In the last decade research funding and interest in stem cell research has moved its focus to tackle hurdles that limit clinical translation to humans, such as reliability and safety of cell sources, and their scaled-up manufacturing. A challenge remains however in the scope of basic science, to understand mechanisms of stem cell biology that have proved to be definitely more complex than maybe anticipated. This would allow better informed human trials. Several thousands of clinical trials of cell therapies have been run and only a few tens of products are currently commercialised. Basic research labs can lead this progress with more flexibility and expertise.
It is already a feature of the stem cell research field how difficult is to translate lab results into the clinic. The FDA has currently (September 2022) approved only 23 products , 19 the EMA , and this after decades of huge public and private funding. It is telling that this is over 15 years since the discovery of induced pluripotent stem cells (iPSCs) , that at first seemed to have a much better chance of being transformed into therapies, as they avoid ethical and sourcing problems of other stem cells types.
Big research structures such as the California Institute for Regenerative Medicine (CIRM), the UK Cell and Gene Therapy Catapult (CGTC), and others around the world were created to support the translation process and to gather relevant insights on how to improve its success. As a result, more R&D funding is now directed to regulatory assessment, preclinical models, and manufacturing, i.e., how reliable and scalable cell sources are. In terms of academic research this usually means bigger projects, access to better facilities and more resources, and partnering with industry and other stakeholders at early stages of research. The CIRM was created in 2004 to accelerate therapy development and was restructured in 2015 to align with key regulatory and product development requirements, a strategy that seems to have work for CIRM  to improve moves to clinical stages of research. CIRM-funded projects have reached phase III clinical trials with overall great returns to the Californian economy . However, it is significant that no therapy developed with CIRM funding has come yet to market. The CGTC has also recently proposed to target manufacturing supply chains and embedding collaborations for the provision of cell and gene therapies in the UK .
These barriers to translation are better defined now and we could finally see many more effective therapies commercialised soon. Still, these barriers related to development, safety, scale-up stages, are not the only barriers faced by stem cell research: huge progress is to be made by understanding why so many of these cellular products fail . Smaller labs such as ours with researchers specialised on regenerative medicine and basic research don’t need the resources required to work towards the preclinical and regulatory challenges noted above. They can focus on elucidating molecular biology mechanisms, for instance those that would explain why animals and cell-culture systems do not recapitulate the relevant human conditions sufficiently well, or how the safety of iPSCs (risk of oncogenic transformation) can be better measured and ensured.
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Current landscape of clinical development and approval of advanced therapies. Iglesias-Lopez, Carolina et al. Molecular Therapy – Methods & Clinical Development, Volume 23, 606 – 618.
Wojciech Zakrzewski, Maciej Dobrzyński, Maria Szymonowicz & Zbigniew Rybak. Stem cells: past, present, and future. Stem Cell Research & Therapy volume 10, Article number: 68 (2019)
Gilberto R. Sambrano, Maria T. Millan. Translating Science into the Clinic: The Role of Funding Agencies. Cell Stem Cell, Volume 26, Issue 4, 2020, Pages 479-481.
The Economic Case for Public Investment in Stem Cell Research. USC Schaffer White Paper by Dana Goldman, PhD, Martha Ryan, Bryan Tysinger, PhD, Adam Rose, Dan Wei and Mark S. Humayun. Published online June 24, 2020.
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