New Imaging Protocol Could Vastly Accelerate Clinical Trials for New ALS Treatments

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lgelb

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Note: there is frequently a long time between a new prognostic marker's development and the FDA or any other regulatory agency's accepting it as a surrogate endpoint. Even as a diagnostic aid or means to monitor disease progression, you also have to look at how costly/difficult it would be for other centers to implement the protocol and the point at which payors will fund it. Still, progress is being made.

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Laurie
 

KarenNWendyn

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That’s absolutely fascinating. Now they need to come up with a way to attach that tracer to a drug that reduces neuroinflammation....
 

Nikki J

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It is interesting and hopefully will find its place. I was in the old study and am doing the new one too.

Dr Atassi said recently, though, not all PALS show this. He theorized that LMND PALS might show in the spinal cord only. Later studies will image the spinal cord
 

Nikki J

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If anyone is able and in the Boston area the study is enrolling PALS, and other MNDs. FALS carriers and healthy controls. You have to lie flat with no breaks for the scan which is at least 90 minutes

https://www.neals.org/als-trials/1381
 

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Note: there is frequently a long time between a new prognostic marker's development and the FDA or any other regulatory agency's accepting it as a surrogate endpoint. Even as a diagnostic aid or means to monitor disease progression, you also have to look at how costly/difficult it would be for other centers to implement the protocol and the point at which payors will fund it. Still, progress is being made.

Best,
Laurie
The protocol is being used in 4 clinical trials run by the NCRI. Whether or not it’s recognized by a regulatory agency as an endpoint, it can allow researchers to discard ineffective compounds/recognize potential treatments earlier in the process.

There is no reference to the cost/difficulty of the protocol, but considering the dramatic patient benefits of earlier diagnosis (I.e. efficacy of edavarone, more clinical trial options), I think the cost/difficulty threshold would need the be very high for centers to ignore it.
 
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lgelb

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1) BeachBum, please don't quote previous posts -- our members often find reading difficult.

2) The cost and logistics are significant in this case, and will be a limiting factor even outside of trials. Moreover, as Nikki points out, the utility across the ALS spectrum is still in research.

3) As I mentioned in my PM to you, our policy is that since you are not diagnosed (and fortunately, are not likely to be), you must stay in your own thread, though we allowed you to open this one in the spirit of learning. To allow otherwise is unfair to others in the same category. Any posts you add from here on in any thread besides your own will therefore be deleted. Thank you for your understanding.
 

Nikki J

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The issue with availability is a big one. The PET MRI machines are not available in very many places AND as noted in the article a cyclotron is needed on site to make the tracer. It has to be made and used immediately.

I believe what is happening with the 4 trials is that is an option given to participants who are enrolled in a site geographically accessible to a place that has the ability. ( MGH obviously and I think UMass is sending people to MGH- I don’t know where else if anywhere). People enrolled at other locations are not given the option and it is not part of the outcome measures.

It is interesting and important but more study is needed. They haven’t even started imaging spinal cords which I think will be an important piece if Dr Atassi is right that Lower motor issues show up there.
 

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We heard about and saw images of this technique at the ALSA Advocacy conference in May and the images were quite dramatic I thought it was one of the more promising developments I heard about there. if it proves itself to the FDA and scientific community (and works with spinal cord imaging) I would think the cost would be offset by the efficiency gained in conducting clinical trials, whose costs we hear about often.

Ed
 

lgelb

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The problem is that the centers who would have to fund capital expenditures are not the ones who bear clinical trial cost -- that is the manufacturers, whose pockets are not as deep as you might think. And again, no one is going to make financial moves without the FDA's blessing, which requires a trial design to approve, absent a policy proclamation.

Still, certainly worth keeping an eye on.
 

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I should have noted in my comment that the costs would certainly limit availability as a tool for early diagnosis, one of the presumed benefits of the technique. I wonder what a facility with the necessary equipment would charge, and what if any insurance coverage would apply. But it's early days yet, and I have no idea how the FDA assesses new diagnostic techniques such as this outside of a clinical trial.

Ed
 
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