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 13, 2012

It's Time To Let Clinical Trial Volunteers Know What's Really Going On (Or Not) In That Scrum


Today many ALS clinical trial volunteers wait.  They wait for trials to be completed.  They wait for data to be released.  They wait for the next phase of a trial to begin.  They wait for news.  They wait for  projects to be funded.  They wait for approvals.  They wait while the ALS clock ticks way too quickly for them and their families.

They can only make educated guesses about what is or isn't going on today.  They are outsiders when it comes to who has the ball, yet they are the most important members of the team.

Surely drug developers have extensive, detailed internal reports about who is doing what today to move their drug candidates closer to market.  How about sharing some specifics with the most important team members, your clinical trial volunteers?

Are we are waiting for an IRB meeting next Thursday? Are there still five trial seats that need to be filled in Omaha? Are statisticians working on the data the rest of the week?  Is a complicated FDA filing is due before the end of the month?  Is nothing really happening until the CEO finds another million dollars?

Knowing some specifics of what exactly is happening today would be a refreshing change for those who put their lives on the line for a clinical trial.  A peek into the scrum seems only fair.  It might even turn out to be good business.

Thursday, December 6, 2012

Seldom Am I This Speechless

This week the PRO-ACT database was announced.  It is wonderful that ALS clinical trial data will be shared and opened up for researchers to access. 



One of the sponsoring organizations had a big PRO_ACT blurb on its facebook page.  It certainly was a logical place for someone to ask if there might be some synergy between the PRO-ACT database of ALS clinical trial patients' data and the U.S. National ALS Registry of ALS patients' data.  After all, this is the 21st Century and data silos should be a thing of the past, right?

The exchange below leaves me at a loss for words.


The National ALS Registry is not a database of information for patients and their families.  It is a registry of virtually all cases of ALS in the United States.  It is meant to be a repository that can be useful for researchers.  It is an epidemiological tool.

There are two clear problems here --
1. The messaging about the U.S. National ALS Registry still isn't working.
2. Our ALS organizations still don't talk to each other very well.

"Watch Out! She's Gonna Blow."

It's a classic television line from Murphy Brown.  It fits my knee-jerk reaction to some reports I read regarding something presented at #alssymp -- the big International ALS MND Symposium.

There seemed to be some speaker criticism of people with ALS yesterday because of the low percentages who enroll in clinical trials.

My knee-jerk reaction to that was to want to ask the speaker to please look around the room.  Perhaps the ALS powers-that-be forget to invite people with ALS to participate in clinical trials the same way they forgot to invite them to be a part of the fabric of this symposium.

Surely clinical trial enrollment is a serious problem, but it will never be solved until ALS authorities do some introspection with the help of those people with ALS who experience inconsistent and mixed messaging regarding clinical trials.

But let's go back to my knee-jerk reaction.  It was based on a tweet and on a blog summary of what the speaker said.  I certainly appreciate the tweets and reports.  They are our only window into the proceedings; however, I really wish I had heard the exact words of the speaker... and the context.  The reports we get online come through the lens of a writer (who is often hurriedly struggling to stuff a thought into 140 characters) who works for a stakeholder.

Thanks very much to those who are providing us with their peeks into the proceedings.  Next year I hope that those peeks and insights will continue, along with some options to experience the words and contexts directly ourselves (and ask some questions).  It will make a much more robust, dynamic picture of the state of ALS research for all stakeholders.

In the meantime, if you are on twitter, I encourage you to follow @paullikeme today.  He has been outstanding in giving us a glimpse into #alssymp .






Wednesday, December 5, 2012

Look Who's Not Listening

This week a who's-who of leaders and researchers in the fight against ALS is holding a series of global meetings in Chicago.  It's a big deal.  Results of many studies are being unveiled and discussed this week.

Organizations are holding internal meetings.  Business deals are being pitched.  Scientists are networking.  "Promising" theories are being touted.

Two years ago when the meetings were in Orlando, some people with ALS showed up and learned and subsequently helped to fill clinical trials and explain some of the science to their peers throughout the year.

Last year in Sydney, patients were provided with a webcast of an "Ask the Experts" session that answered a few questions and left many more unanswered.  At least there was a minimal webcast provision that acknowledged that there are people with ALS who have skin in this game.

This year in Chicago the "Ask the Experts" session was not webcast.  An estimated 150 healthcare professionals and around a dozen people with ALS attended per a tweet from @alslesturner .  This in a city that is within a couple hundred miles of a huge patient population.

Tweets (hashtag #alssymp ) and sporadic online postings are our only window into the proceedings.

For a person to attend the symposium in person (other than the Ask The Experts session) required a substantial registration fee. There was no discount or waiver for people with ALS.  There was no encouragement for people with ALS to be engaged in person or from afar.

Here are a couple of tweets that we saw from organizations attending yesterday's Allied Professionals' program --

  • @mndaustralia Ruston Australia: Communication Technology: 88% of respondents use internet 'to be disabled without technology is unthinkable'
  • @alstdi Gudjon (Iceland MND Society) "stay in touch with patients, they (we) are the specialists in this condition". 
So technology is king for those dealing with ALS and our organizations recognize the critical role of people with ALS as the specialists in their condition.  Such wisdom uttered from a bunker inside of a Sheraton with no webcasts and no welcoming patient participation seems a little odd, eh?

Every day people with ALS and their loved ones search for the newest and best science.  Every day they encounter challenges that scientists don't even realize.  To have these two groups separated by a "Do Not Disturb" sign is unproductive.  Let them engage with each other directly, please.  Let them learn from each other directly.

Next year, please webcast.  Please encourage people with ALS to be there.  Please allow for their questions to be answered. These are bright people who want to learn and be a part of the solutions.  They are not the unclean masses.  They rely on technology and are the specialists in their condition. 

After all, it's all about the patients, right?