Anup Patel, MD: That’s a great idea. Let’s shift gears a little. Elizabeth, I want you to address some of the challenges that we have in this population of Dravet and LGS [Lennox-Gastaut syndrome] as far as taking medications. What are some of the barriers that these kids have and how can we address them?
Elizabeth A. Thiele MD, PhD: Actually, for a lot of patients, there are several barriers. Start with these kids, most of them are on more than just one medication. If we stop and think about the number of pills we’re actually asking these kids to take a couple of times a day, sometimes it’s shockingly overwhelming, quite a large number. And oftentimes it’s a problem. Some kids can’t swallow pills. Some kids refuse to swallow pills, also some people might just have difficulty with oral-motor function—being able to swallow.
Our nurses are actually very good at working with patients trying to figure out what strategies will work. I’m always impressed that parents can also be pretty creative. We have one of my kids who’s probably on 4 medications a day, refuses to take them. The only way the child will take them is if the parents crush up everything and put all the pills together in 1 capsule, they’re able to get the child to take that. So that’s a huge amount of effort for that family to do that every day, which is I think one reason we and also the pharmaceutical industry realize that we need formulations other than pills for many of our patients.
Anup Patel, MD: What about when you try to have them mix it with food? I’m always concerned. What if they don’t eat the whole portion of the food that has the medication?
Elizabeth A. Thiele MD, PhD: Oh no, that’s a big concern, or if they put the medication in liquid, what if it doesn’t get all of it or what if some of the medication sticks to the container? I think it’s a big concern, and that’s one way, I guess you consider if it was always a reliable loss, you could kind of guestimate by following levels of the medications, etcetera. But I think that does concern the parents. The other thing is gee, if Johnny always will take his medication in applesauce, because he likes applesauce, what if then starts hating applesauce because the medication is always in it? That also happens not infrequently. The child will become averse to different foods, even their favorite foods because that’s how they’ve been receiving their medicine.
Anup Patel, MD: Well, Elizabeth talks about how pharmaceutical companies have now started to address this in a more novel way. Eric, talk about some of the newer formulations and ideas that have been brought forward recently.
Jesus Eric Pina-Garza, MD: Well, one of the most recent that is very attractive and I think is a goldmine if you’re a parent, you don’t have to have Lennox-Gastaut. If you give your antibiotic, sometimes you find your spouse holding and the other one giving the dose, and it’s a battle. Both my wife and I are pediatric epileptologists, we think about our patients who have to do this day after day, and you have to put yourself in that position.
One of the delivery systems that are new and could potentially be helpful, not just for anticonvulsants but for medications in general, is the Aquestive Therapeutics technology, the PharmFilm delivery. I actually tested it in a couple of patients with Lennox-Gastaut because they offer a particular challenge. One, just like kids, they don’t like to take medications many times. Sometimes if they are cognitively impaired, which is often comorbidity, they don’t understand why it’s so important, so there’s not like reasoning about taking it. So the medication may fly. And we talk about dysphagia, and dysphagia with liquid is worse. Liquid is just a possible option for the person who prefers liquid over the tablet, which is used for the child, but not for someone with dysphagia.
But having a film technology that dissolves on your tongue and you’re not able to spit it out is incredible. That hopefully will be something that expands to other medications. At this point, it’s just trying to have absorption through the GI [gastrointestinal tract], but in the future maybe this technology will be a rescue medication because they have something called permeating engineering technology, which can go through the mucosa. So that’s a great thing. Short from that, we do what was mentioned earlier. My kids like Nutella, so if you can crush it, and put it in a small amount of Nutella, they take it and it hides the flavor. So you have to find something like that. The tricky part is that the kids are smart, even with cognitive regression. If you try too hard to give medication, they know something is there, so you have to be very careful about how to deliver medication, and it’s clearly a challenge.
Elaine C. Wirrell, MD: And just to say, a lot of our kids also are on the ketogenic diet, and so that inhibits our ability to use things like Nutella. That’s an extra layer of complexity.
Anup Patel, MD: Yes. That’s a really good point. Elizabeth, I wonder if you could spend a couple of minutes on the challenges of dietary therapies in combination with some of the medicines and how you overcome some of these barriers in your practice.
Elizabeth A. Thiele MD, PhD: I have been a zealot for dietary therapy for epilepsy now for 20 years. It’s been a big focus of what I’ve always done, both the classic ketogenic diet and low glycemic index treatment. I think both diets can be wickedly effective in treating epilepsy, almost any type of epilepsy, including Lennox-Gastaut, including Dravet. The difficulty when it comes to medications is many of our medications contain carbohydrates. So being on a diet, the child can’t get on any liquid formulation because they all have sugar, unless we have it compounded. And even some of our solid pill form medications have a lot of carbs. So that goes into the calculation of the diet. But that’s something that centers who do the diet know how to do, and that’s usually pretty easy to troubleshoot. So we frequently use the diet in combination with our medications.