Summary: A phase 1 clinical trial from the Mayo Clinic reports that stem cells taken from patients’ own fat tissue were safely administered and were associated with measurable improvements in sensation and movement for people with traumatic spinal cord injuries. Of the 10 adults treated, seven improved by at least one level on the ASIA (American Spinal Injury Association) Impairment Scale, showing gains in touch and pinprick sensation, muscle strength, and bowel-related function. No serious adverse events were observed.
These early results challenge the longstanding idea that spinal cord injury recovery is impossible beyond a limited early window and point toward the potential of cell therapy as part of future treatment strategies. The study underscores the need for larger, controlled trials to determine which patients benefit most, the extent of functional recovery possible, and how best to combine stem cells with other therapies.
Key Facts:
- Seven of 10 participants had measurable improvement in sensory and motor function after receiving autologous, adipose-derived mesenchymal stem cells delivered intrathecally.
- No serious adverse events were reported; the most common side effects were headache and musculoskeletal pain that resolved with over-the-counter treatment.
- The findings support continued study in larger randomized, controlled trials to evaluate efficacy, dosing, timing, and patient selection.
Source: Mayo Clinic
A Mayo Clinic phase 1 trial finds intrathecal delivery of adipose-derived mesenchymal stem cells may be safe and associated with neurological improvement after traumatic spinal cord injury.
The study appears in the journal Nature Communications and offers early clinical evidence about the potential role of autologous adipose-derived mesenchymal stem cells (AD-MSCs) in treating traumatic spinal cord injury (SCI), a condition with few options for restoring lost function.
In this small clinical trial of 10 adults with traumatic SCI, seven participants improved on the ASIA Impairment Scale. Improvements included better responses to pinprick and light touch testing, increased strength in affected muscle groups, and recovery of voluntary anal contraction—an important marker for improved bowel control. Each of the seven improved by at least one ASIA grade. Three participants showed no change but did not worsen.
“This study documents the safety and potential benefit of stem cells and regenerative medicine,” said Mohamad Bydon, M.D., Mayo Clinic neurosurgeon and first author. He emphasized that spinal cord injury is complex and that future research will examine whether stem cells, possibly combined with other interventions, could form part of a new treatment paradigm to improve outcomes.
No serious adverse events were reported. The most common side effects—headache and musculoskeletal pain—were temporary and responsive to simple analgesics. As a phase 1 trial, the primary goal was to establish safety; secondary measures examined motor and sensory changes but require cautious interpretation until larger controlled studies confirm efficacy.
Participants ranged from 18 to 65 years old and sustained injuries from motor vehicle accidents, falls, and other traumas. Six had cervical (neck) injuries and four had thoracic or upper back injuries. Researchers harvested a small sample of adipose tissue through a 1- to 2-inch incision in the abdomen or thigh, expanded the cells in culture to about 100 million cells over roughly four weeks, and administered them intrathecally into the lumbar spine. Each participant returned for 10 follow-up visits over two years.
While AD-MSCs are known to migrate to areas of inflammation, their precise interactions with injured spinal cord tissue remain incompletely understood. The research team analyzed MRI changes, cerebrospinal fluid markers, and sensory response testing to identify cellular processes that might support regeneration and functional recovery.
Notable outcomes included two patients with cervical injuries who received treatment 22 months after injury and nonetheless improved by one ASIA level. Additionally, two of three patients with complete thoracic injuries—cases where no feeling or movement exists below the injury level—improved by two ASIA grades, regaining some sensation and voluntary movement below the injury. Based on current understanding, spontaneous recovery for complete thoracic injuries is uncommon; researchers estimate only about 5% experience meaningful return of feeling or movement.
Research continues into stem cells for spinal cord injuries
In the United States, stem cell therapies for spinal cord injury remain primarily investigational, and adipose-derived stem cell treatment for SCI is considered experimental by the Food and Drug Administration. Worldwide, the World Health Organization estimates 250,000 to 500,000 new spinal cord injuries occur each year, underscoring a major global need for effective treatments.
An important next step is a larger randomized, controlled trial that assigns participants to receive either the stem cell therapy or a placebo procedure to better evaluate effectiveness, refine patient selection, and define optimal timing and dosing. Dr. Bydon noted that for decades treatment centered on stabilization and rehabilitation, but evolving evidence is beginning to challenge the belief that recovery beyond an early window is impossible. This research represents a meaningful step toward improving outcomes for people with SCI.
Dr. Bydon holds the Charles B. and Ann L. Johnson Professorship of Neurosurgery. The trial received support from multiple Mayo Clinic benefactors and foundations and was partially funded by a Mayo Clinic Transform the Practice grant.
For full author and funding details, consult the published study in Nature Communications.
About this stem cell and SCI research news
Author: Megan Luihn
Source: Mayo Clinic
Contact: Megan Luihn – Mayo Clinic
Image credit: Neuroscience News
Original Research: Open access. “Intrathecal delivery of adipose-derived mesenchymal stem cells in traumatic spinal cord injury: Phase I trial” by Mohamad Bydon et al., Nature Communications.
Abstract
Intrathecal delivery of adipose-derived mesenchymal stem cells in traumatic spinal cord injury: Phase I trial
This Phase I trial (ClinicalTrials.gov: NCT03308565) enrolled 10 patients who had ASIA grade A or B injuries at the time of injury. The primary objective was to evaluate safety, measured by the type and frequency of adverse events. Secondary outcomes assessed changes in motor and sensory scores, imaging findings, cerebrospinal fluid markers, and somatosensory evoked potentials. Manufacturing and delivery of autologous AD-MSCs were successful for all participants. Headache and musculoskeletal pain were the most commonly reported adverse events (8 patients). No serious adverse events occurred. At final follow-up, seven patients showed improvement in ASIA grade compared with baseline. The study met its primary safety endpoint, demonstrating that harvesting and intrathecal administration of autologous AD-MSCs were well tolerated in this cohort of patients with traumatic SCI.