ALS Advocacy

ALS Advocacy
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.

Saturday, December 6, 2014

#alssymp Day +1 -- Research Symposium

The first big day started in a lovely convention center.  After several introductions (including an appearance by the Prince... not the singer, but the real Prince) the presentations began.

The first general presentation was by Dr. Sandrock of Biogen IDEC.  He spoke of clinical trial design and some better principles for Phase II trials.  Some of what he said reminded me of what a gentleman with ALS suggested several years ago -- larger, more definitive Phase II trials.   He also alluded to a preliminary observation period.  I see the value in that, but why eat up more clinical trial time.  I still think that on day of diagnosis patients should have the option of opting in to having their data available for clinical trial use.  That would give any trial a trajectory of any patient's ALS before the start of any trial therapy or placebo.  And we learned at other sessions yesterday how important and variable patient trajectories are with ALS.  I really have trouble with the word "progression."  Sandrock gave a summary of the characteristics of an optimal Phase II trial.  (The slide is in my twitter stream and unfortunately I am not able to post pictures in Blogger from this connection.)  Unfortunately without reliable biomarkers, the characteristics that he described can't be delivered.  I'm pessimistic that we'll see another trial from Biogen Idec until that kind of trial can be designed and delivered.

The first breakout I attended included Dr. Benetar speaking on diagnosis.  There is an approximate 12-month diagnosis delay for people with ALS and that has been the case for more than a decade.  That is outrageous in my opinion.  He indicated that much of the delay is attributable to a period between the primary care physician and getting the patient to an ALS neurologist.  I wish they would crowdsource this sometime and find the real reasons.  I suspect that could reveal some of the weaknesses of American healthcare delivery, including inept scheduling.  He showed data on interventional delay, too. Again, I wonder if that's not as much an indictment of American healthcare delivery as physician behavior.  Have you ever tried to get a riluzole prescription and then get it filled?  Benetar showed some disease trajectories and showed what many of us have also observed -- ALS trajectories are often not linear as neurologists often describe.  Some patients have a linear decline followed by a sudden dropoff.  They hit a wall.  It was nice to see some data that admit that.

The next presenter was interesting, but so help me, I thought I would scream when I heard clinical trial volunteers called "subjects."  Time to move on from that term.  And while we're at it, "controls" is a pretty disrespectful word for people who volunteer their data and parts of their lives for medical research.  Let's use references that respect all who volunteer for clinical trials as team members who are as valuable as those paid to do the research.  And while we're at it, every acknowledgement slide at these meetings would do well to thank the patients first.  I'm glad that at this meeting the importance of the data from those people who volunteer for the control group has been emphasized.

Dr. Schoenfeld gave an interesting presentation on trial design when the treatment being tested poses some potential harm in its delivery mechanism and therefore puts placebo patients at risk.  That's a big deal in the world where stem cells and other things are being placed in bodies.  He suggested that an add-on trial design could be a solution to some of the placebo dilemma in these cases.  And again the concept of an observation period in a trial came up and I ask why we aren't tracking all with ALS from day of diagnosis.

Dr. McGrath from Neuraltus spoke on the biomarker that they were tracking in the NP001 trial.  I was interested in this because some friends participated in this trial and crashed during the washout period when the biomarker was being tracked.  He showed the LPS+ data they tracked and how responders' data did correlate.  I asked about whether they would design their Phase II trial differently if they knew then what they know now.  That washout period with no further therapy was cruel to patients who had responded well during the therapy period.  He indicated that the trial design was based on budget.  They had not funds to do anything more than the six-month trial regardless of whether the drug worked or not.  OK, lesson learned, folks.  Every patient who volunteers for a trial needs to ask, "What happens to me if this stuff really works?"

My day moved on to some presentations on communication devices (while I watched Steve Gleason's entertaining and effective twitter stream on communication device comments to CMS).  There was a brain-interface presentation that interested me, and I learned that this kind of brain interface requires some gazing.  I wonder if some of the problems that people have with today's eye-gaze devices will carry forward to that technology.

I hope that all of the poster presenters will upload their data to the sharable site where we can view in more detail and with more energy than we had at the end of a long day.

Your best tidbits from this symposium will come from following #alssymp on twitter. 

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