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Adipose MSC for Spinal Cord Injury: ASIA Scores Improvement

Posted on December 4, 2019 Written by nhriordan Leave a Comment

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.”

 


Mayo Clin Proc. 2019 Nov 26. pii: S0025-6196(19)30871-7. doi: 10.1016/j.mayocp.2019.10.008.

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, Moinuddin FM, Alvi MA, Goyal A, Yolcu Y, Hunt CL, Garlanger KL, Reeves RK, Terzic A, Windebank AJ, Qu W.

Abstract

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.

PMID: 31785831


 

Filed Under: Uncategorized Tagged With: adipose, mesenchymal stem cells, spinal cord injury

Treatment with Mesenchymal Stem Cells Shown to be Safe for Dog Muscular Dystrophy

Posted on May 22, 2016 Written by nhriordan Leave a Comment

Duchenne Muscular Dystrophy (DMD) is a degenerative genetic disorder that affects the muscles in the body in a progressive manner, leading to eventual death. Mesenchymal stem cells (MSC) are able to reach the affected muscle cells and to start tissue regeneration and repair, making them an attractive treatment option for DMD. This study by Pelatti et al. demonstrates the safety of MSC to treat Golden Retriever Muscular Dystrophy – an animal disorder with remarkable similarities to human dystrophy. The treatment was well-tolerated and no long-term adverse effects were reported.


Stem Cell Rev. 2016 May 19. doi: 10.1007/s12015-016-9659-3

Transplantation of Human Adipose Mesenchymal Stem Cells in Non-Immunosuppressed GRMD Dogs is a Safe Procedure.

Pelatti MV, Gomes JP, Vieira NM, Cangussu E, Landini V, Andrade T, Sartori M, Petrus L, Zatz M.

Abstract

The possibility to treat Duchenne muscular dystrophy (DMD), a lethal X-linked disorder, through cell therapy with mesenchymal stromal cells (MSCs) has been widely investigated in different animal models. However, some crucial questions need to be addressed before starting human therapeutic trials, particularly regarding its use for genetic disorders. How safe is the procedure? Are there any side effects following mesenchymal stem cell transplantation? To address these questions for DMD the best model is the golden retriever muscular dystrophy dog (GRMD), which is the closest model to the human condition displaying a much longer lifespan than other models. Here we report the follow-up of 5 GRMD dogs, which were repeatedly transplanted with human adipose-derived mesenchymal stromal cells (hASC), derived from different donors. Xenogeneic cell transplantation, which was done without immunosuppression, was well tolerated in all animals with no apparent long-term adverse effect. In the present study, we show that repeated heterologous stem-cell injection is a safe procedure, which is fundamental before starting human clinical trials.

PMID: 27193781

Filed Under: Uncategorized Tagged With: adipose, dmd, duchenne muscular dystrophy, fat, mesenchymal stem cells, stem cells

Neil Riordan, PhD

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Recent Posts

  • Amnion-derived cells for regenerative medicine January 27, 2020
  • Vitrification preserves Wharton’s Jelly up to a year January 21, 2020
  • Adipose MSC for Spinal Cord Injury: ASIA Scores Improvement December 4, 2019
  • UCMSC secretions (exosomes) for Perinatal Brain Injury October 3, 2019
  • Immunomodulation of UCMSC in Rheumatoid Arthritis September 26, 2019

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