Part II: "DIPG is not a rare disease. It's a neglected disease."
If you haven’t already, I encourage you to read Part I of my story here, for important context on my late daughter Juliana, and her grueling experience in a pediatric clinical trial for DIPG.
As I mentioned in Part I, I spent nearly 30 years as a professor and scientist in infectious and vector borne diseases. I know how to run clinical trials, as I participated in my own human studies during my years in academia. So, when my nine-year-old daughter, Juliana, was diagnosed in February 2018 with a terminal brain cancer called DIPG (diffuse intrinsic pontine glioma), we looked at various clinical trials that could extend her life, because DIPG is inoperable and has very few to no treatment options.
Unfortunately, Juliana’s clinical trial experience was painful and ineffective for her. She died one year after her diagnosis, just shy of 11 years old. I walked away from a successful career and a wonderful lab that I built, to focus my energy on pediatric brain tumors and clinical trials, along with support for the DIPG Advocacy Group and the Marc Jr Foundation. We are fighting for change at the federal level, and below I outline why, using my experiences as both a grieving father and a scientist.
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“We need more investments from the federal government”
We have all these kids dying every year from DIPG, a disease they call “rare.” But it’s not a rare disease. It’s neglected. I worked on neglected tropical diseases. Why do I call them neglected? Because there’s very little investment in them. But organizations don’t want to hear that. Is malaria neglected? No, because it gets so much money. And then you leave the rest with minimal funding. But the rest affect a lot of people. They’ll say, “There isn’t any money to be made. Why invest in something that only kills 400 people a year?” That’s the rationale we’re working with. And it’s unacceptable. If someone said, “We have two airplanes full of kids, crashing every year and killing 400. Are we going to do something?” Absolutely. They’re going to move everything to change that. But rare diseases? Nope.
Regarding pediatric brain tumors, we need more investments from the federal government to advance new treatments…investments that are consistent and representative of the size of our pediatric population. Roughly 22% of the US population is pediatric (0-18 year-olds). So how come only 4% of funding goes to pediatric research? 96% goes to adult cancers. Why don't pediatric cancers get an amount of money that is at least comparable to its population size? Wouldn't that be [more] fair? And not only that, but the deadliest cancers get almost nothing. I’m not joking when I say that.
This is why I’m now working on the advocacy side of things, on Juliana’s behalf. The DIPG community has a couple of social platforms with over a thousand members worldwide. Specifically, I am a member of the DIPG Advocacy Group, founded by Janet Demeter who also lost her son to DIPG 10 years ago.
The DIPG Advocacy Group has been pushing for Congress to designate a National DIPG Awareness Day. We are currently in our fifth Congress, attempting to get the legislation approved. We’ve been denied multiple times, despite us having the majority vote in favor of it. The Office of Speaker of the House wouldn’t put it on the floor. Why? Her office cited legislation passed in the 90’s, which banned the designation of commemorative dates in the House, due to situations where “the commemorative may only be incidental to the overall purpose of the measure.” We have been told moving forward with this legislation “would break precedent.”
There’s even a pediatrician in Congress, and not even she would sign on as co-sponsor, because of “precedent.” I guess breaking precedent so the deadliest pediatric tumor no one knows about gets a day of awareness is totally out of order.
“We need to make things more logical & not overburden patients who are already suffering”
In 2016, the 21st Century Cures Act was approved, created to help spur innovation in medicine, especially for patients who need treatment options fast. We should be working with that and make things more logical. We need to modernize clinical trials. We need to make it easier to get to clinical trial centers, and easier to communicate with the clinicians, in a way that is not going to be demanding and overburden patients who are already suffering. Will these changes bring immediate cures? Of course not. But they will bring a little more peace of mind and a little more humanity to some of the families that have to go through this.
We can certainly rely on telehealth for clinical trials; several telehealth platforms already exist, and data indicates they can make a significant difference. Consider the pandemic. We quickly adapted and proved telehealth worked well. Why can't we implement some of that here? In February 2020, I was teaching a college course called Emerging Infectious Diseases, and we predicted what would happen with the pandemic. We knew the cases would spike and there’d be a huge rush to hospitals. Telehealth exploded and proved it’s something we could rely on. I wrote a white paper pushing for telehealth, even before the pandemic started…just to show the scientific rationale behind it and to lay the groundwork for what’s possible. It’s been shown that telehealth can increase patients numbers and diversity in a clinical trial, reduce attrition rates, it is more economical, and can be significantly more humane, among other benefits.
