Stem Cells for Epilepsy

Recruiting in Jacksonville (>99 mi)

Overseen By Megan J Gauthier, AS

Age: 18+

Sex: Any

Trial Phase: Phase < 1

Sponsor: Sanjeet S. Grewal

Must be taking: Anti-seizure medications

Disqualifiers: Intracranial tumor, Pregnancy, Cancer, Diabetes, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 3

5 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. However, since the trial involves patients with drug-resistant epilepsy, it is likely that you will continue your current anti-seizure medications.

What data supports the effectiveness of the treatment Allogenic Adipose-derived Mesenchymal Stem Cells for epilepsy?

Research shows that adipose-derived stem cell transplantation can reduce seizures and improve learning and memory in animal models of epilepsy. Additionally, stem cell therapy, including mesenchymal stem cells, has shown promise in controlling seizures in drug-resistant epilepsy patients. ^{1 2 3 4 5}

Is stem cell therapy safe for treating epilepsy?

Stem cell therapy, including mesenchymal stem cells (MSCs), appears to be safe for use in humans, as no severe side effects directly related to the therapy were reported in clinical studies. These studies, although limited, suggest that MSC therapy is generally safe and may reduce seizure frequency in patients with drug-resistant epilepsy. ^{1 3 4 6 7}

How is the treatment with Allogenic Adipose-derived Mesenchymal Stem Cells different from other epilepsy treatments?

This treatment uses stem cells derived from fat tissue, which can help reduce seizures by modulating the immune system and promoting brain repair, unlike traditional epilepsy medications that mainly focus on controlling symptoms. It offers a novel approach by potentially addressing the underlying causes of epilepsy, especially in cases where inflammation plays a significant role. ^{1 3 5 6 8}

What is the purpose of this trial?

This study is a non-randomized, open label, phase 1 clinical trial to evaluate the fesibility and safety of intrathalamic delivery of MSCs during standard of care DBS surgery for epilepsy. Subjects will be screened at our outpatient clinic and interested qualified subjects will be consented and offered participation in this trial. Once consent has been obtained, patients will undergo a standard preoperative evaluation which includes baseline laboratory values and a high-definition MRI. Patients will then undergo a stereotactic procedure for bilateral thalamic implantation of DBS leads through the ClearPoint® system. After the thalamic target for DBS is identified, cells will be infused directly into the anterior nucleus of the thalamus previous to lead implantation. Patients will be followed in the outpatient setting for up to a year after therapy application. Surgical, clinical, and radiographic data will be obtained during these visits

Research Team

Sanjeet Grewal, MD

Principal Investigator

Mayo Clinic

Eligibility Criteria

This trial is for adults over 18 with Drug-Resistant Epilepsy planning to undergo deep brain stimulation (DBS) surgery. They must have normal or slightly elevated blood clotting times, liver function tests within certain limits, and adequate kidney function. Participants need a hemoglobin level of at least 9 g/dL, platelets count above 100 x 10^9/L, and an absolute neutrophil count over 1.5 x 10^9/L.

Inclusion Criteria

I am getting a specific brain surgery for DBS leads in the thalamus.

My kidney function tests are within normal limits.

My epilepsy does not respond to medication.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Preoperative Evaluation

Standard preoperative evaluation including baseline laboratory values and high-definition MRI

1-2 weeks

1 visit (in-person)

Surgery and Treatment

Stereotactic procedure for bilateral thalamic implantation of DBS leads and infusion of AMSCs

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, including surgical, clinical, and radiographic data collection

12 months

Multiple visits (in-person)

Treatment Details

Interventions

• Allogenic Adipose-derived Mesenchymal Stem Cells

Trial Overview The study is testing the safety and feasibility of injecting Mesenchymal Stem Cells (MSCs) into the thalamus during DBS surgery in epilepsy patients. It's an early-phase trial where participants receive stem cells before having electrodes implanted in their brains to see if this can improve outcomes.

Participant Groups

Group I: AMSCs Experimental Treatment 1 Intervention

A single dose of 5x10\^6 AMSCs will be resuspended in 1ml of LRS and infused via intraparenchymal at the time of DBS surgery

Who Is Running the Clinical Trial?

Sanjeet S. Grewal

Lead Sponsor

Trials

1

Recruited

5+

Findings from Research

In a Phase I clinical trial involving patients with drug-resistant epilepsy, the use of autologous mesenchymal stem cells (MSC) was well tolerated and did not lead to severe adverse effects, indicating a good safety profile for this therapy.

The MSC therapy showed promising efficacy, with 30% of patients achieving seizure remission and 50% becoming responders to anti-epileptic drugs, compared to only 17% in the control group, highlighting the potential of MSC as a treatment option for epilepsy resistant to conventional medications.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Treatment of refractory epilepsy patients with autologous mesenchymal stem cells reduces seizure frequency: An open label study. Hlebokazov, F., Dakukina, T., Ihnatsenko, S., et al.[2018]

References

1. United States pubmed.ncbi.nlm.nih.gov

Intrathecal Infusion of Autologous Adipose-Derived Regenerative Cells in Autoimmune Refractory Epilepsy: Evaluation of Safety and Efficacy. [2022]

2. Switzerland pubmed.ncbi.nlm.nih.gov

Stem Cell Therapy in Treating Epilepsy. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

A cell-free extract from human adipose stem cells protects mice against epilepsy. [2015]

4. Netherlands pubmed.ncbi.nlm.nih.gov

Treatment of refractory epilepsy patients with autologous mesenchymal stem cells reduces seizure frequency: An open label study. [2018]

5. Netherlands pubmed.ncbi.nlm.nih.gov

Adipose-derived stem cell transplantation improves learning and memory via releasing neurotrophins in rat model of temporal lobe epilepsy. [2021]

6. United States pubmed.ncbi.nlm.nih.gov

Mesenchymal stem cell therapy for focal epilepsy: A systematic review of preclinical models and clinical studies. [2022]

7. United Arab Emirates pubmed.ncbi.nlm.nih.gov

Reversal of Neuropsychiatric Comorbidities in an Animal Model of Temporal Lobe Epilepsy Following Systemic Administration of Dental Pulp Stem Cells and Bone Marrow Mesenchymal Stem Cells. [2023]

8. Netherlands pubmed.ncbi.nlm.nih.gov

Bone marrow mononuclear cells reduce seizure frequency and improve cognitive outcome in chronic epileptic rats. [2013]