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.

Sunday, November 15, 2015

Driving Ahead, Leadership, Risk, and the Importance of the Rear View Mirror

Last week I attended some of the ALS TDI Leadership Summit events.  It is clearly the most
welcoming conference for people with ALS and caregivers who are interested in science. Access to a variety of scientists, especially during break time and lunch, was outstanding.  Many of the conversations were candid and unfiltered, and that's a refreshing concept in the fight against ALS. Some miscellaneous impressions follow.


  • In the last decade it seems like we've gone through a full philosophical cycle -- From trying to understand a complicated disease to trying to throw things at it regardless of the rationale (just put out the fire) back to trying to understand the many things going on with ALS (figure out if it's a grease fire or an electrical fire).
  • The people with the most at stake in the fight against ALS don't have the benefit of a lot of institutional history.  Those with ALS and their loved ones are largely new at it.  Few with ALS live long enough to remember all that had been tried in the past, and organizations are not good at keeping the loved ones engaged for the long-term.  This provides some convenient blind spots when some of the science is explained.  
  • Many promising drugs have been screened and tested over the last decade, and there is an ever-growing dud pool.  Knowing about the volume and variety of duds is helpful to put the difficult road ahead into perspective.  And we have to be realistic that today's "promising" may well end up in the dud pool.  That doesn't mean stop.  It means that we need to smarten up and shorten the route.  
  • Fundraising is essential, and it's a lot easier to raise funds based on "promising" than it is looking at a rear-view mirror full of expensive duds.  Are we distorting the difficult road ahead by doing what's best for fundraising?  
  • People with ALS want to assume more risk, yet the scientists and clinicians aren't likely to step forward and lead that fight (or even lock arms with them as one man with ALS suggested).  I'm more convinced than ever that patient-led movements will cause the change in this area.  Organizations simply won't take the professional, public risks to be that bold in a heavily regulated field.
  • Having a gentleman with professional and academic chops on the afternoon panel was priceless.  The lithium story is one that has been subject to a lot of revisionist history.  Sometimes the establishment tries to tell that story in a way that makes the patients look gullible.  We had a person with ALS remind the panelists that the patients were the ones who showed that lithium didn't work.  People with ALS were the ones who begged everyone to stop wasting money on trials.  They should be respected for what they did.
  • We heard that small ALS trials are largely destined to fail. It sounds more credible when panelists say it.  Some people with ALS have been saying it for years.  We even heard that at one time some clinicians wanted a very small lithium trial destined to fail to make that problem go away.  There was some refreshing candor from Dr. Katz about the hype of some clinical trials.  At the end of that discussion on trial design, some of us in the audience had a serious question as to whether there is anything currently worth the investment in a clinical trial?  And I wondered what would happen to fundraising today if an organization would honestly say that there is nothing currently worth that investment?  

Depressing?  Not at all if you acknowledge the rear-view mirror and decide to take some new roads forward.   We need to learn not only from the duds but also from slow, inefficient processes.  

The father of a remarkable young woman with ALS asked if we are letting the perfect get in the way of the good.  That sums it up.  We need to forge ahead with admission of what's in the mirror and a willingness to take risk ourselves in many new ways so that people with ALS can take the risks that they accept (with eyes wide open) in order to move the science ahead faster.



Saturday, November 7, 2015

You Wouldn't Think Of Letting That Elevator Door Close


Especially when the waiting person is in a wheelchair.

Yet we close the door in the faces of those with ALS all the time.

Here's an example from twitter today.


It was an interesting article on the virus theory that has been in the news lately.  A woman with ALS responds below.  She's interested in the trial cited in the article.  The article failed to contain trial contact information for those who actually have ALS.


On a long shot, I searched clinicaltrials.gov.  Odds were that I wouldn't have the right keyword for the products involved and wouldn't find it.  I got lucky and found it.



Everything is hard for those with ALS.  Life would be so much better for them (and quite frankly, trials would be filled much sooner) if people would simply make sure that they're holding the door open for those who don't need more barriers in their lives.

After all, they are why you're doing the science that is published in the first place.


Friday, November 6, 2015

It's Much More Than A Game

I live in an NFL city that has a team that set high expectations and has performed terribly this year.

Everybody has an opinion about the problem --

The GM is the problem.
The coach is the problem.
The offensive coordinator is the problem.
The QB is the problem.




And with every assessment of "the problem," there is an expected action that someone will be let go and changes will be made.

I live in a world of ALS that has teams that have set high expectations and have yet to deliver a big win.

It's much more than a game.

Do we ever assess "the problem" and take expected actions?

Ever?

Thursday, November 5, 2015

There Really Is Educational TV

Last night Charlie Rose featured a segment with Dr. Collins of the NIH and then another with PALS Jay Fishman and Dr. Rothstein.  It was late.  After a few hours of sleep, a few very random thoughts are stuck in my mind.

  • NIH funding is precious.
  • Why were NIH funds needed for this new report?  ATSDR has generous funding for the Registry.
  • The delay to diagnosis for ALS is outrageous.  Charlie Rose grasped it.  Jay Fishman lived it.  Dr. Rothstein didn't want to cast aspersions on the fellow physicians who fail to diagnose it. That delay to diagnosis is costly in many ways, and the patient is the one who pays the price, yet we give physicians a professional free pass when they miss it, and they often miss it.
  • The ALS Ice Bucket Challenge missed opportunities.
  • We need better accounting and accountability in the fight against ALS.
  • Money won't cure a disease that is short on new ideas.
  • The cure isn't "around the corner."  I took that to mean that Dr. Rothstein has no impending  treatment for Mr. Fishman.  I appreciated the candor.
  • Too bad the conversation didn't then jump on shortening "the corner" by getting experimental drugs to PALS much more quickly.
  • Charlie Rose is on too late in my city. 
  • Jay Fishman has an attitude that reminds me of my mother when she faced ALS.

We need to have more serious conversations and more action to change the status quo.  Thanks, Mr. Fishman.