Isolation of DMD mesoangioblasts for the development of novel ATIMP

• Research type

  Research Study

• Full title

  Isolation of mesoangioblasts from patients affected by Duchenne Muscular Dystrophy with a skippable mutation of exon 51 for the development of a novel ATIMP (Advanced Therapeutic Investigational Medicinal Product): a non randomized, open label, phase I study.

• IRAS ID

  225682

• Contact name

  Giulio Cossu

• Contact email

  giulio.cossu@manchester.ac.uk

• Sponsor organisation

  The University of Manchester

• Duration of Study in the UK

  0 years, 6 months, 0 days

• Research summary

  The body needs a protein called dystrophin to make our muscles work and keep them strong. Dystrophin is not made in Duchenne Muscular Dystrophy (DMD) and patients lose the ability to use most of their muscles. Currently, there is no efficacious treatment for DMD.
  Our research aims at finding a way of taking specific cells from a muscle of a patient with DMD, then using a method we are currently developing in our laboratory, making changes to the cells in a special way so that they can begin to make the dystrophin protein when the cells are injected back in to the foot and hand muscles. We believe that the modified cells could potentially strengthen these muscles and allow the patient to use these muscles for simple tasks that they have been unable to perform e.g. the holding of a pen. For this preliminary study we plan to recruit one patient only.

  Before such a treatment can be trialled in DMD patients, we need to prove that the cells extracted from a foot muscle of a patient with DMD can be processed to potentially produce the dystrophin protein in a laboratory before we can use this method in patients.

  To do this we need to obtain muscle from one DMD patient. This muscle can only be obtained by a surgical procedure (biopsy). We are asking to use a specific foot muscle for two reasons: (1) the foot muscle is one that the patient does not use; (2) the foot muscle is one of the best preserved muscles in the human body despite the effect of the disease.

  One patient only will be recruited.

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  17/LO/0975

• Date of REC Opinion

  23 Aug 2017

• REC opinion

  Further Information Favourable Opinion