Idea for Clinical Trial

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stephen15
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Joined: Sun May 23, 2021 4:00 pm

Idea for Clinical Trial

Post by stephen15 »

Why not do a clinical trial for people with 1 or 2 APOE4 variants who are younger and asymptomatic (e.g. 20-50) and follow them and a group who did not receive gene therapy to see if replacing the variant(s) with APOE2 decreases the likelihood of developing Alzheimer's? The trial could answer the following questions:

1. Does gene therapy decrease the likelihood of developing Alzheimer's?
2. Does receiving gene therapy at an earlier age further decrease the likelihood of developing Alzheimer's?
3. Are there any adverse effects of gene therapy, specifically replacing APOE4 with APOE2?

I don't see any such trials right now, but I think that would make more sense than looking for people who are already symptomatic and in which the disease may be irreversible. The only APOE4 --> APOE2 replacement trials I've seen so far only include symptomatic patients with 2 variants, like this one:

https://clinicaltrials.gov/ct2/show/NCT ... w=2&rank=1
Sara Barthel
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Location: Texas Hill Country

Re: Idea for Clinical Trial

Post by Sara Barthel »

stephen15 wrote:Why not do a clinical trial for people with 1 or 2 APOE4 variants who are younger and asymptomatic (e.g. 20-50) and follow them and a group who did not receive gene therapy to see if replacing the variant(s) with APOE2 decreases the likelihood of developing Alzheimer's? The trial could answer the following questions:

1. Does gene therapy decrease the likelihood of developing Alzheimer's?
2. Does receiving gene therapy at an earlier age further decrease the likelihood of developing Alzheimer's?
3. Are there any adverse effects of gene therapy, specifically replacing APOE4 with APOE2?

I don't see any such trials right now, but I think that would make more sense than looking for people who are already symptomatic and in which the disease may be irreversible. The only APOE4 --> APOE2 replacement trials I've seen so far only include symptomatic patients with 2 variants, like this one:

https://clinicaltrials.gov/ct2/show/NCT ... w=2&rank=1
Hi stephen15, a warm welcome to apoe4.info and I completely agree! Thank you for your thoughtful approach to a needed area of research - I believe you have found the right platform to share these ideas. As someone who falls into that category, I hope to never get to the point of being symptomatic years down the road and would love to study a study such as that. I also wanted to share a few useful links with you to help familiarize you with this site and stay engaged going forward.

First is the How-to-guide, it will help you get the most out of the ApoE4.info website. It is created to help new (and not so new) members navigate the site. This area explains how to use the quotes button so that the other member receives an email notification of your reply, how to search for topics, how to subscribe to topics of interest and more.

My favorite is the Primer, written by one of our member physicians, Stavia, who is APOE4/4. It is great introduction to ApoE4, biochemistry, and possible prevention strategies in addition to exploring areas such as the science behind the ApoE4 gene and the lifestyle factors that impact its expression. It is a wealth of information - a great place to start and to come back to time and again!

Lastly our wiki Index offers a wealth of additional information and details the muti-factorial approach to taking control of your brain health. Useful for searching specific topics of interest.

And, we would love to know more about you and what led you to join, we invite you to share your journey by creating a new topic in Our Stories. This is something that we encourage all new members to do so they get the opportunity to engage further on the site, especially if their first post is low on details.

Enjoy your learning experience in the forum

Sara Barthel
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