Mesenchymal Stem Cell Therapy for Early Alzheimer's Disease

Plain English Summary

Stem Cell Therapy for Early Alzheimer's Disease is a Phase 2 clinical trial sponsored by Paul E Schulz studying Cognitive Dysfunction. This trial tests if stem cells from a patient's own fat can reduce brain inflammation and improve brain activity in people with early memory or thinking problems. It is for adults aged 60-80 who have been diagnosed with mild cognitive impairment (MCI) due to Alzheimer's disease. Participation involves a small fat sample collection, receiving four stem cell infusions over 12 weeks, and regular clinic visits for checkups and tests. Currently, there are no standard stem cell treatments for early Alzheimer's; standard care focuses on managing symptoms and slowing progression. The trial aims to enroll 12 participants.

Official Summary

The goal of this clinical trial is to learn if stem cell therapy works to treat brain inflammation in adults. Inflammation in the brain may be involved in adults who have memory or thinking problems. The stem cells will be taken from participant's fat samples, processed and given back to participants, so they are their own donor. The main questions this trial aims to answer are: * Does stem cell therapy reduce inflammation in the brain? * Does stem cell therapy improve brain activity? * Does stem cell therapy slow down progression to Alzheimer's disease? Participants will: * Have a small fat biopsy taken at a doctor's office to process stem cells * Receive 4 infusions of stem cells, through a vein in the arm over 12 weeks * Visit the clinic every 2-4 weeks for the first 4 months and then every 1-2 months for 8 months for checkups and tests

Who Can Participate

Here is what you need to know about eligibility for this trial. You can join if you are between 60 and 80 years old and have been diagnosed with mild cognitive impairment (MCI) due to Alzheimer's. You must have specific markers of brain inflammation and amyloid in your brain, and a certain score on a cognitive test (MMSE >= 22). You cannot join if you have other neurological conditions, severe organ disease, a recent history of cancer, or are pregnant. This trial is studying Cognitive Dysfunction, so participants generally need a confirmed diagnosis. The trial is currently accepting new participants.

What They're Measuring

The primary outcomes measure how stem cell therapy affects brain inflammation and activity, aiming to see if it can reduce markers of inflammation and improve brain function. The specific primary outcome measures are: Change in TSPO levels, measured by the PET scan, from baseline to midpoint and baseline to end of study (Baseline, midpoint (169 days from 1st infusion)); Inflammatory cytokines in CSF following adMSC therapy (Baseline, midpoint (169 days from 1st infusion)). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.

About This Phase

This trial is in Phase 2, which tests whether the treatment actually works against the target condition. Phase 2 trials involve 100-300 patients and continue to monitor safety while evaluating effectiveness. This phase often tests different dosages to find the optimal amount. About 33% of Phase 2 drugs advance to Phase 3. If successful, the treatment will move to large-scale Phase 3 trials needed for FDA approval.

Why This Trial Matters

This trial addresses the need for new treatments for early Alzheimer's by investigating if a patient's own stem cells can reduce brain inflammation, a key factor in cognitive decline. Phase 2 success would typically lead to larger Phase 3 trials needed for regulatory approval. This research targets Cognitive Dysfunction, where improved treatment options are needed.

Investor Insight

This Phase 2 trial, focusing on a novel stem cell therapy for a large and growing market, signals potential for a new treatment approach if successful in demonstrating safety and efficacy. Phase 2 trials have approximately a 15-20% chance of eventually gaining FDA approval.

Is This Trial Right for Me?

Ask your doctor about the risks and benefits of stem cell therapy and if this trial is right for you. Be prepared for a small procedure to collect fat, regular IV infusions, and frequent clinic visits for about a year. Understand that this is an experimental treatment, and its effectiveness and safety are still being studied. This trial is currently recruiting participants. The trial is being conducted at 1 site. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.

AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.

