Yesterday Was A Lemon Of A Day

Early yesterday morning financial-market tweets started mentioning Biogen Idec, and the news wasn't good.  Their most prominent of the "promising" ALS treatment candidates, Dexpramipexole, had failed to show efficacy in its large, Phase III clinical trial.  As an ALS advocate soon posted on facebook, "Dex is done."

Nobody believed that this would be a miracle treatment, but most of us believed that for a drug candidate to come this far over the last decade, surely it would help slow the disease in some patients.

No. Yesterday people with ALS felt a devastating blow.

Today we need to figure out how to make some lemonade out of a lemon of a day.  Over 900 clinical trial volunteers were involved in the clinical trial for this drug which was dubbed the "Empower" trial.  Today we need to empower their efforts by learning lessons from the trial process and making positive changes.

If a drug company has a dud in the pipeline, it's good both for the company and for the patients to eliminate the dud.  Good, but how could we have identified this dud sooner?  Time is money for a pharmaceutical company.  Time is lives for those dealing with ALS.

The traditional clinical trial sequence is to have two safety phases followed by efficacy phases.  That makes a lot of sense for most diseases, but ALS isn't most diseases.  We need to face the fact that even a Phase I or II safety trial is an efficacy trial in the eyes of volunteers with ALS.  They're looking for improvements (or more typically, a sense of getting worse less quickly) from the very first dose.  Last year a scientist with ALS who had participated in a Phase II trial asked a very interesting question.  Should we not have larger, more definitive Phase II ALS trials designed to discern safety and some efficacy more clearly?  Risk is tolerable.  You could ask the man who made this suggestion, but his funeral was four months ago.

Yesterday we also saw a quote from the ALS Association's Chief Scientist in the Wall Street Journal --

"The important thing we're learning about ALS is it's a spectrum of diseases," Lucie Bruijn, chief scientist at The ALS Association, said recently. "There are some who respond to treatment and some who don't; at the moment, we lump all the people together and that might be one of the reasons why the trials are failing."

What's the action plan to fix the next clinical trials so that they recognize the spectrum of diseases?  Months ago another man with ALS who had participated in a Phase II trial asked why clinical trials did not insure genetic diversity to prevent the exact kinds of failures described in the quote.  The "spectrum of diseases" concept isn't new.  It has been around for years.  The problem needs an action plan to fix the problem for the next clinical trials.

While were thinking about lemonade recipes, how about giving some other researchers access to that large set of Empower clinical trial patient data?  Biogen Idec has made a huge investment in ALS.  Yesterday they emphasized that they're still committed.  Can they lead by opening up access to the clinical trial data in hopes that something there will add insight to scientific research?  Can they be bold and start an open data standard for ALS clinical trials?  The volunteers surely deserve to have their data be useful since most of them will not survive for any product to make it to market.

Let's prove that the "Empower" word was more than a catchy name to promote clinical trial enrollment. Let's make some lemonade out of yesterday's lemon of a day.  Let's learn from what just happened.  Let's act.