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Mitigating the risks of stem cell therapy: Scientists call for action

Mitigating the risks of stem cell therapy: Scientists call for action
Credit: University of Sheffield

An international workshop of scientists, regulators, and industry experts led by Prof Ivana Barbaric convened to address the risks of genetic mutations in stem cell therapy.

The group has produced a highlighting the need for definitive guidelines in hPSC-based therapy, which has been in Cell Stem Cell.

Human pluripotent stem cell (hPSC) based therapy holds great potential for treating a range of life-changing diseases including Parkinson's, deafness and blindness. Clinical trials of hPSC therapy have shown promising results in treatments, but the emergence of mutations in the stem cells have raised a real concern over how these would affect a patient—potentially transforming healthy cells into malignant ones. The challenge lies in accurately identifying which variants are dangerous and predicting their long-term effects—weighing the risks over benefits for patients.

The white paper includes proposed solutions:

  • Creating centralized, open-access databases to share data on genetic variants.
  • Developing advanced to assess the tumor-causing potential of these variants.
  • Utilizing machine learning to analyze the mass of data generated.
  • Increased funding and interdisciplinary collaboration to accelerate research.

By implementing these measures, researchers aim to establish a framework for ensuring the safety and efficacy of hPSC-based therapies.

The is essential as hPSC therapy has the key to transform lives across the globe—being prepared and fully understanding the risks of the therapy will ultimately lead to its success.

"Understanding the impact of genetic variants in human pluripotent stem cells is paramount for the future of cell therapy. This white paper represents a call for immediate and collaborative action, proposing focused strategies towards establishing definitive guidelines in hPSC-based therapy. Fulfilling this action will have a transformative impact by overcoming a critical challenge to the broad application of hPSC-based therapy," said Prof. Barbaric.

More information: Nissim Benvenisty et al, A call to action for deciphering genetic variants in human pluripotent stem cells for cell therapy, Cell Stem Cell (2025).

Journal information: Cell Stem Cell

Citation: Mitigating the risks of stem cell therapy: Scientists call for action (2025, September 30) retrieved 4 October 2025 from /news/2025-09-mitigating-stem-cell-therapy-scientists.html
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