Monthly Archives: October 2013

The human microbiome: a new approach to treating non-infectious diseases

The fault lies not in our genes, but in our microbiome

Conceptualizing humans as an organism, the microbes living within and on our bodies are often neglected.  A recent trend in academia has been the research into the wide variety of micro-organisms living within and upon humans.  Overlooked until recently, differences in the human microbiome are implicated in many disease states from auto-immune disorders to obesity.  Some day is may be possible to lose a few pounds just by consuming the right mix of bacteria to colonizes your gut!

plaque--bacteria in the mouth microbiome

Many different bacteria from the mouth microbiome

Given the advent of low-cost, high-throughput sequencing and the relatively smaller genomes of micro-organisms, a plethora of studies have been done comparing microbiomes of healthy and sick patients.  First academia must answer the chicken-or-the-egg-question:  do people have these diseases because of the combination of bacteria they have (or don’t have) or does having the condition lead to the combination of bacteria?  Does the microbiome of a sick patient arise from having a specific microbiome or does being sick lead to the presence of the specific microbiome?

To test this question, sickened laboratory animals have been inoculated with the microbiome of healthy animals.  Generally, the answers point to the idea that the right combination of microbes can alleviate the symptoms of a disease.  The list of non-infectious diseases (allergies, asthma, diabetes, and obesity) that have been effectively treated by reconstituting the microbiome in laboratory animals is growing. While academic research on this area is booming, what practical progress has been made on this front?

Compare the number of clinical trials using fecal transplants (46) to those using a cell therapy (29,268).  Clearly there is much room for growth in the practical application of the human biome to disease states.

Additionally, these initial clinical studies are looking only at the microbiome of the lower gut.  This leaves many areas of the human anatomy unstudied.  What about the skin, upper gastrointestinal tract, hair, eyes, nose and other mucus membranes?  While academia is well on the way to working on these issues, little has yet been seen in the way of practical application of this as evidenced by the lack of clinical trials.

DeeAnn Visk, Ph.D., is a freelance science writer, editor, and blogger. Her passions include cell culture, molecular biology, genetics, and microscopy. DeeAnn lives in the San Diego, California area with her husband, two kids, and two spoiled hens. You are welcome to contact her at deeann.v@cox.net

Risk-based clinical trial monitoring

Increasing efficiency in clinical trials

While it sounds intimidating, risk based management is simply applying common sense in a systematic way.  Defining, evaluating, and managing risk is easily accomplished by using a documented approach to evaluate the risks.  Which needs closer monitoring:  a simple clinical trial in the United States at a reputable site previously used with much success or a complex trial in Zimbabwe at a location never utilized before?

Obviously, the trial with the greater unknowns is higher risk.

risk management for the FDA clinical trial

Evaluating risk to monitor clinical trials

Recently, the FDA came out with guidelines on risk-based management for clinical trials.  “The overarching goal of this guidance is to enhance human subject protection and the quality of clinical trial data by focusing sponsor oversight on the most important aspects of study conduct and reporting.”

What this means is merely commonsense in monitoring of clinical trials based on which ones have a higher risk of problems. 

 Another factor to consider beyond experience with a site and the complexity of the trial are the patient outcomes.  Will your trial involve life and death situations? 

Quantifying this can be done using a matrix of categories such as geographical location of the study, Good Clinical Practice compliance (GCP), and the relationship of the sponsor with the investigator.

 Understanding these concepts is crucial to increase efficiency and decrease costs of clinical trials.  As with any change in a regulatory environment, the industry is grappling with how to implement it. Pilot programs explore the implementation and thousands are waiting anxiously for “the answer”. The challenge is that the answer—as with everything within clinical research—is that it depends.

 Laurie Halloran’s presentation on this and other topics in clinical trials made them easy to grasp.  She presented at the San Diego Clinical Research Network meeting last week Tuesday at the Sheppard Mullin law firm.

DeeAnn Visk, Ph.D., is a freelance science writer, editor, and blogger. Her passions include cell culture, molecular biology, genetics, and microscopy. DeeAnn lives in the San Diego, California area with her husband, two kids, and two spoiled hens. You are welcome to contact her at deeann.v@cox.net