Category: Evidence
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?
The bugs we live with
Remember in elementary school science class when we put samples of our hair and saliva in Petri dishes to see what grew? We even used cotton swabs to test the surfaces of our desks and bacteria grew in a few days. These experiments were designed to show us that we have lots of bacteria inside and on our bodies – and all around us. There are microbes (bacteria, viruses, fungi, etc.) everywhere in our “built environment” – the buildings we live and work in – as seen in this incredible animation.
Research suggests that the microbes in our guts play an important role in the development of disease. This collection of organisms, referred to as the microbiome (although technically the collection of organisms is called the microbiota and the genes of those organisms are called the microbiome), may play a role in the development of many diseases including diabetes, obesity, rheumatoid arthritis, asthma, allergies, Crohn’s disease, autism and cancer.
Vaccines and trust
Many years ago I worked in a travel clinic advising people about the immunizations they needed before visiting other countries. Sometimes shots were required for entry into a country, such as the yellow fever vaccine. But we also made sure that MMR (measles-mumps-rubella) and other vaccines were up-to-date. The reason was that while immunizations have been very successful in getting rid of measles (and other childhood illnesses) in the US, many countries still have outbreaks. The booster shots were not necessary in the US because we no longer had cases of measles. Until now.
We are seeing cases of measles (and other childhood illnesses) again because parents are increasingly refusing to get their kids vaccinated. The current outbreak of measles in California is causing a lot of public debate about how to force people to get their kids vaccinated. While we may need to find new ways to enforce vaccination, we also need to restore trust – people increasingly don’t trust doctors, pharmaceutical companies, government agencies or payers.
Health is life
When I was an infectious diseases specialist, most of the patients I saw were hospital inpatients but I also saw a few outpatients. They came to see me because of weeks or months of symptoms that their doctors couldn’t figure out and were often worried that they had a mysterious infection that was hard to diagnose. All of these patients had real symptoms – they were extremely tired, had headaches, muscle pains and sore throats. They generally arrived with stacks of medical records – numerous lab tests and notes from other doctors. I also noticed that many of them had serious “real life” problems – bad marriages, difficulties at work, housing problems, sick relatives and more. Perhaps they really did have an infection that I couldn’t find but I also began to wonder if their symptoms were caused by the stress.
My intuition was that many of these patients would benefit from speaking with a social worker, marriage counselor, psychologist, an expert in finding affordable housing or a financial planner. Unfortunately, these services were not part of our health care system. I suspected that many of my patients would get better if we were able to treat the whole patient, not just the symptoms.
The gut and the brain
We don’t normally think about the gut and the brain being connected. And yet many of us have gotten a stomach ache, nausea or diarrhea from stress or a feeling of “butterflies” from excitement. Or we may experience pleasure from certain foods or feel a need to eat when under stress.
The vagus nerve travels between the brain and other organs in the body and can transmit messages in both directions. The brain can send messages to the gut through chemicals (called neurotransmitters). The gut has its own nervous system (called the enteric nervous system or ENS) that controls digestion. But scientists now think that the ENS can also produce neurotransmitters to send to the brain.
The big question is whether the gut can actually cause symptoms and diseases of the nervous system.
The power of placebos
Lots of people talk about the placebo effect but what exactly is it?
The most reliable clinical studies compare a treatment that is being tested with a fake treatment (called a placebo). Generally, half the people in the study get the treatment and half get the placebo and the then the two groups are compared. In the case of pills the placebo is often a sugar pill. Researchers can even test the effectiveness of a surgical procedure by comparing it with a sham or fake procedure. In these studies (called randomized controlled trials or RCTs), patients (and their healthcare teams) don’t know who is getting the pill or procedure being studied and who is getting the placebo. The reason for this is that patients sometimes get better when they are given a placebo because they believe they will get better (called the “placebo effect”) or because their disease got better on its own.
So can patients get better just by believing they will get better? And can doctors actually prescribe placebos to help people get better?
Research in real time
This past week, I was reminded of the problem of fraud in medical research when the British courts ruled that the mother of a child with autism had lied about her child’s symptoms. The woman was a supporter of Dr. Andrew Wakefield, the British doctor who published studies connecting the Measles Mumps and Rubella – MMR – vaccine to autism based on “data” he made up.
Published medical studies, especially randomized controlled trials, remain the most reliable way for doctors to make treatment decisions. But, in addition to fraud, there are many other problems with medical research studies including the fact that they cost a lot of money to perform and take a long time to complete. An even bigger problem is that research studies have not been done for most of the questions that doctors and patients want answered. And even if there is a relevant published study, the people in the study may be very different from you.
Paying patients for their expertise
Thanks to the work of organizations like the Society for Participatory Medicine and patient advocates like e-patient Dave, the voice of the patient is being heard. And I’ve written before about organizations like the ImproveCareNow network where patients and families are treated as equal partners in quality improvement efforts.
I love that patients and families are being recognized for their expertise and that healthcare organizations are starting to involve patients as team members from the beginning of projects. I also love that organizations like PCORI (Patient Centered Outcomes Research Institute) are recognizing the importance of asking patients the research questions and outcomes that are most important to them.
So this is all really good, right? Yes, but…
The dark side of bacteria
Bacteria are our friends…but not all the time.
While I believe that we need to keep the bacteria in our bodies happy and that the improved cleanliness of modern life may be causing problems, there is also no question that bacteria are our enemies as well. You don’t have to look very far to see examples of how bacteria can cause serious illness or even death – meningococal meningitis, pneumococcal pneumonia, salmonella and tuberculosis to name a few. In most cases, antibiotics are required to treat these infections (or vaccines to prevent the infections).
Loving your bacteria
When my kids were little, I used to joke that bacteria are our friends, so I didn’t worry much when their pacifiers fell on the ground. As an infectious diseases specialist I had also seen the harmful effects of using too many antibiotics – emergence of bad bacteria like MRSA (methicillin resistant Staphylococcus aureus) and development of diseases due to killing off of good bacteria (like Clostridium difficile colitis, a serious inflammation of the gut caused by antibiotics). So I also tried to avoid giving them antibiotics. New research suggests that bacteria may be more helpful than we ever knew.