Stem Cell Therapy for Neurological Disorders

(NEST Trial)

Recruiting at 5 trial locations

Overseen By Steven Levy, MD, Study Director

Age: 18+

Sex: Any

Trial Phase: Academic

Sponsor: MD Stem Cells

Disqualifiers: Pregnancy, Medically unstable, others

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

Compensation: Varies

Number of Visits: 2

Duration: 1 month

500 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether stem cell therapy can improve neurological function in individuals with certain nerve-related conditions. The treatment uses cells from the participant's own bone marrow, delivered through the bloodstream and nose, to assess its potential in aiding nerve damage. It suits those who have experienced nerve issues for over six months without improvement from current treatments. Participants must be medically stable and able to safely undergo the procedure. As an unphased trial, this study provides a unique opportunity to contribute to groundbreaking research that could lead to new treatments for nerve-related conditions.

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.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that using bone marrow stem cells (BMSC) through a vein or the nose is generally safe for treating brain and nerve problems. One study found that administering these cells to patients did not cause major issues. The cells can help reduce brain swelling and repair damage. Another study supports their use for brain and nerve diseases, indicating that this treatment is well-researched and backed by early studies. However, more research is needed to determine its effectiveness in larger groups. Overall, the treatment is considered safe, with few reports of serious side effects. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Unlike the standard treatments for neurological disorders, which often focus on managing symptoms with medications or physical therapy, this innovative approach uses bone marrow-derived stem cells (BMSC). Researchers are excited because BMSC can be administered both intravenously and intranasally, potentially allowing for more direct and effective delivery to the brain. This method taps into the body's own regenerative capabilities, offering a new way to repair damaged tissues. The potential for BMSC to target the root causes of neurological disorders, rather than just alleviating symptoms, sets it apart from current therapies.

What evidence suggests that this trial's treatments could be effective for neurological disorders?

Research has shown that bone marrow stem cells (BMSCs) might help treat brain and nerve disorders. In this trial, participants will receive BMSCs both intravenously and intranasally. Some studies have found that administering BMSCs through a vein or the nose can improve brain functions, particularly in diseases like Parkinson's. Patients have reported better balance, more control over facial muscles, and fewer symptoms such as a stiff facial expression. Animal studies also support these findings, demonstrating improved brain function after stem cell treatments. However, for some conditions like stroke, other studies have not observed significant improvement. Overall, while early results are promising, effectiveness can depend on the specific condition. ^{2 6 7 8 9}

Who Is on the Research Team?

Steven Levy, MD

Principal Investigator

MD Stem Cells

Jeffrey Weiss, MD

Principal Investigator

Coral Springs, Florida

Are You a Good Fit for This Trial?

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

Timeline for a Trial Participant

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)

What Are the Treatments Tested in This Trial?

Interventions

• Intranasal BMSC
• Intravenous and Intranasal BMSC
• Intravenous BMSC

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.

How Is the Trial Designed?

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).

Intranasal BMSC is already approved in United States for the following indications:

Approved in United States as Intranasal BMSC for:

• Neurologic conditions
• Spinal cord injuries
• Neurological damage

Who Is Running the Clinical Trial?

MD Stem Cells

Lead Sponsor

Trials

6

Recruited

1,500+

Published Research Related to This Trial

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]

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]

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]

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]

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]

In a phase II trial involving 120 patients with subacute ischemic stroke, intravenous infusion of autologous bone marrow mononuclear stem cells (BMSCs) was found to be safe, with no significant adverse events reported.

However, the study showed no significant improvement in stroke outcomes, as measured by the Barthel Index and modified Rankin scale, indicating that BMSCs did not provide a beneficial effect compared to the control group.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Intravenous autologous bone marrow mononuclear stem cell therapy for ischemic stroke: a multicentric, randomized trial. Prasad, K., Sharma, A., Garg, A., et al.[2022]

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]

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]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12123819/

Outcomes of autologous bone marrow mononuclear cell ... This study evaluated the effectiveness of intrathecal autologous bone marrow mononuclear cell (BMMNC) therapy combined with education compared with education ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT02795052?cond=Parkinson%20Disease%20stem%20cells&aggFilters=status:rec&viewType=Table&rank=10

Neurologic Stem Cell Treatment Study (NEST) Intravenous administration of BMSC is a well-established approach to neurologic disease and injury with much support for its effectiveness in the pre-clinical ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7186734/

Effects of bone marrow mesenchymal stem cells ... Several experimental animal studies have demonstrated BMSC transplantation to significantly improve neurological function following ischemic injury, promoting ...

4. mdstemcells.com mdstemcells.com/neurological-stem-cell-treatment-study/

Neurological Stem Cell Treatment Study (NEST) Early results are promising: patients with Parkinson's Disease have reported improvements in balance, facial musculature, reduction of the characteristic mask- ...

5. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0014488620303733

Combinatorial intranasal delivery of bone marrow ... BMSC transplantation for ischemic stroke has failed to manifest any significant amelioration of neurological outcomes in multiple clinical trials (Prasad et al.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11725325/

Safety and Efficacy of Autologous Bone Marrow Derived ... This study aims to assess the safety and efficacy of autologous bone marrow-derived mononuclear cell (BM-MNC) therapy in patients with CP, traumatic SCI, and ...

7. sciencedirect.com sciencedirect.com/science/article/pii/S2405844024010818

Effects of bone marrow mesenchymal stromal cells-derived ... There has been evidence to suggest that MSC(M) and MSC(M) derived extracellular vesicles (EVs) diminish neuroinflammation, decrease blood-brain ...

8. link.springer.com link.springer.com/article/10.1007/s10072-023-07274-x

Safety and efficacy of bone marrow mononuclear cell ... Our meta-analysis demonstrated that the transplantation of BMMNCs was safe; however, the efficacy of this procedure requires further validation in larger RTCs.

9. researchgate.net researchgate.net/publication/311959922_Neurologic_Stem_Cell_Treatment_Study_NEST_using_bone_marrow_derived_stem_cells_for_the_treatment_of_neurological_disorders_and_injuries_study_protocol_for_a_nonrandomized_efficacy_trial

Neurologic Stem Cell Treatment Study (NEST) using bone ... The Neurologic Stem Cell Treatment Study (NEST) utilizes bone marrow derived stem cells (BMSCs) for neurological diseases and injuries to ...