Gene Therapy for Alzheimer's Disease
Trial Summary
Will I have to stop taking my current medications?
The trial requires that you stop taking certain medications, such as systemic immunosuppressants, corticosteroids, monoclonal anti-amyloid therapies, and anti-coagulant medications. If you are on these medications, you may need to stop them before participating.
What data supports the effectiveness of the treatment LX1001, AAVrh.10hAPOE2 for Alzheimer's disease?
Research shows that similar gene therapy approaches using adeno-associated virus (AAV) vectors have been effective in delivering therapeutic genes to the brain, improving symptoms in Alzheimer's disease models. For example, AAV-mediated delivery of therapeutic agents has been shown to reduce amyloid plaques and improve cognitive function in animal models.12345
Is the gene therapy treatment AAVrh.10hAPOE2 safe for humans?
How is the treatment LX1001 unique for Alzheimer's disease?
LX1001 is a gene therapy that uses a virus to deliver a protective gene variant, APOE2, directly into the brain, which is different from most Alzheimer's treatments that focus on reducing amyloid plaques or inflammation. This approach aims to modify genetic risk factors and potentially prevent or reverse neurological damage in patients with a high-risk APOE4 gene variant.35689
What is the purpose of this trial?
This trial tests a gene therapy injected into brain fluid for Alzheimer's patients with two APOE4 genes. The therapy aims to change a harmful brain protein to a less harmful version, potentially improving symptoms. Gene therapy is rapidly emerging as a powerful therapeutic strategy for a wide range of neurodegenerative disorders, including Alzheimer's disease.
Research Team
Lexeo Clinical Trials
Principal Investigator
Lexeo Therapeutics
Eligibility Criteria
This trial is for APOE4 homozygotes with mild to moderate Alzheimer's-related cognitive impairment or dementia, who can consent and agree not to share study details on social media. Participants should not be on experimental meds or have active infections like hepatitis or HIV, and must avoid pregnancy during the study.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive intrathecal administration of AAVrh.10hAPOE2 (LX1001) to assess safety and toxicity
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- LX1001
Find a Clinic Near You
Who Is Running the Clinical Trial?
Lexeo Therapeutics
Lead Sponsor
Alzheimer's Drug Discovery Foundation
Collaborator
Weill Medical College of Cornell University
Collaborator