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Gene Therapy Trial for ApoE4 Homozygotes

Posted: Sat Sep 14, 2019 12:00 pm
by Julie G
The Department of Genetic Medicine at Weill Cornell Medicine would like to invite you to learn more about a gene therapy trial for individuals who are ApoE4/E4 and diagnosed with mild cognitive impairment (MCI) or Alzheimer’s disease (AD). The gene therapy is comprised of an adeno-associated virus gene transfer vector coding for ApoE2 which will be delivered intracisternally. The E2 allele is the rarest form of ApoE and carrying even one copy appears to reduce the risk of developing Alzheimer’s by up to 40%. The goal of the trial is to add the ApoE2 gene into the central nervous system to compensate for the effects of ApoE4, and thus prevent or delay the progression of AD.
 
The trial takes place in New York City and consists of an initial examination, the gene therapy administration and multiple follow-up visits over the course of a year. The initial examination will determine an individual’s eligibility to receive the gene therapy and includes a PET scan, an MRI scan and a spinal tap. For more information and a short video.
 
If you would like to learn more about the trial, contact Denesy Mancenido at: dem2026@med.cornell.edu.
 

Research Opportunity for 4/4's with MCI/AD

Posted: Thu Oct 24, 2019 1:22 pm
by Bettylacy
:D Hi all,

I just received this invitation and thought some of you might be interested or know someone.
Sending all my brothers, sisters and non binary friends a big hug!

Bettyg


I am a researcher at Weill Cornell Medicine and would like to invite you to collaborate with us in our gene therapy trial for APOE4 homozygotes.

Based on our studies over the past 6 years and the epidemiologic data that APOE2 confers benefits and decreases risk of AD, we are initiating a clinical trial using an adeno-associated virus gene transfer vector coding for human APOE2 to treat APOE4 homozygotes. The goal is convert the CNS from APOE4/E4 to APOE2/E4. The trial is an open label, dose-ranging study and will enroll individuals who are APOE4 homozygotes with clinical diagnosis of MCI or AD. The study is listed on clinicaltrials.gov: NCT03634007.

Do you have patients that may benefit from trial participation?

Best regards,
Denesy

Denesy Mancenido
Clinical Research Administrator
Director, Clinical Operations & Regulatory Affairs

Weill Cornell Medicine
Department of Genetic Medicine
1305 York Avenue, 13th Floor | New York, NY 10021
(P) 646.962.5583 | (F) 646.962.0380
dem2026@med.cornell.edu

Re: Gene Therapy Trial for ApoE4 Homozygotes

Posted: Fri Oct 25, 2019 8:49 am
by Fiver
Hi Julie and BettyLacy. Thanks. I'd been following this. At one point I saw that there was an age range criteria. I wondering if that's been dropped or expanded?

Re: Gene Therapy Trial for ApoE4 Homozygotes

Posted: Sat Oct 26, 2019 7:14 am
by NF52
Fiver wrote:Hi Julie and BettyLacy. Thanks. I'd been following this. At one point I saw that there was an age range criteria. I wondering if that's been dropped or expanded?

The latest update of this Stage 1 trial was on October 14 on the NIH website: Clinical Trials. It continues to show several requirments, including Apoe 4/4, age 50 or older, diagnosis of MCI or any severity level of AD with positive amyloid on a PET scan. Informed consent can be given by the participant or a legally authorized individual. It does require the participant to be able to undergo MRI, PET and CSF (spinal tap) procedures and to not have heart failure, active infections, more than 4 cerebral micro-bleeds and various other disqualifying conditions.

Last month I spoke briefly with a researcher who said that ApoE 2 does confer some risks, including increased risk of diabetes. But this person also was excited by the concept of replacing some, but not all ApoE 4, with ApoE 2. I especially liked this person's comment that "We don't want to replace all ApoE4; it has some benefits itself."

A Stage 1 trial is for safety and efficacy of dosage amounts. It's where the really big risks are taken by participants in early-stage human trials. This one appears to have three dosing levels, and a 2 year commitment. for the 15 people who enroll. I admire anyone who decides to enroll.

Re: Gene Therapy Trial for ApoE4 Homozygotes

Posted: Sat Oct 26, 2019 7:17 am
by Fiver
Yes, those are the same criteria I saw. I don't qualify (yet). Thanks!

Re: Gene Therapy Trial for ApoE4 Homozygotes

Posted: Wed Oct 30, 2019 3:17 pm
by mike
I'm hoping to never qualify!

Re: Gene Therapy Trial for ApoE4 Homozygotes

Posted: Mon Dec 02, 2019 10:16 am
by Fiver
Talked with Denesey, who seems very nice. While they might be flexible on the age requirement they are focusing only on individuals with some diagnosed MCI. Makes sense. They want to be able to show improvement, if it occurs. But they are still looking for participants.

Re: Gene Therapy Trial for ApoE4 Homozygotes

Posted: Mon Dec 09, 2019 8:43 pm
by Fredsbrain
Is this trial still opened? I was diagnosed with mild cognitive impairment by a neurologist after an evaluation by a psychologist several months ago. But I didn't have any scans. Would I have to get a scan thru my neurologist or how can I get one ordered?

Re: Gene Therapy Trial for ApoE4 Homozygotes

Posted: Tue Dec 10, 2019 6:14 am
by NF52
Fredsbrain wrote:Is this trial still opened? I was diagnosed with mild cognitive impairment by a neurologist after an evaluation by a psychologist several months ago. But I didn't have any scans. Would I have to get a scan thru my neurologist or how can I get one ordered?
Welcome, Fredsbrain!

Based on 60+ years of having friends named Fred--and of course Fred Rogers--I confess to being sure that you and your brain are wonderful!

The clinical trail Gene Therapy for APOE4 Homozygote of Alzheimer's Disease is still recruiting, with an update in the National Institute of Health's website just before Thanksgiving: Clinical Trials.

If you would like to learn more about the trial, you can contact either of these people at Weill Cornell Medical Center in NYC:
Grace W Mammen, Telephone: 646-962-2672 or email: gwm2004@med.cornell.edu
Denesy Mancenido, Telephone: 646-962-5583 or email: dem2026@med.cornell.edu

The good news is that you don't need to go get a PET scan on your own, or an MRI. The study organizers will take care of all of that after first having probably a few meetings with you to be sure you want to begin the screening process. I have had both PET scans and MRIs as part of a 2-year clinical trial that is winding up. Everyone is different, but my impression of a PET scan, which is used to determine if we have amyloid beta plaques in our brains (sometimes called being "amyloid positive") was a breeze. It took an IV and 30 minutes of quiet in a darkened room before a quick, almost silent scan. An MRI is more like a NYC construction crew banging around for 30 minutes--annoying, but not painful, especially when your headphones play soft rock, classical, or whatever radio station you choose! In fact, they had to remind me not to fall asleep during one!

I encourage anyone interested in a clinical trial to remember that you are in the driver's seat and your health is paramount! Your informed consent is required before you even start the screening process, and again before you start the actual study, especially because this study does not include a placebo (inactive treatment) and is an early stage study of safety doses.

I hope you keep us posted on how you are doing, Fred.

Re: Gene Therapy Trial for ApoE4 Homozygotes

Posted: Tue Dec 10, 2019 2:26 pm
by Fredsbrain
I'm brand new to the website. Thanks for the feedback!