Stem Cell Therapy for Neurological Disorders

(NEST Trial)

Recruiting at5 trial locations

Overseen by Jeffrey Weiss, MD

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Academic

Recruiting

Sponsor: MD Stem Cells

Disqualifiers: Pregnancy, Medically unstable, others

No Placebo Group

Trial Summary

What is the purpose of this trial?

This is a human clinical study involving the isolation of autologous bone marrow derived stem cells (BMSC) and transfer to the vascular system and inferior 1/3 of the nasal passages in order to determine if such a treatment will provide improvement in neurologic function for patients with certain neurologic conditions. http://mdstemcells.com/nest/

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, it mentions that you should be stable on your current treatment and unlikely to improve with it, suggesting you may continue your existing medications.

What data supports the effectiveness of this treatment for neurological disorders?

Research shows that delivering bone marrow stromal cells (BMSCs) through the nose can help these cells reach the brain and spinal cord, potentially aiding recovery after strokes and spinal injuries in animal studies. This method has shown promise in improving function and promoting repair in these conditions. ^{1 2 3 4 5}

Is stem cell therapy using bone marrow stromal cells generally safe for humans?

The research includes a study on the safety of intravenous autologous bone marrow mononuclear stem cell therapy for ischemic stroke, which suggests that this method has been explored for safety in humans. ^{1 2 3 6 7}

What makes the intranasal and intravenous BMSC treatment unique for neurological disorders?

This treatment is unique because it uses a non-invasive intranasal route to deliver bone marrow stromal cells (BMSCs) directly to the brain, bypassing the blood-brain barrier, which is a major obstacle for many therapies. This method allows the cells to migrate to the affected areas and potentially improve recovery and regeneration in neurological disorders. ^{1 2 4 5 8}

Research Team

Steven Levy, MD

Principal Investigator

MD Stem Cells

Jeffrey Weiss, MD

Principal Investigator

Coral Springs, Florida

Eligibility Criteria

Adults over 18 with stable neurologic conditions like ALS, MS, stroke, or brain injuries not expected to improve with current treatments. They must be at least 6 months post-diagnosis and cleared by a doctor as safe for the procedure. Pregnant women or those who may become pregnant within 3 months post-treatment are excluded.

Inclusion Criteria

My current treatment for my condition is stable and not expected to reverse my neurological symptoms.

My current treatment for my condition is stable and not expected to reverse any nerve damage.

Be medically stable and able to be medically cleared by their primary care physician or a licensed primary care practitioner for the procedure. Medical clearance means that in the estimation of the primary care practitioner, the patient can reasonably be expected to undergo the procedure without significant medical risk to health

Exclusion Criteria

In the estimation of Dr. Weiss, the BMSC collection and treatment will not present a significant risk of harm to the patient's general health or to their neurologic function

I am willing and able to attend follow-up neurological exams as required.

Patients who are not medically stable or who may be at significant risk to their health undergoing the procedure will not be eligible

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive autologous bone marrow derived stem cells intravenously and intranasally

1 month

1 visit (in-person)

Follow-up

Participants are monitored for changes in neurologic function using the Neuro-QOL questionnaire

12 months

Visits at 1, 3, 6, and 12 months (in-person)

Treatment Details

Interventions

• Intranasal BMSC (Stem Cell Therapy)
• Intravenous and Intranasal BMSC (Stem Cell Therapy)
• Intravenous BMSC (Stem Cell Therapy)

Trial Overview The trial is testing whether injecting patients' own bone marrow stem cells into their bloodstream and nasal passages can improve neurological function in various chronic diseases.

Participant Groups

Group I: Arm 1- Intravenous and Intranasal BMSC Experimental Treatment 1 Intervention

Intervention- Autologous bone marrow aspiration and separation of Bone Marrow Derived Stem Cell (BMSC) fraction then provided intravenously and intranasally (lower 1/3 of nasal passages).

Who Is Running the Clinical Trial?

MD Stem Cells

Lead Sponsor

Trials

6

Recruited

1,500+

Findings from Research

Repeated intranasal delivery of hypoxic preconditioned bone marrow stromal cells (HP-BMSCs) significantly enhances neurovascular repair and functional recovery after ischemic stroke in mice, as shown by improved local blood flow and better performance in behavioral tests.

The study demonstrated that HP-BMSCs express higher levels of regenerative factors, leading to increased cell survival and migration to the affected brain area, indicating a promising non-invasive treatment strategy for stroke recovery.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Delayed and repeated intranasal delivery of bone marrow stromal cells increases regeneration and functional recovery after ischemic stroke in mice. Chau, MJ., Deveau, TC., Gu, X., et al.[2023]

Intranasally delivered bone marrow stromal cells (BMSCs) successfully migrated to spinal cord injuries in rats and contributed to improved hind limb motor function and reduced lesion cavity size over 2 to 4 weeks.

