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Guinea Piggin’

What’s new?

I’m asked “How are you doing?” quite a bit lately, and I always answer with some variation of “about the same” or “not much has changed.” This is the truth, but I don’t mean by this that I’m not seeing progression. I am. But the rate of progression, as far as I can tell, is about the same as it has been.

My left arm and hand continue to weaken; I have early indications and some noticeable atrophy in my left leg and right arm. They are still about two years behind my left arm. On the plus side, there is still no definitive evidence of weakness in my bulbar region (most of the muscles of the face and throat fall into this category), my diaphragm, or in the muscles of my trunk. My speaking, swallowing, and breathing remain unaffected.

At about the two-year mark since the onset of symptoms, I am in pretty good shape.

Guinea piggin’

A week ago today (Thursday, August 4), I joined the phase III trial of dexpramipexole. I took my first dose of what is either a drug that seems to be the next best-hope as an effective ALS treatment (it’s not a cure), or just a cleverly-disguised phony. Yes, there’s a 50-50 chance that I’m swallowing two placebos a day (to go along with the two doses of the not-very-effective riluzole[1] that I started taking at the beginning of June).

Knopp Neurosciences is developing dexpramipexole, and issued a press release in March to mark the enrollment of the trial’s first patient. An earlier press release provided more detail about the hopeful results of a two-part phase II trial that was completed in October 2009. In the first part of this phase II trial, 102 patients were randomized and received either the placebo or one of three dosage levels of the drug. Results were very encouraging (from Knopp Neurosciences):

In the case of ALSFRS-R, the number of treatment failures, defined as the loss of 6 points or greater in ALSFRS-R scores from baseline, totaled 9 subjects (or 33%), in the placebo group; 8 subjects (35%), in the 50 mg/day group, 4 subjects (15%) in the 150 mg/day group, and 2 subjects (8%) in the 300 mg/day group (p=0.014).

In the case of pulmonary function, the number of treatment failures, defined as a reduction in forced vital capacity of 20% or greater from baseline, totaled 8 subjects (30%) in the placebo group, 3 subjects (13%) in the 50 mg group, 3 subjects (12%) in the 150 mg group, and 1 subject (4%) in the 300 mg group (p=0.028).

The pulmonary function data, in particular, are very encouraging. These are press releases, so of course they must be viewed somewhat skeptically. The company is emphasizing positive results. But the numbers are real, and I’m reading that a slowing of progression by an average of 30-40% seems to be a reasonable guess as to what this drug may do. That would be a considerable improvement over riluzole1.

Whether or not I’m getting the real thing right now, it’s a good thing to be in this trial. Knopp has committed to providing to all participants access to the (real) drug from the end of the trial until such time as it hits the market. So, one way or another I will be getting this drug before it is available to the general public. That will be a very good thing if it does prove to be an effective treatment.

I called dexpramipexole the “next best-hope” as an effective treatment for ALS. I meant this in the sense of “next up time-wise.” A little farther down the road is a drug that is in a phase II trial right now, NP001 by Neuraltus Pharmaceuticals. There is a lot of excitement about the potential of this drug, but it is probably years away from the market.


I will enter retirement at the end of this month. My last day of work will be Tuesday, August 30, 2011. I’m starting eleven days of vacation tomorrow morning (going to California tomorrow for two weddings and a birthday), and so I have only five actual workdays left. Wow.

I’ll write more about my retirement on another day.

Recent ALS Bites:
12/17/2019 Steve’s Emergency Communication To-Go
02/28/2019 Unhappy Anniversary
05/12/2017 The Eyes Have It
08/04/2016 YFALS National Corntoss Challenge
07/18/2016 Scopolamine Blues

   or see the  Complete ALS Bites


  1. Riluzole is the only drug approved by the FDA for the treatment of ALS. It’s not been shown to do much good, but some patients may benefit more than others. [^]

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