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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether using a person's own stem cells from fat (Autologous Adipose Derived Mesenchymal Stem Cells) can safely aid recovery from spinal cord injuries. The study examines if these special cells can improve movement and sensation after a spinal injury. Participants will receive either a stem cell injection immediately or after six months of standard care and therapy. Individuals with a blunt, non-penetrating spinal cord injury and a specific level of movement loss may be suitable for this trial. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

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.

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

Research has shown that using a person's own fat tissue to create stem cells is generally safe for individuals with spinal cord injuries. These stem cells are extracted from the person's fat and injected into the spinal canal. Studies have found that this procedure is well-tolerated, with patients not experiencing serious side effects. Another study confirmed that patients handled the treatment well over time. While the results are promising, ongoing research aims to ensure these findings apply to a broader population. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about using autologous adipose derived mesenchymal stem cells (AD-MSCs) for spinal cord injury because they tap into the body's own healing potential. Unlike traditional treatments that mainly focus on physical rehabilitation and symptom management, this approach uses stem cells harvested from the patient's own fat tissue, which are then expanded and delivered directly into the spinal fluid. This method aims to promote neural repair and regeneration, potentially improving outcomes in ways current therapies can't match. By directly targeting the injury site with these biologically active cells, there's hope for more substantial recovery of function.

What evidence suggests that this trial's treatments could be effective for spinal cord injury?

Research has shown that injections of stem cells from fat tissue may help treat spinal cord injuries. In this trial, participants in Treatment Group 1 will receive a single dose of autologous adipose-derived mesenchymal stem cells (AD-MSCs) to aid in repairing nerve damage by encouraging the growth of new nerve tissues. Studies have found that injecting these cells into the spine can improve function and recovery. Early results suggest that these cells can become nerve-like cells, aiding healing and reducing inflammation. Meanwhile, participants in Treatment Group 2 will initially receive the best medical management, including physical and occupational therapy, followed by a single dose of AD-MSCs after six months. While more research is needed, the initial findings are promising for people with spinal cord injuries. ^{2 3 4 5 6}

Who Is on the Research Team?

Wenchun Qu, MD

Principal Investigator

Mayo Clinic

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

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]

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]

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]

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]

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 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]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11394073/

Adipose-Derived Stem Cell Therapy in Spinal Cord Injury Adipose-derived stem cell (ADSC) injections in SCI cases have been investigated and showed promising results.

2. nature.com nature.com/articles/s41467-024-46259-y

Intrathecal delivery of adipose-derived mesenchymal stem ... The effects of adipose tissue-derived mesenchymal stem cell transplantation during the acute and subacute phases following spinal cord injury.

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5137573/

Intrathecal transplantation of autologous adipose-derived ... However, there is currently no effective treatment for SCI. The therapeutic potential of adipose-derived mesenchymal stem cells (ADMSCs) has been emerged.

4. clinicaltrials.gov clinicaltrials.gov/study/NCT01274975

NCT01274975 | Autologous Adipose Derived MSCs ... This study is designed to assess the safety of intravenous autologous adipose derived mesenchymal stem cells transplant in spinal cord injury patients.

5. sciencedirect.com sciencedirect.com/science/article/pii/S2352320424001354

Adipose-derived stem cell therapy for spinal cord injuries ADSCs are a promising regenerative therapy for spinal cord injury and paralysis. •. ADSCs promote neuroregeneration through differentiation, secretion, and ...

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/38561341/

Intrathecal delivery of adipose-derived mesenchymal stem ... Intrathecal delivery of autologous culture-expanded adipose tissue-derived mesenchymal stem cells (AD-MSC) could be utilized to treat traumatic spinal cord ...