Table 1.
Clinical trials using myogenic progenitors for the treatment of Duchenne’s muscular dystrophy.
| Year | N | Donor Cells | Injection | Immuno-Suppression | Results | Conclusions | Reference |
|---|---|---|---|---|---|---|---|
| 1992 | 4 | Allogeneic immunocompatible myoblasts | Intramuscular: tibialis anterior, biceps brachii, and/or extensor carpi radialis longus | No | Variable response. Hybrid myofibers and modest strength increase in 3 of the 4 patient. Slow decay over time. | No signs of immune rejection | [19] |
| 1992 | 8 | Allogeneic immunocompatible myoblasts | Intramuscular: tibialis anterior | Cyclosporin | PCR evidence of hybrid fibers after 1 moth for 3 patients (1 patient tested still positive after 6 months). | Younger patients with less fibrosis presented best outcomes | [20] |
| 1993 | 5 | Allogenic myoblasts | Intramuscular: biceps brachii, left tibialis anterior | No | 0%–36% hybrid fibers after 1 month. Low dystrophin expression. Strong decrease in hybrid fibers at 6 months. No functional recovery. | Transplantation cannot be done without immuno-suppression | [21] |
| 1993 | 8 | Allogeneic myoblasts | Intramuscular: biceps brachii | Cyclosporin | Poor functional recovery and lack of donor-derived dystrophin. | Younger donor cells, regeneration induction and basal laminal fenestration could improve results | [22] |
| 1993 | 1 | Asymptomatic twin sibling myoblasts | Intramuscular: extensor carpi radialis, biceps | No | After 1 year, significant force gain (12%–31%) in wrist extension but not for elbow flexion. Small increase in dystrophin positive and type II fibers. | Small benefit may be due to a low level of spontaneous muscle regeneration | [23] |
| 1995 | 12 | Allogeneic myoblasts | Intramuscular: biceps brachii Injection repeated monthly over 6 months | With and without Cyclosporin | There was no significant change in muscle strength. % of hybrid fiber varied between 10.3 (1 patient), 1 (3) and 0 (8). | Patient age did not correlate with outcome | [11] |
| 1997 | 10 | Allogeneic immune-compatible myoblasts | Intramuscular: tibialis anterior | Cyclosporin | Myoblast survival after 1 month in 3 patients and after 6 month in 1 patient. No recovery symptoms or clinically significant dystrophin expression. | - | [24] |
| 2004 | 3 | Allogeneic myoblasts | Intramuscular: tibialis anterior | Tacrolimus | Hybrid fibers observed in all 3 patients (9%, 6%, 8% and 11%) | - | [25] |
| 2006 | 9 | Allogeneic immuno-compatible myoblasts | Intramuscular Tibalis anterior. High density injections | Tacrolimus | At 4 weeks, 3.5%–26% hybrid fibers | Dystrophin expression restricted to injection site and mostly in short inter-injection distances | [26] |
| 2007 | 1 | Allogeneic myoblasts | Intramuscular Thenar eminence, biceps brachii and gastrocnemius High density injections | Tacrolimus | At 18 months, 34.5% hybrid myofibers in gastrocnemius but almost 0% in biceps brachii. Increased strength only observed in thumb. | - | [27] |
| On-going | - | Mesoan-gioblasts | Intra-arterial | Tacrolimus | Not yet | - | * |
* EudraCT Number: 2011-000176-33; Sponsor Protocol Number: DMD03; Start Date *: 14 February 2011; Sponsor Name: FONDAZIONE CENTRO S; RAFFAELE DEL MONTE TABOR; Full Title: Cell Therapy of Duchenne Muscular Dystrophy by intra-arterial delivery of HLA-identical allogeneic mesoangioblasts.