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Clinical Trails Committee: Building New Paths

May 7, 2019  -  Sandy Morris

Clinical Trials. Two words my family never put together in a sentence before ALS entered our lives. Two words that now embody the hope of cures and the frustration of the now.

The day of diagnosis is forever sketched in my mind--in any ALS patient’s mind. It is the day you are told you are going to die. Not just sometime, but soon. What if it doesn’t have to be that way?

As chilling as ‘diagnosis day’ was, I decided not to ‘puddle.' I chose to fight. I prayed for bravery and asked my tribe to fight harder with me. That’s just what we did. I became a quick study of my own disease. The eternal optimist in me kept saying there is always a way.  I thought, “you just have to try a different path and if that doesn’t work, then try again.”

I quickly joined what I felt was the most promising clinical trial at the time. I was disheartened to learn that ALS clinical trials were much too similar to the mouse models that I had studied in school.  I completed the trial, but the words “what you allow will continue” rang in my ears every step of the way. So I looked into trials in other disease states and realized that we can--we must--do better. It is time to finally bring ALS clinical trials into the 21st century.

The next question was how? There was no path. When you can’t find the path to take, fighters forge ahead and create new ones. My 21-year-old daughter wrote this when she asked her friends to join us in this ALS movement, “my fearless human is changing the game and we are following her into battle.”

So that is exactly what the Clinical Trials team is doing.

This group was formed by individuals with a fierce determination to accelerate the access to treatments by ALS patients (pALS), no matter the pathway. Right to Try, Expanded Access, Clinical Trials--all of these pathways should work for pALS. All should help make the hope of cures real as soon as possible.

So, you may be wondering, how are we making this hope real?

Step one: We are working to improve the design of ALS clinical trials to more closely resemble the humanity and innovation of oncology and HIV trials. We absolutely recognize the importance of a scientifically sound trial, but also believe that ALS clinical trials must be designed for humans.

We have a saying in this group. We. Are. Not. Zebrafish. (WANZ). And because WANZ, we commissioned a poll with ALS-TDI about clinical trials--the results of which will be published on this blog very soon.

We have also dug into trial design in ALS and beyond, and educated ourselves on the history of clinical trials (including the failures of ALS trials so that we don’t repeat the mistakes of the past).

With this training, we have begun outlining how to do trials better, never forsaking science, so that we can be a partner to scientists and companies when they create the trials that we hope will save our lives. We are on a real mission to change outdated ‘default’ clinical trial design and measurement. And we will not rest until we have.

Step two: As a group, are taking what we have learned and are building relationships with all the critical partners. We met with multiple FDA leaders in February, and have been talking to biotech firms and Pharma. We are working with powerful leaders in Congress, with nimble and brave leadership in ALS organizations, with game-changing neurologists and brilliant scientists. We are doing this to ensure that the ALS landscaping changes today, not 15-150 years from today.

Step three: In the coming weeks, we will detail our efforts so far on this blog and set out what we plan to do next. This committed clinical trial crew won’t stop until game-changing paths have been made.  Watch us – better yet, join us.

If this sounds like your kind of group and you would like to join the movement, please reach out to us via [email protected]  Title your email Clinical Trials and let us know you are ready to fight too.

Here’s to this community. We are better together. We will win this fight together.

Sandy

I AM ALS Clinical Trials Committee Members

  • Deb Bellina
  • Nicole Cimbura
  • Shea Harden
  • Phil Green
  • Sandy Morris
  • Bob Hebron
  • Bonnie McKay
  • Bryan Galentine
  • Shelly Hoover
  • Alison Burell
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