Vikram Bhise, MD

Vikram Bhise, MD, Is an Associate Professor at Rutgers – Robert Wood Johnson Medical School.  He specializes in Epilepsy and Pediatric Neuroimmunology, and runs the pediatric demyelinating diseases program, evaluating children with multiple sclerosis, autoimmune encephalopathy, and related diseases.  He trained in Pediatrics and Pediatric Neurology, at Maimonides Medical Center and Montefiore Medical Center, respectively.  He subsequently received additional training in Clinical Neurophysiology with a focus on Epilepsy at SUNY Downstate Medical Center, and in Pediatric Multiple Sclerosis at SUNY Stony Brook Medical Center. Dr. Bhise conducts clinical research focused on biomarkers and quality of life in pediatric multiple sclerosis, as well as studies in epilepsy and neurogenetics. 

Q1. As a specialist who focuses on neuroimmunology, what forms of measurement do you use to make an evaluation or diagnosis for children with multiple sclerosis?

A1. There's a lot that goes into evaluating or diagnosing children with MS. Usually we start off with the story that the family brings to us. We look at what the child is experiencing and what the parents are seeing. Then we do a dedicated examination trying to substantiate the findings that they're describing and look for others they may not even be aware of. If they are having some blurred vision in their eye, can we tell if there's some abnormalities there that are correlating with what they see?

We try to get a good sense of the time-course of things, observing whether this is the first time something's happened or if this has this been going on for a while. Have there been multiple things going on, multiple episodes? We're primarily looking for events called relapses, which are neurologic attacks that are not quick. They don’t last for seconds or hours; they can last for days to weeks, sometimes even months. Individuals will have episodes that tend to get worse and then tend to get better. This is the type of description we’re looking to come from the families.

Once that assessment is complete, we've found that the MRI is one of the best tools in helping us confirm the diagnosis. It's not just diagnostic but the MRI also  has some prognostic potential and we're looking specifically for patterns in the MRIs. For children, that pattern can be a little bit more challenging. Their patterns can often overlap with patterns of other inflammatory diseases of the brain like ADEM for example and make it much more difficult for us to characterize someone as truly having MS.

There are also some other diseases which have been discovered in the past decade or elaborated upon like neuromyelitis optica and MOG antibody disorder, which can look exactly like MS in the early stages. Sometimes,  that's just not all the information we need. Sometimes it's more difficult to make those distinctions and in these cases, we will look at a spinal tap, a lumbar puncture, and for specific studies from those procedures to help us get a better understanding. There may be other ancillary tests that we use, such as evoked potentials, for example.

Evoked potential testing has kind of fallen to the wayside over the past decade because of the MRI studies becoming a much more useful tool, but we may still use the visual evoked potential to see if there are subtle lesions that can't be seen on the MRI. Other methods might include optical coherence tomography, which is another test looking at the eye that gives you a specific look at the retinal nerve fiber layer, which gets thinned after attacks on the eye on the optic nerve.

We may do neuropsychological testing, which is a battery of tests looking at different cognitive domains and trying to get a sense of a person's cognitive profile to see if that matches what we would expect in somebody with MS. This test could be more challenging for a teen and a child, particularly a younger child. When it comes to pediatric neuropsychology, it's a little bit harder sometimes to get good data, particularly from younger kids.

In addition, we have a battery of tests that we do on the serum. Some disorders, like NMO and MOG, have antibodies that help us identify them. We don't have a specific test that says, yes, you have MS and no, you don't have MS. It's really the combination of all the tools.

We do these tests often to look for things that mimic MS. We look for other neuroinflammatory or neurobiological diseases that can look a lot like MS and fool us. Most of the time they don't look exactly like MS, but every now and then you get a case that's virtually indistinguishable. There are other tools which may be in less use, but we put the combination of all these things together to help us make the most informed judgment.

