What side effects are associated with this type of intervention?

Common treatments for Alzheimer's Disease, particularly those targeting APOE isoforms like the gene therapy LX1001, may include side effects such as headaches, dizziness, and injection site reactions. More broadly, Alzheimer's treatments like cholinesterase inhibitors (e.g., donepezil, rivastigmine) can cause gastrointestinal issues such as nausea, vomiting, and diarrhea, while NMDA receptor antagonists (e.g., memantine) may lead to dizziness, confusion, and headaches. It is important to discuss potential side effects with a healthcare provider to tailor the treatment plan to individual needs.

What prior FDA approvals exist?

The FDA has approved several treatments for Alzheimer's Disease, primarily focusing on symptomatic relief rather than disease modification. Cholinesterase inhibitors like donepezil, rivastigmine, and galantamine are commonly used to manage symptoms. Memantine, an NMDA receptor antagonist, is another approved drug that helps with cognitive symptoms. Recently, aducanumab, a monoclonal antibody targeting amyloid-beta plaques, received FDA approval, marking a shift towards disease-modifying therapies. While gene therapy approaches like LX1001, which aims to convert CSF APOE isoforms from APOE4 to APOE2-APOE4, are still under investigation, they represent a promising future direction for potentially altering the disease course at a genetic level.

What other types of research exist?

Promising novel alternatives to Gene Therapy (LX1001) for Alzheimer's Disease patients include: 1) Allopregnanolone for neuron protection and regeneration, 2) T3D-959, a PPAR delta/gamma agonist showing improvements in cognitive function, and 3) patient-specific stem cell therapies aimed at neuroprotection and cognitive enhancement. These approaches are currently under investigation and show potential for future treatment options.

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Gene Therapy

Gene Therapy for Alzheimer's Disease (LEADLTFU Trial)

Phase 1

Recruiting

Research Sponsored by Lexeo Therapeutics

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 260 weeks

Awards & highlights

No Placebo-Only Group

Summary

This trial is a study to evaluate the safety of a gene therapy (LX1001) for people with a specific genetic risk for Alzheimer's disease. The therapy aims to convert a harmful gene variant to a protective one, potentially slowing the disease's progression.

Who is the study for?

This trial is for individuals with Alzheimer's who have the APOE4 gene variant and previously received LX1001 gene therapy. They must not have any medical conditions that could increase risk during the study, and they agree to keep their personal medical data off social media until all related studies are complete.

What is being tested?

The study is monitoring long-term safety of LX1001 gene therapy in treating Alzheimer's. It also looks at changes in brain markers like amyloid levels, tau proteins, and MRI scans to see if there's improvement in cognitive functions over time.

What are the potential side effects?

While specific side effects aren't listed here, participants will be monitored for any long-term safety concerns arising from the previous LX1001 gene therapy treatment.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 260 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~260 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 260 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Incidence of serious adverse events

Incidence of treatment emergent adverse events

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Previously administered LX1001Experimental Treatment1 Intervention

This is a long-term follow-up study to evaluate the safety following LX1001, a gene therapy, for participants who are APOE4 homozygotes with clinical diagnoses varying from MCI or dementia due to AD who have previously received LX1001. Study LX1001-01 was designed to assess the safety of LX1001 at 3 ascending doses (1.4 × 1010, 4.4 × 1010, 1.4 × 1011 gene copy \[gc\]/mL CSF) as per droplet digital polymerase chain reaction methodology, with each group consisting of approximately n=5 individuals for a total of approximately 15 participants for the entire study. In this study, participants who have received LX1001 in the parent protocol (LX1001-01) will be followed for up to 260 weeks post gene therapy administration

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

LX1001

2019

Completed Phase 2

~20

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Alzheimer's Disease (AD) include cholinesterase inhibitors, which work by increasing levels of acetylcholine in the brain to improve communication between nerve cells, and amyloid-beta targeting therapies, which aim to reduce amyloid plaques that are characteristic of AD. Gene therapy, such as LX1001, focuses on converting APOE4 isoforms to APOE2-APOE4 in cerebrospinal fluid, potentially reducing the risk and progression of AD associated with the APOE4 genotype. These treatments are crucial as they target different aspects of AD pathology, aiming to slow disease progression and improve cognitive function and quality of life for patients.

[Pharmacological approaches to the therapy of Alzheimer's disease].Apolipoprotein E: a pharmacogenetic target for the treatment of Alzheimer's disease.