growing scientific momentum around this idea as you probably know – likely better than I if you are dealing with this condition in your daily life. You may note that I have not actually “scoffed” at the science, and I certainly did not scoff at the Rett Syndrome study. What I scoffed at was the company, it’s decision-making behavior, it’s clinical strategy/claims and its lack of corporate governance.

    But, for the record, yes – I am skeptical of GPE/Trofinide. The science is fairly old and it’s neuroprotective/neurodevelopmental function unproven, though I am glad you found benefit. That is encouraging.

    Also, before I continue, Fragile X and Rett, though they have some of the same developmental biology issues, should not even be remotely “clumped” together in assessment as to whether or not a drug has potential to help patients. As you have done. That is precisely the the type of irresponsible, evidence-devoid behavior that this company uses. As a patient stakeholder you should not be sucked in.

    Now, to get to the nub of your comment – which I appreciated by the way – I’d like to offer you two thoughts.

    1) You should be an incensed as I am – if not more – that this company can’t get its act together. If you think that Trofinitide is something special, then you should own a share or two of the company and be present at AGM meetings and ask why the company can’t design robust clinical trials, or use statistical methods that the FDA understands. I agree with you about the Breakthrough designation, incidentally, it’s not the end of the world at all. But what it raises red flags for is that the study design and outcomes did not offer statistical confidence and this is very problematic for the future of this drug. It is YOU who should be asking why, not me, because it will ultimately impact you.

    2) I have children and I couldn’t imagine the heartache of living with some of these conditions. I appreciate how few real options there are. However when companies do a very underwhelming job of taking capital from investors and conducting themselves in a way that doesn’t ultimately result in the best outcome for patients, all it does it make it harder to concentrate resources on those rare diseases, not easier. If Trofinitide is your salvation, then I can tell you that it is currently residing in a commercial vehicle that is unworthy of your hopes, dreams and expectations and it needs to pull its socks up.

    So, to summarise, I appreciate your emotion. I hope that when you reblogged your scathing reply, that you will also – in the interest of balanced dialog – also reblog my response. Ultimately, I write these posts because I want to see quality clinical development benefit patients. I can’t do this as a patient stakeholder, as you can, but I can certainly call on mediocre performance when I see it. And contrary to your assertions, I will not feel remorse when I am proven wrong – I will feel joy for the patients who will benefit.

    For something like this, I am delighted to be proven wrong. Meanwhile, I continue to do what I do – which is attempt to hold companies to a higher standard of performance.

    Thanks for engaging.

    • melelllan says:

      I have complete confidence in Neuren. Not getting Breakthrough means nothing of the sort of a “red flag”as you have noted. No company is mandated to announce its application for this, and yet Neuren did, a bold move and just because the FDA did not understand the reporting method or rather because it was noted to be an unconventional reporting method, does not make it invalid. Many drugs travel this same path and ultimately get approval.

      Also, if you understood the science behind Trofinetide and IGF1, you would see how the underlying pathways, though ultimately a reversal of each other in Rett syndrome and Fragile X, will benefit and can be repaired.

      This company has the fortitude to tackle what many won’t touch for the very reason that it is so difficult to prove efficacy and get these drugs to market, and yet efficacy IS proven, safety is proven and while the number of participants can be seen as small, it represented a fairly large percentage of Rett women in the United States. What you see as futile or mismanagement, I see as an amazing trial structure given the shortage of available candidates and the size of the company.

      Your arguement also only hinges on Breakthrough Status, very closeminded given that Orphan Drug status and Fast Track Status have been granted, both evidence that the FDA DOES acknowledge both clinical evidence of efficacy and need.

      So, we will agree to disagree, but as a parent and widely read person in this area I in no way see Neuren as a mediocre company, I see it as groundbreaking and innovative and have complete and utter confidence in Treagus and others to ultimately give my daughter a voice and a chance.

      • cbehrenbruch says:

        You are dead wrong about the “elective” reporting of Breakthrough status. They had no choice because they primed the market for it by announcing that they had applied for it.

        I think you and I are talking at odds. I agree with you about breakthrough status, and there was no need for Neuren to take the statistical approach it did.

        I wish you every luck.

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