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Maryam Fouladi, MD, remembers the moment she decided to devote her career to pediatric neuro-oncology. It happened during her second year of residency, while visiting a young patient with a rare brain cancer she had never heard of. A mentor had invited her to join him as he delivered news that no parent or child ever wants to hear. The 5-year-old girl’s tumor was incurable.
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“I couldn’t believe this was the state of the field — that there was nothing we could do for this child,” recalls Dr. Fouladi, Chair of COG’s Central Nervous System (CNS) Tumor Committee. “I remember thinking, ‘If you’re going to dedicate your life to doing something, this is it — make things better for patients like this.’”
Dr. Fouladi has seen remarkable strides in research and treatment of CNS cancers over the past few decades. Survival rates for the most common pediatric brain cancer have increased to 70–80%. Rare CNS cancers, however, still claim far too many young lives. We met with Dr. Fouladi to learn about COG’s leadership in CNS research and the critical role philanthropy plays in groundbreaking discoveries that give more young people with cancer a better chance to live long, healthy lives.
What are some advances that COG’s CNS Committee has worked on?
For a long time, it was difficult to determine exactly what type of brain tumor a child had and what treatment plan would be their best option. COG’s Molecular Characterization Initiative is a major advance that enabled scientists to figure out what subtype of tumor patients had, upfront. Now, molecular subtyping gives us a really comprehensive, integrated diagnosis that allows us to tailor therapy with much greater specificity. This way, we can give children exactly the treatment they need.
What are some studies that have significantly improved treatments and outcomes for kids with central nervous system cancers?
Our studies on high-risk medulloblastoma showed that adding a drug called carboplatin to radiation increased survival in a very specific molecular subtype. It didn't help other groups, but it helped this particular group by leaps and bounds. We can now recognize which subgroups of patients do well with lower doses of radiation. We can also identify which groups need more therapy to improve their survival.
Dr. Fouladi has seen remarkable strides in research and treatment of CNS cancers over the past few decades. Survival rates for the most common pediatric brain cancer have increased to 70–80%. Rare CNS cancers, however, still claim far too many young lives. We met with Dr. Fouladi to learn about COG’s leadership in CNS research and the critical role philanthropy plays in groundbreaking discoveries that give more young people with cancer a better chance to live long, healthy lives.
What are some advances that COG’s CNS Committee has worked on?
For a long time, it was difficult to determine exactly what type of brain tumor a child had and what treatment plan would be their best option. COG’s Molecular Characterization Initiative is a major advance that enabled scientists to figure out what subtype of tumor patients had, upfront. Now, molecular subtyping gives us a really comprehensive, integrated diagnosis that allows us to tailor therapy with much greater specificity. This way, we can give children exactly the treatment they need.
What are some studies that have significantly improved treatments and outcomes for kids with central nervous system cancers?
Our studies on high-risk medulloblastoma showed that adding a drug called carboplatin to radiation increased survival in a very specific molecular subtype. It didn't help other groups, but it helped this particular group by leaps and bounds. We can now recognize which subgroups of patients do well with lower doses of radiation. We can also identify which groups need more therapy to improve their survival.
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Another successful trial improved the standard of care for atypical teratoid/rhabdoid tumor (AT/RT). Kids with this type of cancer historically had a very dismal prognosis. In our AT/RT study, patients who received a two-part chemotherapy treatment ended up having a much better outcome. That outcome is still sitting in the 40% range. It's much better than prior, but it's still not good enough.
What is a current study that you’re excited about? There’s a large-scale COG study of low-grade gliomas, the most common brain tumor in children. We’ve randomized the study between conventional chemotherapy and a more targeted therapy that may be less toxic. With this study and others, we are focusing more on personalized therapy and making sure we have an integrated diagnosis with a molecular understanding of a specific patient's tumor and trying to really deliver therapy for that particular patient. |
What are the committee's top priorities for the next five years?
We’re always future-focused at COG, striving to strike the right balance between large groups of patients who need therapy and smaller groups with rarer tumors. We can decrease therapy to keep the same great outcomes for the low-risk groups while minimizing long-term toxicities. Or we can intensify treatment for higher-risk groups with targeted therapies that are less toxic, or additional chemotherapy or immunotherapy, or different ways of delivering therapy, such as directly injecting drugs into the fluid around the brain and spinal cord. We are always trying to treat as many patients as possible close to home, so they don't have to travel far to specialized centers to get novel therapies.
COG has been a great equalizer in that regard. The beauty of COG is our ability to not only enroll large numbers of patients in trials for the more common tumors, but also those with rarer tumors, because we have over 200 research sites. This allows us to enroll the critical masses of patients needed to get definitive answers leading to potentially paradigm-shifting therapies.
We’re always future-focused at COG, striving to strike the right balance between large groups of patients who need therapy and smaller groups with rarer tumors. We can decrease therapy to keep the same great outcomes for the low-risk groups while minimizing long-term toxicities. Or we can intensify treatment for higher-risk groups with targeted therapies that are less toxic, or additional chemotherapy or immunotherapy, or different ways of delivering therapy, such as directly injecting drugs into the fluid around the brain and spinal cord. We are always trying to treat as many patients as possible close to home, so they don't have to travel far to specialized centers to get novel therapies.
COG has been a great equalizer in that regard. The beauty of COG is our ability to not only enroll large numbers of patients in trials for the more common tumors, but also those with rarer tumors, because we have over 200 research sites. This allows us to enroll the critical masses of patients needed to get definitive answers leading to potentially paradigm-shifting therapies.
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How does philanthropy support the committee's work?
Philanthropy is critical. Only about 4% of National Cancer Institute (NCI) funds go to any kind of pediatric cancer research. None of us who have ideas could get this done without philanthropic support. That’s as true for researchers studying basic science as it is for those doing translational studies, turning discoveries into novel treatments. We’re very lucky at COG to have the support of the NCI, which allows us to have this infrastructure. This is very different from most other countries in the world, where that kind of national and federal support doesn't exist. |
“Recent compelling research has suggested that using two chemotherapies in combination could potentially improve outcomes in patients with a particularly high-risk form of ependymoma, a type of brain tumor that makes up roughly 5% of pediatric brain tumor diagnoses each year. Because this drug combination is not yet proven, insurance will not pay for it. Since no other proven therapy currently exists to help patients with this high-risk, rare tumor, philanthropic support for COG is the only way to assess this drug combination while sparing patient families from bearing the burden of the cost of these drugs.” - Adam Esbenshade, MD, COG CNS Committee Member |
What are some hard-to-fund needs that additional donor support could help meet?
It’s always very challenging to get funding for studies to reduce long-term health issues that can compromise learning, movement, eyesight, and the like for CNS cancer survivors. These are the things families talk about. Yes, it's really important to cure my child. It’s also important to make sure that my child can succeed at school, can grow up normally, can socialize, can have good hearing, good vision. It’s critical that we capture the data and assess what our treatments are doing to patients, so we can find better ways of improving care.
Learn more in Q&As with other COG Committee Chairs.
It’s always very challenging to get funding for studies to reduce long-term health issues that can compromise learning, movement, eyesight, and the like for CNS cancer survivors. These are the things families talk about. Yes, it's really important to cure my child. It’s also important to make sure that my child can succeed at school, can grow up normally, can socialize, can have good hearing, good vision. It’s critical that we capture the data and assess what our treatments are doing to patients, so we can find better ways of improving care.
Learn more in Q&As with other COG Committee Chairs.