We first reported a successful case of treating spinal cord injury with umbilical cord mesenchymal stem cells (UC-MSC) back in 2009. Fast forward 10 years to November 2019: the Mayo Clinic in Rochester is now doing a clinical trial with mesenchymal stem cells derived from adipose tissue (fat) to treat spinal cord injury. In this paper, they report the amelioration of a 53-year-old male patient with grade C trauma to the spinal cord at the time of enrollment. He received 100 million of his own AD-MSC and experienced no adverse events save a moderate headache. He was followed for 1.5 years after treatment: the authors report improvements in neurological function, as indicated in several categories of ASIA (American Spinal Injury Association) scores.
The authors call for larger trials to demonstrate the safety and efficiency of this therapeutic option. Interestingly, they conclude noting that “It is important to demonstrate the successful translation of novel therapeutics using a multimodal approach at a time when such therapeutic options are attracting scrutiny by the US Food and Drug Administration.”
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.
Spinal cord injury (SCI) is a devastating condition with limited pharmacological treatment options to restore function. Regenerative approaches have recently attracted interest as an adjuvant to current standard of care. Adipose tissue-derived (AD) mesenchymal stem cells (MSCs) represent a readily accessible cell source with high proliferative capacity. The CELLTOP study, an ongoing multidisciplinary phase 1 clinical trial conducted at Mayo Clinic (ClinicalTrials.gov Identifier: NCT03308565), is investigating the safety and efficacy of intrathecal autologous AD-MSCs in patients with blunt, traumatic SCI. In this initial report, we describe the outcome of the 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. Although he improved to an American Spinal Injury Association grade C impairement classification, the individual continued to be wheelchair bound and severely debilitated. After study enrollment, an adipose tissue biopsy was performed and MSCs were isolated, expanded, and cryopreserved. Per protocol, the patient received an intrathecal injection of 100 million autologous AD-MSCs infused after a standard lumbar puncture at the L3-4 level 11 months after the injury. The patient tolerated the procedure well and did not experience any severe adverse events. Clinical signs of efficacy were observed at 3, 6, 12, and 18 months following the injection in both motor and sensory scores based on International Standards for Neurological Classification of Spinal Cord Injury. Thus, in this treated individual with SCI, intrathecal administration of AD-MSCs was feasible and safe and suggested meaningful signs of improved, rather than stabilized, neurologic status warranting further clinical evaluation.