The 2012 Nobel Prize Award in Physiology or Medicine emphasizes the expectation that scientists and physicians have for the potential of using engineered adult stem cells to heal humans.

Stem cells are regenerative medicine

As chief scientific officer of a leading stem cell preservation bank involved in stem cell research, I was intrigued and more than a little excited that the 2012 Nobel Prize in Physiology or Medicine was awarded to a pair of scientists involved in stem cell research. The award is significant because it emphasizes the hope that scientists and physicians have for the beneficial use of stem cells in regenerative medicine.
Sir John B. Gurdon (left) and Shinya Yamanaka (right) were jointly recognized for discovering that mature cells can be reprogrammed to become pluripotent. The technical name for the stem cells they engineered is Induced Pluripotent Stem (iPS) Cells. iPS cells are created by taking mature, differentiated cells from virtually any part of the body — skin, blood, umbilical cord, etc. — and figuratively turning back the clock to create cells in an embryonic-like state.

Perfect match

Once in the embryonic state, the cells can be differentiated into cells of any tissue type. The discovery is groundbreaking because iPS cells could be created from any individual and serve as a reservoir to make new, healthy cells and tissues that are HLA-matched for that individual in the future. It is important to note that Gurdon and Yamanaka’s research involved adult stem cells, not embryonic stem cells. Although implementation of this discovery is years, perhaps decades away from being useful for patients, it is none-the-less very exciting and emphasizes the expectation that stem cells will have a profound impact on regenerative medicine and the ultimate quality of life as our population ages⁽¹⁾.

Still more work needed

Although none of the ethical concerns exist with iPS cells that are present with the use of embryonic stem cells:

• considerable regulatory hurdles must be overcome to demonstrate that the process is safe and does not cause tumor formation
• improvements are required to make the process more efficient and easier to perform in the laboratory
• cost-saving measures need to be identified to make the technology widely available

It is not clear if some cell types are better than others at generating iPS cells and/or if the age of the starting cell is important. A recent report from scientists at the Salk Institute used cord blood cells to generate iPS cells and convert them into neuron-like cells.

“This study shows for the first time the direct conversion of a pure population of human cord blood cells into cells of neuronal lineage by the forced expression of a single transcription factor,” said Juan Carlos Izpisua Belmonte, a professor in Salk’s Gene Expression Laboratory, who led the research team. The study was published on July 16, 2012, in the Proceedings of the National Academy of Sciences.
Someday, this may prove valuable for the treatment of a wide range of neurological conditions, including stroke, traumatic brain injury and spinal cord injury.

Our hope for the future

We share the hope that stem cells may one day be routinely used to cure a wide variety of diseases. Cord blood has become standard of care for treatment of leukemia, lymphoma, metabolic storage disease, immune deficiencies and dozens of other debilitating diseases. The use of cord blood stem cells as therapy does not have the ethical concerns associated with embryonic stem cells, and has a clear path through the regulatory process.

We are actively looking for new uses for stem cells derived from cord blood, cord tissue and menstrual blood.

Our own research efforts include setting up new studies involving the use of menstrual stem cells in wound healing; two FDA-approved clinical trials that are studying the use of menstrual stem cells to treat Type I Diabetes and liver cirrhosis; and pre-clinical studies to explore the use of cord blood stem cells to treat neurological disorders that include stroke and ALS. The latter is being conducted in conjunction with Saneron-CCEL Therapeutics, our Tampa, Florida-based research and development partner that cultivates and advances cellular therapies for deadly diseases that lack adequate treatment options.