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, August 23, 2015

Is It Wrong To Yell "Fire Extinguisher..."

...in a crowded, burning theatre where the doors are all locked?

Of course not if you have a fire extinguisher in your hands and are trying to clear a path to help.

It would be very wrong if you have a great blueprint for an awesome  extinguisher (that you consider to be a "breakthrough" in snuffing fires)  and a successful experiment and a patent application but have yet to go through rigorous testing, certification, approvals, pilot production, or production.  You would set a really cruel expectation amidst a lot of people facing death.

Last week was an interesting one in the fight against ALS.  We had already seen the news published August 7 about TDP-43 in Science https://www.sciencemag.org/content/349/6248/650.abstract

That was certainly another good scientific step forward.

In a tenuous attempt to link the scientific good news to last August's ALS Ice Bucket Challenge, we started getting crafty statements.   What better way to encourage donations this year than to associate it with a recent advance in understanding ALS?

Media coverage was being cultivated, and this makes a feel-good story about the marvelous, fun phenomenon of August, 2014.  It makes a wonderful feel-good story.

Then there was the Reddit AMA exchange with one of the paper's authors followed by some national interviews.  A tie of the #ALSicebucketchallenge to this project was emphasized.  Wow.  We did that with the ice, eh?  A young scientist almost seemed giddy with enthusiasm about the significance of his discovery.  Double-wow.  And this was emanating from one of the finest academic medical institutions in the nation, Johns Hopkins.

As Answer ALS aptly described in a statement on Facebook, "The headline seen this evening had the phrase 'A Cure for ALS Could Be Coming Soon.'  As the last few days have gone on, the headlines seemed to create themselves... "




And for families craving optimistic sound-bites as they deal with ALS, were they ever getting the optimistic sound-bites, right from the scientist's mouth.


And the ecstatic news went on and on.  Mission accomplished?


Perspective, anyone?

  • When did this work start?
  • How much did the project cost and exactly who supplied what funding? 
  • What are the next steps?
  • How much money is needed for the next steps?
  • What is the most optimistic timeline for the next steps?
  • What are the barriers (scientific or regulatory) to bringing this finding to a therapy?

That clarity and perspective will help us all to understand not only the significance of the findings but also what we all need to do to help bring those headlines to reality.  

Maybe we need to talk about patient-centric p.r. and fundraising.  A respected medical institution needs to celebrate its achievements and raise funds with respect for the patients and caregivers and loved ones who are trapped in a burning theatre.  They just heard "fire extinguisher" on the national news last week.

Wednesday, August 19, 2015

Say What?

It started on August 13 with a tweet from an ALSA executive, "#ALSIceBucketChallenge donations have led to a significant ALS research discovery! bit.ly/1DQByYZ "

Wow.  Impressive.  Holy cow.  That was fast.  Say what?

The press release was very carefully couched and didn't quite mesh with the cause-and-effect excitement of the tweet, but the seed was planted.

Then it continued with organizational tweets with wording that matched exactly.


Amazing. Remarkable. Cool.  Say what?

Then yesterday there was the ALSA mass emailing -- Subject line: "This is amazing news!"


Yes, amazing.  Fabulous. Incredible.  Say what?

The cause-and-effect implication is clear.  IBC donations to ALSA led to this discovery?  That's fast science.  How refreshing.  Say what?

We didn't get a good list of  #ALSicebucketchallenge checks written to researchers last year. We saw numbers related to committed funds.  In these lists of funded research, I don't see this project listed in the 2014 file but Dr. Wong is listed as a $160,000 grant recipient in 2015. 

The full research paper on this "significant #ALS research discovery" sits behind a paywall.  My curiosity won last night.  I bought access. Let's see how this breakthrough was funded with IBC funds, was discovered, was written up, was reviewed, and was submitted to a journal between August. 2014, and March, 2015.  That's cool fast science.  Say what?

Acknowledgements at the end of the paper included that the project was funded in part by
  • The Robert Packard Center for ALS Research
  • Muscular Dystrophy Association
  • The Amyotrophic Lateral Sclerosis Association
  • Target ALS
  • The Johns Hopkins University Neuropathology Pelda Fund
  • The Johns Hopkins University Alzheimer's Research Center (NIH P50AG05146)
  • The Samuel I. Newhouse Foundation

Unfortunately we don't know how many dollars were involved or how much each agency contributed or the dates of the grants.

The paper itself is complicated and involved experiments and evaluations on mice followed by more work on deceased PALS' brains.  There isn't a timeline but it would have taken a miraculous change in the ALS research paradigm for any substantial amount of this discovery work to have been carried out in Q4 of 2014.  I would be amazed if the illustrations alone could have been made and approved in six months, let alone all of the underlying work.

And there was even mention in the paper of a patent application filed by two of the authors. 

So the "Say what?" response keeps going off when we read the tweets and the emails.  How can this be?  Is it a deceitful way to make people feel good about their last year's IBC generosity so they'll do it again?  Or was there really a scientific funding-to-discovery-to-journal miracle in six months?