The goal is to be able to have honest discussions with families that these tools are just tools that we're trying to play catch up with a disease and try to make a decision as fast as possible to prevent someone from going untreated.

Q2.  How does a diagnosis of multiple sclerosis affect the overall quality of life for a child/teen, and how does it affect their overall psychosocial health? Education?  Transition needs? Etc.

A2. It can be quite profound, just hearing the diagnosis can be truly life changing for most folks. It would really depend on the family the first time that we meet them. If they have no suspicion that this is what's going on, that can be a shock. Other families may be more aware of what’s going on. Perhaps another physician has already suggested it, or they came from the ER which had already done some of the baseline tests like the MRI, and they had some kind of  suspicion. Maybe they googled it and they saw something to be worried about, so they may be prepared. But even then,  once you confirm the diagnosis, it's really like the sky falling at that point.

Past the diagnosis stage, there's really an adjustment phase that we see, and we've been doing some work in this. We started doing some work looking at quality of life. We've interviewed a large  number of families and asked them some key questions such as, “What's important to you?” It is key for them to tell us rather than us telling them. By doing this, we’re finding out things that may not have been on the forefront of our minds, although it was certainly in the forefront of their minds,  so it's a good learning opportunity.

These may be things that we've seen in other quality of life studies in other diseases, but you also have to consider each disease unique and make sure you're looking at this from the perspective of the people that are really being affected. One of the great examples was that the teens really cared more about visible symptoms. For example, an adult with MS may have fatigue, severe fatigue. They may be unable to perform well in their job and that could be a game changer for them. Yet if they had a mild limp, they'd say, yeah, it's kind of embarrassing but I can keep going forward. I can hang in there and my colleagues at work might even support me; but for a teenager, they may care less about the fatigue and way more about having this limp that all their peers can now notice. The symptoms that are important to them can be totally different depending on the age group.

What we found is that teenagers look at things quite differently in trying to optimize their outcomes, and we don't just want them to be medically well. We want them to succeed in school, we want them to succeed in getting into college, or going into the workforce. So, we asked a lot about what it takes to get you there. We asked a lot of the young adults who had pediatric onset MS if they were  successful? And if you were, what got you there; and the ones who hadn't reached that yet we asked-- what do you need?

When it came to transition needs, by far, we’ve found almost complete silence on the teenager’s part, which was a little surprising for us. We thought that there would be a little bit of discussion. They didn’t understand what a 504 is. We don't expect the average individual to know, but we thought that they might understand what the tools were, yet they really had no language for discussing that with us. We realized that the start of our transition talks had to be focused on the things that we use for that language.

For example, if I wanted to get a ding in my car fixed. I had to spend 20 minutes explaining to the dealership what I wanted. It was a regular car dealership, so it was integrated. But I had to find the right words to say. I want “auto body.” If I said vehicle repair, they said, oh, you want your car tires replaced? No, no, no. so, it’s very important to speak the right language just to get the process started. Those are some of the things that we found.

Q3. In what ways do environmental and genetic risk factors influence therapeutic decisions in pediatric patients with MS? 

A3. They really play a big role in terms of the risk. We find that the more risk factors you have, likely we're dealing with a more severe disease. It doesn't necessarily always work that way, but you may be prepared to use a more potent therapy for individuals that are hitting more of the categories of concern.

But in addition to just the main disease modifying medications and MS, we look at vitamin D. And the data is yet to come out on that. There are some big studies that are trying to confirm or refute if vitamin D really has a therapeutic role, but we find that our teens and our kids have lower than average low vitamin D levels. We know that kids have low vitamin D levels nationwide in this country, but our patients are even lower than that. And that's one thing that we try to supplement and hope that by supplementing it, that it's going to be helpful. Maybe it's not as potent to therapy as the main medications, but we're hoping that's something to add on.

Q4. Overall, what are some advances, trends or recent studies regarding therapies that might support positive outcomes in children with MS? 

