In the field of cancer research, solid tumors pose an especially difficult challenge compared to blood-borne cancers like leukemia. In most solid tumors, the proteins driving cancer growth are buried far inside the nuclei of their cells. Because they are so difficult to reach, these targets have been considered “non-druggable.” The result is a bleak reality: Current treatments fail for many children with solid tumors.
But the outlook for solid tumor treatments is changing, thanks to breakthroughs pioneered by Children's Hospital of Philadelphia researchers.
Led by CHOP physician-scientists John Maris, MD, and Yael Mossé, MD, the research team has developed ways to reach these buried targets with a new class of immunotherapies. Funded by a $1 million grant from the Children’s Cancer Research Fund (CCRF), Dr. Maris will soon begin conducting clinical trials to test the new therapy’s effectiveness.
Expanding on immunotherapies
When it comes to treating blood cancers, the past 15 years have brought about game-changing advancements. For the treatment known as CAR T-cell therapy, a patient’s T cells — a type of white blood cell — are re-engineered so they can find and destroy cancer cells. In 2012, CHOP treated the first-ever pediatric leukemia patient using CAR T-cell therapy, and that patient, Emily Whitehead, remains cancer-free to this day.
CAR T-cell therapy is effective for blood cancers because the targets exist on the surface of the blood cancer cells. But no one could figure out how to get this type of therapy inside a solid tumor — until now.
‘An enormous opportunity’
The first type of solid tumor the CHOP team will study is a childhood cancer called neuroblastoma, which forms in nerve cells and often comes back, or relapses, after treatment. “We think that this new immunotherapy can potentially provide curative therapy for relapsed neuroblastoma, which is currently not curable,” Dr. Maris explains.
The goal is that the initial findings in neuroblastoma will extend to other cancers, such as brain cancers, bone tumors and more. Dr. Maris sees great potential: “There is an enormous opportunity to replace our existing standards of care with more precise, potently effective and safer treatments harnessing the body’s own immune system.”
Having a philanthropic partner offer support for these types of early-stage trials accelerates discoveries. “Children’s Cancer Research Fund is committed to funding doctors and scientists who bring powerful impact in the treatment and cure of childhood cancers,” says Elizabeth Crippen Allen, CCRF’s Chief Executive Officer. “We are proud to support Dr. Maris’ clinical trials, bringing hope to families of children facing a relapse of neuroblastoma. We believe, together, we can make hope bigger than heartbreak.”
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In the field of cancer research, solid tumors pose an especially difficult challenge compared to blood-borne cancers like leukemia. In most solid tumors, the proteins driving cancer growth are buried far inside the nuclei of their cells. Because they are so difficult to reach, these targets have been considered “non-druggable.” The result is a bleak reality: Current treatments fail for many children with solid tumors.
But the outlook for solid tumor treatments is changing, thanks to breakthroughs pioneered by Children's Hospital of Philadelphia researchers.
Led by CHOP physician-scientists John Maris, MD, and Yael Mossé, MD, the research team has developed ways to reach these buried targets with a new class of immunotherapies. Funded by a $1 million grant from the Children’s Cancer Research Fund (CCRF), Dr. Maris will soon begin conducting clinical trials to test the new therapy’s effectiveness.
Expanding on immunotherapies
When it comes to treating blood cancers, the past 15 years have brought about game-changing advancements. For the treatment known as CAR T-cell therapy, a patient’s T cells — a type of white blood cell — are re-engineered so they can find and destroy cancer cells. In 2012, CHOP treated the first-ever pediatric leukemia patient using CAR T-cell therapy, and that patient, Emily Whitehead, remains cancer-free to this day.
CAR T-cell therapy is effective for blood cancers because the targets exist on the surface of the blood cancer cells. But no one could figure out how to get this type of therapy inside a solid tumor — until now.
‘An enormous opportunity’
The first type of solid tumor the CHOP team will study is a childhood cancer called neuroblastoma, which forms in nerve cells and often comes back, or relapses, after treatment. “We think that this new immunotherapy can potentially provide curative therapy for relapsed neuroblastoma, which is currently not curable,” Dr. Maris explains.
The goal is that the initial findings in neuroblastoma will extend to other cancers, such as brain cancers, bone tumors and more. Dr. Maris sees great potential: “There is an enormous opportunity to replace our existing standards of care with more precise, potently effective and safer treatments harnessing the body’s own immune system.”
Having a philanthropic partner offer support for these types of early-stage trials accelerates discoveries. “Children’s Cancer Research Fund is committed to funding doctors and scientists who bring powerful impact in the treatment and cure of childhood cancers,” says Elizabeth Crippen Allen, CCRF’s Chief Executive Officer. “We are proud to support Dr. Maris’ clinical trials, bringing hope to families of children facing a relapse of neuroblastoma. We believe, together, we can make hope bigger than heartbreak.”
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