In the white paper, one of the major caveats I mention - something that I’ll admit is over my head - is the issue related to state rights and how we do licensing for clinicians. Because it all varies state to state. But again, using the pandemic as an example, the FDA allowed for universal licensing to go across the country. So, it's something that can definitely be worked out. The FDA has the ability to waive the licensing issue for clinical trials, especially in situations where the disease is terminal and there are no other options. Further, years ago Congress passed legislation on the patient’s right to try: a right for patients - particularly those inflicted by terminal diseases - to try drugs that their physician feels can benefit them, even if the treatment hasn’t been proven.
Another issue to address is drug pricing and insurance approvals. In Juliana’s case, among the various drugs we tried to reduce the effects of the inflammation caused by the tumor, and to try to slow down the tumor's advance, two drugs in particular each reaching very close to $30 thousand dollars per a 30-day treatment regimen. In other words, that’s basically $1k per pill. This is not trivial in terms of money, so obviously insurance denied it. They claimed that these drugs hadn’t been tested for pediatric cancer.
To get coverage for these drugs, I needed to write an appeal to the insurance company. Neither the hospital staff nor the clinical staff treating the patient are not allowed to write appeals; it has to come from the individual or their relatives/caregiver. So, I thought, this is what I’ve done all my life as a scientist, write papers to describe the work. Prior to publishing their work, scientists go through a peer-review process in which other scientists review and critique what has been submitted. There is often a back-and-forth regarding the data. Once all the details are cleared, then a paper can be accepted for publication and life continues. Here, I figured I’d be facing a similar situation, which I had to overcome with a combination of a personal justification backed by hard data.
I went ahead and after a couple of days of research and writing, I submitted a five-page appeals letter to insurance, outlining the rationale for why the drugs we requested for Juliana should be covered. The appeals letter was almost immediately approved and Juliana received the medications. To my surprise, the hospital even asked if my letter could be used as a template.
“We don’t have a true health care system. It’s disease care.”
Our healthcare system as a whole needs to shift. Currently, for any treatment, the approach is hierarchical and individualistic. A leader is appointed and they make the decisions. Their voice is the rule. This has become normal. But it’s just wrong. And this idea of the U.S. “healthcare” system. It’s actually an oxymoron. We don’t have a true health care system. It's disease care/management. Because drugs to treat disease make money. The FDA has approved drugs that show no clinical benefit to patients. I've seen some of the data…it's kind of ridiculous. But they benefit drug companies, because they make them money. And they can charge whatever they want. As an example, a drug to treat a particular vector-borne parasitic disease costs $50 overseas and around $50k in the US. This is the exact same drug here and elsewhere. It is the only FDA-approved drug for this particular disease. But if you happen to travel overseas, say to Costa Rica, and come back with the disease and would like to use this drug, be ready to fork over 1000 times what your friends in France, India, Brazil would pay for the same drug!
We need to change the paradigm with what’s called “systems medicine.” It’s a team-based approach. Folks in the lab studying the data. Nurses observing the patients and communicating that back to the team. It’s an environment where everyone’s thoughts are valid, and everyone listens. It’s a collaborative approach to try to understand the underlying issue of each individual. We have the capabilities to predict disease… to establish the mechanisms that will potentially reduce or eliminate infection or exposure. So instead of just treating the disease, you really approach it at all angles to try to get to the root of it.
We got lucky that Juliana's primary clinician was collaborative with me. I told her from the beginning, you're the clinician - you've seen these patients before - and I have not. But I'm going to throw some ideas at you. I'm going to ask you questions based upon my understanding of immunology and microbiology. If I'm wrong, please tell me, but if I'm right, let's continue the conversation. She was open to that dialogue, which was great, because we both knew that Juliana's condition was terminal. There were no clear options.
“Clinical trials need to provide more support”
At least I've been in this business for 30 years. So, I know how and where to look for information…how to navigate things, even if I don’t have all the details. But even I still had to fight an uphill battle. So can you imagine the people who don’t have the same experience as me, having to do things like write an appeal letter to their insurance company? That's where the clinical trials need to provide more support.
For a deeper dive into the critical need for modernizing clinical trials, you can read my white paper here.