A4  Interestingly, we just don't have a lot of that research in kids. There's been tons and tons of great research in adults. Like many other fields, you take what you learn from that and you apply it to the teens and kids. But we've learned time and time again they're not just little adults. They're truly a separate group, and we must consider them as such, and we really need those studies in kids.

The first study that came out confirmed that fingolimod was a good and effective therapy in children. But does that mean that you're only limited to using the only FDA approved option, or do you really want to try to offer families the litany of choices that you do for an adult with MS? When I meet families for the first time, we’re spending a good hour just talking about the different treatment choices with them and looking at the risks, the benefits, why one option might be chosen over another,  how it's going to affect their lifestyle, and how it might fit into their life.

We want to still be able to make those decisions. I think we can make a more informed decision with fingolimod, but we don't want to just jump to conclusions with all those other therapies. We're a little bit behind the mark when it comes to therapies with kids, and we really need all those studies. They are active, and they are being done now; we're really waiting for those results to come out. That's going to be a huge change. Basically, that's the real trend. We're now going to see those studies in adults being replicated in kids one by one. Every time a new therapy comes out for adults, it must be validated in children as well.

Part of the regulations now do stipulate that these studies must be done. If you do a study in the adult population, you must see if you can do it in the pediatric population. You can't just say, hey, you know we're done. That's really what we're looking for in terms of getting the big therapeutic outcomes.

Vikram Bhise, MD, Is an Associate Professor at Rutgers – Robert Wood Johnson Medical School.  He specializes in Epilepsy and Pediatric Neuroimmunology, and runs the pediatric demyelinating diseases program, evaluating children with multiple sclerosis, autoimmune encephalopathy, and related diseases.  He trained in Pediatrics and Pediatric Neurology, at Maimonides Medical Center and Montefiore Medical Center, respectively.  He subsequently received additional training in Clinical Neurophysiology with a focus on Epilepsy at SUNY Downstate Medical Center, and in Pediatric Multiple Sclerosis at SUNY Stony Brook Medical Center. Dr. Bhise conducts clinical research focused on biomarkers and quality of life in pediatric multiple sclerosis, as well as studies in epilepsy and neurogenetics. 

Q1. As a specialist who focuses on neuroimmunology, what forms of measurement do you use to make an evaluation or diagnosis for children with multiple sclerosis?

A1. There's a lot that goes into evaluating or diagnosing children with MS. Usually we start off with the story that the family brings to us. We look at what the child is experiencing and what the parents are seeing. Then we do a dedicated examination trying to substantiate the findings that they're describing and look for others they may not even be aware of. If they are having some blurred vision in their eye, can we tell if there's some abnormalities there that are correlating with what they see?

We try to get a good sense of the time-course of things, observing whether this is the first time something's happened or if this has this been going on for a while. Have there been multiple things going on, multiple episodes? We're primarily looking for events called relapses, which are neurologic attacks that are not quick. They don’t last for seconds or hours; they can last for days to weeks, sometimes even months. Individuals will have episodes that tend to get worse and then tend to get better. This is the type of description we’re looking to come from the families.

Once that assessment is complete, we've found that the MRI is one of the best tools in helping us confirm the diagnosis. It's not just diagnostic but the MRI also  has some prognostic potential and we're looking specifically for patterns in the MRIs. For children, that pattern can be a little bit more challenging. Their patterns can often overlap with patterns of other inflammatory diseases of the brain like ADEM for example and make it much more difficult for us to characterize someone as truly having MS.

There are also some other diseases which have been discovered in the past decade or elaborated upon like neuromyelitis optica and MOG antibody disorder, which can look exactly like MS in the early stages. Sometimes,  that's just not all the information we need. Sometimes it's more difficult to make those distinctions and in these cases, we will look at a spinal tap, a lumbar puncture, and for specific studies from those procedures to help us get a better understanding. There may be other ancillary tests that we use, such as evoked potentials, for example.

