When patients are diagnosed with epilepsy, the first line of treatment is usually anti-seizure medication, which controls seizures in most cases.
However, about a third of epilepsy patients are drug-resistant, meaning their seizures can’t be controlled with medication.
These patients might have to try other forms of treatment, like lifestyle changes or surgery. Some might decide to join clinical trials.
Q&A about clinical trials
Michael Gelfand, MD, PhD, a physician at Penn Medicine, discusses some of the trials happening at Penn—and what patients need to know before signing up for a trial.
Q: What makes someone a good candidate for a clinical trial?
Dr. Gelfand: There isn’t one basket—all trials are different, just like all patients are different. So, it really depends on the specific trial and on the patient’s goals.
For example, some patients can’t find a medication that stops their seizures, so they might be good candidates for a trial of a new daily medication of device to prevent seizures. Other patients can feel an “aura,” or a sense that a seizure is coming, and might be good candidates for a trial of a medication to help prevent that aura from turning into a larger seizure.
Q: What are the main benefits of participating in clinical trials?
Dr. Gelfand: I’d say there are two big ones. The main one is possibly finding something that works.
People who don’t get control over their seizures, or feel like epilepsy is lowering their quality of life, are always looking for something new. Something that will help even though other things have failed in the past.
There’s also a more altruistic benefit. Patients realize that they have benefited from research done in the past and often want to give back. The epilepsy community is very tight-knit. People with well-controlled epilepsy know people who have epilepsy that isn’t as well-controlled, and they want to help.
Q: What is the process of joining a clinical trial?
Dr. Gelfand: The first step is discussing it with your physician and deciding if it’s the best option for you. Then the team conducting the trial will review your case and speak with you to determine if the trial is appropriate.
They may want to know what other treatments you’ve tried for epilepsy or what other medical problems you have. If you’re a good candidate, you’ll come to clinic for a more detailed discussion and enrollment
Q: How long does that process take?
Dr. Gelfand: It’s actually a pretty quick enrollment process—usually less than a month. The trials themselves can last anywhere from a few days to a few years.
Q: Who is the ideal trial participant?
Dr. Gelfand: The ideal patient is someone who is reliable. If you want to do a trial, you have to reliably take your medicine, or have family that will help you take it. You need to be able to reliably report if you’re having seizures, so we can tell if a treatment is working.
Q: Is there anyone who shouldn’t participate in a trial?
Dr. Gelfand: There isn’t really a type of person who shouldn’t join. Many of our patients have other conditions besides epilepsy that could affect their eligibility for joining certain trials. But it depends on the specific trial.
Q: Can patients do more than one trial at a time?
Dr. Gelfand: In most cases, no, particularly if the study involves the use of a new or experimental medication, since this might make the results difficult to interpret.
It might be possible to do a second “observational” study that does not involve new treatments. But it is always very important to discuss these decisions with the study team—both for accuracy and to ensure personal safety.
Q: What are some of the concerns that patients have?
Dr. Gelfand: Some patients are concerned about having enough time, getting transportation—the logistics.
But the main concern patients have is definitely safety. They may be scared because new medications or devices, like all treatments, can have side effects. One of our primary goals in conducting trials is understanding and minimizing side effects, to keep our patients safe while trying to treat their epilepsy.
Q: Does that mean the trials aren’t safe?
Dr. Gelfand: Not at all. Drugs have to go through significant testing before being approved for a large clinical trial. While no treatment is 100% safe, patients in clinical trials are chosen and monitored very carefully to minimize risks and maximize possible benefits.
Q: What are some of the trials happening now at Penn Medicine?
Dr. Gelfand: We have a trial of a new daily medication which we hope will help some of that one-third of patients who continue to have seizures on existing medications. Another trial is studying a possible treatment for patients with seizure clusters—multiple seizures happening over and over for a few hours, which can be dangerous and distressing to patients.
There are also non-drug-based trials. We test devices like responsive neurostimulation (RNS) that have a lot of hope for patients whose seizures don’t respond to medications.
And then, we have a few smaller-scale trials starting, such as one evaluating a smartwatch that can detect convulsions, to allow patients to get help faster.
Also read: Can New Therapy Stop Epileptic Seizures?
Learn about Seizures and Epilepsy Treatments and Procedures
Q: What do you see in the future for epilepsy clinical trials?
Dr. Gelfand: The biggest problem in epilepsy is that a large number of patients continue to have seizures despite trying all existing treatments. Medications just don’t work for them.
There are many new devices and surgical treatments, but we often are just beginning to understand how to use them. My hope is that trials in the future will help us understand how to combine these approaches with medical therapy to close that gap and make more patients seizure-free.