The development of clinical-grade stem cell lines has been accomplished thanks to the efforts of National Institutes of Health (NIH), moving forward the advancement of medical applications and cell-based therapies to treat millions of people suffering from remotely incurable ailments such as Alzheimer’s disease, Parkinson’s disease, spinal cord injury, diabetes and muscular dystrophy. The clinical-grade stem cells have been manufactured by the Maryland branch of chemicals and biotechnology manufacturing company, Lonza, which is currently being supported by NIH.
According to James M Anderson, MD, PhD, Director, Division of Program Coordination, Planning, and Strategic Initiatives, “…providing access to clinical-grade stem cells remove a significant barrier in the development of cell-bases therapies.”
On March 9, 2009, President Obama issued Executive Order 13505 , Removing Barriers to Responsible Scientific Research Involving Human Stem Cells. The executive order also stated that the Secretary of Health and Human Service can conduct stem cell research using a human Embryonic Stem Cell (hESC), as permitted by law.
Internal NIH policies and procedures, consistent with Executive Order 13505 and these guidelines, will oversee the intramural NIH stem cell research. According to Executive Order 13505, there was an eligibility criteria set for the use of human embryonic stem cells.
Stem cells are a class of un-differentiated cells having the potential to differentiate themselves into specialized cell types. Generally, there are two types of stem cells depending upon their sources; embryonic adult. Stem cells obtained through human umbilical cord blood cells followed by a normal healthy birth, are taken out and then forced back into their pluripotent state in which they are incapable of differentiating themselves into any cell type.
Cells developed using these methods are known as induced Pluripotent Stem Cells (iPSC’s). However, these clinical-grade stem cells (iPDSs) are different from laboratory-grade stem cells because they are manufactured under a set of regulations known as current Good Manufacturing Practices (cGMP) that are approved by the Food and Drug Administration (FDA).
These regulations are designed to ensure the safety as well as the potential capability of the stem cells to be enrolled for use in human clinical trials. The NIH Common Fund’s Regenerative Medicine program is currently supporting the manufacturing of this cell line.
Anderson said that the process of developing new tools and techniques in the biomedical research community through strategic investment in high-impact research of stem cells is vital. He added that the aim of the NIH Common Fund is to promote this research healthily.
The question arises is, if taking laboratory stem cells to clinical grade level is following a cutter approach? And the answer is no. So far, the research conducted on stem cells, on one or any other level faces many barriers such as meeting the criteria of these guidelines, which is a time-consuming and costly process. Therefore the aim of the Stem Cell Translation Laboratory (SCTL) is to remove these barriers holding back the therapeutic applications of iPSCs and promote their efficacy and usefulness in pre-clinical and clinical trials.
The use of stem cell therapy on lab animals is already in progress — so far, researchers have been successful in undoing diabetic conditions by using iPSCs. Similarly, they are halfway towards the restoration of limb functioning in mice suffering from spinal cord injuries. This time, the target is a human, which is far more challenging for researchers, however the development of research-grade stem cells provides them hope of advancement in the development of medical therapies for human patients.
“As part of our longstanding commitment to providing critical bio specimens 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.
Sheldon added, considering the therapeutic benefits of these clinical-based stem cells, they are in high demand.
Currently RUCDR Infinite Biologics distribute the laboratory-grade cell lines among researchers made by the NIH Regenerative Medicine Program. Additionally, these clinical-based stem cells are stored and distributed by the National Institute of Neurological Disorders and Stroke (NINDS) Human Cell and Data Repository (NHCDR). The use and development of the stem cells is currently being supported through an NINDS grant to RUCDR Infinite Biologics at Rutgers University, Piscataway, New Jersey.