Evoked potential testing has kind of fallen to the wayside over the past decade because of the MRI studies becoming a much more useful tool, but we may still use the visual evoked potential to see if there are subtle lesions that can't be seen on the MRI. Other methods might include optical coherence tomography, which is another test looking at the eye that gives you a specific look at the retinal nerve fiber layer, which gets thinned after attacks on the eye on the optic nerve.

We may do neuropsychological testing, which is a battery of tests looking at different cognitive domains and trying to get a sense of a person's cognitive profile to see if that matches what we would expect in somebody with MS. This test could be more challenging for a teen and a child, particularly a younger child. When it comes to pediatric neuropsychology, it's a little bit harder sometimes to get good data, particularly from younger kids.

In addition, we have a battery of tests that we do on the serum. Some disorders, like NMO and MOG, have antibodies that help us identify them. We don't have a specific test that says, yes, you have MS and no, you don't have MS. It's really the combination of all the tools.

We do these tests often to look for things that mimic MS. We look for other neuroinflammatory or neurobiological diseases that can look a lot like MS and fool us. Most of the time they don't look exactly like MS, but every now and then you get a case that's virtually indistinguishable. There are other tools which may be in less use, but we put the combination of all these things together to help us make the most informed judgment.

The goal is to be able to have honest discussions with families that these tools are just tools that we're trying to play catch up with a disease and try to make a decision as fast as possible to prevent someone from going untreated.

Q2.  How does a diagnosis of multiple sclerosis affect the overall quality of life for a child/teen, and how does it affect their overall psychosocial health? Education?  Transition needs? Etc.

A2. It can be quite profound, just hearing the diagnosis can be truly life changing for most folks. It would really depend on the family the first time that we meet them. If they have no suspicion that this is what's going on, that can be a shock. Other families may be more aware of what’s going on. Perhaps another physician has already suggested it, or they came from the ER which had already done some of the baseline tests like the MRI, and they had some kind of  suspicion. Maybe they googled it and they saw something to be worried about, so they may be prepared. But even then,  once you confirm the diagnosis, it's really like the sky falling at that point.

Past the diagnosis stage, there's really an adjustment phase that we see, and we've been doing some work in this. We started doing some work looking at quality of life. We've interviewed a large  number of families and asked them some key questions such as, “What's important to you?” It is key for them to tell us rather than us telling them. By doing this, we’re finding out things that may not have been on the forefront of our minds, although it was certainly in the forefront of their minds,  so it's a good learning opportunity.

These may be things that we've seen in other quality of life studies in other diseases, but you also have to consider each disease unique and make sure you're looking at this from the perspective of the people that are really being affected. One of the great examples was that the teens really cared more about visible symptoms. For example, an adult with MS may have fatigue, severe fatigue. They may be unable to perform well in their job and that could be a game changer for them. Yet if they had a mild limp, they'd say, yeah, it's kind of embarrassing but I can keep going forward. I can hang in there and my colleagues at work might even support me; but for a teenager, they may care less about the fatigue and way more about having this limp that all their peers can now notice. The symptoms that are important to them can be totally different depending on the age group.

What we found is that teenagers look at things quite differently in trying to optimize their outcomes, and we don't just want them to be medically well. We want them to succeed in school, we want them to succeed in getting into college, or going into the workforce. So, we asked a lot about what it takes to get you there. We asked a lot of the young adults who had pediatric onset MS if they were  successful? And if you were, what got you there; and the ones who hadn't reached that yet we asked-- what do you need?

When it came to transition needs, by far, we’ve found almost complete silence on the teenager’s part, which was a little surprising for us. We thought that there would be a little bit of discussion. They didn’t understand what a 504 is. We don't expect the average individual to know, but we thought that they might understand what the tools were, yet they really had no language for discussing that with us. We realized that the start of our transition talks had to be focused on the things that we use for that language.

