Several therapeutic applications are now being studied in preclinical tests using induced pluripotent stem cell (iPSC) derived cells for their potential to produce functional cells capable of replacing damaged or dysfunctional tissues in patients. The first clinical trial using iPSCs started in 2008, and now in 2020, the number has risen to 53.
The early clinical trials did not involve the transplant of iPSCs into humans, but rather, the creation and evaluation of iPSC lines for clinical purposes. Within these trials, iPSC lines were created from specific patient populations to determine if these cell lines could be a good model for a disease of interest.
iPSC Clinical Trials Involving Cell Therapeutics
Today, there are now several companies progressing iPSC-derived cell therapeutics into clinical trials, including:
- Cynata Therapeutics – CYP-001, its iPSC-derived MSCs
- Fate Therapeutics – FT500, its iPSC-derived NK cell product
Within Japan, there are also several physician-led studies underway investigating the use of iPSC-derived cellular products within human patients. While some of these researchers are patiently waiting for the results of their clinical studies, others are pursuing new therapeutic strategies. For example, with CRISPR-Cas9 technologies, stem cell biologists are making attempts to repair mutated genes in human iPSCs before using iPSC-derived cells to treat patients in need.
There has also been tremendous interest among researchers in the application of iPSC technologies to human disease modeling, in particular, to the modeling of neurologic diseases using patient-specific iPSCs. In the future, Parkinson’s disease, Alzheimer’s disease, and spinal cord injuries may be treated with iPSC therapy or replacement tissues obtained from iPSCs.
As of today, iPSCs research has rapidly evolved to offer the possibility of replacing regenerated and non-regenerating tissues, such as the heart, pancreas, and brain, and other critical cell types.
Figure. Number of Clinical Trials Involving iPSCs by Year, 2006-2020
iPSC Progress in Japan
In a surgical procedure performed in October 2018, neurosurgeons from Kyoto University implanted 2.4 million cells into the brain of a patient with Parkinson’s disease. The iPSCs obtained from peripheral blood cells of a donor were reprogrammed into induced pluripotent stem cells (iPSCs) and then differentiated into dopaminergic precursor cells, which researchers hope will boost dopamine levels and improve the patient’s symptoms.
Japanese researchers have also launched several clinical trials to evaluate the efficacy of iPSCs in heart disease and macular degeneration of the eye. Scientists around the world are studying ways to turn iPSCs into treatments for everything from endometriosis to spinal cord injury. So far, only a small number of patients have receiving iPSC-based treatments within clinical trials.
In 2014, a Japanese woman who had macular degeneration of the eye received a transplant of iPSC-based retinal cells developed from her own cells. The woman did not show any apparent improvement in her vision, but the safety of the iPSC-derived cells was confirmed in the study. In 2017, five patients were treated with iPSC-derived retinal cells for macular degeneration and one patient developed an adverse reaction.
In 2019, three patients with heart disease received iPSC-derived cardiomyocytes. By 2022, six Parkinson’s disease patients are to receive iPSC-derived neurons.
As of March 2020, 53 clinical trials have been reported for iPSCs in the website of ClinicalTrials.gov.
To learn more about the market for iPS cells, view the “Global Induced Pluripotent Stem Cell (iPS Cell) Industry Report 2020.”
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