What side effects are associated with this type of intervention?

Common treatments for Alzheimer's Disease include cholinesterase inhibitors (e.g., donepezil, rivastigmine) and NMDA receptor antagonists (e.g., memantine). Side effects of cholinesterase inhibitors often include nausea, vomiting, diarrhea, and muscle cramps, while memantine can cause dizziness, headache, confusion, and constipation. For treatments similar to BDNF gene therapy, which focus on neuroprotection and activation of brain cells, potential side effects could include immune reactions, local inflammation, and off-target effects due to the viral vectors used in gene therapy. It is important to discuss these potential risks with a healthcare provider.

What prior FDA approvals exist?

FDA-approved treatments for Alzheimer's Disease primarily include cholinesterase inhibitors (donepezil, rivastigmine, and galantamine) and an NMDA receptor antagonist (memantine). These drugs focus on symptom management rather than neuroprotection or activation of brain cells. While there are no FDA-approved treatments directly analogous to BDNF gene therapy, research continues into neurotrophic factors and gene therapies aimed at slowing disease progression and protecting brain cells.

What other types of research exist?

Promising alternatives to BDNF gene therapy for Alzheimer's Disease include: 1) Antisense oligonucleotide therapies, which target specific genetic mutations to reduce harmful protein production. 2) Investigational oral compounds like ONC201, which may affect mitochondrial metabolism and stress response pathways. 3) Chimeric antigen receptor (CAR)-expressing T cells targeting specific brain cell markers. 4) Oncolytic viruses like DNX-2401, which are designed to selectively infect and kill diseased cells. These therapies are in various stages of clinical trials and show potential for neuroprotection and activation of brain cells.

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Virus Therapy

AAV2-BDNF Gene Therapy for Alzheimer's Disease

Phase 1

Recruiting

Led By Mark Tuszynski, M.D., Ph.D.

Research Sponsored by Mark Tuszynski

Eligibility Criteria Checklist

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

Must have

Minimum age 50

Diagnosis of dementia due to Alzheimer's Disease (AD) by National Institute of Aging (NIA) - Alzheimer's Association (AA) criteria for AD

Must not have

Major depression or another major psychiatric disorder within the past two years

History of active malignancy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 24 months

Awards & highlights

No Placebo-Only Group

Summary

This trial tests a new gene therapy that uses a virus to deliver a protective protein called BDNF into the brains of people with early Alzheimer's Disease and Mild Cognitive Impairment. The goal is to help brain cells survive, function better, and form new connections. The therapy aims to slow or prevent further brain cell loss. Brain-derived neurotrophic factor (BDNF) has been explored for its potential to support neuron survival and function in neurodegenerative diseases.

Who is the study for?

This trial is for people aged 50-80 in San Diego, Orange Counties, California or Ohio with early Alzheimer's or Mild Cognitive Impairment. They must speak English without communication issues and have a caregiver. Participants need to be generally healthy, not pregnant, and have stable medication use for at least one month.

What is being tested?

The trial tests AAV2-BDNF Gene Therapy as a potential treatment for Alzheimer's Disease and Mild Cognitive Impairment. It involves using a harmless virus to deliver BDNF protein into the brain to protect against cell loss and activate remaining cells.

What are the potential side effects?

As this is a first-in-human trial of gene therapy, specific side effects are unknown but may include reactions related to the immune system's response to the viral vector or new protein production.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am 50 years old or older.

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I have been diagnosed with Alzheimer's Disease according to NIA-AA criteria.

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My recent brain scans show no signs of disease.

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I can see and hear well enough for brain function tests.

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I am between 50 and 80 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have not been diagnosed with major depression or a significant psychiatric disorder in the last two years.

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I have a history of cancer.

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I do not have any major neurological conditions.

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I have had a condition where my lymphocytes grow abnormally.

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I have not experienced severe mental health issues like psychosis or extreme agitation in the last three months.

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I have had thoughts of suicide in the last 3 months.

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I do not have any severe illnesses or unstable health conditions.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 24 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~24 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 months for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Memory change tested on Benson Complex Figure Draw and Memory

Memory change tested on Ray Auditory Verbal Learning Task

Safety as assessed by mumber of participants with treatment-related adverse events assessed on MRI scan

Secondary study objectives

Change in Biomarkers including CSF amyloid, tau and neurofilament

Efficacy on PET scan reflected by change in fluorodeoxyglucose (FDG) PET scan

Memory change tested on mini-mental status examination (MMSE)

+1 more

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: Gene transfer of AAV2-BDNFExperimental Treatment1 Intervention

Up to 12 subjects will receive open-label AAV2-BDNF

0 / 12Eligibility criteria met

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 often aim to protect brain cells and enhance their function. The gene therapy with Brain-Derived Neurotrophic Factor (BDNF) being studied aims to slow or prevent cell loss and activate still-functioning brain cells by continuously delivering BDNF into the brain. BDNF supports the survival of existing neurons and encourages the growth and differentiation of new neurons and synapses. This neuroprotective and neuroactivating approach is crucial as it directly targets the underlying neuronal degeneration in Alzheimer's Disease, potentially slowing disease progression and improving cognitive function. Other treatments, such as cholinesterase inhibitors and NMDA receptor antagonists, work by enhancing neurotransmitter activity to improve communication between neurons, but they do not directly address neuronal survival and regeneration.