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 21, 2017

My Random Walk Through #ALSSYMP

This was my fourth International Symposium on ALS MND.

After a couple of weeks, a few things have stood out to me to distinguish this one... and a few other things that remind me of the same old same old that needs to change.

The Symposium itself has some preliminary undercards to get us warmed up for the three days of hard-core science.

Ask the _______

They call it "Ask the Experts.  That name needs to change.  Actually, the event format itself could use some change.  Last year in Dublin the three neuroscientists on the panel gave crisp, timely, short presentations followed by time for Q and A.  This year the format reverted to that of my first two experiences where the neuroscientists repurposed longer presentations and talked too long, questions that were pre-submitted via email were never addressed, and the session finished with unasked questions still in the room.  It happens on a day that is convenient for the meeting schedule, but most inconvenient for people with ALS and caregivers who want to attend the three days of scientific symposium, too.

One of my questions made it to the panel -- What would it take to get fairly inexpensive potential biomarkers like serum creatinine or retina thickness (that have been suggested for years) to a definitive "yes it is or no it isn't" decision?  The answer was very vague.  Again, later in the scientific symposium, creatinine came up, Vitamin D came up, TSH came up.  These are not expensive things to test, but nobody is stepping up to come up with a definitive answer.

ENCALS Meeting

I appreciate that this meeting of European researchers the afternoon before the start of the scientific symposium was open for anyone to register and there was no charge.

It was enlightening.  One presentation from Dr. Tang took old Rilultek data and mapped it to the Kings ALS Staging.  Contrary to my intuition, the responders were in the Stage IV group (most advanced).  That seems to me to be a big deal as people with ALS choose among a very sparse set of treatment options.

ALS Patient Fellows Dinner

This year a program was started thanks to the help of ALSTDI and PatientsLikeMe and some motivated individuals to offer fellowships to the scientific symposium to people with ALS and caregivers.  They submitted applications showing their willingness and ability to participate in the science and move some patient voice closer to the scientists actually working in labs and clinics.  The patient fellows were fabulous -- smart, informed, insightful, challenging.  Our dinner was a highlight as we got acquainted in person and talked about the symposium and ALS in general.  Special thanks to Rob Goldstein and Paul Wicks.  I think that their later interactions and questions at the symposium proved the value of reducing barriers (such as the substantial fee for patients and caregivers) to have them be part of the fabric of the symposium.

28th International Symposium on ALS MND

The abstracts are all available online for those who want to dig in.  https://www.mndassociation.org/symposium/programme/abstracts-online/

The keynote from Dr. Rosenfeld was a refreshing dose of cold water in our faces.  He suggested some significant paradigm changes at defining ALS.  He even suggested that we have failed in the past, so it's time to change.   Unfortunately the whole paradigm shifting idea seemed to be lost after the next coffee break.  We fell back into our same old same old ruts.

We started seeing the use of the word "subjects" for people participating in ALS research in the first prize-winning presentation.  Oops.  It was better than in the past, but we still see that word used by far too many neuroscientists who should know to have more respect for the most important people on their clinical research teams.

A few of the typical acknowledgement slides at the ends of presentations included people with ALS.  Even fewer had them at the top where they belong.  Something so simple, so respectful, so appropriate is so difficult with this group.

During a panel on telemedicine and precision medicine, we heard again of the silos in precision medicine programs.  If this were a corporate shareholders' meeting, the crowd would have risen to say, "Fix the silos. Now."  This crowd nods and moves on.  I had my hand up for the "Fix the silos now" suggestion but time for questions ran out right before my moment.

A neurologist asked about how they would get paid for telemedicine.  Surely a practical question, but certainly if more people with ALS were present, it may have not been on the front burner.

At a breakout session (I was not present), one of the patient fellows evidently asked a mic-drop question to a panel about the cost/value proposition in comparing supportive care for vented people and expensive meh drugs for ALS.  You can consider his thoughts on this and another important topic, placebo groups  here.

And speaking of placebo trials, we saw to instances where placebo groups seemed to do relatively well.  Overachieving placebo groups spoil the spotlight for the drug.  So... live by the placebo sword, die by the placebo sword?  I wonder if randomization put the real drug in the hands of those who were in the overachieving placebo group if a meh drug would have been found to be wildly effective.

I learned the term "enrichment strategy."  It's a way of picking trial participants to make the drug more likely to show that (or look like) it works.

In the session on recent clinical trial results, it was a lot of "more study needed."  To my eye, either the drugs aren't working or the trial designs aren't working.

There was a chippy moment when a well-known neurologist suggested that reproducibility of trial results was needed for edaravone.  The drug company doc didn't like the suggestion.  Chippy moments are a lot more constructive when we have good discourse and not indignation.

There was a panel on Right to Try, Expanded Access, etc. that included an ethicist, an entrepreneur,

and a neurologist.  Does anyone notice who is missing in this discussion?  Dr. Bateman-House from NYU was quite good, and I appreciate her tweeting answers back to me while she sat on the panel on the big stage.

I saw one presentation that I didn't think was very solid science, and it was an area where I feel I have some background.  A neurologist who is vested in the project got the mic at the end and said, "Very nice work..." before his question.  That spoke volumes to me.

An ALS advocate at the symposium mentioned more than once that she got the best information from symposium participants at the hotel bar.  That's true sometimes.  I learned of a study on lithium that showed actual efficacy in a specific subgroup.  I saw a communications technology expert help one of the patient fellows who had been led down the wrong technology path.  Much goes on outside the sessions here.  I even learned on an elevator that my tweets about the word "subjects" are useful in educating students to avoid that term.

There was another mic-drop moment in a large session when a man with ALS asked a presenter if people with ALS were included in the study design process.  Nope.  Awkward.

There was much, much more.  Poster sessions were not as accessible as they had been at past symposia, and I felt like I missed a lot of material and good conversation there.

I realize that it takes far too much time and effort to implement even the simplest changes in the fight against ALS. I feel like as much as we rely on placebo trials, they aren't so stellar that we should stop looking for alternatives.  I feel now more than ever we need to embrace people with ALS and caregivers who are interested in the science at these symposia.  And I am especially grateful for the inaugural Patient Fellows who made the trip and added to some important conversations.


Late addition:  Oh, and take your Vitamin D.  One study it as a possible biomarker to disease progression.  More study needed.  A neurologist asked a refreshing, common-sense question -- "Why not test people with ALS for Vitamin D deficiency?  It's not expensive.  Vitamin D supplement is low risk.  Just do it."  At times like this, if all neurologists exercised such common sense and reported findings, we could figure out if it's a helpful biomarker or not (and perhaps help some people with ALS along the way).








Sunday, December 3, 2017

Meet The ALS Clinical Research Swear Jar

Last week on a webinar, I heard a clinical investigator puzzled at the low enrollment in a particular trial.  How about dropping the word "subjects" for the most important people on your clinical research team, Doc?

Nobody aspires to be called "subject."  It's not respectful.  It's objectifying.  It conjures up images of royal underlings.  People who put their lives on the line to advance science deserve respect.  Words matter.

Reference to "subjects" has long been banished in many disease areas and industries.  ALS researchers don't seem to have gotten the message.  Maybe this will help.  Let's have a jar where ALS scientists have to drop a Benjamin every time they slip with the "s" word for a valued clinical research participant?

ALS MND Symposium presenters, use a more respectful word or have your wallets ready.