Manufactured Stem Cells to Advance Clinical Research

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

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Researchers supported by the National Institutes of Health developed a clinical-grade stem cell line, which has the potential to accelerate the advance of new medical applications and cell-based therapies for millions of people suffering from such ailments as Alzheimer’s disease, Parkinson’s disease, spinal cord injury, diabetes, and muscular dystrophy. The stem cells were developed by isolating human umbilical cord blood cells following a healthy birth, and coaxing them back into a pluripotent state, or one in which they have the potential to develop into any cell type in the body. Cells developed in this manner are called induced pluripotent stem cells (iPSCs). With NIH support, these cells were manufactured by Lonza, Walkersville, Maryland, and described in a publication by Behnam Baghbaderani, Ph.D., and colleagues in Stem Cell Reports.

“…providing access to clinical-grade stem cells removes a significant barrier in the development of cell-based therapies”

—James M. Anderson, M.D., Ph.D., Director, Division of Program Coordination, Planning, and Strategic Initiatives

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 current good manufacturing practices (cGMP), a set of stringent regulations enforced by the U.S. Food and Drug Administration 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, M.D., Ph.D., 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)(link is external)that 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.”

The Regenerative Medicine Program supported the manufacturing of the clinical-grade stem cell line as part of its mission to serve as a national resource for stem cell science to accelerate the development of new medical applications and cell-based therapies. Another avenue through which the Regenerative Medicine Program is fulfilling its mission is through the Stem Cell Translation Laboratory (SCTL) that is funded by the Common Fund and administered by the NIH’s National Center for Advancing Translational Sciences (NCATS). The aim of the SCTL is to remove barriers that currently impede the therapeutic application of iPSCs, which include the lack of highly reproducible and well-defined procedures required to generate, characterize and differentiate patient-specific iPSCs in a safe fashion for pre-clinical and clinical use. In parallel to developing an integrated stem cell research program within NCATS, the SCTL will soon be soliciting collaborations from the research community in order to address the most pressing impediments towards stem cell therapies.