The International Society for Stem Cell Research (ISSCR) has submitted a formal response to a request for information (RFI) issued by the National Institutes of Health (NIH) regarding the suspension of new submissions to the NIH Human Embryonic Stem Cell (hESC) Registry and considerations for reducing reliance on hESCs in federally supported research.
ISSCR, representing approximately 5,000 scientists, clinicians, ethicists, and industry leaders around the world, emphasizes that continued investment in hESC research is essential to sustain scientific progress and advance life-saving treatments.
hESCs: the basis for scientific discovery and clinical progress
Human embryonic stem cells remain the cornerstone of modern biomedical research due to their status as the “gold standard” for human pluripotency, the ability to self-replicate indefinitely and differentiate into all cell types in the human body. This multipotency has enabled innovative advances in understanding human development, modeling disease, and developing regenerative medicine approaches.
Decades of NIH investments have built a robust research ecosystem around hESCs, supporting widely shared cell line repositories, standardized protocols, and expertise. These resources have laid the foundation for a growing pipeline of clinical applications, including Phase III clinical trials targeting diseases such as Parkinson’s disease, type 1 diabetes, and drug-resistant epilepsy.
As of late 2024, nearly half of the world’s clinical trials using hESC-derived products will be conducted in the United States, highlighting both scientific momentum and the importance of continued support from the federal government.
Irreplaceable scientific resources
Although new technologies such as induced pluripotent stem cells (iPSCs) and computational models are expanding the research toolkit, ISSCR emphasizes that these systems are not a replacement for hESCs. Instead, we rely on hESC as a key benchmark for validation and comparison. Other cell sources, such as adult stem cells, have fundamentally different properties, including more limited differentiation potential and challenges in scaling in culture.
Furthermore, hESCs play a key role in the development of human-relevant drug discovery systems and new approach methodologies (NAMs) aimed at reducing reliance on animal experiments while improving predictive accuracy in preclinical research. hESCs serve as a stable and well-characterized reference material that allows direct comparison and benchmarking in the development of rigorous and reproducible NAM platforms.
Policy stability is important at turning points
ISSCR warns that restricting access to hESCs or disrupting the NIH registry could have far-reaching implications. After years of basic research, the field is now at a critical juncture where continued stability in policy will determine whether scientific discoveries lead to approved treatments for patients.
Restricting access not only risks derailing ongoing clinical programs, it could undermine billions of dollars of prior federal investment and slow progress across multiple areas of biomedical research.
Strong ethical oversight is already in place
ISSCR also emphasizes that hESC research is conducted under a strict ethical and legal framework. NIH guidelines, along with complementary oversight from major scientific institutions, ensure that federal funds are not used for research that involves the derivation of new hESC lines or the destruction of human embryos.
ISSCR calls for evidence-based policy
ISSCR concludes that, based on scientific and translational evidence, reducing NIH support for hESC research or suspending new submissions to the NIH Registry is not justified. The Society urges the NIH to lift the current moratorium on registry submissions and reaffirm its longstanding commitment to funding scientifically valuable research in this area.
Human embryonic stem cells are an essential and irreplaceable component of the biomedical research ecosystem. Maintaining access to these critical resources is essential to advancing discovery, supporting innovation, and ultimately delivering new treatments to patients.
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International Stem Cell Research Association
