Research Trial Debate

ScottSmith

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I am trying to decide between the Healey Platform Trial and the Phase 2 Adipose Derived Mesenchymal Stem Cell Trial at Mayo. I have already been screened and gotten into the Mayo trial. But I can opt out of that sill and get into Healey. I am bulbar onset, diagnosed last year, sporadic, ALSFR 43, 39 years old.

Stem Cell Pros - No placebo. It's my own stem cells so its relatively safe. I've heard a great anecdotal story from a PALS who did it and halted progression for 8 months. It's 4 treatments (1 every 3 months).

Stem Cell Cons - A long lead in time (wouldn't get stem cell treatment till July 2021). 6 hour travel. At least one trip a month. They aren't adding neurotrophic factors like NurOwn did. No open label extension.

Healey Pros - I have a clinic doing it right here in Kansas City. I could get treatment as early as March. Open label extension. Relatively short (opportunity to get into another trial).

Healey Cons - 25% chance of placebo (still pretty good but worse than 0%). Don't get to choose the therapy (I'm wanting the CNM-Au8, very strong pre-clinical data).

I know that I'm really fortunate to have options. I don't want to pretend I have a real dilemma here. But it does feel like a big decision to guess right on. After all, many of us only get one shot at this. Thanks for your help and support! I'm curious to hear the feedback.
 

lgelb

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Just as the best vax is in your arm, the best ALS treatment is probably in your body. I would not get revved up by preclin data in ALS, which has been notoriously unreliable.

100% in July vs. 75% in March, you can do the math. Another way is how sad you would be if you found out you were in the Healey placebo group. In other words, is your motivation more about you or more about science. You don't have to answer that, just acknowledge to yourself what your values are in this decision.

And you're right, with many trials using 2y past dx as a threshold, how long you're committed is another criterion, though some trials can accept you concomitantly and others will want a washout period. So every trial should be something that you really want to do, for any combination of reasons.

Not knowing all your prognostic factors, you know that bulbar is less likely to go slow, though that is less true at younger ages like yours. I would run through the model: diagnostic delay, FVC, FTD, how close you are to loss of speech, closely correlated with loss of walking and overall survival in bulbar onset. Naturally, a feeding tube and BiPAP as soon as needed extend life.
 

ScottSmith

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Thank you lgelb for your response. No lie here, my primary objective is to get lucky with a trial therapy that slows my progression. Of course, it is also a duty to contribute to data that may help others in the future. I forgot to mention that with the stem cell trial I will have to cease my Radicava infusions, which would not be the case with Healey.
 

Nikki J

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If you opt out does that reopen a slot ? Or will you be counted as a drop out? .
 

ScottSmith

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It will reopen that spot. I would dropout before the trial start date. I haven't even signed the consent form yet. I've just been guaranteed the spot from a phone screening.
 

ScottSmith

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I would be curious to hear your thoughts Nikki? You have a lot of science knowledge and I respect your opinion.
 

Nikki J

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It is a personal choice. I would ask how fast the Healey trial would get you to drug/ placebo point. The Healey drugs all have solid scientific bases and it is considerably more convenient. That may become much more of an issue later on. I know somebody who is traveling for open label visits and it is becoming burdensome

stem cells are tempting of course but there is no open label as you say You are fortunate to have a choice but I don’t see it as a slam dunk either way.
 

njk0407

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Hi Scott,

I am so sorry to hear of your diagnosis. I know firsthand how devastating it is. My husband was diagnosed in 2016 at the age of 36. Our daughter had just turned 1 and our son was 5. I can completely empathize with how you and your wife are feeling. I wanted to offer you some helpful information although this current thread is not about that. Shortly after my husbands diagnosis he able to make a “legacy video” for our children free of charge from the comfort of our home, which is priceless. The company Thru My Eyes offers free legacy videos to terminal people with young children. I will post their info below. Also, please make as many fun family memories as possible. The Gleason foundation offers grants to families called “Team Gleason Adventures”. The grant allows you to take a memorable trip anywhere you’d like and they cover all of your expenses. Also, Paint for a Cure offers financial gift donations to families with ALS. They gifted us $5k this past December, shortly before my husband passed. Then in February I was able to team up with Paint for a Cure to surprise my dear friends the Beremans still a lot of life to live and memories to be made. I know it’s gut wrenching and terrifying, but please remember that.
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-Nikki Thompson
 
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Dvholmes

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Sounds like a difficult decision. I think I’d opt for the Healey trial with OLE. If you go on Stem Cell and it doesn’t work your stuck on it. If it does work you’ll then get cut off the drug at the end and then what?
 
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