If you haven’t already, I encourage you to read Part I of my story here, for important context on my late daughter Juliana, and her grueling experience in a pediatric clinical trial for DIPG.
As I mentioned in Part I, I spent nearly 30 years as a professor and scientist in infectious and vector borne diseases. I know how to run clinical trials, as I participated in my own human studies during my years in academia. So, when my nine-year-old daughter, Juliana, was diagnosed in February 2018 with a terminal brain cancer called DIPG (diffuse intrinsic pontine glioma), we looked at various clinical trials that could extend her life, because DIPG is inoperable and has very few to no treatment options.
Unfortunately, Juliana’s clinical trial experience was painful and ineffective for her. She died one year after her diagnosis, just shy of 11 years old. I walked away from a successful career and a wonderful lab that I built, to focus my energy on pediatric brain tumors and clinical trials, along with support for the DIPG Advocacy Group and the Marc Jr Foundation. We are fighting for change at the federal level, and below I outline why, using my experiences as both a grieving father and a scientist.
“We need more investments from the federal government”
We have all these kids dying every year from DIPG, a disease they call “rare.” But it’s not a rare disease. It’s neglected. I worked on neglected tropical diseases. Why do I call them neglected? Because there’s very little investment in them. But organizations don’t want to hear that. Is malaria neglected? No, because it gets so much money. And then you leave the rest with minimal funding. But the rest affect a lot of people. They’ll say, “There isn’t any money to be made. Why invest in something that only kills 400 people a year?” That’s the rationale we’re working with. And it’s unacceptable. If someone said, “We have two airplanes full of kids, crashing every year and killing 400. Are we going to do something?” Absolutely. They’re going to move everything to change that. But rare diseases? Nope.
Regarding pediatric brain tumors, we need more investments from the federal government to advance new treatments…investments that are consistent and representative of the size of our pediatric population. Roughly 22% of the US population is pediatric (0-18 year-olds). So how come only 4% of funding goes to pediatric research? 96% goes to adult cancers. Why don't pediatric cancers get an amount of money that is at least comparable to its population size? Wouldn't that be [more] fair? And not only that, but the deadliest cancers get almost nothing. I’m not joking when I say that.
This is why I’m now working on the advocacy side of things, on Juliana’s behalf. The DIPG community has a couple of social platforms with over a thousand members worldwide. Specifically, I am a member of the DIPG Advocacy Group, founded by Janet Demeter who also lost her son to DIPG 10 years ago.
The DIPG Advocacy Group has been pushing for Congress to designate a National DIPG Awareness Day. We are currently in our fifth Congress, attempting to get the legislation approved. We’ve been denied multiple times, despite us having the majority vote in favor of it. The Office of Speaker of the House wouldn’t put it on the floor. Why? Her office cited legislation passed in the 90’s, which banned the designation of commemorative dates in the House, due to situations where “the commemorative may only be incidental to the overall purpose of the measure.” We have been told moving forward with this legislation “would break precedent.”
There’s even a pediatrician in Congress, and not even she would sign on as co-sponsor, because of “precedent.” I guess breaking precedent so the deadliest pediatric tumor no one knows about gets a day of awareness is totally out of order.
“We need to make things more logical & not overburden patients who are already suffering”
In 2016, the 21st Century Cures Act was approved, created to help spur innovation in medicine, especially for patients who need treatment options fast. We should be working with that and make things more logical. We need to modernize clinical trials. We need to make it easier to get to clinical trial centers, and easier to communicate with the clinicians, in a way that is not going to be demanding and overburden patients who are already suffering. Will these changes bring immediate cures? Of course not. But they will bring a little more peace of mind and a little more humanity to some of the families that have to go through this.
We can certainly rely on telehealth for clinical trials; several telehealth platforms already exist, and data indicates they can make a significant difference. Consider the pandemic. We quickly adapted and proved telehealth worked well. Why can't we implement some of that here? In February 2020, I was teaching a college course called Emerging Infectious Diseases, and we predicted what would happen with the pandemic. We knew the cases would spike and there’d be a huge rush to hospitals. Telehealth exploded and proved it’s something we could rely on. I wrote a white paper pushing for telehealth, even before the pandemic started…just to show the scientific rationale behind it and to lay the groundwork for what’s possible. It’s been shown that telehealth can increase patients numbers and diversity in a clinical trial, reduce attrition rates, it is more economical, and can be significantly more humane, among other benefits.
