Stories about: childhood cancer

Probing the brain’s earliest development, with a detour into rare childhood cancers

In early brain development there is an increase in ribosomes, contained in these nucleoli
Nucleoli, the structures in the cell nucleus that manufacture ribosomes, are enlarged in very early brain development, indicating an increase in ribosome production. Here, a 3D reconstruction of individual nucleoli. (Kevin Chau, Boston Children’s Hospital)

In our early days as embryos, before we had brains, we had a neural fold, bathed in amniotic fluid. Sometime in the early-to-mid first trimester, the fold closed to form a tube, capturing some of the fluid inside as cerebrospinal fluid. Only then did our brains begin to form.

In 2015, a team led by Maria Lehtinen, PhD, Kevin Chau, PhD and Hanno Steen, PhD, at Boston Children’s Hospital, showed that the profile of proteins in the fluid changes during this time. They further showed that these proteins “talk” to the neural stem cells that form the brain.

In new research just published in the online journal eLife, Lehtinen and Chau shed more light on this little-known early stage of brain development.

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A bold strategy to enhance CAR T-cell therapies, capable of targeting DIPG and other tough-to-treat cancers

CAR T-cell therapy uses a patient's own genetically modified T cells to attack cancer, as pictured here, where T cells surround a cancer cell.
T cells surround a cancer cell. Credit: National Institutes of Health

A Boston-based team of researchers, made up of scientists and pediatric oncologists, believe a better CAR T-cell therapy is on the horizon.

They say it could treat a range of cancers — including the notorious, universally-fatal childhood brain cancer known as diffuse intrinsic pontine glioma or DIPG — by targeting tumor cells in an exclusive manner that reduces life-threatening side effects (such as off-target toxicities and cytokine release syndrome). The team, led by Carl Novina, MD, PhD, and Mark Kieran, MD, PhD, of the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, calls their approach “small molecule CAR T-cell therapy.”

Their plan is to optimize the ability for CAR T-cell therapies, which use a patient’s genetically modified T cells to combat cancer, to more specifically kill tumor cells without setting off an immune response “storm” known as cytokine release syndrome. The key ingredient is a unique small molecule that greatly enhances the specificity of the tumor targeting component of the therapy.

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Pediatric cancers and precision medicine: The feasibility question

precision cancer medicine personalized medicine pediatric oncology childhood tumors
What is precision cancer medicine all about? See the full infographic at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center.

A family walks into their oncologist’s office and sits down. Their son’s care team is there, ready to discuss the sequencing report they received about the tumor in his leg.

“We think we have something,” the oncologist says. “We found a known cancer-associated mutation in one gene in the tumor. There’s a drug that targets that exact mutation, and other children and adults whose tumors have this mutation have responded well. We’ll have to monitor your son closely, but we think this is a good option.”

This hypothetical conversation, while common in adult oncology, happens rarely (if at all) on the pediatric side. This kind of personalized, genomics-driven medicine (where the genetic alterations in a patient’s tumor drive therapy, not the tumor’s location) isn’t a standard approach for childhood cancers yet.

Note that I said yet. The door to personalized pediatric genomic cancer medicine is cracking open, in part because three recent papers — including one out of Dana-Farber/Boston Children’s Cancer and Blood Disorders Center — are starting to convince the field that clinical genomics can indeed be done in pediatric oncology.

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