#cell therapy

1.2 billion allogeneic MAPC (MultiStem) IV 24–48 h after stroke. MASTERS (double-blind, 137 randomised, 33 UK/US sites) was safe, but the day-90 primary endpoint failed in the 126 analysed (OR 1.08, p=0.83). Plus the 9 years since.

At eight UK sites, 20 million allogeneic neural stem cells (CTX0E03) were injected into the putamen of 23 stroke patients by stereotactic neurosurgery. The primary endpoint was not met, the randomized trial meant to confirm the findings stopped at 15 patients, and the developer went through administration and delisted. Here is what a single-arm study can and cannot say, read straight from the numbers.

A phase 3 trial in ALS of autologous mesenchymal stem cells induced to secrete high levels of neurotrophic factors (NurOwn). The primary endpoint was not met (33% vs 28%, OR=1.33, P=.45). The signal in the less-impaired subgroup was not statistically significant and is only hypothesis-generating, yet the CSF biomarkers showed a clear biological effect. A piece for reading expectation and reality apart.