In the white paper, one of the major caveats I mention - something that I’ll admit is over my head - is the issue related to state rights and how we do licensing for clinicians. Because it all varies state to state. But again, using the pandemic as an example, the FDA allowed for universal licensing to go across the country. So, it's something that can definitely be worked out. The FDA has the ability to waive the licensing issue for clinical trials, especially in situations where the disease is terminal and there are no other options. Further, years ago Congress passed legislation on the patient’s right to try: a right for patients - particularly those inflicted by terminal diseases - to try drugs that their physician feels can benefit them, even if the treatment hasn’t been proven.
Another issue to address is drug pricing and insurance approvals. In Juliana’s case, among the various drugs we tried to reduce the effects of the inflammation caused by the tumor, and to try to slow down the tumor's advance, two drugs in particular each reaching very close to $30 thousand dollars per a 30-day treatment regimen. In other words, that’s basically $1k per pill. This is not trivial in terms of money, so obviously insurance denied it. They claimed that these drugs hadn’t been tested for pediatric cancer.
To get coverage for these drugs, I needed to write an appeal to the insurance company. Neither the hospital staff nor the clinical staff treating the patient are not allowed to write appeals; it has to come from the individual or their relatives/caregiver. So, I thought, this is what I’ve done all my life as a scientist, write papers to describe the work. Prior to publishing their work, scientists go through a peer-review process in which other scientists review and critique what has been submitted. There is often a back-and-forth regarding the data. Once all the details are cleared, then a paper can be accepted for publication and life continues. Here, I figured I’d be facing a similar situation, which I had to overcome with a combination of a personal justification backed by hard data.
I went ahead and after a couple of days of research and writing, I submitted a five-page appeals letter to insurance, outlining the rationale for why the drugs we requested for Juliana should be covered. The appeals letter was almost immediately approved and Juliana received the medications. To my surprise, the hospital even asked if my letter could be used as a template.
“We don’t have a true health care system. It’s disease care.”
Our healthcare system as a whole needs to shift. Currently, for any treatment, the approach is hierarchical and individualistic. A leader is appointed and they make the decisions. Their voice is the rule. This has become normal. But it’s just wrong. And this idea of the U.S. “healthcare” system. It’s actually an oxymoron. We don’t have a true health care system. It's disease care/management. Because drugs to treat disease make money. The FDA has approved drugs that show no clinical benefit to patients. I've seen some of the data…it's kind of ridiculous. But they benefit drug companies, because they make them money. And they can charge whatever they want. As an example, a drug to treat a particular vector-borne parasitic disease costs $50 overseas and around $50k in the US. This is the exact same drug here and elsewhere. It is the only FDA-approved drug for this particular disease. But if you happen to travel overseas, say to Costa Rica, and come back with the disease and would like to use this drug, be ready to fork over 1000 times what your friends in France, India, Brazil would pay for the same drug!
We need to change the paradigm with what’s called “systems medicine.” It’s a team-based approach. Folks in the lab studying the data. Nurses observing the patients and communicating that back to the team. It’s an environment where everyone’s thoughts are valid, and everyone listens. It’s a collaborative approach to try to understand the underlying issue of each individual. We have the capabilities to predict disease… to establish the mechanisms that will potentially reduce or eliminate infection or exposure. So instead of just treating the disease, you really approach it at all angles to try to get to the root of it.
We got lucky that Juliana's primary clinician was collaborative with me. I told her from the beginning, you're the clinician - you've seen these patients before - and I have not. But I'm going to throw some ideas at you. I'm going to ask you questions based upon my understanding of immunology and microbiology. If I'm wrong, please tell me, but if I'm right, let's continue the conversation. She was open to that dialogue, which was great, because we both knew that Juliana's condition was terminal. There were no clear options.
“Clinical trials need to provide more support”
At least I've been in this business for 30 years. So, I know how and where to look for information…how to navigate things, even if I don’t have all the details. But even I still had to fight an uphill battle. So can you imagine the people who don’t have the same experience as me, having to do things like write an appeal letter to their insurance company? That's where the clinical trials need to provide more support.
For a deeper dive into the critical need for modernizing clinical trials, you can read my white paper here.
If you haven’t already, I encourage you to read Part I of my story here, for important context on my late daughter Juliana, and her grueling experience in a pediatric clinical trial for DIPG.
