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(Cellular Immunology, 2009) Mesenchymal Stem Cells as Anti-inflammatories: Implications for Treatment of Duchenne Muscular Dystrophy

1Thomas E. Ichim, 2Doru T. Alexandrescu, 3Fabio Solano, 3Fabian Lara, 4Rosalia De Necochea Campion, 5Eugenia Paris, 6Erik J Woods, 7Michael P Murphy, 8Constantin A Dasanu, 9Amit N Patel, 10Annette M Marleau, 1*Neil H. Riordan

1Medistem Inc, San Diego, USA; 2Georgetown Dermatology, Washington DC, USA;3Institute for Cellular Medicine, Panama City, Panama; 4University of California, San Diego, California; 5Hospital CIMA, San Jose, Costa Rica; 6General Biotechnology, Indianapolis, Indiana; 7Division of Medicine, Indiana University School of Medicine, Indiana, USA; 8Department of Hematology and Medical Oncology, St Francis Hospital and Medical Center, Hartford, CT; 9Dept of Cardiothoracic Surgery, University of Utah, Salt Lake City, Utah, USA; 10Department of Surgery, University of Nebraska Medical Centre, Omaha Nebraska

Duchenne muscular dystrophy (DMD) is a lethal X-linked musculodegenerative condition consisting of an underlying genetic defect whose manifestation is augmented by inflammatory mechanisms. Previous treatment approaches using gene replacement, exon-skipping or allogeneic cell therapy have been relatively unsuccessful. The only intervention to mediate improvement in survival, albeit minor, is glucocorticoid treatment. Given this modality appears to function via suppression of underlying inflammation; we focus this review on the inflammatory response as a target for mesenchymal stem cell (MSC) therapy. In contrast to other cell based therapies attempted in DMD, MSC have the advantages of: a) ability to fuse with and genetically complement dystrophic muscle; b) possess anti-inflammatory activities; and c) produce trophic factors that may augment activity of endogenous repair cells. We conclude by describing one practical scenario of stem cell therapy for DMD.

...."Case Report of Stem Cell Therapy for DMD

We report a case study of a 23 year old male diagnosed with DMD at age 3, who manifested a progressive decrease in muscular strength and became wheelchair bound at age of 12. Supportive treatment with intermittent courses of prednisone, pain management, along with physical therapy was provided. Frequent respiratory infections secondary to a poor respiratory effort with decrease clearance of the secretions were managed with standard antibiotic therapy. On August 5-14th, 2008, the patient was treated with a combination of ERC and CD34 umbilical cord blood, mixed lymphocyte reaction-matched positive cells,subsequently on November 25-28, the patient received another course of therapy including placental matrix derived mesenchymal stem cells (Table 1). Cells were prepared and administered as previously described by us [148, 149]. No adverse events were associated with the stem cell infusion. A significant increase in muscle strength occurred in all the muscle groups, and was accompanied by an increase in the functional capacity of the patient. Thus, a pre-transplantation strength of 2-2.5/5 in the neck, shoulder, upper, and lower extremities began to improve after each of the two stem cell administrations, and reached a final 4/5 level 1 month after second transplantation treatment. The increments in muscle strength after the two stem cell administration appeared to be additive, with most benefit recorded after the second. Upper extremity improvement in strength evolved from the incapacity to lift against
gravity before the transplantation towards the ability to lift 2 lbs weights after the procedure. Trunk balance and strength were also markedly improved. The patient gained 20 lbs, along with an increased general activity level. The frequency of respiratory infections decreased from 3-4/year before stem cell therapy to none. The inspiratory effort improved from -32 to -40 cm H20. A muscle biopsy taken in January 2009 demonstrated normal (>50%, normal = 50-100% expression of normal-molecular size) levels of muscular dystrophin. The improvement in muscular strength, clinical respiratory function, and general level of activity are maintained to date.
To our knowledge this is the first report of profound dystrophin expression occurring in a non-ambulatory DMD patient after ERC treatment .......

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Seems like stem cels are the answer with all the studies I've read. So how come it's not more commonly researched and how come we are still in beginning phases of any treatments or cures. I fear that these stages of progress (because of FDA mainly), will not be available for my Nicolas who is 7. It becomes very frustrating, sitting and waiting.
Hard to believe. I looked at the whole paper and there is not real data in it. Only claims, but no evidence offered to support them. I have never seen a journal publish a research paper without primary data! Without data it is impossible for anyone to critically evaluate the claims, including the journal editors. Sounds incredibly fishy

I don't know the protocol for a "case study", but it seems that something like this couldn't possibly be approved for study in a human when exon skipping and utrophin upregulation was required to be studied in mice, dogs, llamas, humpback whales and little green martians before it was approved to be studied in humans.
Mesenchymal stem cell for DMD are being reserached in India too and some days back improvements were reported in the media. The resercher (Dr Gururaj Rao) explained that the improvements were seen after the first treatment in the upper arms and there are 4 more treatments to happen for 2 brothers.

On the whole Mesencymal stem cells are being exlpored and are reporting sysmilar findings. This one is startling in claims. 20lbs gain in a 23rd year old, 50% dystrophin.
These kind of claims come up on a regular basis.

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