While the intranasal route showed some efficacy, the results were not as pronounced as those achieved with intrathecal BMSC administration, indicating that while intranasal delivery is a viable option, it may be less effective than direct spinal delivery.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Intranasal delivery of bone marrow stromal cells to spinal cord lesions. Ninomiya, K., Iwatsuki, K., Ohnishi, Y., et al.[2015]

In a study involving 40 adult male rats with traumatic brain injury, both intravenous injection of bone marrow mesenchymal stem cells (BMSC) and mobilization of hematopoietic stem cells using G-CSF showed significant functional improvements compared to a control group.

There was no significant difference in functional outcomes between the BMSC and G-CSF treatment groups, indicating that both methods may be equally effective in promoting recovery after TBI.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Comparison of transplantation of bone marrow stromal cells (BMSC) and stem cell mobilization by granulocyte colony stimulating factor after traumatic brain injury in rat. Bakhtiary, M., Marzban, M., Mehdizadeh, M., et al.[2021]

Intrathecal injection of bone marrow stromal cells (BMSCs) in a rat model of Alzheimer's disease led to the successful migration and differentiation of these cells into cholinergic neurons, which are crucial for cognitive function.

The treatment resulted in an increase in healthy neurons and a decrease in damaged neurons, suggesting that BMSC therapy could be a promising approach for Alzheimer's disease management.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The effect of intrathecal delivery of bone marrow stromal cells on hippocampal neurons in rat model of Alzheimer's disease. Eftekharzadeh, M., Nobakht, M., Alizadeh, A., et al.[2022]

Intranasal administration of stem cells offers a promising noninvasive method to bypass the blood-brain barrier (BBB) and deliver therapeutic agents directly to the brain, which is crucial for treating neurological disorders.

This review discusses various cell types and diseases that could benefit from intranasal stem cell therapy, highlighting both the potential advantages and challenges of this innovative treatment approach.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Advances in intranasal application of stem cells in the treatment of central nervous system diseases. Zhang, YT., He, KJ., Zhang, JB., et al.[2021]

Intravenous administration of bone marrow stromal cells (BMSC) showed similar therapeutic benefits for spinal cord injury (SCI) as intraspinal administration, with both methods improving motor function as measured by the BBB scale in a study involving adult female rats.

Immuno-histochemical analysis revealed that BMSCs administered intravenously differentiated into both neurons and glial cells, while intraspinally administered BMSCs primarily differentiated into glial cells, suggesting a potential advantage of the intravenous route in promoting neuronal repair.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A comparative study of therapeutic benefits of intraspinal and intravenous bone marrow stromal cell administration to spinal cord injuries. Khalatbary, AR., Tiraihi, T.[2016]

Transplanting bone marrow stromal cells (BMSCs) embedded in a platelet-rich plasma (PRP) scaffold significantly improves neurological function in rats after intracerebral hemorrhage compared to BMSCs administered in saline.

The use of PRP scaffolds not only enhances the survival of transplanted BMSCs but also promotes endogenous neurogenesis, leading to better integration of the cells and improved recovery outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cell therapy with bone marrow stromal cells after intracerebral hemorrhage: impact of platelet-rich plasma scaffolds. Vaquero, J., Otero, L., Bonilla, C., et al.[2018]

References

1. England pubmed.ncbi.nlm.nih.gov

Delayed and repeated intranasal delivery of bone marrow stromal cells increases regeneration and functional recovery after ischemic stroke in mice. [2023]

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

Intranasal delivery of bone marrow stromal cells to spinal cord lesions. [2015]

3. Iran pubmed.ncbi.nlm.nih.gov

Comparison of transplantation of bone marrow stromal cells (BMSC) and stem cell mobilization by granulocyte colony stimulating factor after traumatic brain injury in rat. [2021]

4. Iran pubmed.ncbi.nlm.nih.gov

The effect of intrathecal delivery of bone marrow stromal cells on hippocampal neurons in rat model of Alzheimer's disease. [2022]

5. England pubmed.ncbi.nlm.nih.gov

Advances in intranasal application of stem cells in the treatment of central nervous system diseases. [2021]

6. Iran pubmed.ncbi.nlm.nih.gov

A comparative study of therapeutic benefits of intraspinal and intravenous bone marrow stromal cell administration to spinal cord injuries. [2016]

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

Intravenous autologous bone marrow mononuclear stem cell therapy for ischemic stroke: a multicentric, randomized trial. [2022]

8. England pubmed.ncbi.nlm.nih.gov

Cell therapy with bone marrow stromal cells after intracerebral hemorrhage: impact of platelet-rich plasma scaffolds. [2018]