NIH Deputy Director: manufactured stem cells to advance clinical research

According to the researchers, the stem cells were developed by isolating human umbilical cord blood cells after birth and “coaxing them back into a pluripotent state​” – creating induced pluripotent stem cells (iPSCs).

These cells were then manufactured by Lonza, with support from NIH, who has already received inquiries from companies about obtaining the cGMP clinical-grade cells.

To learn more about the stem cell line and its potential to accelerate new medical applications, Outsourcing-Pharma.com talked with James M. Anderson, M.D., Ph.D., Deputy Director, NIH Division of Program Coordination, Planning, and Strategic Initiatives.

Melissa Fassbender, Outsourcing-Pharma.com: Specifically, what is likely to be the biggest impact of the stem cell line?​

James M. Anderson​: The goal of developing a clinical-grade stem cell line was to enable development of new therapies and accelerate early-stage clinical research. The nature of scientific research and discovery makes it difficult to predict when (or if) such therapies may be widely available, but these cells will enable many researchers and companies to develop therapies that they may not otherwise have been able to develop.

Fassbender: What makes the cell line unique and how does this help clinical researchers? ​

Anderson​: Researchers working to uncover new biological pathways, develop new paradigms, and discover new cures perform their experiments on laboratory-grade cells. Laboratory-grade simply means that there are no regulations or quality control measures to ensure that the cells are safe for human use, and this is acceptable because laboratory-grade cells serve as a model to test hypothesis and potential drugs before translating the finding to humans.

For cells to be used for clinical applications in humans (i.e. clinical-grade cells) they must be generated – from the very first step all the way through to the very last step – under 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.

These regulations are called current Good Manufacturing Practices (cGMP) and complying with these standard presents a significant barrier for many researchers; a significant time and financial investment in addition to special facilities and equipment is required to generate and maintain cGMP stem cells.

By providing cGMP stem cells, at a fraction of the costs to generate them, directly to the research community, NIH is removing those barriers and the hope is that it will accelerate the development of stem cell cures for candidate ailments such as Alzheimer’s disease, Parkinson’s disease, spinal cord injury, diabetes, and muscular dystrophy.

Fassbender: Who is able to order the stem cells? ​

Anderson​: Both non-profit investigators and for-profit companies can order the cGMP clinical-grade stem cells. However, because these cells must be maintained at all times under current good manufacturing practices (an FDA requirement for the cells to maintain their “cGMP” status) only those researchers with the proper facilities and equipment for maintaining cGMP clinical-grade cells should order them.

Researchers lacking the necessary facilities and equipment may still order cGMP clinical-grade stem cells if they collaborate with an entity who can properly maintain the cells under cGMP conditions.

The NIH Common Fund supported the development of the clinical-grade stem cells because removing the barriers described above to accelerate the translation of stem cell research into therapies is aligned with the Common Fund’s mission of funding catalytic, transformative, and unique research.

A clinical-grade stem cell line is a unique tool for the biomedical research community that we envision will catalyze the development of new therapies by removing a significant time-intensive and costly barrier in the development of clinical-grade stem cells.

Although NIH is excited to be able to provide cGMP clinical-grade stem cells to researchers to enable development of new therapies and accelerate early-stage clinical research, it is important to keep in mind that on the timeline towards treatments there are hidden uncertainties and unexpected obstacles; it is not unusual for pharmaceutical companies to spend 10-15 years bringing a new treatment to market.