Study Design

• Study Type: INTERVENTIONAL
• Allocation: NA
• Model: SINGLE_GROUP
• Masking: NONE
• Enrollment: 12 participants

Interventions

• BIOLOGICAL: adMSC — IV-infusion of autologous, adipose-derived, Mesenchymal Stem Cells (adMSCs), of approximately 2x10(8) adMSCs in 250mL saline.

Primary Outcomes

• Change in TSPO levels, measured by the PET scan, from baseline to midpoint and baseline to end of study (Baseline, midpoint (169 days from 1st infusion))
• Inflammatory cytokines in CSF following adMSC therapy (Baseline, midpoint (169 days from 1st infusion))

Secondary Outcomes

• Number of participants with treatment-related adverse events (Baseline - End of Study (337 days from 1st infusion))
• Changes in neurofilament light chain (Nf-L) in CSF following adMSC therapy (Baseline, midpoint (169 days from 1st infusion))
• Changes in Glial fibrillary acidic protein (GFAP) in CSF following adMSC therapy (Baseline, midpoint (169 days from 1st infusion))
• Changes in Total Tau/phosphor-Tau ratios in CSF following adMSC therapy (Baseline, midpoint (169 days from 1st infusion))
• Changes in cerebral metabolism activity via FDG PET imaging from image baseline to midpoint (Baseline, midpoint (169 days from 1st infusion))

Full Eligibility Criteria

Inclusion Criteria:

1. Has signed an informed consent form before any assessment is performed as part of the study.
2. Be male or female between 60 and 80 years old.
3. Subject has been or is in process of being clinically diagnosed with late pre-symptomatic or mild cognitive impairment (MCI) due to AD (prodromal AD).
4. Mini-Mental State Examination (MMSE) score of ≥ 22
5. Has an MRI to evaluate AD pathology (may use previous if within 6mo.)
6. Has APOE status to evaluate AD pathology (may use previous result)
7. Proficiency in English is required because cognitive tests are administered in English only.
8. Has evidence of brain amyloidosis via PET Scan or Aβ42/40 ratios in CSF.
9. Has evidence of peripheral inflammatory profile based on CRP (≥ 8 mg/L), IL-6 (≥ 3.1 pg/mL), TNF-α (10 pg/mL), or erythrocyte sedimentation rate (ESR) (≥20 mm/h) in blood assays.
10. Is in the opinion of the Investigator, in good general medical health based upon medical history, physical examination, laboratory tests, vital signs and EKG.

Exclusion Criteria:

1. Current medical or neurological condition that might impact cognition or performance on cognitive assessments. (e.g., traumatic brain injury (TBI), Parkinson's disease (PD), multiple sclerosis, etc.)
2. Inability or unwillingness of patient to undergo neuropsychological testing.
3. Advanced, severe, progressive or unstable disease that may interfere with the safety, tolerability and study assessments, or put the subject at special risk. (e.g., significant cardiac disease, severe renal impairment, severe hepatic impairment, autoimmune disease, etc.)
4. History of malignancy of any organ system within the past 60 months, that in the opinion of the investigator would impede evaluation or interpretation of subject safety or study results.
5. Females of childbearing potential must not be pregnant.
6. Inability or unwillingness to undergo PET Scans.
7. Inability or unwillingness to undergo MRI Scans.
8. Positive blood test for either HIV, Hepatitis B, Hepatitis C or Syphilis
9. Positive for TSPO SNP rs6971
10. Inability or unwillingness to undergo Lumbar Punctures.
11. Inability or unwillingness to undergo infusions.
12. Any condition, which in the opinion of the investigator, would put the subject at undue risk or would interfere with evaluation of the investigational product or interpretation of subject safety or study results.

Trial Locations

• The University of Texas Health Science Center at Houston (UTHealth), Houston, Texas, United States

Frequently Asked Questions

Related Conditions

• Alzheimer's Disease
• Mild Cognitive Impairment (MCI)
• Cognitive Dysfunction
• Neuroinflammation
• Dementia
• Amyloidosis
• Brain Health
• Neurological Disorders
• Geriatric Medicine
• Regenerative Medicine

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