MSB Trading 2021 - paradigm shift, page-3883

Dear Wilba,

following please find full-text link to Dr Joanne Kurzberg's 2020 review of the updated Expanded Access Protocol (Protocol 275) for remestemcel-l:

https://www.tctjournal.org/article/S1083-8791(20)30059-8/fulltext

The EAP is the manifestation of MSB's 'compassionate use' program, as I understand it. I'm not aware of any more recent published info on the Protocol. MSB has received no credit for this work from the FDA, as far as I'm aware.

A (hopefully) relevant extract:

" A total of 242 subjects at 50 sites in 8 countries (the United States, Canada, United Kingdom, Italy, Finland, Spain, New Zealand, and Australia) participated in this expanded access program (EAP). The study was approved by the Institutional Review Board or Ethics Committee of each participating institution. The study was conducted in accordance with the principles of the Declaration of Helsinki and the International Conference on Harmonization Good Clinical Practice Guidelines....The methodology of this study has been described in detail in the previous publication of the results in 75 subjects.

Eligible subjects were age 2 months to 17 years, inclusive, with aGVHD secondary to allo-HSCT or donor lymphocyte infusion who had failed to respond to systemic steroid therapy for grade B-D aGVHD using the Center for International Blood and Marrow Transplant Registry grading scheme."