Lou Gehrig's Disease - Motor Neuron Disease - Amyotrophic Lateral Sclerosis
Thought it had been cured by now? Still no known cause. Still no cure. Still quickly fatal. Still outrageous.

Tuesday, May 22, 2012

We All Need To Pay Attention To The Rest Of The Story

Almost a year ago, people with ALS were anticipating several important clinical trial opportunities.  One was a phase III trial for Biogen Idec's Dexpramipexole  The other was a phase II trial for Neuraltus' NP001.  We had been hearing high promise for Dexpramipexole for years.  NP001 was new on the radar.

Both trials filled enrollment quickly.

Patients talk online.  Normally with ALS clinical trials, patients try to figure out if they are less worse taking the test drug than they would have been without.  The bar is low.

NP001 (or an incredible placebo) actually made some clear positive differences for some patients.  That is a first in the 15+ years I've been paying attention to ALS.  Now what?

The trial design called for six months of test therapy followed by a three-month "monitoring period."  Patients were to get no drug during the monitoring period by trial design.

Because of all of the years of poor (or no) results from clinical trial drugs, the expectations were low and the monitoring period didn't seem harmful based on all of the dud drugs we've encountered.  But... until the whole cohort of patients in that phase II trial clears the six months of therapy plus the three months without drug, the data are not able to speak to scientists and regulators.

This isn't scientific, but I've met five patients in my small e-circle who had experienced improvements or plateaus while on NP001 (or the incredible placebo) who are now crashing during the monitoring period.  Really crashing.

I heard a leading ALS organization dismiss the situation because Neuraltus is not as well funded as Biogen Idec.  Sorry, but a money excuse is specious. The trial had a design.  Is there not room for some kind of adaptable trial design?  Will leading ALS organizations allow future trial designs to maintain a barrier between patients and the drug should it appear to perhaps be effective?  Does mixing companion diagnostic aspects with efficacy trials set patients up for a crash period (that we hadn't considered before since nothing had worked before)? Are our organizations really paying attention and willing to lead?

NP001 may not be the treatment we've dreamed of, but it (or the incredible placebo) was doing something. How do we sleep nights when patients who volunteered to test it are allowed to crash?

Yesterday we heard more coverage of patients who are trying to take the science into their own hands so that future patients may fare better.  Guess what drug the featured patient trialed?


  1. Make that 6 people. My FRS has dropped 14 pts since I've been off the infusions and I need to start bipap.

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  3. Laura, Thanks for chiming in. This is just nuts! It seems like we have some people with heads in the sand on this one. Looking the other way isn't the kind of leadership we need.

    I understand how you can't just change a clinical trial willy nilly, but certainly more people need to know about this situation. ALS should be the poster disease for having compact, meaningful clinical trials that offer some protection to the patients who volunteer!

  4. I have been on the phase III trial for Biogen Idec's Dexpramipexole since 10/2011 (one of the last to get in). September, I am rolling into month 12 and just found out today I will NOT roll over to the real open-label drug until later in October '12. Since taking the pills, I have had no symptoms and have only gotten considerably worst. So I presume (and I hope for everyone's sake) I have the placebo. I wonder what good DEX might do now. (sorry for the off topic post, just a bit disappointed today)