Stem Cell Therapy for Spinal Cord Injury

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Mayo Clinic

Must not be taking: Immunosuppressants, Antirheumatics, Steroids, others

Disqualifiers: Pregnancy, Non-traumatic SCI, Infections, others

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

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 1

Duration: 1 day

40 Participants Needed

Trial Summary

What is the purpose of this trial?

The purpose of this research study is to investigate the safety and potential therapeutic effects of autologous, culture-expanded, adipose derived mesenchymal stem cell intrathecal injections in the treatment of spinal cord injury.

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications, such as anti-rheumatic drugs and systemic steroids, at least 3 months before enrolling. If you are currently on chronic immunosuppressive therapy or taking riluzole for ALS, you will not be eligible to participate.

What data supports the effectiveness of the treatment for spinal cord injury?

Research shows that using adipose-derived stem cells (cells taken from fat tissue) can help improve recovery in spinal cord injury. Studies in rats and some human cases have shown that these cells can aid in functional recovery, suggesting potential benefits for patients with spinal cord injuries. ^{1 2 3 4 5}

Is stem cell therapy using adipose-derived mesenchymal stem cells safe for humans?

In a clinical trial, a patient with a spinal cord injury received adipose-derived mesenchymal stem cells and tolerated the procedure well without any severe adverse events, suggesting it is safe for humans. ^{1 2 3 6 7}

How is the treatment with Autologous Adipose Derived Mesenchymal Stem Cells different from other treatments for spinal cord injury?

This treatment is unique because it uses stem cells derived from a patient's own fat tissue, which are then infused back into the body to help repair spinal cord damage. Unlike other treatments, it focuses on using the body's own cells to promote healing and regeneration, offering a personalized approach to recovery. ^{1 2 3 4 7}

Research Team

Mohamad Bydon, MD

Principal Investigator

Mayo Clinic

Eligibility Criteria

Adults aged 18+ with traumatic, non-penetrating spinal cord injuries (SCI) graded A or B at injury time, who've seen no significant improvement for a year. Participants must understand and commit to the study's requirements including home exercises and follow-ups. Women of childbearing age need a negative pregnancy test and must use contraception during the study.

Inclusion Criteria

I am not pregnant, can use birth control, and am of childbearing age.

My spinal cord injury was severe at first but improved within a year, though it has not gotten better since.

If a woman becomes pregnant during the study, she will be closely watched to make sure both she and the baby are healthy. Any serious issues related to the pregnancy will be noted.

Exclusion Criteria

I have taken medication for rheumatic disease in the last 3 months.

I have had treatments involving stem cells, gene therapy, or exosomes.

I can have an MRI and am willing to undergo MRI procedures.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Patients receive a single dose of autologous, adipose derived mesenchymal stem cells

1 day

1 visit (in-person)

Observation

Patients are observed over six months while attending physical and occupational therapy

6 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 months

Treatment Details

Interventions

• Autologous Adipose Derived Mesenchymal Stem Cells
• Best Medical Management: Occupational and Physical Therapy

Trial Overview The trial is testing if one's own adipose-derived mesenchymal stem cells can safely improve SCI outcomes when injected into the spinal fluid, alongside standard occupational and physical therapy treatments.

Participant Groups

Group I: Treatment Group 1: AD-MSC Injection Experimental Treatment 1 Intervention

Patients will receive a single dose of autologous, adipose derived mesenchymal stem cells one time. The cells are isolated from patient's adipose tissue and expanded for intrathecal delivery.

Group II: Treatment Group 2: Best Medical Management Active Control 2 Interventions

Patients will be observed over six months while attending physical and occupational therapy. After six months, patients will receive a single dose of autologous, adipose derived mesenchymal stem cells one time. The cells are isolated from patient's adipose tissue and expanded for intrathecal delivery.

Who Is Running the Clinical Trial?

Mayo Clinic

Lead Sponsor

Trials

3,427

Recruited

3,221,000+

Findings from Research

Intravenous transplantation of adipose-derived stem/stromal cells (ASCs) in rats with spinal cord injury (SCI) led to improved functional recovery and reduced spinal cord damage, although the long-term survival of the transplanted cells was not achieved.

The study identified that the cytokine CINC-1, secreted by ASCs, played a significant role in promoting recovery by activating key signaling pathways (ERK1/2 and Akt) in the spinal cord, suggesting a mechanism through which ASCs exert their therapeutic effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Intravenous infusion of adipose-derived stem/stromal cells improves functional recovery of rats with spinal cord injury. Ohta, Y., Hamaguchi, A., Ootaki, M., et al.[2018]

In a study involving 10 patients with posttraumatic paraplegia, co-infusion of autologous adipose-derived mesenchymal stem cells (N-Ad-MSC) and hematopoietic stem cells (HSC) into the cerebrospinal fluid was found to be safe, with no adverse effects reported.