As I mentioned in Part I, I spent nearly 30 years as a professor and scientist in infectious and vector borne diseases. I know how to run clinical trials, as I participated in my own human studies during my years in academia. So, when my nine-year-old daughter, Juliana, was diagnosed in February 2018 with a terminal brain cancer called DIPG (diffuse intrinsic pontine glioma), we looked at various clinical trials that could extend her life, because DIPG is inoperable and has very few to no treatment options.
Unfortunately, Juliana’s clinical trial experience was painful and ineffective for her. She died one year after her diagnosis, just shy of 11 years old. I walked away from a successful career and a wonderful lab that I built, to focus my energy on pediatric brain tumors and clinical trials, along with support for the DIPG Advocacy Group and the Marc Jr Foundation. We are fighting for change at the federal level, and below I outline why, using my experiences as both a grieving father and a scientist.
“We need more investments from the federal government”
We have all these kids dying every year from DIPG, a disease they call “rare.” But it’s not a rare disease. It’s neglected. I worked on neglected tropical diseases. Why do I call them neglected? Because there’s very little investment in them. But organizations don’t want to hear that. Is malaria neglected? No, because it gets so much money. And then you leave the rest with minimal funding. But the rest affect a lot of people. They’ll say, “There isn’t any money to be made. Why invest in something that only kills 400 people a year?” That’s the rationale we’re working with. And it’s unacceptable. If someone said, “We have two airplanes full of kids, crashing every year and killing 400. Are we going to do something?” Absolutely. They’re going to move everything to change that. But rare diseases? Nope.
Regarding pediatric brain tumors, we need more investments from the federal government to advance new treatments…investments that are consistent and representative of the size of our pediatric population. Roughly 22% of the US population is pediatric (0-18 year-olds). So how come only 4% of funding goes to pediatric research? 96% goes to adult cancers. Why don't pediatric cancers get an amount of money that is at least comparable to its population size? Wouldn't that be [more] fair? And not only that, but the deadliest cancers get almost nothing. I’m not joking when I say that.
This is why I’m now working on the advocacy side of things, on Juliana’s behalf. The DIPG community has a couple of social platforms with over a thousand members worldwide. Specifically, I am a member of the DIPG Advocacy Group, founded by Janet Demeter who also lost her son to DIPG 10 years ago.
The DIPG Advocacy Group has been pushing for Congress to designate a National DIPG Awareness Day. We are currently in our fifth Congress, attempting to get the legislation approved. We’ve been denied multiple times, despite us having the majority vote in favor of it. The Office of Speaker of the House wouldn’t put it on the floor. Why? Her office cited legislation passed in the 90’s, which banned the designation of commemorative dates in the House, due to situations where “the commemorative may only be incidental to the overall purpose of the measure.” We have been told moving forward with this legislation “would break precedent.”
There’s even a pediatrician in Congress, and not even she would sign on as co-sponsor, because of “precedent.” I guess breaking precedent so the deadliest pediatric tumor no one knows about gets a day of awareness is totally out of order.
“We need to make things more logical & not overburden patients who are already suffering”
In 2016, the 21st Century Cures Act was approved, created to help spur innovation in medicine, especially for patients who need treatment options fast. We should be working with that and make things more logical. We need to modernize clinical trials. We need to make it easier to get to clinical trial centers, and easier to communicate with the clinicians, in a way that is not going to be demanding and overburden patients who are already suffering. Will these changes bring immediate cures? Of course not. But they will bring a little more peace of mind and a little more humanity to some of the families that have to go through this.
We can certainly rely on telehealth for clinical trials; several telehealth platforms already exist, and data indicates they can make a significant difference. Consider the pandemic. We quickly adapted and proved telehealth worked well. Why can't we implement some of that here? In February 2020, I was teaching a college course called Emerging Infectious Diseases, and we predicted what would happen with the pandemic. We knew the cases would spike and there’d be a huge rush to hospitals. Telehealth exploded and proved it’s something we could rely on. I wrote a white paper pushing for telehealth, even before the pandemic started…just to show the scientific rationale behind it and to lay the groundwork for what’s possible. It’s been shown that telehealth can increase patients numbers and diversity in a clinical trial, reduce attrition rates, it is more economical, and can be significantly more humane, among other benefits.
