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.

Wednesday, September 27, 2023

Holding Hope Hostage


It's so important to families dealing with ALS.  It's what keeps many of them going.  You hope for a better day tomorrow.  You hope for an effective treatment.  You hope for a miracle.  Hope is more precious to you than almost anything.  You look for it everywhere.  

Today, thousands of families dealing with ALS have found hope in an investigational therapy called NurOwn being developed by Brainstorm Cell Therapeutics, Inc.  It is the beam of light they have sought every day they deal with the stinking ALS.

Brainstorm completed a Phase III trial over three years ago.  If failed to meet its endpoints.  It failed royally.  Unfortunately many ALS trials do.

NurOwn represents a huge amount of hope because many of us have seen people who were in the trial actually feel improvement.  That is literally miraculous in ALS.  Unfortunately every time we go back and look at that summary report from the trial, the trial failed.  But hope in ALS Land isn't easily smothered when something good has happened to someone.  That hope is precious to thousands.

Through social media and a lot of hype,  the hope has grown and reached thousands more people.  And then thousands more.  

But every time we go back to that report, the trial was a failure. 

Brainstorm didn't step up and run another trial on those it suspects are the responders.  Instead it decided to rearrange the data from the trial and convince us that they have the hope that every person with ALS seeks.  If you remove the people that NurOwn didn't work for, it works!  That's not great science, but it sure stokes the hope business.

And there have been all kinds of excuses for not doing another trial or providing expanded access to their unapproved therapy.  People don't want to challenge those excuses.  They don't want to mess with the holder of their hope.

For the last three months, since I made a comment quoted in a national publication that I suspected that the reason for never telling their trial participants (from a trial that ended in 2020 and has added no new data since) if they got placebo or treatment might have something to do with some of the public miracle cases having received placebo, my online life has become nothing short of a nightmare.  Clearly many think I deserve it because I'm being critical of their ray of hope.  

No, I'm being critical of a company that is holding their hope hostage.

I've learned a lot about adult cyber bullies.  I've learned even more about parents and grandparents who teach the kids to be kind and then say some terrible things about another person online.  They see me as stealing their hope -- a hope that they feel is so close to delivering them their miracle.

In reality, there is much that Brainstorm could have been doing to deliver some of the fruits of that hope  sooner and to offer some scientific proof to back up the miracles.  They have instead decided to hold hope hostage for one and only one ransom -- FDA approval to sell NurOwn to people with ALS at a price of their choosing.

Today, September 27, there is an FDA Advisory Committee meeting to help the FDA make its decision.  

Two days ago we first saw the documents related to that meeting.  The FDA briefing document was stunning.  It took me  hours to wade through the problems they found in Brainstorm's data, and perhaps of even more concern, the problems in their manufacturing processes.  The normally noisy online ALS world supporting NurOwn was quiet.  Journalists were busy pumping out coverage of a remarkable document.  I suspected that the NurOwn supporters were reading and stunned, too.  Silly me.

Yesterday we saw organizations completely ignoring the FDA briefing document and doubling-down on supporting approval.  Didn't they even read what the FDA had found?  Perhaps they don't want to be the ones to suggest we refuse to pay the ransom of approval of a product that represents precious hope but does not represent good science yet.

An online mob thinks I am trying to steal their hope. Brainstorm says the only way to get the hostage hope out of that room is FDA approval.

Today we will see people critical of anyone (including the FDA) who wont capitulate to the company that holds their hope hostage.  The facts won't matter

I wish that more people would actually read the reports and think for themselves.

A company is holding their hope hostage rather than having used the last three years to find the scientific truth.

All with ALS deserve better than hope that isn't moored in truth.

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