Manufactured stem cells to advance clinical research

Clinical-grade cell line will enable development of new therapies and accelerate early-stage clinical research.

These clinical-grade stem cells are different from the more common laboratory-grade cells — those used in most scientific publications — because unlike laboratory-grade stem cells, clinical-grade stem cells can be used for clinical applications in humans. The distinctive feature of this cell line is that it was developed under cGMP, a set of stringent regulations enforced by the FDA, which ensures each batch of cells produced will meet quality and safety standards required for potential clinical use. The NIH Common Fund’s Regenerative Medicine program supported the manufacturing of this cell line.

“The Common Fund aims to accelerate research progress by developing new tools and resources for the biomedical research community through strategic investments in high-impact research,” said James M. Anderson, director of the NIH Division of Program Coordination, Planning, and Strategic Initiatives, which houses the Common Fund. “Since meeting cGMP guidelines is very time-intensive and costly, providing access to clinical-grade stem cells removes a significant barrier in the development of cell-based therapies.”

Significant progress with stem cell therapy in mice is already underway. Researchers have reversed diabetic conditions in mice using iPSC-generated insulin-producing cells and have partially restored limb function in mice with spinal cord injuries. Translating these studies into humans is the next challenge, and by making clinical-grade stem cells available, NIH hopes to speed up the development of new stem cell therapies for patients.

The clinical-grade stem cells, as well as research-grade cells cultured from the same cell line, are available for order and will be stored and distributed by the National Institute of Neurological Disorders and Stroke (NINDS) Human Cell and Data Repository (NHCDR), which is supported through a NINDS grant to RUCDR Infinite Biologics at Rutgers University, Piscataway, New Jersey. RUCDR also distributes laboratory-grade cell lines made by the NIH Regenerative Medicine Program. Laboratory-grade cells can be used for research that lays the foundation for eventual use of clinical-grade cells, such as determining the conditions necessary to guide the iPSCs to become specific cell types like neurons, insulin-producing beta-cells, or heart cells.

“As part of our longstanding commitment to providing critical biospecimens of the highest quality to investigators around the world, we share the excitement of being able to provide access to this resource,” said Dr Michael Sheldon, director of the Stem Cell Center at RUCDR. “The Regenerative Medicine Program’s laboratory-grade stem cells are frequently requested by researchers. Given the therapeutic potential of the cGMP clinical-grade stem cells we anticipate a strong demand from both the academic and corporate sectors.”