CELLTOP Clinical Trial: Phase 1 Findings on Adipose-Derived Stem Cell Therapy for Spinal Cord Injury Paralysis

Spinal cord injuries (SCI) are life-altering conditions with limited treatment options. While rehabilitation and medical management can provide some improvements, regenerative medicine is emerging as a promising alternative. The CELLTOP study, an on-going multidisciplinary phase 1 study conducted at the Mayo Clinic, is investigating the safety and efficacy of adipose tissue–derived mesenchymal stem cells (AD-MSCs) to aid in spinal cord recovery. 

In this initial report, Bydon et al. describe the outcome of the study’s first treated patient – a 53-year-old survivor of a surfing accident who sustained a high cervical American Spinal Injury Association Impairment Scale grade A SCI with subsequent neurologic improvement that plateaued within 6 months following injury.

The CELLTOP Trial Stem Cell Treatment Process 

Nine months after his injury, the patient enrolled in the CELLTOP study. An abdominal fat tissue sample was collected, and stem cells were isolated, expanded, and preserved. Eleven months after the injury, the patient received an injection of 100 million AD-MSCs through a lumbar puncture at the L3-4 level.

Safety and Tolerability: Minimal Side Effects Observed in Trial

According to the authors, the procedure was well tolerated. The only reported side effect was a mild to moderate headache on the second day, which resolved with over-the-counter medication. No severe adverse effects were observed during the 18-month follow-up.

Observed Neurological Improvements 

Following the stem cell injection, the patient showed notable improvements in motor and sensory function over 18 months, including:

• Motor Function: The patient’s upper limb motor scores improved from 35 at baseline to 44 at 18 months. Lower limb motor scores increased from 36 to 49. These improvements were observed in both sides of the body.
• Sensory Function: Sensation, measured through pinprick and light touch scores, nearly doubled. The pinprick score increased from 45 to 95, and the light touch score improved from 54 to 96.
• Upper Extremity Capabilities: The patient’s ability to use his arms and hands improved significantly, particularly in tasks requiring pulling, pushing, and finger dexterity.
• Quality of Life: The patient’s physical and mental health scores improved, as measured by the Patient-Reported Outcomes Measurement Information System (PROMIS) questionnaire.

Observed Improvements in Physical Therapy Performance 

Over the 18-month follow-up, the patient also demonstrated significant progress in mobility and strength, including:

• His walking speed improved from 0.17 m/s to 0.43 m/s.
• Walking distance increased from 635 feet in 12.8 minutes to 2200 feet in 34 minutes.
• Shoulder flexibility improved, with greater range of motion in both arms.
• Grip strength and hand dexterity showed notable gains.

How Stem Cells Aid in Spinal Cord Repair

SCI leads to significant nerve damage and scarring that inhibit natural healing. Stem cells offer potential benefits by:

• Reducing Inflammation: AD-MSCs have anti-inflammatory properties that may create a more favorable environment for nerve regeneration.
• Promoting Tissue Repair: These cells can support the growth of new nerve cells and blood vessels, enhancing recovery.
• Enhancing Neuroprotection: Stem cells may help preserve existing nerve function and prevent further deterioration.

Future Prospects of Regenerative Medicine for SCI 

While this case study presents promising results, further research is necessary. The CELLTOP study continues to evaluate the effectiveness of stem cell therapy in more patients. Future related studies will explore optimal dosing, timing, and potential combination therapies to enhance recovery further.

Based on these initial results, the authors conclude that regenerative medicine -particularly stem cell therapy – holds significant promise for treating SCI. The first patient in the CELLTOP study demonstrated meaningful neurological improvements, suggesting that AD-MSC therapy could offer new hope for those with SCI. Continued research and clinical trials will determine whether this treatment can become a standard option for spinal cord injury recovery.

Source: Bydon M, Dietz AB, Goncalves S, Moinuddin FM, Alvi MA, Goyal A, Yolcu Y, Hunt CL, Garlanger KL, Del Fabro AS, Reeves RK, Terzic A, Windebank AJ, Qu W. 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. Mayo Clin Proc. 2020 Feb;95(2):406-414. doi: 10.1016/j.mayocp.2019.10.008. Epub 2019 Nov 27. PMID: 31785831.