In the white paper, one of the major caveats I mention - something that I’ll admit is over my head - is the issue related to state rights and how we do licensing for clinicians. Because it all varies state to state. But again, using the pandemic as an example, the FDA allowed for universal licensing to go across the country. So, it's something that can definitely be worked out. The FDA has the ability to waive the licensing issue for clinical trials, especially in situations where the disease is terminal and there are no other options. Further, years ago Congress passed legislation on the patient’s right to try: a right for patients - particularly those inflicted by terminal diseases - to try drugs that their physician feels can benefit them, even if the treatment hasn’t been proven.
Another issue to address is drug pricing and insurance approvals. In Juliana’s case, among the various drugs we tried to reduce the effects of the inflammation caused by the tumor, and to try to slow down the tumor's advance, two drugs in particular each reaching very close to $30 thousand dollars per a 30-day treatment regimen. In other words, that’s basically $1k per pill. This is not trivial in terms of money, so obviously insurance denied it. They claimed that these drugs hadn’t been tested for pediatric cancer.
To get coverage for these drugs, I needed to write an appeal to the insurance company. Neither the hospital staff nor the clinical staff treating the patient are not allowed to write appeals; it has to come from the individual or their relatives/caregiver. So, I thought, this is what I’ve done all my life as a scientist, write papers to describe the work. Prior to publishing their work, scientists go through a peer-review process in which other scientists review and critique what has been submitted. There is often a back-and-forth regarding the data. Once all the details are cleared, then a paper can be accepted for publication and life continues. Here, I figured I’d be facing a similar situation, which I had to overcome with a combination of a personal justification backed by hard data.
I went ahead and after a couple of days of research and writing, I submitted a five-page appeals letter to insurance, outlining the rationale for why the drugs we requested for Juliana should be covered. The appeals letter was almost immediately approved and Juliana received the medications. To my surprise, the hospital even asked if my letter could be used as a template.
“We don’t have a true health care system. It’s disease care.”
Our healthcare system as a whole needs to shift. Currently, for any treatment, the approach is hierarchical and individualistic. A leader is appointed and they make the decisions. Their voice is the rule. This has become normal. But it’s just wrong. And this idea of the U.S. “healthcare” system. It’s actually an oxymoron. We don’t have a true health care system. It's disease care/management. Because drugs to treat disease make money. The FDA has approved drugs that show no clinical benefit to patients. I've seen some of the data…it's kind of ridiculous. But they benefit drug companies, because they make them money. And they can charge whatever they want. As an example, a drug to treat a particular vector-borne parasitic disease costs $50 overseas and around $50k in the US. This is the exact same drug here and elsewhere. It is the only FDA-approved drug for this particular disease. But if you happen to travel overseas, say to Costa Rica, and come back with the disease and would like to use this drug, be ready to fork over 1000 times what your friends in France, India, Brazil would pay for the same drug!
We need to change the paradigm with what’s called “systems medicine.” It’s a team-based approach. Folks in the lab studying the data. Nurses observing the patients and communicating that back to the team. It’s an environment where everyone’s thoughts are valid, and everyone listens. It’s a collaborative approach to try to understand the underlying issue of each individual. We have the capabilities to predict disease… to establish the mechanisms that will potentially reduce or eliminate infection or exposure. So instead of just treating the disease, you really approach it at all angles to try to get to the root of it.
We got lucky that Juliana's primary clinician was collaborative with me. I told her from the beginning, you're the clinician - you've seen these patients before - and I have not. But I'm going to throw some ideas at you. I'm going to ask you questions based upon my understanding of immunology and microbiology. If I'm wrong, please tell me, but if I'm right, let's continue the conversation. She was open to that dialogue, which was great, because we both knew that Juliana's condition was terminal. There were no clear options.
“Clinical trials need to provide more support”
At least I've been in this business for 30 years. So, I know how and where to look for information…how to navigate things, even if I don’t have all the details. But even I still had to fight an uphill battle. So can you imagine the people who don’t have the same experience as me, having to do things like write an appeal letter to their insurance company? That's where the clinical trials need to provide more support.
For a deeper dive into the critical need for modernizing clinical trials, you can read my white paper here.
If you haven’t already, I encourage you to read Part I of my story here, for important context on my late daughter Juliana, and her grueling experience in a pediatric clinical trial for DIPG.
