Mesoangioblast-based gene therapy for Duchenne Muscular Dystrophy
Research type
Research Study
Full title
Mesoangioblast-mediated exon 51 skipping for genetic correction of dystrophin, based upon a single injection in individual skeletal muscles of five non ambulant patients affected by Duchenne Muscular Dystrophy: a non randomized, open label, phase I/IIa study.
IRAS ID
262828
Contact name
Mary Imelda Hughes
Contact email
Sponsor organisation
The University of Manchester
Eudract number
2019-001825-28
Duration of Study in the UK
2 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 have developed in our laboratory, making changes to the cells in a special way so that they can begin to make the dystrophin protein when 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.
To do this we need to obtain one muscle from five DMD patients. 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. Cells will be isolated, genetically corrected in culture and, once controlled for being safe, injected in the contra-lateral foot muscle. If 10% or more of the level of dystrophin present in normal muscles will be detected, then other cells will be injected in the muscle of the thumb, whose force of contraction will be measured before, during and after the treatment. An increased or stabilised force of thumb contraction would impact on the quality of patient life.
A 9 year safety follow up is proposed as an observational study.REC name
North East - York Research Ethics Committee
REC reference
19/NE/0216
Date of REC Opinion
15 Aug 2019
REC opinion
Further Information Favourable Opinion