Friday, 31 March 2017

Just Not Me

There will be two people specially remembered in the world of ALS; the last person to die from this disease, and the first person to be cured of this disease. These two people, and their two individual events, will likely not happen at the same time. People will die from ALS for many years after there are treatments. It will take a long time before there is a cure. It will happen, some day, most likely too late for me. But it is possible that those people who will be cured are amongst us right now.

There have been a couple of interesting developments in the ALS treatment arena, and one very disturbing change. The disturbing change is in the area of stem cell treatment. There is no proven correlation between stem cell treatment and recovery from ALS. I know PALS who have spent the tremendous amounts of money needed to go abroad for these treatments. They are all dead now, or they have had no change in their ALS and are on the same path as before.

What is disturbing these days is a series of ads I have been seeing on Facebook which make it look like this is a working treatment. These ads tell the stories of people who are trying to raise money for treatments at a clinic in Bangkok; this is not a place noted for its research and treatment into ALS. The ads are very professionally done, made up to look like news stories, implying a breakthrough in ALS treatment, carefully worded to avoid the promise of a cure, worded well enough to instill hope, and, most importantly, create new customers. This is the worst kind of preying on the sick, the most venal of attempts to sell an unproven, snake oil treatment. I fear these ads the most.

Then there are the legitimate drug companies seeking permission to enter North America with new treatments, a process which can take many years. One of these is a company from Isreal called BrainStorm, with a drug called NurOwn, which in turn is a part of a legitimate stem cell treatment process. This drug creates a special kind of cell, based on the patients spinal cord stem cells, in the blood which can protect neurons from toxins. It has shown effectiveness in animal models of ALS, sciatic nerve injury, Parkinson’s disease, multiple sclerosis, optic nerve ischemia and Huntington’s disease. The drug has been tested on people and has been shown to slow ALS progress.

A second drug seeking human trials in North America is Masitinib, made by AB Pharmeceuticals in France. According to AB Pharmeceuticals, Masitinib helps to slow progression of ALS by reducing inflammation. A kinase inhibitor, masitinib blocks mast cell-mediated degranulation, the release of cytotoxic substances that might further damage the motor nerves. In other words, it won't cure ALS, but it might slow it down a bunch.

The problem with both of these drugs is the need for Phase 3 Clinical Trials. This is the phase where a new drug has already been demonstrated as safe for human consumption and potentially effective in treating the targeted disease. Both NurOwn and Masitinib are ready for stage 3 in North America. They are waiting for government approval. The problem now is simply one of time.

It often takes up to two years for the US government to approve a Phase 3 Clinical Trial. These clinical trials are large, consisting of hundreds and perhaps thousands of people. So once the Phase 3 Clinical trial is approved, a process which might be foreshortened for an ALS treatment, the trial itself must be completed, a process of up to 3 years. After all of that, the drug must be submitted for FDA approval in the US, and Health Canada approval in Canada. In the US, these approvals can take a year or two. In Canada they take even longer; Canada adds the additional wrinkle of each province having its own approval process as well.

So even though there are viable treatments on the horizon, treatments which have a pretty fair chance of slowing, or even stopping, ALS progession, they are years away from getting to me, mostly thanks to slow and insensitive government organizations. They are, in battling disease, the same as in battling wars, inefficient and arbitrary.

All of this is to say that there are a great many things out there which could get my hopes up, discoveries which "should" make me excited. Except I know the process. It is highly unlikely that I will be the first to be cured. It is decidedly likely that I will not be the last to die. At least the progress being made will help someone, someday. Just not me.


  1. Hey Richard,

    I've only recently stumbled upon your blog during my own research for myself. I was finally diagnosed about a year ago at the Mayo Clinic after 2 years of thinking it was something else. So I've had it over 3 years now and I'm noticing from reading your blog and watching your video we have many similarities and I wish you well. One thing I've noticed it that you somehow get off your ass most days and do tons of stuff! I'm still working toward that. Anyway, in reference to this blog entry, I am seriously considering going the the Hadassah in Israel for the BrainStorm, NurOwn treatment. First I'll go see the medical team at Mass. General and see if they think I'd be a good candidate for it. I'm also researching a phase 2 study for the investigational drug NP001 which sounds a bit like Mastnib which you mentioned. I'm currently on the Tirasemtiv Phase 3 Trial. No real idea if I'm on it or the placebo and of course that's very frustrating. I believe that I am, but that's just my guess. So frustrating to not know if what I'm feeling is progression or something else. So... this is my reason for wanting to go to Israel and pay for the treatment and know I'm getting it. The same familiar story I read everyday as I learn more and more of others with our predicament.

    Thanks for being so open and speaking freely about your daily experiences, it really helps someone like me that struggles to ask for help and share my daily misery as I just try and put on a smiling face for everyone to see. Your honesty is refreshing.

    Greenwich, CT USA

  2. I am Karen Baird, I was diagnosed of ALS (Lou Gherigs Disease) in 2014, the doctor told me there is no permanent cure for the disease, i was given medication to slow down the progress of the disease, at the initial stage it was not so bad till it progressed to the end middle stage were i had difficulties going about my daily functions as i constantly felt weakness in my legs, ankles and feet, i was totally devastated till my daughter’s co-worker told us about a herbal doctor from west Africa who have herbal medicines for all kind of diseases including ALS, when i contact this herbal doctor via his email, he sent me two bottles of ALS herbal medicine through courier service, when i received this herbal medicine, he gave me step by instructions on how to apply it, when i applied it as instructed, i was cured of the disease within 20-24 days of usage. Contact this great herbal doctor via his email at abumereherbalcentre @ gmail . com
    www . abumereherbalcentre . simplesite . com