Since the discovery of induced pluripotent stem cells (iPSCs) in 2006, a large and thriving research products market has emerged, largely because the cells are non-controversial and can be generated directly from adult cells. It is clear that iPSCs represent a lucrative market segment, because methods for commercializing this cell type are expanding every year and clinical studies investigating iPSCs are swelling in number.
Therapeutic applications of iPSCs are also emerging. In 2013, RIKEN launched the world’s first study of an iPSC-derived cell therapy product, treating the first patient in 2014 with iPS cell-derived retinal sheets. By 2016, Cynata Therapeutics launched the world’s first formal trial of an allogeneic iPSC-derived cell product (CYP-001) for the treatment of GvHD. Cynata is now testing these cells in the treatment of GvHD, critical limb ischemia (CLI) and osteoarthritis (OA).
Within United States, the NIH is undertaking the first U.S. clinical trial of an iPSC-derived therapeutic. This Phase I/IIa clinical trial will involve 12 patients with advanced-stage geographic atrophy of the eye. Riding the momentum within the immunotherapy field, Fate Therapeutics is developing iPSC-derived NK and CAR-T cell product candidates.
Numerous studies are also underway in Japan, with iPSC-derived cell therapeutics being used for the treatment of Parkinson’s disease, heart disease, spinal cord injury, and platelet production.
iPSC Cell Applications in 2021
While the therapeutic progress is exciting, other methods of commercializing iPS cells have also expanded exponentially.
Today, there are at least eight distinct iPS cell applications that include:
- Research Products: Dozens of market competitors provide iPSC specific tools to scientists worldwide, including human iPSC lines and differentiated cells types, as well as optimized reagents, protocols, differentiation kits and more.
- Drug Development & Discovery: iPSCs have the potential to transform drug discovery by providing physiologically relevant cells for compound identification, target validation, compound screening, and tool discovery.
- Cellular Therapy: iPSCs are being explored in a diverse range of cell therapy applications for the purpose of reversing injury or disease.
- Toxicology Screening: iPSCs can be used for toxicology screening, which is the use of stem cells or their derivatives (tissue-specific cells) to assess the safety of compounds or drugs within living cells.
- Personalized Medicine: The use of techniques such as CRISPR enable precise, directed creation of knock-outs and knock-ins (including single base changes) in many cell types. Pairing iPSCs with genome editing technologies has added a new dimension to personalized medicine.
- Disease Modelling: By generating iPSCs from patients with disorders of interest and differentiating them into disease-specific cells, iPSCs can effectively create disease models “in a dish.”
- Stem Cell Banking: iPSC repositories provide researchers with the opportunity to investigate a diverse range of conditions using iPSC-derived cell types produced from both healthy and diseased donors.
- Emerging Applications: Other applications for iPSCs include areas like tissue engineering, 3D bioprinting, clean meat production, wildlife conservation (preserving endangered species), and more.
Since the discovery of iPSC technology 15 years ago, exponential progress has been made in stem cell biology and regenerative medicine.
New pathological mechanisms have been identified and explained, new drugs identified by iPSC screens are in the pipeline, and the first clinical trials employing human iPSC-derived cell types have been initiated.
What do you think the next 15 years will hold? Let us know in the comments below.