As I mentioned in Part I, I spent nearly 30 years as a professor and scientist in infectious and vector borne diseases. I know how to run clinical trials, as I participated in my own human studies during my years in academia. So, when my nine-year-old daughter, Juliana, was diagnosed in February 2018 with a terminal brain cancer called DIPG (diffuse intrinsic pontine glioma), we looked at various clinical trials that could extend her life, because DIPG is inoperable and has very few to no treatment options.
Unfortunately, Juliana’s clinical trial experience was painful and ineffective for her. She died one year after her diagnosis, just shy of 11 years old. I walked away from a successful career and a wonderful lab that I built, to focus my energy on pediatric brain tumors and clinical trials, along with support for the DIPG Advocacy Group and the Marc Jr Foundation. We are fighting for change at the federal level, and below I outline why, using my experiences as both a grieving father and a scientist.
“We need more investments from the federal government”
We have all these kids dying every year from DIPG, a disease they call “rare.” But it’s not a rare disease. It’s neglected. I worked on neglected tropical diseases. Why do I call them neglected? Because there’s very little investment in them. But organizations don’t want to hear that. Is malaria neglected? No, because it gets so much money. And then you leave the rest with minimal funding. But the rest affect a lot of people. They’ll say, “There isn’t any money to be made. Why invest in something that only kills 400 people a year?” That’s the rationale we’re working with. And it’s unacceptable. If someone said, “We have two airplanes full of kids, crashing every year and killing 400. Are we going to do something?” Absolutely. They’re going to move everything to change that. But rare diseases? Nope.
Regarding pediatric brain tumors, we need more investments from the federal government to advance new treatments…investments that are consistent and representative of the size of our pediatric population. Roughly 22% of the US population is pediatric (0-18 year-olds). So how come only 4% of funding goes to pediatric research? 96% goes to adult cancers. Why don't pediatric cancers get an amount of money that is at least comparable to its population size? Wouldn't that be [more] fair? And not only that, but the deadliest cancers get almost nothing. I’m not joking when I say that.
This is why I’m now working on the advocacy side of things, on Juliana’s behalf. The DIPG community has a couple of social platforms with over a thousand members worldwide. Specifically, I am a member of the DIPG Advocacy Group, founded by Janet Demeter who also lost her son to DIPG 10 years ago.
The DIPG Advocacy Group has been pushing for Congress to designate a National DIPG Awareness Day. We are currently in our fifth Congress, attempting to get the legislation approved. We’ve been denied multiple times, despite us having the majority vote in favor of it. The Office of Speaker of the House wouldn’t put it on the floor. Why? Her office cited legislation passed in the 90’s, which banned the designation of commemorative dates in the House, due to situations where “the commemorative may only be incidental to the overall purpose of the measure.” We have been told moving forward with this legislation “would break precedent.”
There’s even a pediatrician in Congress, and not even she would sign on as co-sponsor, because of “precedent.” I guess breaking precedent so the deadliest pediatric tumor no one knows about gets a day of awareness is totally out of order.
“We need to make things more logical & not overburden patients who are already suffering”
In 2016, the 21st Century Cures Act was approved, created to help spur innovation in medicine, especially for patients who need treatment options fast. We should be working with that and make things more logical. We need to modernize clinical trials. We need to make it easier to get to clinical trial centers, and easier to communicate with the clinicians, in a way that is not going to be demanding and overburden patients who are already suffering. Will these changes bring immediate cures? Of course not. But they will bring a little more peace of mind and a little more humanity to some of the families that have to go through this.
We can certainly rely on telehealth for clinical trials; several telehealth platforms already exist, and data indicates they can make a significant difference. Consider the pandemic. We quickly adapted and proved telehealth worked well. Why can't we implement some of that here? In February 2020, I was teaching a college course called Emerging Infectious Diseases, and we predicted what would happen with the pandemic. We knew the cases would spike and there’d be a huge rush to hospitals. Telehealth exploded and proved it’s something we could rely on. I wrote a white paper pushing for telehealth, even before the pandemic started…just to show the scientific rationale behind it and to lay the groundwork for what’s possible. It’s been shown that telehealth can increase patients numbers and diversity in a clinical trial, reduce attrition rates, it is more economical, and can be significantly more humane, among other benefits.
