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.

Thursday, December 4, 2014

#alssymp Day -1 -- Ask the Experts

For the next few days I'll share impressions as I attend the big global ALS MND Symposium.


The events involve a complicated (at least for me) series of "insider" meetings for organization employees and healthcare professionals.  The culmination will be three days of research meetings for which anyone who can put up the substantial registration fee can register.


Yesterday there was also a public opportunity -- An "Ask the Experts" session held by the host ALS organization.  I knew about the concept from past symposia and asked if I could attend.  Local patients and caregivers are the main audience.  There was no webcast this year.  When I inquired, I was advised that they would accept submitted questions, and I did so on behalf of several people with ALS who participate in an online research forum.  See the blog post dated November 30 ("What Should I Talk About").


An email advised me that Ask the Experts would start at 2pm yesterday.  When I got there shortly before 2, the crowd was sparse.  A few families were there with members who appeared to have ALS.  People trickled in and the session itself didn't start until 2:50.  Fortunately my phone battery was good and I could still tweet.  They provided video and audio translations from the presenters' English to Dutch and French.  My crowd-size guess is around 50 people with around a dozen people with ALS.


The format was one that they had used in the past.  There were three scientists --
Dr. Hardiman (Ireland)
Dr. Van Den Berg (Belgium)
Dr. Bruijn (US)


Each gave a presentation followed by a very short question and answer period.


Dr. Hardiman
Presentation was around the mouse model.  Not much new. She emphasized the cost of human trials, mentioning that the recent Dexpramipexole trial that plopped cost $100 million.  A rather discouraging note was that good drugs fail because of flawed trial design. The solutions cited were cleverer subgrouping and subtyping making more personalized trials.  Tricals is an ENCALS group which is working on better trial matching to patients.  She had a really nice bubble chart showing the various known genetic variations in ALS.


Q&A
Questions -- Why not have individual trials?  What about compassionate use?
A (VDB) -- If you have compassionate use, then you can't do trials.  Without a biomarker we can't measure efficacy in a very small trial.  If in a small trial everybody got better, then compassionate use wouldn't be a problem.
A (Hardiman) -- Lithium was an example of something that people thought might help and the trial proved it hurt.
My thought cloud -- Lithium was actually a great example of public data showing that the drug didn't work for ALS far before the formal clinical trial was finished.


Van Den Berg
He spoke of Project MinE and identifying the spectrum of disease variants and associated risks of having genes vs risks of getting the disease.  The goal for Project MinE is to sequence 15000 people with ALS and 7500 controls.  He made some very bold statements about combining all ALS biobanks in the world, using standard formats, etc.  I hope he's right, but I wonder if it's wishful thinking.  The lure of volume pricing isn't going to be enough to get some researchers out of their silos in my opinion.
Q&A
Question -- What are you doing with all this information.   Are you just collecting it?
A (several) bridged the conversation to iPS models and the ability to screen drugs against a lot of variations of ALS.


Bruijn
The four main IBC research grants were described.  There was a reference to "ALS Association Research Institute" which sounds grand but I think is just a title to get all their "prongs" to sound more like research.  The big news I heard was that the four IBC granted projects will use a GUID as a patient-level identifier.
Q&A
Question (me) -- Is anybody using GUID for control groups to make them more rich and versatile?
A (several) -- Got a lot of feedback on the importance of control groups and heard a foggy from Bruijn that they would look at that.  Hardiman thought it would be unethical since you have to deidentify control data.  I think that GUID is considered to be deidentified.
Followup Question (me) -- Is anybody tracking the control group members to see if they develop ALS?  (I think I detected an eyeroll from VDB?).


Question -- Nobody has discussed stem cells.  Why not?
A (Bruijn) -- That wasn't within the scope of her prepared presentation.  It's not because they are not important.
The discussion livened up a little and there were some references to concerns in Europe, are stem cells the answer, etc.  This was a topic that patients obviously wanted to hear about and discuss.


And time ran out.


There was no indication that the experts got the questions that were presubmitted.


Maybe next year the host organization will make this session more productive and patient-focused and accessible.  Ask PALS, CALS what format works for them.  Get the questions that they want answered and then have some lively discussions (like the one that was just starting on stem cells when time ran out). Presenters were all fighting the clock trying to use presentations that were too long and not much discussion was stimulated. There are better ways to do this.


The patients and caregivers really are the experts.















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