TreeFrog Therapeutics, a leading biotech company specialising in regenerative medicine, has made significant strides in developing a scalable cell therapy for Parkinson’s disease, according to a study published in Neurotherapeutics. The study highlights the successful bioproduction of the therapy, which resulted in full behavioural recovery in a pre-clinical model, 16 weeks after transplantation.
Utilising its proprietary C-Stem™ technology platform, TreeFrog Therapeutics achieved this milestone by producing a cryopreserved 3D microtissue format in a 10-litre bioreactor. The therapy contains mature dopaminergic neurons, which are critical in Parkinson’s treatment, and the results mark a breakthrough in tackling the key challenges facing cell therapy development for the disease.
The potential of cell therapy for Parkinson’s has been widely recognised since the 1980s, with early research focusing on fetal cell transplants. Over time, the development of induced pluripotent stem cells (iPSCs) solved the problem of cell sourcing, but challenges in bioproduction and industrialisation have hindered progress. To date, no cell therapy for Parkinson’s has advanced beyond the clinical stage.
Commenting on the publication, Jens Schroeder, Chief Medical Officer at TreeFrog Therapeutics, expressed optimism about the future: “This paper showcases how we’ve addressed some of the most complex challenges in Parkinson’s treatment. Using our C-Stem™ platform, we’ve created a therapy that contains mature dopaminergic neurons in a 3D format, ensuring cell survival post-graft with proven efficacy in pre-clinical trials. I am proud of Nicolas Prudon, the lead author, and the entire team for their outstanding work.”
One of the key obstacles for existing investigational cell therapies is the format of the product. Many therapies use single-cell suspensions, which carry a high risk of cell death due to anoikis, compromising both the survival and potency of the treatment after transplantation. TreeFrog’s 3D microtissue format, however, protects the delicate, mature neurons, increasing their survival rate and, potentially, the overall efficacy of the therapy.
Another challenge has been the bioproduction process. Most current strategies rely on manual, labour-intensive scale-out methods, which introduce variability. In contrast, TreeFrog’s C-Stem™ platform utilises a scale-up method with a stirred-tank bioreactor, enhancing productivity while reducing costs. The 10-litre bioreactor is designed for commercial-scale production, capable of generating doses for hundreds of patients at a time, providing a significant advantage for future treatments.
Cryopreservation has also posed a major hurdle in cell therapy development, as fresh cell products typically show better performance, while cryopreserved products often lead to a delayed therapeutic effect. However, the study revealed that TreeFrog’s cryopreserved product demonstrated the same efficacy as fresh cells, without any delays in time-to-effect, addressing a key limitation in the field.
The full paper, which details the bioproduction process, product characterisation, and pre-clinical efficacy results, is now available in Neurotherapeutics. These findings could pave the way for future advancements in cell therapy, bringing new hope for the treatment of Parkinson’s disease.
