What does the future of stem cell science look like?
Jo-Anna Reems, Ph.D., a University of Utah research associate professor in the Division of Hematology and Hematologic Malignancies, was an important part of a discussion related to that question recently at the Regenerative Medicine Partnering Forum, an annual event held in California. The forum was part of a broader initiative called the Stem Cell Meeting on the Mesa.
Organized by the Alliance for Regenerative Medicine, the California Institute for Regenerative Medicine and the Sanford Consortium for Regenerative Medicine, the recent Stem Cell Meeting on the Mesa brought together the regenerative medicine industry with the scientific research community to advance discussion about moving stem cell science into cures.
In addition to work as a research associate professor, Reems —who presented at the conference — also serves as scientific director of the University of Utah Health Care's Cell Therapy and Regenerative Medicine Facility (CTRM).
She shared her thoughts on the emerging regenerative medicine field in a Q&A with Health Feed.
Q: You practice in the field of regenerative medicine. For those unfamiliar with the subject, how would you describe what you do on a day-to-day basis as well as what the field of regenerative medicine entails?
To begin this discussion, we need to define what is meant by the term "regenerative medicine", a relatively new field that involves the repair and/or replacement of damaged or defective cells and tissue. The first successful regenerative medicine therapy occurred in 1956 when stem cells from the bone marrow of an identical twin were used to restore their sibling's bone marrow after they had undergone irradiation. The transplanted stem cells, capable of growing and differentiating into a number of various cell types, succeeded in achieving a complete remission of the sibling's leukemia by replenishing the cellular elements of their blood stream.
At first stem cells were obtained from the bone marrow of normal donors, later they were obtained from stem cells mobilized into the peripheral blood stream of a donor. And, around 1990, a third source of stem cells from umbilical cord blood was reported that facilitated the restoration of hematopoiesis. All of these stem cell sources are typically used to restore blood forming cells, but have also been used for non-hematopoietic disorders. More recently, other sources of stem cells with the capability of restoring non-blood forming cells have been identified. These stem cell sources include embryonic stem cells, induced pluripotent stem cells (iPS) and pluripotent mesenchymal stromal cells (MSCs). The potential of these stem cells as treatment modalities to repair and replace defective cells in a variety of disease states are currently under study. In particular, there are numerous clinical trials exploring the potential of the MSC to treat disease states that involve inflammatory bowel disease, musculoskeletal injuries, liver damage, burns and skin injuries, and spinal cord injury.
The University of Utah's CTRM was first established in 1990 to support the blood and marrow transplant (BMT) programs at the Huntsman Cancer Institute and Primary Children's Hospital. The day-to-day efforts of staff in supporting the BMT program include the collection of stem cells from donors/patients, transporting products to the CTRM facility where the quality of products is assessed and the stem cell product is prepared for infusion into patients. In some cases, stem cell products are frozen and stored at <-150 °C for later use for transplantation.
To include applications of novel cellular therapy products for the repair of diseased or injured non-hematopoietic tissues, the scope of the CTRM was expanded in 2006 by leveraging the infrastructure already in place for the blood and marrow transplant program. This expansion included, but was not limited to preparing cells for the treatment of diseases such as amyotrophic lateral sclerosis, liver damage and back pain etc. The process of delivering novel cell therapies is a long, arduous and labor intensive endeavor that ultimately leads to filing an investigational new drug (IND) application with the FDA to perform clinical trials. Steps along this process include transfer of technology from an academic or industrial partner to the CTRM and then optimization of procedures by the CTRM that outline the procurement, isolation, culture strategy, freezing and storing methods and transportation of cells. All of this is completed prior to submitting an IND application. Upon IND activation and under cGMP conditions products are then prepared for use in a clinical trial by the CTRM.
Q: Why are you passionate about regenerative medicine? What drew you to the field? What do you love about your job?
The healing abilities of the body are amazing and the possibility that it may one day be possible to harness this capability is not only fascinating, but also carries a reward of having the potential to profoundly influence the quality of life and possibly the life expectancy of individuals. What I love the most about my job is that it is a privilege to be part of this cutting edge science and to have the opportunity to work with highly dedicated and skilled individuals at the CTRM.
Q: What are some of the challenges and pressing issues facing the field of regenerative medicine right now?
A major challenge for the field of regenerative medicine is overcoming the difficulty in getting novel cell therapies to patients. Due to tight regulations in the U.S., getting a novel cellular therapy to the patient in the U.S. is lagging behind other countries. This is resulting in an increase in individuals seeking treatments in foreign countries for stem cells.
A major challenge for the CTRM is obtaining financial support that will accelerate innovations focused on the development of regenerative medicine in Utah as well as establishing working teams at the University of Utah that are focused on translating novel cell therapies specifically for treating patients under various medical disciplines. For example, core working teams dedicated to delivering a cell therapy for liver damage under gastroenterology or for macular degeneration under ophthalmology.
Q: What do you think the next big breakthrough with stem cells will be? What's on the horizon?
Wow, there are so many possibilities. Given the number of clinical trials that are currently underway using MSCs, I expect that the next big breakthrough will be that these cells have successfully and reproducibly alleviated the symptoms of several specific diseases.
On the horizon is the development of new culturing technologies that support in vitro self-renewal of stem cell populations. The development of such new methods will permit growing stem cells in the laboratory in large quantities that will be sufficient for clinical use.
Q: What's significant about the University of Utah's regenerative medicine program? What are some examples of breakthroughs this program has had, and where do you see it headed in the future?
The growth in the field of regenerative medicine is predominately in the public and private sector. The CTRM at the University of Utah is one of only a few academic centers with a Cell Therapy and Regenerative Medicine facility. Future goals of the CTRM include developing and delivering novel cell products via university collaborations and business partnerships as well as to build an internal core group of scientist, clinicians and bioengineers focused on developing, engineering and providing patients with the highest quality clinical grade cellular and tissue engineered products.