TreeFrog Therapeutics, a regenerative medicine biotech focused on using its proprietary GMP compliant technology platform, C-Stem, to develop cell therapies, published an article in Neurotherapeutics, demonstrating the first successful bioproduction of a Parkinson’s Disease cell therapy in a scalable bioreactor, leading to full behavioral recovery 16 weeks after transplantation using a cryopreserved 3D microtissue format.
The potential of cell therapy for Parkinson’s Disease is well documented – dating back to pioneering studies in the 1980s using fetal cell transplants – and scientific progress over the last decades provides great hope for the future. The emergence of induced pluripotent cells (iPSCs) opened new pathways and solved one of the first major challenges, cell sourcing. However, multiple hurdles remain, particularly around industrialization and no cell therapy for Parkinson’s Disease has advanced beyond clinical stage to date.
“Today’s publication demonstrates how TreeFrog Therapeutics has overcome the most complex challenges of developing a successful treatment for Parkinson’s disease using our C-Stem platform technology and producing a therapy containing mature dopaminergic neurons with a unique 3D format that promotes cell survival post-graft with proven pre-clinical results. We are excited to present the paper to the scientific community and look forward to many discussions on our scientific and bioproduction progress. I am proud of all the work done by Nicolas Prudon, lead author, alongside colleagues at TreeFrog”. Jens Schroeder, Chief Medical Officer, TreeFrog Therapeutics.
The paper published online today reinforces the potential of TreeFrog’s cell therapy to overcome several complex challenges, one of which being the limitation of the format. Most other existing investigational cell therapies use single-cell suspensions which have an elevated risk of inducing cell death through anoikis affecting survival and/or potency of the product post-transplantation. The results achieved with the unique 3D format of TreeFrog’s cell therapy product demonstrate the potential to circumvent this limitation, providing protection to the more mature, sensitive cells that are neurons.
Another challenge facing companies is the bioproduction strategy. While currently only scale-out methods have been employed, which increase the potential of variability due to the manually intensive methods required, C-Stem production use a scale-up strategy with a stirred-tank bioreactor enhancing productivity and reducing costs. The system is commercial scale ready for the Parkinson’s disease cell therapy, with doses for hundreds of patients in one 10L bioreactor.
Finally, as in downstream processing, cryopreservation has proven to be a major challenge in the cell therapy space with fresh product performing better, while cryopreserved product resulted in a delayed time-to-effect.