Patients showed variable and sustained improvements in their mobility and function over an average follow-up of nearly 3 years, suggesting that stem cell therapy may enhance recovery even after a significant duration post-injury.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Infusion of autologous adipose tissue derived neuronal differentiated mesenchymal stem cells and hematopoietic stem cells in post-traumatic paraplegia offers a viable therapeutic approach. Thakkar, UG., Vanikar, AV., Trivedi, HL., et al.[2020]

Transplanting human adipose tissue-derived mesenchymal stromal cells (hADSCs) into a rat model of spinal cord injury (SCI) resulted in better functional recovery and significant increases in brain-derived neurotrophic factor (BDNF) levels compared to human bone marrow-derived mesenchymal stromal cells (hBMSCs).

hADSCs demonstrated higher proliferative activity and enhanced expression of growth factors, leading to improved angiogenesis, preserved axons, and reduced lesion cavity formation, suggesting they may be a more effective treatment option for SCI than hBMSCs.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Comparison of mesenchymal stromal cells from human bone marrow and adipose tissue for the treatment of spinal cord injury. Zhou, Z., Chen, Y., Zhang, H., et al.[2018]

Transplanting rat adipose tissue-derived mesenchymal stem cells (rAT-MSCs) significantly improved tissue regeneration and functional recovery in spinal cord injury (SCI) models, as shown by evaluations using the BBB rating system and immunostaining for regeneration markers.

The timing of rAT-MSC transplantation was less critical than expected, as immediate transplantation after injury was not significantly more effective than transplantation 9 days post-injury, indicating the adaptive capabilities of these stem cells in promoting recovery.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The Effects of Adipose Tissue-Derived Mesenchymal Stem Cell Transplantation During the Acute and Subacute Phases Following Spinal Cord Injury. Aras, Y., Sabanci, PA., Kabatas, S., et al.[2018]

In a study involving rat models of spinal cord injury, both undifferentiated adipose tissue-derived stromal cells (uADSCs) and differentiated ADSCs (dADSCs) showed significant improvements in locomotor function compared to a saline control, suggesting that both types of cells can aid recovery.

Interestingly, the undifferentiated uADSCs and those differentiated using Protocol 2 (dADSC-P2) led to better functional recovery than those differentiated by Protocol 1 (dADSC-P1), indicating that pre-transplant differentiation may not be necessary for effective treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effects of differentiated versus undifferentiated adipose tissue-derived stromal cell grafts on functional recovery after spinal cord contusion. Zhang, HT., Luo, J., Sui, LS., et al.[2021]

The CELLTOP study is investigating the safety and efficacy of intrathecal injections of autologous adipose tissue-derived mesenchymal stem cells (AD-MSCs) in patients with spinal cord injury, with initial results showing that the first patient tolerated the procedure well without severe adverse events.

After receiving 100 million AD-MSCs, the patient demonstrated meaningful neurological improvement over 18 months, suggesting that this regenerative approach may offer potential benefits for restoring function in spinal cord injury cases.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

CELLTOP Clinical Trial: First Report From a Phase 1 Trial of Autologous Adipose Tissue-Derived Mesenchymal Stem Cells in the Treatment of Paralysis Due to Traumatic Spinal Cord Injury. Bydon, M., Dietz, AB., Goncalves, S., et al.[2020]

In a study involving 14 patients with spinal cord injury, intrathecal transplantation of autologous adipose-derived mesenchymal stem cells (ADMSCs) showed mild improvements in neurological function, with 5 patients experiencing increased ASIA motor scores after 8 months.

The procedure was generally safe, with no serious adverse events reported, although some patients experienced mild side effects like urinary tract infections and nausea, indicating that further research is needed to optimize patient selection and treatment protocols.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Intrathecal transplantation of autologous adipose-derived mesenchymal stem cells for treating spinal cord injury: A human trial. Hur, JW., Cho, TH., Park, DH., et al.[2018]

References

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

Intravenous infusion of adipose-derived stem/stromal cells improves functional recovery of rats with spinal cord injury. [2018]

2. India pubmed.ncbi.nlm.nih.gov

Infusion of autologous adipose tissue derived neuronal differentiated mesenchymal stem cells and hematopoietic stem cells in post-traumatic paraplegia offers a viable therapeutic approach. [2020]

3. England pubmed.ncbi.nlm.nih.gov

Comparison of mesenchymal stromal cells from human bone marrow and adipose tissue for the treatment of spinal cord injury. [2018]

4. Turkey pubmed.ncbi.nlm.nih.gov

The Effects of Adipose Tissue-Derived Mesenchymal Stem Cell Transplantation During the Acute and Subacute Phases Following Spinal Cord Injury. [2018]

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

Effects of differentiated versus undifferentiated adipose tissue-derived stromal cell grafts on functional recovery after spinal cord contusion. [2021]

6. England pubmed.ncbi.nlm.nih.gov

CELLTOP Clinical Trial: First Report From a Phase 1 Trial of Autologous Adipose Tissue-Derived Mesenchymal Stem Cells in the Treatment of Paralysis Due to Traumatic Spinal Cord Injury. [2020]

7. England pubmed.ncbi.nlm.nih.gov

Intrathecal transplantation of autologous adipose-derived mesenchymal stem cells for treating spinal cord injury: A human trial. [2018]