Stem cell therapy is creating new avenues for treating and curing disease. Small molecules, compounds, or other chemical entities have been the first line of defense in fighting many diseases for centuries. This practice has been intensified in the last century through the development of targeted drugs and inhibitors that ameliorate disease symptoms. However, because of the limitations of drug-to-target binding, only about 10% of cellular targets can be modulated in this way. The promise of cell therapy expands therapeutic options significantly. But as previously discussed, stem cell therapy has many challenges to overcome, and clear strategies to advance them into the clinic are emerging.1 But what can stem cell therapy learn from the decades of pharmaceutical development pipelines?
Limitations facing stem cell therapy include:
Unknown requirements for factors: what is controlling stem cell regulation? How is that contributing to a “cure”?
Heterogeneity of the stem cell population: Are only a small percentage of the stem cells therapeutically “capable” or “active” – how can we harness these cells?
In vivo differentiation requires further characterization and is dependent upon the microenvironment of the recipient, which is highly variable.
Lessons from pharmaceutical development suggest that two major practices will assist in the development of stem cell therapy applications:
Define Measurable Outcomes and Criteria
Complexity is rampant in stem cell therapy development. It is in the cells, the patients either donating or receiving cells, and the processing and manipulation of the cells. How can measurable clear outcomes be defined to standardize treatments? Using a multifactorial analysis to assess a series of different parameters, we can specify clear outcomes. An excellent example of this was described by a HemaCare Scientific Advisory Board member, Dr. Stephen Minger of GE Healthcare. GE Healthcare has used high content imaging to determine and measure as many variables as possible in creating consistent high quality stem cells on a massive scale.
Genomic variability amongst patients can determine how safe and effective a drug is for a given patient. As drug development has become more sophisticated, determining patient outcomes based on their genetic makeup is dictating drug selection and dosing. Similar pharmacogenomic profiling will advance stem cell therapy applications. A patient’s immune response to certain cell types could be predicted. Expression profiling of both the stem cell therapy, as well as the patient could be compared to allow for synergies or incompatibilities and for improving outcomes.
The horizon for new stem cell therapy applications promises treatments and cures for a variety of diseases. As the challenges facing this new technology are overcome, new strategies are implemented to move it into the clinic. And as standardization of the therapies progresses, a new era of medicine is opening.
1. Srijaya, T.C. et al. (2014) Advancing stem cell therapy from bench to bedside: lessons from drug therapies. Journal of Translational Medicine, 12:243