People with ALS and their caregivers have been pressing hard for the FDA to speed up approvals. They get pushback from organizations including ALSA that good science takes a long time.  Then we get self-serving fundraising messages that our donations led to a "breakthrough" in under six months in some remarkable fast science.  Say what?

People are dying.  They deserve some clarity.

If my "Say what?" reaction was out of line, I'm all ears.

Monday, August 10, 2015

"Do You Know What The Worst Part Is?"

#whatwouldyougive

That's a movement that originated with @speed4sarah.  You can read up on it on her blog http://www.speed4sarah.com/sarahs-blog/

I decided to give it a try.  I would try to give up something that ALS takes from people.  It would raise some awareness.  It would raise some funds.

One thing that really stuck with me when Mom had ALS is that we take so much for granted -- taking a sip of water without choking, working a door lock, talking to one another.  There was such a spectrum of things that I could "give up" for #whatwouldyougive.

Mom still had good grip strength during her ALS but it was terribly difficult for her to lose the dexterity in her fingers.  The inability to work a lamp switch or button a button was so frustrating for her.  More on that later.

I set my #whatwouldyougive bar very low.  I didn't want to fail.  My left hand in a ski mitten  for a day shouldn't be that tough.  I'm right-handed.  I picked a weekend day when I wouldn't have to do my day job. I didn't want to have to ask anyone for help, so I kept it simple.  One free hand would work.  I would be safe driving.  The bar was so low that I felt like a sissy.

Enter Sunday morning.  I put the mitten on first thing.  Immediately I realized I couldn't scratch the dog like she expects every morning.  Not a big deal, right?  On to fix my toast and tea.  It wasn't pretty when I realized that I could not slice my toast the way I've sliced my toast since I was six years old and learned to make my own toast.  Thirty minutes into my experiment, I said, "Boy, I'll be glad when this day is over."  Ninety minutes in, I was totally frustrated.  I wanted my old life back where I could slice my toast my way.

I've broken my right wrist twice in my life and I was never this frustrated (there's that word again).  I decided that immobilization feels good when something hurts and there is healing going on.  That's not how this was.  It was just exasperating.  I couldn't clean my eyeglasses.  I couldn't floss.  My right arm itched.  It was incredibly frustrating.

For my sanity, I decided to try some distraction and went to the grocery store.  It went well until I needed a produce bag.  Ah, plastic bags were my bane all day, at the store, in the wastebasket, on the dog walk.

By late morning I was really hungry and had no desire to frustrate myself more by cooking.  I stopped in a local restaurant that was still serving breakfast.  It was one of the best experiences of the day.  We talked about the mitten and the hashtag and they fixed me a good breakfast that required no knife dexterity.

I had been making a list of the day's challenges and finally stopped listing them.  We take so much for granted.

The first time Mom asked me to help her button her blouse, she asked, "Do you know what the worst part is?"

No, I didn't.

"When I've lost the ability to do something, I know I'll never do it again."

She had buttoned her last button.

At the end of my experiment I took off my mitten.  My life goes on.  My toast is right this morning.  And how many mothers with ALS buttoned their last buttons yesterday?

_____

Please set a challenge for yourself and try it.  Low bars are fine.  Trust me, it's not easy.  And then support https://www.razoo.com/story/Sarah-Coglianese-Fundraising-For-Whatwouldyougive

Or give directly to @speed4sarah 's research cause at www.als.net 
Or give to www.teamgleason.org that supplies technology to those with ALS to help compensate for those abilities that ALS steals.
Or do all of the above.  After all, we take so much for granted.
Thanks.





Wednesday, August 5, 2015

Let Me In, Whee-Ooh

"Let Me In"
The Sensations - 1962
Let me in whee-ooh (whee-ooh, whee-ooh, hoop-whee-ooh) 
(Whee-ooh, whee-ooh, hoo-ooh-oop-whee-ooh, whee-ooh) 

I can see the dancin' (let me in) 
The silhouettes on the shade 
I hear the music (music), all the lovers on parade 
Open up (let me in), I wanna come in again 
I thought you were my friend 





The big ALS symposium is coming up in December.  Already we're seeing news of abstracts accepted and discoveries that will be presented.

The registration fee of approximately $600 goes up even more after August 19.  http://www.mndassociation.org/research/international-symposium/registration/

There are people with ALS and their loved ones who are interested in good science.  They spend countless hours searching.  A gentleman I knew with ALS attended the symposium in 2010 and found an interesting drug candidate and enrolled in the clinical trial as soon as it was available.

Why would we not want people with ALS or their loved ones at such a symposium?

Why would there be so many barriers between PALS and this festival of information about good science?

The fee without any consideration for people with ALS and caregivers is one of the big barriers.

The lack of welcoming information for those who aren't insiders is another.

The spirit and letter of the Patients Included movement needs to be in the fabric of every ALS meeting, especially the premier scientific symposium.  It's past time for this meeting to be physically and virtually accessible to every PALS or loved one who is interested.