The Journey

Dear Arthur and others,

Yes, we know of the terribly unfortunate and too early death of the mother of Maria Mouskos. May the soul of Paradisa Mouskos rest in peace. I believe she is the third person involved with Team Iplex to pass away during the period of November 8, 2008 until January 1, 2009. That represents the time in which those seeking Iplex have been waiting for Insmed to release it under the extraordinarily difficult and time consuming measures specified.

The article you have forwarded: I personally feel it is well-written and a relevant view that should be considered. It has been submitted by three well-qualified representatives of the ALS neurological establishment and there are many substantiated quotations and references from other documents and resources. And, left to the article only, there could be a foundation for saying “we are all wrong in embracing this treatment called Iplex”.

I also feel it is a clear example of the ALS neurological establishment attempting to disprove or contravene the efforts of the ALS community to be self-directed in their treatment protocols when they (the ALS community) have been unable to secure appropriate treatment through their physicians. Please don’t misunderstand—that lack of “appropriate treatment” may still be, and probably is, due to the lack of any efficacious medication or other intervention devised to deal with ALS. It isn’t because of some unholy cabal that is, in any way, attempting to thwart the well-being of those with ALS. No one could possibly be that callous or lacking in humanity to do such a thing.

But there is another alternative as to why this situation exists—a “possible” pharmaceutical treatment that is embraced by the patient base and ignored or even rejected by the ALS neurological establishment. That has to do with the medical and technological barriers between physicians and patients (ALS and otherwise) that have been breaking down in the approximate 11 years of internet access by patient populations. During that time, the information boundaries that previously existed have been gradually breaking down, more patients and their families have become more aggressive in determining their own treatments, and there has actually been an osmosis of data and knowledge that has been diffused in both directions—the previously single-directed transmission of information from physician to patient to a now-existing transmission of data in both directions. There is a price to be paid for this, or rather “prices to be paid for this”. Those include: possibly erroneous information transmitted, inappropriate use of otherwise good information transmitted, a lynch-mob mentality that possibly forces the medical establishment to embrace or at least reluctantly provide such treatment, and a gradual softening of the prior hard line of “I prescribe/you listen” mentality of the medical community. I doubt that any professional group would take that lightly, least of all the medical community that has regularly “earned its stripes” through many years of training, practice and frequent sacrifice—both personal and professional. There are exceptions, of course, to this and any generalization.

Which brings me to another form of “sacrifice”—that experienced by the ALS patient community and their loved ones, caregivers, families and friends. In this form of sacrifice, they and we have watched the decline of ALS bodies from prior normalcy to diminished and malnourished shadows of their former selves. We and they have watched and suffered as their respiration, eating, drinking, limb usage, muscle strength, ability to speak, walk, sleep and countless other functions have declined or disappeared. Those of us who fall into the category other than “patient” have been to too many funerals, wakes, shivas, memorial services, dedications, remembrances and otherwise to sit by and say, “that’s ok.” And far more significantly, while we, as caregivers or family or bystanders are able to walk away muttering, our loved ones don’t walk away at all.

So what is the “disconnect” and what is the “issue”? How and why should this situation be resolved? What is going on that causes three well-intentioned researchers to write an article denouncing or at least severely minimizing a grass roots approach for the use of a biotech treatment for which some very real claims of efficacy, or at least hopes of efficacy, exist? The answers, which are so apparent, are as follows:

1. The disconnect comes from the inability of the medical establishment to think, or even imagine, that a grass roots movement, developed by non-professionals via the internet and email, could possibly recognize a relevant treatment for a previously untreatable disease. That disconnect happened with HIV-AIDS and it is still the circumstance with ALS. But we all know that without the movement engendered by the HIV-AIDS patient community and their loved ones, that disease would still not have become a condition with which people live, as opposed to that from which they die.
And ALS is, unfortunately and horribly, a condition from which everyone still dies.

2. The issue is simply whether or not the ALS community should have the right to try a medication or other treatment protocol that has at least a suggestion of efficacy and a probability of safety at least as defined as the likelihood of disrepair and death with which they are otherwise faced. Is Iplex safe—“indisputably”, in the words of an FDA representative and clearly defined in the original safety studies of Iplex, which was, after all, approved for use by infants affected with severe short growth stature at the same mg/Kg dosage levels now proposed for use by those with ALS. I think that none of you reading this will forget that those with ALS, certainly one of the most under-served and ill-protected patient communities in the history of mankind, have too much to risk and too little to gain by being the “good patient” any longer—quietly and surely succumbing to this most devastating of diseases while following the only protocol that is absolutely approved and totally without any possibility of undue expense or possible side effects—nothing. I think Iplex is the better alternative.

Best Wishes,

Stephen Byer
bsbyer@mhtc.net
ALS WORLDWIDE