In the white paper, one of the major caveats I mention - something that I’ll admit is over my head - is the issue related to state rights and how we do licensing for clinicians. Because it all varies state to state. But again, using the pandemic as an example, the FDA allowed for universal licensing to go across the country. So, it's something that can definitely be worked out. The FDA has the ability to waive the licensing issue for clinical trials, especially in situations where the disease is terminal and there are no other options. Further, years ago Congress passed legislation on the patient’s right to try: a right for patients - particularly those inflicted by terminal diseases - to try drugs that their physician feels can benefit them, even if the treatment hasn’t been proven.
Another issue to address is drug pricing and insurance approvals. In Juliana’s case, among the various drugs we tried to reduce the effects of the inflammation caused by the tumor, and to try to slow down the tumor's advance, two drugs in particular each reaching very close to $30 thousand dollars per a 30-day treatment regimen. In other words, that’s basically $1k per pill. This is not trivial in terms of money, so obviously insurance denied it. They claimed that these drugs hadn’t been tested for pediatric cancer.
To get coverage for these drugs, I needed to write an appeal to the insurance company. Neither the hospital staff nor the clinical staff treating the patient are not allowed to write appeals; it has to come from the individual or their relatives/caregiver. So, I thought, this is what I’ve done all my life as a scientist, write papers to describe the work. Prior to publishing their work, scientists go through a peer-review process in which other scientists review and critique what has been submitted. There is often a back-and-forth regarding the data. Once all the details are cleared, then a paper can be accepted for publication and life continues. Here, I figured I’d be facing a similar situation, which I had to overcome with a combination of a personal justification backed by hard data.
I went ahead and after a couple of days of research and writing, I submitted a five-page appeals letter to insurance, outlining the rationale for why the drugs we requested for Juliana should be covered. The appeals letter was almost immediately approved and Juliana received the medications. To my surprise, the hospital even asked if my letter could be used as a template.
“We don’t have a true health care system. It’s disease care.”
Our healthcare system as a whole needs to shift. Currently, for any treatment, the approach is hierarchical and individualistic. A leader is appointed and they make the decisions. Their voice is the rule. This has become normal. But it’s just wrong. And this idea of the U.S. “healthcare” system. It’s actually an oxymoron. We don’t have a true health care system. It's disease care/management. Because drugs to treat disease make money. The FDA has approved drugs that show no clinical benefit to patients. I've seen some of the data…it's kind of ridiculous. But they benefit drug companies, because they make them money. And they can charge whatever they want. As an example, a drug to treat a particular vector-borne parasitic disease costs $50 overseas and around $50k in the US. This is the exact same drug here and elsewhere. It is the only FDA-approved drug for this particular disease. But if you happen to travel overseas, say to Costa Rica, and come back with the disease and would like to use this drug, be ready to fork over 1000 times what your friends in France, India, Brazil would pay for the same drug!
We need to change the paradigm with what’s called “systems medicine.” It’s a team-based approach. Folks in the lab studying the data. Nurses observing the patients and communicating that back to the team. It’s an environment where everyone’s thoughts are valid, and everyone listens. It’s a collaborative approach to try to understand the underlying issue of each individual. We have the capabilities to predict disease… to establish the mechanisms that will potentially reduce or eliminate infection or exposure. So instead of just treating the disease, you really approach it at all angles to try to get to the root of it.
We got lucky that Juliana's primary clinician was collaborative with me. I told her from the beginning, you're the clinician - you've seen these patients before - and I have not. But I'm going to throw some ideas at you. I'm going to ask you questions based upon my understanding of immunology and microbiology. If I'm wrong, please tell me, but if I'm right, let's continue the conversation. She was open to that dialogue, which was great, because we both knew that Juliana's condition was terminal. There were no clear options.
“Clinical trials need to provide more support”
At least I've been in this business for 30 years. So, I know how and where to look for information…how to navigate things, even if I don’t have all the details. But even I still had to fight an uphill battle. So can you imagine the people who don’t have the same experience as me, having to do things like write an appeal letter to their insurance company? That's where the clinical trials need to provide more support.
For a deeper dive into the critical need for modernizing clinical trials, you can read my white paper here.
Representation Matters
Increasing diversity in clinical trials builds trust, promotes health equity, and leads to more effective treatments and better outcomes (NEJM). But there is much work to be done - and barriers to break - to improve awareness and access for all people.
Do you know someone who is a member of a marginalized community who has participated in a clinical trial? If so, we’d love to meet them and share their story. We hope to represent the many faces of clinical trials through this project, and inspire others by shining a light on their experience.
They can contact us here.
