Induced Pluripotent Stem Cells Industry Methods Reduce Risks

The original technique for making iPS cells involved expressing genes via retroviruses, which introduced risks of mutations. Researchers have since developed non-integrating methods using viruses, mRNA, proteins or small molecules that work without permanently altering the genome. In 2020, scientists in Japan achieved the first human iPS cell derivation using only mRNA, avoiding all risks from gene insertion. Other teams improved efficiency by developing episomal vectors and minicircle DNA that enable reprogramming with fewer promoter copies. These safer techniques bring iPS cell therapies closer to clinical use.

Patient-Specific cell Models Advance Disease Understanding

By reprogramming cells from patients with various diseases, researchers can generate cell models to study disease mechanisms in a dish. In 2021, scientists in the UK created the first retinal organoids from Global Induced Pluripotent Stem Cells of patients with retinitis pigmentosa to better understand causes of vision loss. Chinese researchers also produced iPS cell-derived cardiomyocytes from genetic heart disease patients, revealing new insights into arrhythmias. Such models allow preclinical testing of new drugs without animal use or risky human trials. Many global teams are establishing biobanks of iPS cells to aid research on disorders like ALS, Parkinson’s and Alzheimer’s disease.

Cell Therapy Trials Demonstrate Real promise

With safety enhancements to reprogramming methods, initial iPS cell therapy trials are showing promise. In 2018, a team in Japan reported successful treatment of age-related macular degeneration using iPSC-derived retinal pigment epithelium cells, representing the first reported use of iPS cells to treat human disease. Other milestones include a 2021 trial in Germany using iPSC-cardiac patches to repair heart attack damage. Meanwhile, a Phase 1 trial in China is evaluating iPSC-neural progenitors for treating spinal cord injury. While longer-term data is still needed, such pioneering studies demonstrate iPS cells can restore lost function without rejection – a massive breakthrough for regenerative medicine.

Scaling Up Manufacturing for Widespread Application

For iPS cell therapies to benefit large patient numbers, methods are needed to produce clinical-grade cells at an industrial scale. Researchers worldwide are optimizing bioprocess techniques. In the US, a team developed a scalable stirred-tank production approach to generate billions of retinal pigment epithelium cells from a single iPSC line in a matter of weeks. Meanwhile, a partnership between Japanese and Canadian researchers resulted in a new defined xeno-free media for large-scale expansion of hepatocyte-like cells from iPSCs. Such process advances are crucial to fulfill the vast therapeutic potential of these versatile cells through widespread availability of regenerative medicines.

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