Friday, March 6, 2009

Well, it's only been 4 days...

But is seems to me like an eternity since I've posted.
There have been several interesting things going on in the ALS community.
The most being the fact that the FDA has continued to stonewall all IND (Investigational New Drug) applications for IPLEX for all ALS patients. Other than the fact that it is a ridiculous government agency that has no heart, I really don't understand the rationale behind complete lack of human compassion and decency.

So do you know what an IND is? It is the authorization of a drug by the FDA on an individual basis in a "last ditch effort" to help someone that is suffering a diagnosis with no more alternatives as determined by their physician. It is an application that must be submitted by a licensed doctor with good reason and in the best interest of his patient.

Historically, the FDA only denies IND requests when they fear that the drug might kill the patient or cause significant harm. This, of course, is very ironic for ALS patients. Many have reminded the FDA that there is no treatment, there is no cure, and everyone dies from this disease eventually, most within 5 years after having been paralyzed for 60% of their post-diagnosis life.

I don't think that there is an ALS patient on the planet that would prefer to die from ALS than take their chances with some sort of possible, undocumented, never reported reaction to a drug that is already approved by the very same agency for use in infants and small children with short stature syndrome.

What is the agenda of this agency? Why do they prevent people from trying this drug? These are people who would most certainly sign any kind of waiver of responsibility either to the FDA, their doctors or to the company that produces it. Why?

There were several theories circulating initially. One was that there was this "fear of deadly consequences" that loomed over the drug, which of course does not hold any water for ALS patients, who are already holding a death sentence decree much worse than any reaction to a drug might be.

A second one was that in some sense this group "The Bloggers" would use the information derived form its use to form unfounded conclusions as to it's efficacy and the drug company sell more???? Please. Even the most ignorant political dinosaur should know that at this point the Web/Internet community is large enough and educated enough to sniff out blatant commercialization of almost anything. I personally am an Internet veteran with 15 years of experience and connections througout the technology, media, publishing, social media, and advertising communities. Nothing is going to get past me and my group of peers that are connected and discussing these issues every day. It would be a disaster for any drug company to try and pull this off.

I now believe that the latest, ridiculous conclusion is essentially "if we give IND's to everyone now requesting them, there will be no one left to do a double blind study to prove or disprove it's value in treating ALS patients." Does anyone NOT see the shocking irony here? Let me rephrase this (as I understand it) "Let's deny dieing people a drug that we have already deemed safe to give in large doses to infants, so we can actually give it to 1/2 of a control group and test whether or not it works for real."

Let me state an obvious fact: People are dieing of ALS every DAY. These people have been lieing motionless for months, if not years, with only their perfectly healthy minds and their caregivers to help them get through this. Some of them, as I've recently learned, are abused by those closest to them, and they are helpless to save themselves.

There is no time to wait around and see if this works. It is a new world, people can share information. Scientists should seek to leverage the worldwide communication platforms that exist now rather than fear them. Please don't let academic arrogance kill people in the name of good science. Figure out how you can distribute this drug and others to a wider group, figure out how you can gather control data without being sadistic to people that are already suffering an unfathomable pain. Stand up and honor your vows of compassion and those "to do no harm."

And if you are an official that has not had to take those vows, or a drug company executive seeking profit or power, rethink your pathetic stance and have some compassion for the thousands of people and their families suffering from this horrible disease. Think about my post of last week. You, Mr. High and Mighty official, YOU could wake up tomorrow and not be able to turn your ignition or button that last button on your shirt. You, my friend, will then wish you had not been such a complete arrogant jerkoff.

My friends tell me "you catch more..(what...jerks?) with honey than you do with vinegar", so let me rephrase that last statement. Just forget for a second that I called you an arrogant jerkoff... "Please look past yourself to the pain of others and let people that need to try this drug try it." You have nothing to lose, if it works, you are a hero, if it doesn't then so be it. Our situation will not be harmed. You will have given us hope for at least some small period of time. A gift of hope. I have already bought this gift many times without efficacy AND without remorse or demand for compensation.

If you read my blog, you know i'm not, generally, an angry person. This situation really pisses me off though, and I apologize, once again, if I offended you.

PS, believe me, Jerkoff was NOT my first choice of words. I've become a bit more judicious in my choice of words, really only so this post might appeal to a wider range of email filters.

PSS, will someone please forward this to the human being that keeps stamping "REJECTED" on all the IND's for IPLEX that come across his desk? I would welcome his response to this blog at any time.


  1. As if battling ALS isn't bad enough, the hope of seeing a drug come to market lies in the hands of morons. No one knows for sure what will work- but why - if a drug is already on the market for use in infants, can it not be tried on adults with a fatal disease... who are willing to try it?
    And what will happen if say ALS-TDI find efficacy in the lab on something they discover? Imagine the excitement squashed. I hate to say it, add to the list, on top of care giving, fighting with insurance companies, fund raising, awareness campaigns and research, we must also stand up against the FDA and The Government. This Blog explains the frustrations- and helps further the discussion on WHAT SHOULD WE DO to prepare for a treatment and cure?

  2. I have just found your blog and am very grateful that I have. You are speaking for so many of us out here who sometimes don't have the words. I have been recently diagnosed and I have felt cheated, angry, sad, angry, angry and angry over some of the stuff going on with our lives. 140 years - no cure - no treatment! Angry. Then when something does come along offering some small hope, wait, we have to see if we can kill half the people in the study before we can cure the rest of you. Who among us would not say, just give it to me. I am a 58 year old woman who has felt cheated out of a good 20 years. I look at the picture of your wife and daughters and feel ashamed of my anger. What a beautiful family you have. So young, so vibrant, so hopeful of the future. Then this comes into your life. I am so sorry. Sorry for you, sorry for your daughters and most of all sorry for your wife who has been robbed of herself. I will follow this blog and hope that your journey is a loving one. I know there has to be something to learn from all this. I pray that there is.

  3. Nancy, thank you for your comment. This is one of my angrier posts and I hope you have found some of the more hopeful ones as well.
    Thanks for finding it and thanks for commenting. Best wishes in your battle. Try to stay strong and know that we are pulling for you too.