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Is myoblast transplantation effective?

Nature Medicine volume 4, pages 1208–1209 (1998)Cite this article

To the editor— Nature Medicine has emphasized the controversial program of myoblast transplantation into Duchenne muscular dystrophy (DMD) patients undertaken by the Cell Therapy Research Foundation (CTRF) in Memphis1,2. We have previously suggested3 that the confrontational aspects of this work are unnecessary and easily resolved by simple investigation. In particular, we have argued that it can be misleading to use immunostaining with a single polyclonal antibody to demonstrate donor dystrophin4,5. This is because many biopsies of DMD muscle contain variable numbers of so called 'revertant' fibers that contain immunochemically demonstrable dystrophin6. The mechanism underlying this expression of 'revertant' dystrophin is unknown, but, where the patient has an identified genomic deletion from his dystrophin gene, it is possible to show that the 'revertant' dystrophin lacks exons encoded by the deleted region7,8. We now report findings on biopsy samples volunteered by the family of one of the subjects of the CTRF program.

The subject, a boy less than 6 years of age, diagnosed with DMD, was grafted at the CTRF with 12.5 × 1012 myoblasts (obtained from an unrelated donor), via approximately 200 injections into muscles of the upper body and limbs. According to the protocol, he was immunosuppressed with cyclosporin. One biceps brachialis muscle was 'sham-injected' with the carrier medium in accordance with the randomized double-blind trial procedure being done at that time.

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Acknowledgements

The authors acknowledge the support of the Muscular Dystrophy Group of Great Britain, The Leopold Muller Foundation, The Medical Research Council and the Parent Project for Duchenne Muscular Dystrophy Research.

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Authors and Affiliations

  1. Muscle Cell Biology Group MRC Clinical Sciences Centre Imperial College School of Medicine, London, UK

    Terry Partridge & Qi Long Lu

  2. North East Wales Institute MRIC Biochemistry Group , Wrexham, UK

    Glenn Morris

  3. Department of Molecular Genetics and Biochemistry University of Pittsburgh School of Medicine, Pittsburgh, PA, USA

    Eric Hoffman

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  1. Terry Partridge
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  2. Qi Long Lu
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  4. Eric Hoffman
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Partridge, T., Lu, Q., Morris, G. et al. Is myoblast transplantation effective?. Nat Med 4, 1208–1209 (1998). https://doi.org/10.1038/3167

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