From ALS Bites

I Want a New Drug, III (but not this one)


Biogen Idec logoBiogen Idec announced this morning that the dexpramipexole trial failed.

[ Here's a better piece from the Boston Globe. ]

I am mildly surprised. Not shocked, but somewhat surprised. Based on bits of information leaked from various places over the past six months, I was pretty optimistic it would be found marginally effective. Not that it would be anything close to a cure, but that it would provide at least some early slowing of disease progression. But not so.

This is a disappointment to be sure. I took dexpramipexole or a placebo for 12 months starting in August of 2011, and have been taking the real drug since early September of 2012. I guess I took my last dose of it this morning.

How does this affect me personally?

I know that friends and family will be concerned that this is a setback for me personally. I don’t really see it that way. My study doctor and nurse both felt I was on the real drug from the beginning of the trial (this due to some minor side effects). Additionally, when all trial participants started on the real drug in September I didn’t notice any changes to indicate this was my first exposure to the drug. So my progression is what it is, with or without the drug. I have enjoyed a relatively slow progression, and today’s announcement does nothing to change that. This is a blow, really, to those who were hoping it was the drug that was responsible for my slower-than-average progression. If it was helping me, it might help them. I feel for these patients who really have lost a source of hope today.

What is next?

There is still the drug NP001, which is basically sodium chlorite.[1] It certainly failed its phase II trial in one respect. Researchers had hoped it would “flip a switch” (a macrophage) leading to a permanent change in the central nervous system’s immune response, which it targets. This clearly did not occur. In fact, patients who received infusions for six months and experienced what seems to be a significant slowing of the disease, saw their progression speed up during the six months they were followed after this period of infusions. Some, I know, died right on schedule. Still, there is considerable hope among some in the ALS community that this drug will ultimately prove to be a winner. Expectations for it have changed, however. At a minimum, we know that six months of infusions will not do the trick. The drug company Neuraltus will presumably design their next study differently. It is unclear whether this will require another phase II trial, or if they can proceed to phase III. Even if it can prove to be of long-term benefit, it is almost certainly several years away from the market.

Stem cell research is ongoing. One trial, at Emory University in Atlanta, is in an early phase I trial. The state of California last year committed 18 million dollars to study the use of stem cells to treat ALS, but it will be at least a decade before anything comes of this. Sadly, the promise of stem cell treatment for ALS and other diseases was set back significantly by the G.W. Bush administration.[2] Any benefits from these therapies will come far too late for me and the thousands of others currently affected by the disease.

So, more of the same. Lots of research is underway. By all accounts, progress in understanding some of the underlying causes of neuron death in ALS has been made. But it is an extraordinarily complicated disease. It probably isn’t really one disease, and there probably won’t be one therapy found to be effective against all varieties.

Recent ALS Bites:
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Notes

  1. A few tenacious ALS patients understood the active ingredient in NP001 to be sodium chlorite from reading scientific journals, patent applications and other materials. Some of them tried creating their own concoctions of the drug. A few tried taking sodium chlorite orally, with very disappointing results. See this interesting account of these quixotic patient-led “clinical trials”. [^]
  2. In August of 2001, President Bush first disallowed the use of federal dollars for research on any embryonic stem cells except for the limited lines of cells that had already been harvested from destroyed embryos. Embryos are routinely destroyed by fertility clinics. His ban did nothing to stop this practice, it just prevented stem cells from these discarded embryos from being used. Twice more during his administration, he vetoed bills which would have lifted this ban. Though President Obama reversed this shortly after taking office (and his decision directly influenced California’s $18 million investment in stem cell-related ALS research), various groups continue the fight to deny funding for important research that relies on embryonic stem cells. It is not true that adult, or pluripotent, stem cells can be used as a replacement. In fact, a ban on embryonic stem cell research would also derail related research on adult stem cells. I hope sanity prevails. [^]