By Ralph Tarantino, PhD
Pharmaceutical Consultant and Principal, SteriTech Solutions, LLCMiddletown, NJ Most would agree that mapping of the human genome during the last few years of the 20th century was a key technological advance in a century that saw the most technological advances in history. Knowledge of the human genome makes pharmacogenomics possible, and pharmacogenomics makes personalized medicines possible. Personalized medicine—hype not withstanding—is a model for pharmacotherapeutics that cannot be ignored by those interested in advancements in the treatment of disease. It most often refers to use of an individual’s genetic information to optimize treatment or prevent disease for that individual. Crizotinib (Xalkori) and vemurafenib (Zelboraf) are notable personalized medicines approved by the FDA in 2011.
In oncology, the well established link between the overexpression of the HER2 tyrosine kinase and breast cancer is probably the best example of how personalized medicine can be effective for female patients. The use of trastuzumab to inhibit HER2 completes the loop of diagnosis to treatment. A recently published study (Slamon D et al. N Engl J Med. 2011;365(14):1273) showed an increase in survival rate in patients with breast cancer administered trastuzumab as adjunctive therapy. Personalized medicine is, however, not without its critics.
Some clinicians have expressed concerns that investment into successful widespread treatments should be the priority. Others have pointed out that clinical study design to establish the safety and efficacy of personalized medicine is too complex to be practical. It may not be easy to disagree with these opinions, but innovation is never easy, and there are threats to the innovative mindset needed to advance research in this area. The threats are from those who see a multitude of ethical issues involved and from those who control research and development budgets.
Will people be tested when there is no treatment? Who owns the information? Will it be used for discrimination? What is the obligation to inform family members of such information? These are common “ethical” issues raised by the usual people. My question to you as practitioners is how valid are they as issues when the object here is to save or at least extend human life? You should get involved; your opinions should be known.
Make no doubt about it, innovation is not everything; it’s the only thing in pharmaceutical research and development. Using the human genome to foster innovation in the treatment of disease is a no-brainer. Isaac Asimov was quoted as saying, “The saddest aspect of life right now is that science gathers knowledge faster than society gathers wisdom.” Dr. Asimov may be correct, but it’s something those of us in health care should not readily respect; we have the responsibility to be wiser.
Isn’t it time we filtered out the true ethical issues and stop this industry within an industry of bottom dwellers that feeds off fears of unwarranted litigation? Isn’t it time we put pressure on those who control R&D purse strings to channel funds for more mechanistic research? Isn’t time we pushed our professional organizations to take on these issues rather than get fat on our dues and contributions from Pharma? It is well past time I believe.