Tag: N of 1 experiments
Is the microbiome the key to health?
Interest in the microbiome – those trillions of bugs (bacteria and other microorganisms) in your gut – is increasing and according to an article in the NY Times, drug companies are trying to get in on the action.
As I’ve written before, these bugs may be important in the development of chronic disease leading to the possibility that you can “transplant” healthier bugs into someone with a disease. In fact, transferring the feces (poop) from one person to another has been shown to cure cases of Clostridioides difficile (C. diff) colitis (a life threatening infection of the gut caused by overuse of antibiotics). This is called fecal microbiota transplantation (FMT). Studies are ongoing to see if FMT can be used to treat inflammatory bowel disease (IBD), irritable bowel syndrome (IBS) and other chronic conditions. But there are a limited number of places doing FMT so people are doing their own transplants by harvesting the feces of a friend or family member and using a home blender (I’ll spare you any additional details but the DIY instructions are readily available online).
Patient-collected data
In April 2018, I participated in the 2018 Quantified Self (QS) Symposium on Cardiovascular Diseases held in San Diego. I was reminded of that session several weeks ago while attending the 2nd Annual Meeting of the Society for Participatory Medicine. In both conferences I was struck by the power of patients’ observations and measurements to manage their own diseases.
I first learned about the Quantified Self movement a few years ago while reading about Larry Smarr, an astrophysicist and computer scientist who started tracking his own exercise and weight but ultimately began expanding his self-tracking to include blood tests when he was told he had “pre-diabetes”. He ultimately diagnosed his own Crohn’s disease long before he had any symptoms based on analyzing his own blood and stool tests (including twice weekly stool microbiome analysis). He has since published a how-to guide in a biotechnology journal and participated in planning his own bowel resection for Crohn’s disease in 2016.
Personal experiments
During my medical training, we were taught that if a patient responds to a treatment, it doesn’t necessarily mean that every patient will respond in the same way. The results in a single patient might be due to chance so it was important to look at the results of well-designed research studies before we could conclude that the treatment really worked. In statistics, “N” refers to the sample size in an experiment so we referred to these individual observations as “N-of-1” experiments (and we did not look at them very favorably).
Unfortunately, there are lots of problems with research studies – they take a long time to complete, the patients in the studies are very carefully selected and may be very different from you, there is often bias in the way the results are interpreted, etc.
What if doctors and patients had tools that allowed them to design high-quality experiments specifically for the individual patient?
New types of evidence
It can be difficult to figure out how to use the results of research studies (randomized controlled trials or RCTs) to make a healthcare decision. There are many other problems with RCTs that may be less obvious.
First, to perform an RCT can take years – you need to get approval from the hospital where you are performing the study because you are doing research on humans. Then you need to get funding for the study so you may need to apply for some grants. After the study is completed, the results need to be analyzed; and then a paper needs to be written and submitted for publication to a journal. It could take years from the time the results are known until the time they are published.