April 2016, Vol. 5, No. 3
Personalized Medicine’s SkepticsThe Last Word
The premise of personalized medicine is actually rather simple. If the diagnosis is correct at the molecular level, the prescribed treatment has a greater potential to be more accurate, efficacious, and safe. And if treatments are more accurate, patients will benefit from improved outcomes, and the health system will expend fewer resources on therapies that don’t work while targeting treatments that do on patients who will benefit.
Enormous progress has been made based on that promise. In 2008, the Personalized Medicine Coalition counted 13 products that it considered personalized medicines. Today there are over 150 on the market making a difference in patients’ lives. Last year alone, the FDA approved 13 new drugs with biomarker strategies in their labels, 28% of the total.
President Obama, inspired by the field’s advancement, has put in place a Precision Medicine Initiative to organize research on individual variation, which he and others contend “gives us one of the greatest opportunities for new medical breakthroughs that we have ever seen.”
All of this progress, however, has been met with renewed skepticism among some critics. They say personalized medicine represents more hype than hope. While few argue that the goals of personalized medicine are less than noble, these critics also believe that they are too ambitious, too costly, and unrealistic.
Three recent articles in the mainstream press illustrate the argument against personalized medicine.
In a front-page story in the New York Times on March 12 entitled “Genetics Often Muddle Options in Cancer Care,” Gina Kolata notes that many physicians don’t know how to manage the plethora of data they now have available to them. Basing her story on a single case, she suggests that our newfound ability to understand the genetic drivers of breast cancer confuses rather than improves care. Curiously, she ignored the results of promising research on personalized medicine, including a study presented at the 10th European Breast Cancer Conference in Amsterdam the week before, that demonstrated how, with an easy-to-use genetic test, researchers had successfully predicted which breast cancer patients could avoid chemotherapy without their cancers recurring after surgery, sparing those patients painful treatment and the health system unnecessary costs.
Similarly, Jeneen Interlandi writes in “The Paradox of Precision Medicine,” published in Scientific American in mid-March, that for every proponent of personalized medicine there is a critic who thinks that it is “a waste of time and money.” She questions especially the value of the Precision Medicine Initiative’s effort to collect and aggregate data from 1 million Americans to determine what works for whom, contending that the challenges of integrating the derived data into clinical care will be insurmountable. “Most physicians,” she writes, echoing Kolata’s argument, “are not trained to make sense of existing genetic tests, and so far no one has come up with a good way to train them.” As have others, she argues that it would make more sense to invest in less costly public health initiatives-diet, exercise, and smoking cessation-than in understanding and treating disease, as if public health and personalized medicine are locked into a zero-sum exercise.
In an essay he calls “The Limits of Personalized Medicine,” published in The Atlantic on March 16, Timothy Caulfield also argues that personalized medicine, which he equates with “using genetic information to motivate healthy lifestyles,” is inherently limited. Citing selected social science research, he says that knowing one’s risk does not persuade people to change unhealthy behaviors and, like Interlandi, contends that it may even dampen public support for public health initiatives like encouraging better diets, more exercise, and less substance abuse. Like Interlandi, he assumes that public health and personalized medicine are locked into a zero-sum game when clearly they are not.
In brief, what these 3 articles have in common is that they demonstrate that the bar for proving the value of personalized medicine is higher than for other medical initiatives. Clearly, the personalized medicine community needs to better document the evidence of its successes because skeptics have only to challenge its contentions to slow progress. And, equally important, proponents of personalized medicine need to educate healthcare providers and patients by explaining how patients will benefit from the integration of new methods of clinical care that stress getting the diagnosis right at the molecular level before therapies are prescribed.
The number of genes associated with breast cancer risk continues to increase. Recently, through whole exome sequencing, 2 groups of researchers have demonstrated that mutations in RECQL increase the risk of breast cancer.1 The first paper, by Cybulski and colleagues, was published online in Nature Genetics in April 2015, and [ Read More ]
A new biomarker can identify the subgroup of Class 1 uveal melanomas most likely to metastasize, according to a retrospective study published in Clinical Cancer Research, a journal of the American Association for Cancer Research (AACR). Among Class 1 uveal melanomas, those with high levels of PRAME mRNA were more [ Read More ]