For example, if I wanted to get a ding in my car fixed. I had to spend 20 minutes explaining to the dealership what I wanted. It was a regular car dealership, so it was integrated. But I had to find the right words to say. I want “auto body.” If I said vehicle repair, they said, oh, you want your car tires replaced? No, no, no. so, it’s very important to speak the right language just to get the process started. Those are some of the things that we found.

Q3. In what ways do environmental and genetic risk factors influence therapeutic decisions in pediatric patients with MS? 

A3. They really play a big role in terms of the risk. We find that the more risk factors you have, likely we're dealing with a more severe disease. It doesn't necessarily always work that way, but you may be prepared to use a more potent therapy for individuals that are hitting more of the categories of concern.

But in addition to just the main disease modifying medications and MS, we look at vitamin D. And the data is yet to come out on that. There are some big studies that are trying to confirm or refute if vitamin D really has a therapeutic role, but we find that our teens and our kids have lower than average low vitamin D levels. We know that kids have low vitamin D levels nationwide in this country, but our patients are even lower than that. And that's one thing that we try to supplement and hope that by supplementing it, that it's going to be helpful. Maybe it's not as potent to therapy as the main medications, but we're hoping that's something to add on.

Q4. Overall, what are some advances, trends or recent studies regarding therapies that might support positive outcomes in children with MS? 

A4  Interestingly, we just don't have a lot of that research in kids. There's been tons and tons of great research in adults. Like many other fields, you take what you learn from that and you apply it to the teens and kids. But we've learned time and time again they're not just little adults. They're truly a separate group, and we must consider them as such, and we really need those studies in kids.

The first study that came out confirmed that fingolimod was a good and effective therapy in children. But does that mean that you're only limited to using the only FDA approved option, or do you really want to try to offer families the litany of choices that you do for an adult with MS? When I meet families for the first time, we’re spending a good hour just talking about the different treatment choices with them and looking at the risks, the benefits, why one option might be chosen over another,  how it's going to affect their lifestyle, and how it might fit into their life.

We want to still be able to make those decisions. I think we can make a more informed decision with fingolimod, but we don't want to just jump to conclusions with all those other therapies. We're a little bit behind the mark when it comes to therapies with kids, and we really need all those studies. They are active, and they are being done now; we're really waiting for those results to come out. That's going to be a huge change. Basically, that's the real trend. We're now going to see those studies in adults being replicated in kids one by one. Every time a new therapy comes out for adults, it must be validated in children as well.

Part of the regulations now do stipulate that these studies must be done. If you do a study in the adult population, you must see if you can do it in the pediatric population. You can't just say, hey, you know we're done. That's really what we're looking for in terms of getting the big therapeutic outcomes.

Vikram Bhise, MD, Is an Associate Professor at Rutgers – Robert Wood Johnson Medical School.  He specializes in Epilepsy and Pediatric Neuroimmunology, and runs the pediatric demyelinating diseases program, evaluating children with multiple sclerosis, autoimmune encephalopathy, and related diseases.  He trained in Pediatrics and Pediatric Neurology, at Maimonides Medical Center and Montefiore Medical Center, respectively.  He subsequently received additional training in Clinical Neurophysiology with a focus on Epilepsy at SUNY Downstate Medical Center, and in Pediatric Multiple Sclerosis at SUNY Stony Brook Medical Center. Dr. Bhise conducts clinical research focused on biomarkers and quality of life in pediatric multiple sclerosis, as well as studies in epilepsy and neurogenetics. 

Q1. As a specialist who focuses on neuroimmunology, what forms of measurement do you use to make an evaluation or diagnosis for children with multiple sclerosis?

A1. There's a lot that goes into evaluating or diagnosing children with MS. Usually we start off with the story that the family brings to us. We look at what the child is experiencing and what the parents are seeing. Then we do a dedicated examination trying to substantiate the findings that they're describing and look for others they may not even be aware of. If they are having some blurred vision in their eye, can we tell if there's some abnormalities there that are correlating with what they see?

