Precision Medicine: A Targeted Approach to Treatment

“I want the country that eliminated polio and mapped the human genome to lead a new era of medicine – one that delivers the right treatment at the right time. In some patients with cystic fibrosis, this approach has reversed a disease once thought unstoppable. Tonight, I'm launching a new Precision Medicine Initiative to bring us closer to curing diseases like cancer and diabetes – and to give all of us access to the personalized information we need to keep ourselves and our families healthier.”

            This quote, from President Barack Obama’s State of the Union address on January 28^th 2014, marked the start of a federal effort to shift the way in which diseases are treated. President Obama coined the program the “Precision Medicine Initiative”, and its goal is to shift the paradigm of disease treatment from a “one-fits all” model to a more personalized approach. To support this effort, $215 million has been budgeted for investment in organizations such as the National Institutes of Health (NIH), the FDA, and the Office of the National Coordinator for Health Information Technology.

            With this funding, these organizations have set goals of enrolling millions of individuals in research programs to track and house a multitude of factors that contribute to the individuals’ well being. With this information, a database is to be set up to house all of the data that is collected through the research. The database will contain a multitude of information such as medical records, gene profiles, lifestyle activities and more. From this, the government hopes to generate new advancements in biomedicine that will allow physicians to tailor treatment options to the individual, so that diseases can be diagnosed and treated on an individual and more personal basis. The research will focus on establishing patterns among individuals, and scientists will analyze how individuals are similar in terms of their genetic makeup, lifestyle choices, environmental exposures, and many other factors. Using this information, patients can be classified into subpopulations for which a specific treatment can be developed to be better combat disease.

            The benefits of this approach are plentiful. The most obvious of these is that physicians will be able to fine-tune treatments for individuals so that diseases can be diagnosed and treated in a targeted manner. It will also allow individuals access to their own health profile, giving them a sense of empowerment and control over their health. In turn this could empower individuals and families to take action against destructive lifestyle choices that may be affecting their health. Having this knowledge at one’s own fingertips in this easy of a manner could kick start a desire to change unhealthy habits.

            More importantly in my mind, this program encourages private and public programs to work together. Reading “The Genome War” opened my eyes as to the pride that researchers feel towards their work, as well as the disdain that is held when that work is purely sought after to put money in the pockets of corporations. This program offers incentive for both sides, both academia and the private sector. Research institutions such as NIH will be receiving funding to carry out the research and organization behind the database. What this research uncovers will then pave the way for better treatments and drugs to combat specific forms or types of diseases that are seen in individuals with patterns of genetic and lifestyle characteristics. I view this relationship as a very important and necessary one. Too often is research halted or completely abandoned due to a lack of funding. The private sector has the money to fund such research, but there is no motive from a business standpoint to invest in “basic research” that may not turn up anything of value. This Precision Medicine Initiative is giving researchers the initial funding they need to prove that personalized medicine is the future, and I believe that once the ball gets rolling the private sector will realize the value in the approach. Hopefully this will motivate the private sector to invest in this research without losing sight of the goal: to improve the lives of those living with the diseases that give their products purpose.

When Does Life Begin: The Ethical Dilemma Behind Embryonic Stem Cell Research

At what point is something considered human?  Is the potential of life more important than saving a life?  What level of protection and respect is required for what stages of embryonic development?  These are some of the questions the National Bioethics Advisory Commission had to ask in order to determine whether or not embryonic stem cell research is moral or ethical.

Areas where adult stem cells
can be found

Stem cells can be found in many places in the body and can be used to study diseases, test new drugs, identify how undifferentiated cells become differentiated, and generate cells and tissues that could be used for cell-based therapies.  There are three types of stem cells that scientists understand.  Adult stem cells are differentiated cells that can be found in the brain, heart, and bone marrow.  They can renew themselves and yield some or all of the major specialized cell types of tissue or organ in the human body.  Their primary role is to maintain and repair the tissues in which they are found.  This means that stem cells found in the heart are differentiated to repair heart muscle cells whereas stem cells found in the leg are differentiated to repair leg muscle cells.  The second type of stem cell is induced pluripotent stem cells.  These stem cells are adult cells that have been genetically reprogrammed to an embryonic stem cell-like state through the forced expression of genes and other factors important for embryonic stem cells.  The last type of stem cell is embryonic stem cells.  Embryonic stem cells have the capacity to become any cell in the human body.  They are entirely undifferentiated (pluripotent).  They are easier to grow in a culture than adult stem cells and offer opportunities to learn about the differentiation of cells and the growth of human embryos.  This could pave the road for research in genetic diseases and birth defects.  Embryonic stem cells are derived from embryos.  Most are derived from fertilized eggs (in vitro) that were donated for research purposes.  They are NOT derived from fertilized eggs in a woman's body.  Once the eggs are obtained, they must be grown until about five days after fertilization when the blastocyst has formed and is filled with inner cell mass (embryoblast), the cells that are then used in embryonic stem cell research.  In order to obtain these cells, the blastocyst must be broken and the ethical dilemma arises.

Blastocyst with inner cell mass
in upper right.

Despite the scientific advances embryonic stem cell research could have, the ethical dilemma forces us to choose between two moral principles:

1. The duty to prevent or alleviate suffering
2. The duty to respect the value of human life

This is because in order to obtain the cells, the embryo has to be destroyed, thus destroying a possible human life, despite its potential to alleviate the suffering of thousands of people.  Therefore, the moral question behind embryonic stem cell research is at what point does a human embryo count as a human life and what degree of respect and protection is required at different stages of development?  Some examples of the arguments include the moral status from fertilization onwards and whether or not the embryo has increasing status as it develops.

Arguments against Embryonic Stem Cell Research:	Arguments for embryonic stem cell research:
“Development from a fertilized egg into a baby is a continuous process and any attempt to pinpoint when person-hood begins is arbitrary.”	·         It has not yet been implanted into the uterus ·         Does not have the psychological, physical, or emotional properties associated with being human ·         The embryo does not have the opportunity to become a child without being implanted into a woman’s uterus.  Something with such a low probability of becoming a person should not be treated as if it already were
·         If we judge the moral status of the embryo in order to determine it human-hood, we make an arbitrary decision about who is human.  If human-hood was defined as the formation of the nervous system, we still would not believe that someone who had lost nerve to be any less human. ·         If we cannot determine whether or not an embryo is human than we should not destroy it.  “A hunter does not shoot if he is not sure whether his target is a deer or a man.”	·         Tons of fertilized eggs are lost because of natural causes so what does it matter if we use some for research?

                After much deliberation of the National Bioethics Advisory Commission, it was decided that while the research was ethical, restrictions needed to be set in place in order to provide the public with the assurance that the research would be conducted within an accountable and rigorous system of oversight and review.  The restrictions that were recommended were that first, the research should be limited to using only “cadaveric fetal material” and embryos remaining after infertility treatments.  Second, sponsorship should only occur on an appropriate system of national oversight. 

                In conclusion, while the board was unable to determine when human life truly began or what stages of development require what levels of protection and respect, it was decided that embryonic stem cell research was, in fact, moral as long as it followed the guidelines they set.