Myoblast implantation in Duchenne muscular dystrophy: the San Francisco study.
We evaluated myoblast implantation in 10 boys with Duchenne muscular dystrophy (DMD) and absent dystrophin (age 5-10 years) who were implanted with 100 million myoblasts in the anterior tibial muscle of one leg and placebo in the other. Cyclosporine (5 mg/kg/day) was administered for 7 months. Pre- and postimplantation (after 1 and 6 months) muscle biopsies were analyzed. Force generation (tetanic tension and maximum voluntary contraction) was measured monthly in a double-blind design. There was increased force generation in both legs of all boys, probably due to cyclosporine. Using the polymerase chain reaction, evidence of myoblast survival and dystrophin mRNA expression was obtained in 3 patients after 1 month and in 1 patient after 6 months. These studies suggest a salutary effect of cyclosporine upon muscular force generation in Duchenne muscular dystrophy; however, myoblast implantation was not effective in replacing clinically significant amounts of dystrophin in DMD muscle.
Miller
R G
RG
Department of Neurology, California Pacific Medical Center, San Francisco, USA.
Sharma
K R
KR
Pavlath
G K
GK
Gussoni
E
E
Mynhier
M
M
Lanctot
A M
AM
Greco
C M
CM
Steinman
L
L
Blau
H M
HM
D000328:Adult; D001706:Biopsy; D017690:Cell Transplantation / Q000379:methods; D002478:Cells, Cultured; D002648:Child; D002675:Child, Preschool; D016572:Cyclosporine / Q000627:therapeutic use; D016189:Dystrophin / Q000096:biosynthesis / Q000172:deficiency*; D006650:Histocompatibility Testing; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D008279:Magnetic Resonance Imaging; D008297:Male; D009119:Muscle Contraction; D018482:Muscle, Skeletal / Q000473:pathology / Q000503:physiopathology / Q000637:transplantation*; D009136:Muscular Dystrophies / Q000473:pathology / Q000503:physiopathology* / Q000628:therapy*; D010808:Physical Examination; D014176:Protein Biosynthesis; D014019:Tissue Donors; D014158:Transcription, Genetic
D016189:Dystrophin; D007166:Immunosuppressive Agents; D016572:Cyclosporine