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, October 21, 2021

October 20, 2021 -- Remember The Date

On Wednesday, October 20, a group of people with ALS and caregivers who dub themselves "ALS Problem Solvers" met with nine top NIH NINDS leaders to discuss "Five Things That Will Change Everything."

It wasn't just another meeting.

This was a long time coming, and each of the ALS Problem Solvers knew that the stakes were big.

Over a year ago, Gregg Gonsalves had been on a panel at the NYU conference on pre-approval access to therapies.  As I often do, I asked a question of the panel about how ALS can benefit from such things that are so common for other diseases. Expanded Access Programs are like unicorns in ALS. At the break, I heard a voice behind me as I sought a cup of tea in the foyer, "I want to help."  It was Gregg.  And he has since been generous with his help.

Shortly after that conference, a group of people with ALS and a few caregivers started brainstorming on how we can move ALS research out of its ruts. It seems like other diseases move ahead with concepts and tools that are unused for ALS.  We started meeting on alternating Saturdays with Gregg.  We did a lot of homework.  We did everything good workgroups do, including celebrating some ideas and arguing about others, but we moved forward.

We homed in on the fact that the NIH has implemented basic, successful solutions such as a natural history study for other diseases that are not done for ALS.  Why not ALS?  Why not us?  

For decades ALS has been like the orphan disease that has truly been orphaned by some basic aspects of scientific research.  We were determined to change that, and Gregg encouraged us to make our case directly to top leaders of NIH NINDS.  Directly.  Make the case.  We were on it.  We researched.  We wrote.  We rewrote. Assert nothing without citation. We criticized our own work. We researched more.  We knew we needed to be on solid scientific and academic ground. We wrote the document, "Five Things That Will Change Everything," that described the problems and the solution -- Five cogs that working together would change everything in ALS research.
  • A Natural History Study
  • A Broad Network of Clinical Trials
  • A Framework for Broader Insights - A Parallel Track
  • NIH Leadership
  • Our Relationship

On September 25 our document was presented to Dr. Walter Koroshetz, the head of NINDS, along with a request for a meeting with the ALS Problem Solvers.  He accepted almost immediately and October 20 was the date.

The preparations were intense. Six people with ALS and four caregivers would speak. These words would be short and pithy.  They would reinforce the cogs with life experiences that showed the need. We practiced, we refined the message, we practiced more.  We rehearsed challenges and questions that NINDS might pose.  We were prepared.

We also got the help of others.  Two top ALS neurologists joined us.  Two professionally accomplished friends of one ALS Problem Solver pitched in and made significant contributions to our presentation.  We had a supportive statement from Senator Murkowski who knows NINDS leaders well.  

Along the way we had a few disagreements and a lot of fun. We were prepared.

The meeting was to start at 5 o'clock yesterday.  The ALS Problem Solvers were online fifteen minutes early, all clad in our blue t-shirts with the five cogs.  We were prepared.  At the stroke of five, the waiting room started to fill with NINDS names we knew and a lot of names we didn't know.  It was time to roll.  

Dr. Koroshetz introduced the eight others from NINDS who were there.  He brought the whole a-team.  That was good.  We got some positive feedback on the document.  They had read it.  The reaction that it was a good mix of science and human experience was encouraging.  And the t-shirts even got their attention.

ALS Problem Solver Danielle started the presentation and what we had practiced so many times was 
finally being said to NINDS staff.  They were engaged. Gwen hit it out of the park with her problem statement bolstered with life experiences. Tim spoke to the natural history study as he does so well.  Becky and Glen hit the marks on the need a broad clinical trial network. Sandy's comments on a parallel track were spot on.  It was my turn to deliver the visual on the first three cogs working together. I remembered to unmute. Phew. Caregiver Bob hit the importance of NIH NINDS leadership to make all ALS research more effective, then Phil brought it home with compelling words on teamwork and our direct relationship with NINDS.  The two top ALS neurologists offered supportive comments. The action items were up next.  We were asking for some things that were easier than others, but we knew we had to be specific.  

Now it was NINDS turn to tell us what they thought of our proposal.

We knew that there would likely be concerns just because of government process and bureaucracy, and there were.  We heard that NINDS has a sweet spot with ALS in basic and pre-clinical research.  Yes, and we are grateful for all their work in those areas.  They emphasized that the lack of understanding of ALS makes clinical research risky and prone to failure.  Yes, we understand that.  

We had about 15 minutes left in our one-hour meeting, and I was starting to worry that we were not going to get any specific next steps out of this, but something started to happen.

The concerns about the lack of understanding about ALS that makes clinical research so risky really made the case for a natural history study.  NINDS shared that there was a natural history study with 800 people. I was puzzled.  Then in the chat, we got the thought from one of the neurologists that perhaps CReATe could be expanded.  Ah, CReATe -- I knew that as a study strictly for familial ALS.  Great. People are thinking.  Perhaps we already have an NIH asset that can be leveraged as a large and broad ALS natural history study for everyone with ALS.  Now we're thinking.

NINDS offered us contact points from their meeting participants for each aspect of change we are requesting.

And we have an opportunity to participate in an upcoming strategic planning process.  That will take more time than we want, but we will be there and direct voices and brains of people with ALS will influence those conversations.

And our foot is in the door in a very good way -- NINDS working directly with these people with ALS and caregivers.  ALS Problem Solvers proved that people with ALS belong and can contribute to the science.  This is a new model for co-producing with NIH, and it could not come at a better time.

And as Gregg reminded us last night, the hard work is just starting.

We're ready.

We're grateful.

October 20. Remember the date. It was an important day.


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