Advancing the FDA-approved Phase I clinical study for evaluating the safety and efficacy of a stem cell therapy intervention to reduce brain inflammation and improve clinical outcomes in people with early Alzheimer’s.
Anthony Oliva, Ph.D.
Miami, FL - United States
Individuals with Alzheimer’s typically experience brain inflammation linked to an uncharacteristically active immune system and other causes. Researchers have been studying a special type of stem cell, called mesenchymal stem cells (MSCs), to help reduce such harmful inflammation. These cells are collected from adult bone marrow, and have the potential to develop into many different cell types, including brain cells. MSCs can be used as a treatment as they tend to target sites of injury or inflammation, where they can promote tissue repair.
Scientists have been studying the use of MSCs to treat a wide variety of diseases. In preclinical studies using genetically engineered Alzheimer’s-like mice, MSCs injected into the bloodstream can travel to the brain, where they reduce inflammation and may improve brain function.
Dr. Anthony Andrew Oliva and colleagues have led the way for MSC therapy to treat aging-related conditions, and have recently begun applying their expertise in MSCs as a potential therapy for Alzheimer’s disease.
The team has begun to evaluate the therapeutic potential of MSCs through a Phase I clinical trial, funded by their initial Part the Cloud award. Dr. Oliva and his team have conducted an initial safety run-in phase test on 5 participants, by injecting low doses of MSCs into the bloodstream to ensure the procedure is safe. This has been a successful step so far, establishing safety. The research team has also conducted a preliminary analysis of biological markers (that includes blood and another biological fluid that surrounds the brain and spinal cord called cerebrospinal fluid) for these participants to help the research team understand the impact of the MSC therapy
The preliminary successful results on the 5 participants have enabled Dr. Oliva to expand the study to a larger group of participants. The clinical trial has been converted to a multi-center study that includes 5 different sites allowing the researchers to expand their recruitment efforts. This multi-center expansion has already led to the successful enrollment of an additional 14 participants and puts Dr. Oliva and colleagues on-track to meet their enrollment milestones ahead of originally anticipated. In total, the researchers plan to recruit 30 participants.
Each of the participants is randomly assigned to receive 20 million or 100 million MSCs, or no MSCs. This is a minimally invasive procedure, where the cells are injected into the bloodstream of the participants. Due to the nature of MSCs, they do not require specific donor-recipient matching to be made. For this study, the MSCs are collected from young, healthy, highly screened donors.
All participants are monitored for 12 months after the treatment for any side effects, to ensure safety. During this follow up period, which is ongoing, participants also provide biological samples (blood and cerebrospinal fluid), undergo brain scans, and complete cognitive assessments, to help the researchers understand the impact of the MSC treatment.
This clinical trial is the first step toward testing the effectiveness of MSCs in people with early Alzheimer’s. If successful, the potential therapy will advance to future, larger-scale clinical trials evaluating the efficiency of the MSC treatment.
Made possible through the generous funding from the Part the Cloud, benefiting the Alzheimer’s Association.
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