Hi, I'm Sandy's Friend, Cathy

 We lost Sandy Morris to the stinking ALS this week.

As many of you know, she was a huge influence for the better in what we called ALS Land.  If you didn't know her, just google "Sandy Morris ALS."

I've been working at ALS advocacy for a quarter century.  I'm just one of many, and I work hard pounding away at the boulder.  Sandy made the boulder crack.

Sandy made everyone involved in the fight against ALS better, from us simple advocates to top researchers and heads of state.  People knew her and admired her and listened to her because she was right and did not cling to anything of the past as good enough.

Keep in mind that people come along in ALS Land and push the envelope all the time, but seldom do they make a significant dent in anything.  I knew Sandy was the real deal on July 12, 2018.  ALSA held a meeting in Washington to talk about the FDA ALS Guidance (again).  Many of us were tired of talk, but a group of esteemed scientists and people living with ALS were assembled and talked some more.  During a Q and A a woman from California called in and asked why we didn't just play the transcript from the February 25, 2013 hearing on ALS because we were just rehashing everything that had been said then.  Boom.  I knew that Sandy Morris from California was the real deal.

Many of us miss her terribly this week, mostly as a friend, but also as a leader.  Sandy knew how to run a meeting.  She had charisma, but she didn't depend on that.  She always had an agenda.  We always started on time and finished on time and every person understood the homework to be completed before the next weekly meeting.  Sandy had vision and the human skills to manage projects to move to that vision.  How we miss her.

The tangible accomplishments Sandy delivered are immense, and nobody paid her a dime to do what organizations and staffs have never been able to deliver.  Expanded Access started to happen in ALS Land because of Sandy.  She studied, always wanting to have her facts straight, then she acted with purpose.   Legislative efforts were effective because of her relentless efforts.  And drug developers started to design more humane trials because Sandy insisted and taught us all how to insist.  And the Morris ALS Principles will set the tone and boundaries that have never been established before for those dealing with ALS.

I'm very happy working behind the scenes and it was a treat to help Sandy. I go to conferences with top ALS researchers, and most are oblivious to me.  I learned a couple of years ago to introduce myself, "Hi, I'm Sandy's friend, Cathy."  Suddenly they pay attention. Sandy and I joked that my name is now officially, "I'm Sandy's friend, Cathy."  

Sandy was also a talent scout for ALS Land.  She had people skills that brought some immense talent into the fight to work along side her.

We have a huge void in ALS Land this week.  One person can't fill it.  I just hope and pray that we can all chip in the talents we have to keep making cracks in that boulder that Sandy started so well for us.

We muddle ahead in a grief fog this week, but we have homework that is due next week.  And we must deliver.

Sign Here

We sign forms.  We agree to things in order to get treatments, both approved and experimental.  It's exactly the same thing as signing forms in order to rent a car.  There is a lot of fine print and there are things in that fine print that we don't particularly like (if we have the time and eyesight to read it), but what can you do?  The form is a barrier between you and what you need.  You sign to get past that barrier.  You have no power in the deal when you are at the car rental counter.

Your health data, especially when aggregated with the data of lots of other people, are of significant value to medical researchers.  Imagine how much Company B would like to have data on people taking Company A's product.  The blood tests and clinical data observations over time could be priceless.

Let's go back to those forms you sign.  People with ALS are encouraged more and more to get genetic
testing and counseling.  You sign forms to do that, and the company provides your data to you.  Great.  But what do those forms say about what happens next to your data?

People with ALS sign forms to receive treatment and to participate in research.  Wonderful. But what do those forms say about what happens next to your data?

Most people with ALS want their data shared broadly, and that certainly happens in some situations.  In other situations, their data become a commodity.  If data are eventually sold to the highest bidder, is that not having the opposite result of what a person with ALS intended?

We need to talk more about this problem now so that we can influence what is on those forms. People with ALS can use their power to make sure that they are not voiceless pawns in someone else's business model.

And we need to talk more about the role of agencies like NIH to help us with solutions to neutralize this problem.  If we had a robust NIH natural history that was a "come one, come all" destination for people with ALS to contribute their data, we could be assured of open and equitable access to data that would advance the science rather than advance a few investors' bank accounts.

Open the @#$%^& Curtain

Today we wait.  We wait for some therapy developers to do whatever they're doing.  We wait for regulators to do whatever they're doing.  We sometimes don't even know for whom we are waiting.  We just wait.  

The work goes on behind a big, heavy, black curtain.

Imagine a big corporation designing and delivering a new car. There are dozens of sub teams on the project and there may be contracted engineers and test drivers.  Government regulators having a role to play, too.  Do you think that any of those project members gets to go hide behind a big black curtain and do whatever they do, only to come out whenever they get finished?  Do you think that the team is hidden from what others are doing?  Do you think that somebody who needs more time just gets to take more time and tell others to wait?

Ha.  

Even airlines figured out that a flight delay without a reason is disaster for their gate agents.  Tell the waiting stakeholders what is going on.

In the often dysfunctional world of ALS, we have therapies being tested, analyzed, and evaluated for approval.  Is anybody really back there today?  We have no idea of what is going on.  We just wait.  And if nobody is actually back there doing something, we don't know if we're waiting for something that will never happen.  We just wait.

I'm not asking for gigantic regulatory or process upheaval.  Simply provide some basic transparency.  What is going on behind that black curtain.  Who are we waiting for and what are they doing.  We realize that some jobs are difficult and take a long time.  Just tell us what's going on.  Are contracted statisticians working on data for the next three weeks?  Are regulators analyzing public comments for three days?  Are executives meeting with investors for a two-day conference?  Are we waiting for an investigator's hernia surgery?  What is going on?  Or what is not going on?

