CASE PRESENTATION: This study was approved by the Korea Food and Drug Administration through the Emergency Use
Investigational New Drug Application. A 71-year-old Asian man from South Korea with
progressive supranuclear palsy was treated with five
intravenous infusions (each time 2×108 cells) and four intrathecal infusions (each time 5×107 cells) with autologous adipose tissue-derived mesenchymal stem cells expanded under good manufacturing practice conditions. Clinical examinations were performed immediately before treatment and throughout the six months of follow-up. The tests included: 1)
Progressive Supranuclear Palsy Rating Scale; 2) Berg Balance Scale; 3) Korean Mini Mental State Examination; 4) Modified Barthel Index; 5) grip strength; 6) Box and Block Test; and 7) Nine-Hole Peg Test.The
Progressive Supranuclear Palsy Rating Scale results gradually decreased, and the clinical rating scale scores of the Berg Balance Scale, Korean Mini Mental State Examination, and Modified Barthel Index gradually increased. Grip strength was maintained. Performance in the Box and Block Test and Nine-Hole Peg Test improved after adipose tissue-derived mesenchymal stem cells treatment compared to baseline throughout the six months of follow-up. Except for the intermittent mild
fever and transient elevated blood pressure, the treatment of our patient with
progressive supranuclear palsy with autologous adipose tissue-derived mesenchymal stem cells showed no significant adverse events, and delayed the progression of neurological deficits by achieving functional improvement in the follow-up period.
CONCLUSIONS: These results are encouraging and hopeful for further studies in patients with
progressive supranuclear palsy using autologous adipose tissue-derived mesenchymal stem cells as a safe and effective
therapy. This case report is the first known study of adipose tissue-derived mesenchymal stem cells safely delaying the progression of
progressive supranuclear palsy with functional improvement during the follow-up period.