Revolutionizing Healthcare: How Induced Pluripotent Stem Cells are Paving the Way for Personalized Medicine

By Zachary Liebowitz | May 30th, 2024 

Stem cell research was forever changed following the breakthrough discovery of induced pluripotent stem cells (IPSC’s) by Dr. Shinya Yamanaka in 2006. Why? Because this discovery enabled the scientific community the ability to reprogram easily accessible adult somatic cells into a pluripotent state, all the while maintaining the same genetic composition. That means scientists can remove cells, most commonly skin and blood cells, and differentiate them into any cell variant found in the human body. This discovery dramatically impacted the field of medicine as a whole because it has the potential to alter the shape of future healthcare.

With the goal of developing a treatment with the most benefits alongside the least amount of side effects for the average person, traditional medicine is centered on treating the broad population. Human beings are 99.9 percent genetically similar, but the 0.1 percent variability leads to vast differences. Next time you are in a crowd, consider the variance in height, eye color, and limb length of those around you. But the imperceptible differences among us are even more pronounced at the molecular and genetic level. The variations in genes that code for proteins used in our molecular pathways lead to slight, but critically important, difference in how our individual organs and immune system address disease and metabolize drugs. What is beneficial for one subset of the population could lead to undesirable effects in another subset. Designing drugs and devices for the average person is no longer the best way to tackle disease treatment — hence the need for personalized medicine.

Personalized medicine is the idea of tailoring a treatment to be as unique as the disease present in the individual. Small genetic differences mean that the same disease in two individuals may need very different approaches to be most effective for each patient. IPSC technology allows for the customization of treatment to that level. A personalized medicine approach means that instead of prescribing a general treatment for a disease, cells can be taken from a patient and induced into the desired cell variant and thoroughly analyzed to determine which treatment regime is most effective for it. These cells under scrutiny have the exact genetic makeup of the patient, meaning that any unique nature of the disease specific to the patient can be observed and treated, thus enabling the physician to select the most effective treatment pertaining to the patients’ individual response.

Not only is this model of personalized medicine more clinically relevant than today’s approaches, it is simultaneously more humane, time effective, and cost efficient. 

Humane:  by allowing for the simultaneous screening of multiple treatments in vitro the patient is excused from the potential of spending months at a time on different treatments, experiencing the painful side effects of switching among differing drugs in an effort to find the best option.

Time effective: by running multiple treatments simultaneously the time needed to find the best treatment is dramatically reduced. There is no “in-between” time needed to allow one treatment to clear out of the system as is sometimes the case.   Very often when treating diseases, time is of the essence. 

Cost efficient: With reduced time and effort spent in determining the best treatment option, this could lead to less financial obligations by to the patient for their healthcare costs.  

An additional benefit of using IPSC’s can be seen in the treatment of rare and multifactorial diseases. I am sure you can guess why rare diseases can be hard to treat – it is a matter of numbers. Because only a small sample size exist, they are difficult to study using traditional observational methods such as epidemiology, or experimental methods such as clinical trials.  Here come IPSC’s to the rescue again, because individuals exhibiting rare diseases can have their cells taken and developed into models to study, thereby allowing for a better understanding of diseases and potentially to the development of treatments and a better outcome of life for those unfortunate enough to experience a rare disease. This same idea can be applied to multifactorial diseases such as autism spectrum disorders and type 1 diabetes, diseases in which the genetic identity is not yet known. Instead of spending vast sums of money in an effort to create animal models for these diseases we can instead study direct human models of these diseases with relative ease, allowing for a better understanding of these diseases/ailments that have been hard to study and treat in the traditional manner.

Personalized medicine is the future of healthcare. By utilizing the patients’ own cells through IPSC technology the medical establishment is able to study, develop, and tailor a treatment for a disease as unique as each patient. IPSC’s offer a path into a brighter future for patients and more effective and protective public health measures.

Sources:

Mathur, S., & Sutton, J. (2017). Personalized medicine could transform healthcare. Biomedical reports, 7(1), 3–5. https://doi.org/10.3892/br.2017.922

Chun, Y. S., Byun, K., & Lee, B. (2011). Induced pluripotent stem cells and personalized medicine: current progress and future perspectives. Anatomy & cell biology, 44(4), 245–255. https://doi.org/10.5115/acb.2011.44.4.245

Omole, A. E., & Fakoya, A. O. J. (2018). Ten years of progress and promise of induced pluripotent stem cells: historical origins, characteristics, mechanisms, limitations, and potential applications. PeerJ, 6, e4370. https://doi.org/10.7717/peerj.4370

The views expressed do not necessarily reflect the official policy or position of Johns Hopkins University or Johns Hopkins Bloomberg School of Public Health.

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