The mystery of the black curtain gives an awful lot of cover and leads to a lot of misunderstandings. 

Just open the $%^&* curtain and tell us what's going on. 

Dear FDA Advisory Committee

Below are my comments submitted to the FDA Advisory Committee that will be meeting to address questions related to the Amylyx AMX0035 approval.

_____

Mom was diagnosed with bulbar-onset ALS 25 years ago at age 78 (after a long delay to diagnosis).  She had a fast case and died in 1997.

One might think that a therapy option that slows the speed of a difficult terminal disease might not be of much use to an elderly person in her situation.  I think that just the opposite is true.

One morning, for the first time ever, she asked me to help her button her blouse.  She had strength in her hands but was losing finger dexterity.  As I buttoned those few buttons, she said, "Do you know what the worst part is?"  I simply said that I did not.  She then said, "Once I lose the  ability to do something, I know I'll never be able to do it again."  

She had buttoned her last button. 

I think we all need to let that soak in.

She took riluzole without any pretense that she was going to beat the grim reaper.  She simply wanted to try to keep the independence and dignity of being able to button a button or take care of her personal hygiene as long as possible.

My understanding is that AMX0035 hit its primary endpoint with statistical significance and has a good safety profile.  It's a combination of substances that the FDA knows well.  We are realistic that not every therapy will work for every person with this wicked, heterogeneous disease. I think AMX0035 belongs in a physician's toolbox to try to help a person with ALS who isn't ready to throw in the towel on buttoning that last button.

Thank you very much for the opportunity to comment at an important regulatory moment.  This is the time to apply both the spirit and the letter of the FDA ALS Guidance.

Mislead or Lead

 Dear. CDC and ATSDR,  

You can lead or you can choose to continue to mislead.  The choice is yours.

ALS Prevalence and Incidence in the US

Option 1: Mislead. 

This is unfortunately the path you have chosen.

We sporadically get prevalence reports based on the people with ALS you find rather than the population of people with ALS in the US.  You do not have a population-based registry, yet you issue reports with demographics as if you do.

There are some important examples of how misleading you have been in the past few months --

Dr. Walter Koroshetz, the head of NINDS at NIH, testified the following to Congress in July, 2021:

https://docs.house.gov/meetings/IF/IF14/20210729/113983/HHRG-117-IF14-Wstate-KoroshetzW-20210729.pdf

"Neurodegenerative disorders also include rare but devastating conditions like ALS, a rapidly progressive, fatal disease that affects the nerve cells controlling voluntary movement. The CDC estimates that 12,000–15,000 Americans have ALS, and approximately 5,000 Americans are newly diagnosed with ALS each year."

You misled one of the top neurologists in the country, and as a result you misled the Congress.

News outlets also turn to the CDC as a trusted source.

https://www.wdtv.com/2021/07/28/morgantown-resident-talks-about-living-with-als/

"Amyotrophic Lateral Sclerosis (ALS) affects between 12,000 to 15,000 people across the U.S., according to the CDC."

https://khn.org/news/article/dying-patients-with-rare-diseases-struggle-to-get-experimental-therapies/

"More than 16,000 people in the United States were estimated in 2015 to have ALS..."

More than 16,000, indeed.  Way more.

And there is the draft scoping document that ICER produced for their study on pricing related to two anticipated FDA product approvals.  ICER is in the numbers business and turned to the CDC for a very basic number.

https://icer.org/wp-content/uploads/2022/02/ICER_ALS_Draft-Scope_020222.pdf

"There are about 16,500 people living with ALS in the United States, with a prevalence of 5 to 6 per 100,000 persons."

ALSA issued comments related to this draft but failed to mention the understatement of prevalence of ALS by the CDC.  ALSA is a "partner" (another misleading word since ALSA is actually a paid contractor on the project) and for some reason decided to be misleading, too, by ignoring the problem.  Thank heavens ICER did correct the data based on an individual pointing out the incompleteness. of the CDC's data.

Option 2: Lead

Stop publishing prevalence (and now incidence) reports as if your data are complete.  Be clear that this is a sample of the people you found and you don't know what you don't know.  The people you didn't find don't necessarily look like the people you found.  And based on a past analysis, you miss almost as many people with ALS as you find.  Be clear.  Give us details that are actually meaningful such as how many people self-enroll, how many are in the Medicare files, etc.  And please publish data. simply and promptly.   Leaders understand the importance of meeting due dates.

Clinical Trial Notification

Option 1: Mislead

Again, this is the path you have chosen.

People are encouraged to sign up for clinical trial notification and they infer that you will let them know of trials.  They do not understand that you will inform them of a tiny fraction of enrolling interventional trials.  They sit back, relax, and wait for the magic email from the CDC  telling them of the right trial while their eligibility windows evaporate.  

And your matching capabilities are limited.  The fact that dead people can receive trial notifications is a testament to that problem.

Option 2: Lead

Stop using 1980 email tactics.  You have access to a list of all enrolling interventional trials via clinicaltrials.gov.  Work with your "partners" to design trial notification that is more effective and less onerous than yours.  The best way may not involve you at all.  That's fine.  We need what is best for those with ALS and sponsors.

Summary

In an attempt to constantly celebrate success of the project, it has been easy for you to mislead.  Please stop. 

The CDC and ATSDR should lead, and that means being honest and. clear about what we have and what we need to do.  

Please lead.