We try to get a good sense of the time-course of things, observing whether this is the first time something's happened or if this has this been going on for a while. Have there been multiple things going on, multiple episodes? We're primarily looking for events called relapses, which are neurologic attacks that are not quick. They don’t last for seconds or hours; they can last for days to weeks, sometimes even months. Individuals will have episodes that tend to get worse and then tend to get better. This is the type of description we’re looking to come from the families.

Once that assessment is complete, we've found that the MRI is one of the best tools in helping us confirm the diagnosis. It's not just diagnostic but the MRI also  has some prognostic potential and we're looking specifically for patterns in the MRIs. For children, that pattern can be a little bit more challenging. Their patterns can often overlap with patterns of other inflammatory diseases of the brain like ADEM for example and make it much more difficult for us to characterize someone as truly having MS.

There are also some other diseases which have been discovered in the past decade or elaborated upon like neuromyelitis optica and MOG antibody disorder, which can look exactly like MS in the early stages. Sometimes,  that's just not all the information we need. Sometimes it's more difficult to make those distinctions and in these cases, we will look at a spinal tap, a lumbar puncture, and for specific studies from those procedures to help us get a better understanding. There may be other ancillary tests that we use, such as evoked potentials, for example.

Evoked potential testing has kind of fallen to the wayside over the past decade because of the MRI studies becoming a much more useful tool, but we may still use the visual evoked potential to see if there are subtle lesions that can't be seen on the MRI. Other methods might include optical coherence tomography, which is another test looking at the eye that gives you a specific look at the retinal nerve fiber layer, which gets thinned after attacks on the eye on the optic nerve.

We may do neuropsychological testing, which is a battery of tests looking at different cognitive domains and trying to get a sense of a person's cognitive profile to see if that matches what we would expect in somebody with MS. This test could be more challenging for a teen and a child, particularly a younger child. When it comes to pediatric neuropsychology, it's a little bit harder sometimes to get good data, particularly from younger kids.

In addition, we have a battery of tests that we do on the serum. Some disorders, like NMO and MOG, have antibodies that help us identify them. We don't have a specific test that says, yes, you have MS and no, you don't have MS. It's really the combination of all the tools.

We do these tests often to look for things that mimic MS. We look for other neuroinflammatory or neurobiological diseases that can look a lot like MS and fool us. Most of the time they don't look exactly like MS, but every now and then you get a case that's virtually indistinguishable. There are other tools which may be in less use, but we put the combination of all these things together to help us make the most informed judgment.

The goal is to be able to have honest discussions with families that these tools are just tools that we're trying to play catch up with a disease and try to make a decision as fast as possible to prevent someone from going untreated.

Q2.  How does a diagnosis of multiple sclerosis affect the overall quality of life for a child/teen, and how does it affect their overall psychosocial health? Education?  Transition needs? Etc.

A2. It can be quite profound, just hearing the diagnosis can be truly life changing for most folks. It would really depend on the family the first time that we meet them. If they have no suspicion that this is what's going on, that can be a shock. Other families may be more aware of what’s going on. Perhaps another physician has already suggested it, or they came from the ER which had already done some of the baseline tests like the MRI, and they had some kind of  suspicion. Maybe they googled it and they saw something to be worried about, so they may be prepared. But even then,  once you confirm the diagnosis, it's really like the sky falling at that point.

Past the diagnosis stage, there's really an adjustment phase that we see, and we've been doing some work in this. We started doing some work looking at quality of life. We've interviewed a large  number of families and asked them some key questions such as, “What's important to you?” It is key for them to tell us rather than us telling them. By doing this, we’re finding out things that may not have been on the forefront of our minds, although it was certainly in the forefront of their minds,  so it's a good learning opportunity.

