Authored by Katrina Adlerz, Ph.D., Scientist, Analytics, Product & Process Development
& Josephine Lembong, Ph.D., Scientist, Analytics, Product & Process Development
The 2019 International Society for Cell and Gene Therapy (ISCT) Annual Meeting brought clinicians, regulators, and industry to Melbourne, Australia to collaborate and share progress in the rapidly developing field of Cell & Gene Therapy. Mesenchymal Stem/Stromal Cells (MSCs) were a major focus of the conference with an entire preconference workshop devoted to the Global Clinical Trial Landscape of MSCs as well as multiple sessions and keynotes throughout the conference. RoosterBio was active in the technical sessions and presented new technology on the industrial scale up of both MSCs and MSC-derived extracellular vesicles, as well as announcing a partnership with Tissue Regeneration Therapeutics to make umbilical cord -derived MSCs broadly available. Other key themes at this year’s meeting were: addressing global regulatory compliance, scalability, reducing cost of goods, exciting developments in exosomes/extracellular vesicles and recent advances in many different cell and gene therapy strategies.
MSC Progress in Clinical Trials, Manufacturing, and Comparability
Progress in clinical trials across the globe was the focus of a full day pre-conference workshop. Dr. Robert Mays of Athersys provided an update on their stromal cell product Multistem as they move into Phase III clinical trials for ischemic stroke patients and continue to investigate the treatment’s mechanism of action with immune regulation possibly being one key piece. Dr. Yufang Shi echoed this, highlighting work to pre-condition MSCs to bolster immune responses. Dr. Eleuterio Lombardo of Takeda discussed the development of Alofisel, an allogeneic MSC product that has been approved in Europe for perianal fistulas in Crohn’s disease. Some challenges in clinical trials were highlight
ed, such as inconsistent responses between pre-clinical and clinical studies. Dr. Lombardo pointed out an often-stated public opinion that successes in animal studies are often attributed to the use of “fresher” cells, and that these results may not be repeatable in human clinical trials because clinical trials often use cells that have been expanded and cryopreserved. Dr. Lombardo presented his review of the literature which did not support this opinion. Instead, he found that many studies did not state whether MSCs were cryopreserved or fresh, and surprisingly, it was also very rare that studies reported the Population Doubling Level (PDL). Furthermore, in his recent study where fresh and cryopreserved cells were
|Photo courtesy http://www.isct2019.com/photo-gallery/|