Dr. Jeffrey D. Sharon, MD Clinical Neurotologist and Researcher
This article originally appeared in the Winter 2024 issue of On The Level.
Jeffrey D. Sharon, MD
It’s hard not to get frustrated with the current state of vestibular medicine.
As anyone who has suffered from a vestibular problem will attest, there are inefficiencies and errors in the diagnosis of different vestibular ailments. One doctor might conclude you have vertigo, whereas a second diagnoses labyrinthitis, and then a third is of the opinion that you have atypical migraines.
Furthermore, many treatments are unproven. Even at the best clinics in the world, patients are told to try a medication for several months and if that doesn’t work then they try a second medication, and then a third, and at some point years can go by without any forward progress.
Complicating matters further, some diseases are not understood. Nobody knows what causes Meniere’s disease. How can you treat a disease that you fundamentally don’t understand?
Whenever we are faced with more questions than answers, the solution is research.
Research: A Messy Solution
Research is a process of trying to answer questions using the scientific process.
Because data is inherently messy and because people are inherently complicated, controlled experiments are needed to extract clean data from the real world.
Take Meniere’s disease for example. For years the first line treatment of Meniere’s disease in Europe has been betahistine. In the United States betahistine is available through compounding pharmacies. Based on the European experience, doctors in the US have been prescribing betahistine for decades. And each doctor has some patients who say it works great, some who are not sure, and some who are pretty sure it doesn’t work.
So, is it a good treatment? A reasonable doctor might say that since it has a chance of working, and because it’s a safe medication, it’s worth a try. But finally in 2016 researchers completed the BEMED trial which compared betahistine treatment to a placebo and they found no difference in efficacy. You had the same treatment benefit with the medication and a sugar pill. Both reduced vertigo attacks by the same amount.
However, sometimes one question leads to another. Currently there is a thought that the dose studied in the BEMED trial was too low and that therefore either a higher dose or a combination pill is needed for betahistine to work.
To create the future we want we must shape it. Research is the reason that medical care today is better than it was 50 years ago, and hopefully why medical care in 50 years from now will be better than today.
The Basics of Participating in Research
It can be exciting to be a part of that process. But it’s also important to know what you are getting involved with. Sometimes research comes with risks. And it’s the job of the research team to adequately inform you of all those risks.
Let’s start with the basics:
- Participating in research is always 100% voluntary. That means that you can always say no. You also always have the right to withdraw consent.
- Any research study will have gone through an ethics committee before approval.
- Some studies involve treatment, and some studies don’t. That means that some treatments maybe provide direct benefit to you and some may not.
- As with other areas of life, it can be helpful to understand everyone’s motivations for doing the research.
What to Expect
To highlight what to expect with vestibular research, let me discuss two recent studies of mine.
Example 1
The first study was a randomized, placebo controlled clinical trial.
In that study we treated half of the subjects with a placebo for 3 months and we treated the other half with a drug. In order not to influence the results the research team and the patients were both blind to treatment, meaning nobody knew whether they got the drug or a placebo.
Subjects had to participate for 4 months and come in for 4 visits during that time.
We collected data, including some identifiable data like name and date of birth (almost every study does), and while we keep it secure in a cybersecurity approved cloud database, there is always a risk of data breach.
For that study the other risks relate to the study drug.
Reflecting back, it did feel a little odd to be blinded, not knowing if I was giving someone a placebo or not. On the other hand, it’s really the only way to know for sure if a drug treatment works.
Example 2
In the second study we scanned people’s brains with a functional MRI to try to discover which neural networks are involved with vestibular migraine.
This study was a lot shorter, just a single visit. There was also no treatment involved. But an MRI can be uncomfortable (I do personally find them claustrophobic), and we were showing videos in the scanner to trigger vertigo, so we were temporarily causing the symptoms that we are usually trying to avoid.
Both of these studies were completely paid for, the first by a drug company and the second by a research grant from the American Otological Society. So there was no cost to the participant.
Informed Consent
Whenever you participate in research, you should get a copy of the informed consent form. That’s a detailed document that should answer every single question you have about the study. You should have an opportunity to ask questions and think things over.
The informed consent form should specify:
- Any risks, benefits, and costs
- Who is paying for the study
- What is required of you
- What is the goal of the study
- How many visits there will be
- Whether you get compensated for your time
- Who to contact for any issues or questions
Entry Criteria
Every study will have entry criteria. This is a list of inclusion criteria (specific things that you need to qualify) and exclusion criteria (specific things that will disqualify you from participating).
For example, in a vestibular migraine study the inclusion criteria might specify that you must have a diagnosis of vestibular migraine according to Barany Society criteria, that you are above 18 years of age, and that you are fluent in English (if the study forms haven’t been translated to other languages).
The exclusion criteria might specify that you cannot have had ear surgery, brain surgery, a head trauma, or another vestibular condition.
In other words, inclusion criteria ensure that you are part of the group of patients that the researcher is trying to study, and the exclusion criteria ensure that you don’t have an additional factor that would complicate the data.
Is a Study Right For You?
When evaluating whether a research study is right for you, it’s important to understand how much risk is involved.
If you are getting an experimental treatment that has not been evaluated by the FDA, there can be some unknown risks. On the other hand, if someone is taking a drug that is FDA approved for one purpose and studying it for another disease, the risk is lower.
There are also observational studies where the research team just collects your data without changing your treatment. Presumably the only risk there is related to privacy of information.
How to Find Trials That Need Your Participation
The easiest way to find out if there is a clinical trial for you to participate in is to search clinicaltrials.gov. This is a website that maintains a database of ongoing clinical trials. Most respectable researchers will post their trial to that website, although I do find that it’s common for information to be out of date.
On clinicaltrials.gov you can search by a disease condition. So if you have been diagnosed with bilateral vestibular hypofunction you can search to see what trials are available to you. It’s also always reasonable to ask your doctor if they know of any trials that you could qualify for.
The Challenge of Recruiting
When running a clinical trial recruitment is usually your biggest challenge. Even though you do a lot of calculations ahead of time, once you start a trial it can seem like every person you evaluate either misses one of the inclusion criteria, has an exclusion criterion, or otherwise doesn’t want to participate.
Many trials run too long, run out of money, or don’t finish because of recruitment problems. That can be a pity because the goal of a trial is usually to provide knowledge that helps everyone with the condition of interest.
On the other hand, it can be hard for someone to participate in a clinical trial. There is a fear that you could lose valuable time if you get a placebo. The number of visits can be a burden. You may not want to accept all the inherent risks. You may just want treatment and not to sign a bunch of forms that can seem very serious and daunting.
Why People Choose to Participate in Research
I am personally inspired when, despite all that, one of my patients participates in research. When I ask them why, I usually get the same answer: “What I am going through is terrible and I don’t want anyone else to have to suffer as I did.”
Yes, it is true that sometimes someone participates in research because they tried all the usual treatments and nothing worked so they want to try something experimental. And of course that is more than reasonable, especially considering how disabling vestibular diseases can be.
But I find, more often than not, that my patients participate in research because of altruism. They want to be a part of the fight against disease. They want medical care to advance. They are okay with suffering a little for just the chance of alleviating someone else’s suffering.
I think that my patients are some of the best people that I’ve ever met. Research at its core is a partnership based on trust. It’s critical for the researcher to honor, respect, and cherish the sacred trust that is given to them by study participants.