Personalized medicine set to revolutionize clinical trials

Individuals in the medical community and beyond are paying an increasing amount of attention to personalized medicine, which involves tailoring treatment to meet a specific patient's circumstances and requirements. Even President Barack Obama acknowledged the approach in this year's State of the Union address, noting its potential to cure diseases such as cancer, diabetes and cystic fibrosis.

Universities working to translate the tenets of personalized medicine to clinical trials
The National Cancer Institute recently gave a $10.4-million grant to researchers at Duke University, North Carolina State University and the University of North Carolina at Chapel Hill with the goal of kick-starting a more personalized approach to designing clinical trials and analyzing their results. One of the project's principal investigators, Marie Davidian, spoke with the NC State News about what her team is trying to accomplish.

"Currently, most clinical trials offer a randomized snapshot of one moment in time," Davidian, a professor of statistics at NC State, told the media outlet. "These trials can tell you whether treatment A or B is more effective, on average. But effective treatment of individual patients, especially for a progressive and recurring disease like cancer, requires a physician to make a series of decisions over time while taking the patient's information into account."

Through the application of the tenets of personalized medicine to gather information about patients' biomarker characteristics, genetic profiles and medical histories, the goal of creating and deploying more optimized clinical trials gets closer to being realized. With this approach, trial results will not only indicate the effectiveness of treatments, but will also go a step further to determine which patients respond better than others to particular treatments and why. 

"Clinical trials focused on average, not personalized, responses have numerous shortcomings."

Trials focusing on average responses come with pitfalls, research finds
In a recent piece for the weekly scientific journal Nature, Nicholas Schork, director of human biology at the La Jolla, California-based J. Craig Venter Institute, detailed the shortcomings of clinical trials that focus on average rather than personalized responses. Schork, who is also a professor at the Translational Genomics Research Institute in Phoenix and the University of California, San Diego, explained that the patient recruitment bias toward white, Western trial participants resulted in the creation of drugs that can actually be harmful to individuals who don't fit this profile.

In some instances, negative effects befall patients who don't fit the "norm" established by clinical trials. In other cases, people with characteristics that deviate from those of the control group actually end up better off. Schork presented the leukemia drug Gleevec as an example, noting that it doubled the chances of survival among patients with tumors that have a particular chromosomal abnormality.

To truly grasp the need to apply the tenets of personalized medicine to clinical trials and the healthcare market at large, it is helpful to understand how ineffective the trials that focus on average responses can be. According to Schork, the 10 highest-grossing drugs in the United States only improve conditions for between 4% (in the case of Nexium, for heartburn) and 25% (Humira for arthritis, Remicade for Crohn's disease and Enbrel for psoriasis) of the people to whom they are prescribed.The effectiveness rates for the other six drugs are as follows:

  • Crestor, for high cholesterol, and Advair Diskus, for asthma: 5%, or one in every 20 patients
  • Copaxione, for multiple sclerosis: 6.25%, or one patient in 16
  • Neulasta, for neutropenia: 7.7%, or one patient in 13
  • Cymbalta, for depression: 11%, or one patient in nine
  • Abilify, for depression and schizophrenia: 20%, or one patient in five

With figures like these, it stands to reason that drug companies would be taking a hard look at developing personalized treatment options.

The most effective of the 10 highest-grossing drugs in the United States only improve conditions for one in four patients.The most effective of the 10 highest-grossing drugs in the United States only improve conditions for one in four patients.

Pharma companies' personalized medicine investments set to rise
According to research sponsored by the Personalized Medicine Coalition and conducted by the Tufts Center for the Study of Drug Development, pharmaceutical companies project a 33% increase in their drug development and testing-related personalized medicine investments over the next five years.

"Since 2010, groups have bought into this idea of personalized medicine and really gotten behind that, and it's demonstrated in the industry in terms of the investment in the space, with dollars and resources," stated Glen Ferguson, vice president of corporate development at diagnostic company HealthTell, as quoted by GenomeWeb. "The more we're learning, the better our tools are becoming, [and] the more we're understanding the complexity of the science related to disease and the human body."

The extent of the effect that personalized medicine may bring to clinical trials is still not fully understood, but one thing's for sure: Preliminary indications are encouraging.

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