Gene Therapy Trial for ApoE4 Homozygotes

[Elevation]

I believe this same study is now being offered in a research facility in Orlando.
Same requirements.

Sarah Poissant
Patient Recruitment
100 W Gore Street Suite 202 Orlando FL 32806
Phone: 689-216-3179
Fax: 407-386-9305
Email: Sarah.Poissant@ppd.com
Website: www.ppd.com
Follow us on Facebook PPD Clinical Research Unit - Orlando, FL - Home | Facebook

Elevation

[NF52]

I believe this same study is now being offered in a research facility in Orlando.
Same requirements.

Sarah Poissant
Patient Recruitment
100 W Gore Street Suite 202 Orlando FL 32806
Phone: 689-216-3179
Fax: 407-386-9305
Email: Sarah.Poissant@ppd.com
Website: http://www.ppd.com
Follow us on Facebook PPD Clinical Research Unit - Orlando, FL - Home | Facebook

Elevation

Hi Elevation, I'm not sure what the source of your information was, but it seems inaccurate. The website for PPD Orlando Clinical study opportunities currently shows no trials of any kind.
https://www.ppd.com/participate-in-clin ... rtunities/

The Clinical Trials.gov website for this trial was last updated on April 21 and continues to show only the Weill Cornell Medical Center in NYC as the sole site: https://clinicaltrials.gov/ct2/show/NCT ... 1#contacts

[Elevation]

I received a call from Sarah recently informing me of this study....I questioned her...she assured me it was the same study. I did not proceed so don’t really know more.

[NF52]

I received a call from Sarah recently informing me of this study....I questioned her...she assured me it was the same study. I did not proceed so don’t really know more.

Maybe they haven’t updated their web page. I saw that in another site regarding a study I knew they were doing.
It’s a fascinating study—but I don’t think I’d be brave enough to sign up!

[Brian4]

Results from a second generation investigational gene therapy program show effective delivery of APOE2, the gene associated with protection against the development of Alzheimer's disease, while also targeting and silencing APOE4, the gene that increases risk of developing the disease. A 40% reduction was observed in APOE4 expression, while APOE2 expression was maintained, suggesting APOE2 was resistant to microRNAs (miRNAs) silencing. The efficacy of gene silencing by miRNAs targeting APOE4 was evaluated by inserting miRNA into the AAV vector expression cassette.

Hey ApropoE4, and thanks for this!

I just noticed there are two new gene therapy candidates listed at Lexeo's website:

LX1020 Alzheimer's APOE2+ /E4-
LX1021 Alzheimer's Christchurch

I assume you're referring to LX1020 – though perhaps a different research group is working on something similar.

In any event, very exciting research! (I think I'll wait for the Christchurch variant to be available.)

Brian

[ApropoE4]

I think the study was / is for LX1001, but they just raised $100mm for further trials (I believe including LX1020 and 1021)

https://www.lexeotx.com/post/lexeo-ther ... -financing

[Morazan]

Hi Folks, I am getting ready to head to Orlando to participate in the LX1001 study mentioned above. They have asked that I NOT discuss my experience on "social media," so I won't be posting updates here, but I would be happy to chat with fellow Apoe4x2s via email or other mediums of communication... I'd especially like to be in touch with others who are participating in this, or similar studies. (I think you can send me a private PM on this site, and include your email address or phone.) What do you think is the risk/benefit ratio of joining such a study? Is it better to get in early, or to wait? I don't dare dream of being "cured," but my optimistic side thinks it's worth a Hail Mary pass.
Here is the name and a link to the full description of the study:
Lexeo Therapeutics -- “A 52-Week, Multicenter, Phase 1 Open-label Study to Evaluate the Safety of LX1001 in Patients with APOE4 Homozygote Alzheimer’s Disease”
https://clinicaltrials.gov/ct2/show/NCT03634007

[ApropoE4]

Serious complications in phase 1 clinical trials that are not cancer related are about one in 300.

We could speculate about this particular trial being possibly safer than average due to the nature of the vector, but since there are many unknowns it's best to just stick with that figure and assume those are the odds of something going terribly wrong.

The benefit is of course you might have a couple of years advantage in getting treated, and you help all of mankind!

Another risk you could speculate about is that the effective dosage is determined to be close to the high dose leg, while you might be placed in the low dose leg. I'm not sure if later on it would be possible to effectively correct that, as the immune system would potentially fight off the additional dose more effectively.

[Morazan]

Good to know! I was assuming the risk might be 1 in 100. Another question is the risk over time, e.g. can a brain tumor that shows up five years later be attributed to the treatment? As for the low-dose risk, the doctor said that if there was a clear advantage to the higher dose, that, yes, I could possibly return for a second serving, but we did not discuss a possible immune response. Sigh. My basic assumption is that these clinical trials will take years, and that the resulting treatments will be priced astronomically high, so it's worth the risk for me to get in early. I have not lived a particularly healthy lifestyle. Even if there is no direct benefit, it will be good to get all the brain scans and blood tests I will be getting for free, so I will have a better sense of my trajectory. And yes, benefit all mankind

[grommet]

To add to NF52's great summary - the trial hasn't shown success in 40% of participants - rather it's shown APOE4 expression was down by 40% on average among participants, while APOE2 expression was maintained at least over the months that have passed.

This is a fascinating thread…

Does anyone know how APOE4 **expression** is measured? Is the test available to people (me) who want to pay for it?


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