These may be things that we've seen in other quality of life studies in other diseases, but you also have to consider each disease unique and make sure you're looking at this from the perspective of the people that are really being affected. One of the great examples was that the teens really cared more about visible symptoms. For example, an adult with MS may have fatigue, severe fatigue. They may be unable to perform well in their job and that could be a game changer for them. Yet if they had a mild limp, they'd say, yeah, it's kind of embarrassing but I can keep going forward. I can hang in there and my colleagues at work might even support me; but for a teenager, they may care less about the fatigue and way more about having this limp that all their peers can now notice. The symptoms that are important to them can be totally different depending on the age group.

What we found is that teenagers look at things quite differently in trying to optimize their outcomes, and we don't just want them to be medically well. We want them to succeed in school, we want them to succeed in getting into college, or going into the workforce. So, we asked a lot about what it takes to get you there. We asked a lot of the young adults who had pediatric onset MS if they were  successful? And if you were, what got you there; and the ones who hadn't reached that yet we asked-- what do you need?

When it came to transition needs, by far, we’ve found almost complete silence on the teenager’s part, which was a little surprising for us. We thought that there would be a little bit of discussion. They didn’t understand what a 504 is. We don't expect the average individual to know, but we thought that they might understand what the tools were, yet they really had no language for discussing that with us. We realized that the start of our transition talks had to be focused on the things that we use for that language.

For example, if I wanted to get a ding in my car fixed. I had to spend 20 minutes explaining to the dealership what I wanted. It was a regular car dealership, so it was integrated. But I had to find the right words to say. I want “auto body.” If I said vehicle repair, they said, oh, you want your car tires replaced? No, no, no. so, it’s very important to speak the right language just to get the process started. Those are some of the things that we found.

Q3. In what ways do environmental and genetic risk factors influence therapeutic decisions in pediatric patients with MS? 

A3. They really play a big role in terms of the risk. We find that the more risk factors you have, likely we're dealing with a more severe disease. It doesn't necessarily always work that way, but you may be prepared to use a more potent therapy for individuals that are hitting more of the categories of concern.

But in addition to just the main disease modifying medications and MS, we look at vitamin D. And the data is yet to come out on that. There are some big studies that are trying to confirm or refute if vitamin D really has a therapeutic role, but we find that our teens and our kids have lower than average low vitamin D levels. We know that kids have low vitamin D levels nationwide in this country, but our patients are even lower than that. And that's one thing that we try to supplement and hope that by supplementing it, that it's going to be helpful. Maybe it's not as potent to therapy as the main medications, but we're hoping that's something to add on.

Q4. Overall, what are some advances, trends or recent studies regarding therapies that might support positive outcomes in children with MS? 

A4  Interestingly, we just don't have a lot of that research in kids. There's been tons and tons of great research in adults. Like many other fields, you take what you learn from that and you apply it to the teens and kids. But we've learned time and time again they're not just little adults. They're truly a separate group, and we must consider them as such, and we really need those studies in kids.

The first study that came out confirmed that fingolimod was a good and effective therapy in children. But does that mean that you're only limited to using the only FDA approved option, or do you really want to try to offer families the litany of choices that you do for an adult with MS? When I meet families for the first time, we’re spending a good hour just talking about the different treatment choices with them and looking at the risks, the benefits, why one option might be chosen over another,  how it's going to affect their lifestyle, and how it might fit into their life.

We want to still be able to make those decisions. I think we can make a more informed decision with fingolimod, but we don't want to just jump to conclusions with all those other therapies. We're a little bit behind the mark when it comes to therapies with kids, and we really need all those studies. They are active, and they are being done now; we're really waiting for those results to come out. That's going to be a huge change. Basically, that's the real trend. We're now going to see those studies in adults being replicated in kids one by one. Every time a new therapy comes out for adults, it must be validated in children as well.

Part of the regulations now do stipulate that these studies must be done. If you do a study in the adult population, you must see if you can do it in the pediatric population. You can't just say, hey, you know we're done. That's really what we're looking for in terms of getting the big therapeutic outcomes.