Cancer Currents Highlights: Recent Payoffs in Childhood Cancer Research
National Cancer Institute (NCI)
The National Cancer Institute, part of the National Institutes of Health, is the lead federal agency for cancer research
In his Pulitzer Prize-winning book The Emperor of All Maladies, Siddhartha Mukherjee recounts the story of a team of NCI scientists—led by “The Emils,” Emil Freireich and Emil Frei—as they ran clinical trials testing combinations of chemotherapy drugs in the hopes of curing children with leukemia.
At that time, in the early 1960s, leukemia was uniformly fatal. Even so, the researchers came under intense scrutiny and criticism for wanting to give sick children multiple drugs—or “poisons,” as some called them—at the same time.
Indeed, the treatments initially did make the children in the trial much sicker. “Then, unexpectedly—at a time when it was almost unbearable to look for it—there was a payoff,” Mukherjee wrote. “The leukemia went into remission. The bone marrow biopsies came back one after another—all without leukemia cells.”
Now, most kids with acute lymphoblastic leukemia, the most common childhood cancer, are cured. And although chemotherapy is still a mainstay of treating children with many forms of cancer, today’s treatment can include cutting-edge medications, such as personalized cell therapies and cleverly designed antibodies that can be stunningly effective.
But scientists aren’t only discovering ways to help children survive cancer. They’re also helping kids go on to live healthy lives by finding ways to limit or avoid the long-term side effects that have plagued many survivors of childhood cancers.
So, in case you missed them the first time around, the Cancer Currents stories below capture some of this progress. We hope you find these stories inspiring and informative.
As always, we welcome your feedback on Cancer Currents . And if you know somebody with an interest in these topics, please feel free to forward them this newsletter.
Carmen Phillips
Managing Editor, Cancer Currents
Tovorafenib Approved for Some Children with Low-Grade Glioma
FDA has granted an accelerated approval to tovorafenib (Ojemda) for kids and teens who have low-grade glioma with changes in the BRAF gene. In a small clinical trial, the drug shrank or completely eliminated tumors in nearly 70% of patients.
Blinatumomab Increases Survival for Infants with an Aggressive Type of ALL
Giving the drug blinatumomab (Blincyto) after standard chemotherapy substantially increased survival for infants with an aggressive form of acute lymphoblastic leukemia (ALL), a recent study showed. If confirmed in larger studies, the treatment may become standard therapy for infants with ALL caused by KMT2A rearrangements.
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Liquid Biopsies on the Horizon for Children with Solid Cancers
Results from a new study highlight the progress being made toward developing liquid biopsies specifically for use in children with solid cancers like Ewing sarcoma and Wilms tumor. The tests can help detect and diagnose cancer and monitor for response to treatment and recurrence.
Dexrazoxane Protects the Heart Long Term in Kids Being Treated for Cancer
Doxorubicin is used to treat many types of childhood cancer, but it can damage the heart. Giving dexrazoxane (Zinecard) before each dose substantially decreases a child’s risk of treatment-related heart problems in adulthood, new study results show.
Drug Combination Shows Promise for Rhabdomyosarcoma, but Can It Get to Clinical Trials
An NCI study in mice has identified a drug combination that may help treat children with rhabdomyosarcoma. But one of the drugs, ganitumab, is no longer being made. Based on the study's promising results, the NCI researchers who led the study want to test the combination in humans.
Sodium Thiosulfate Approved to Reduce Chemo-Related Hearing Loss in Children with Cancer
The chemotherapy cisplatin often causes permanent hearing loss. Sodium thiosulfate (Pedmark) is the first treatment approved by FDA that can reduce the risk of hearing loss and the severity of damage to the inner ear in children treated with cisplatin.
Additional Resources from NCI
To read more news and research updates from the National Cancer Institute , visit the Cancer Currents blog.
GCC Distinguished Cancer Scholar, Professor and Director, Cancer Biology Program, Department of OB/GYN, Morehouse School of Medicine, Atlanta, Ga 30310
3moAs an EwingSarcoma researcher, I support NCI mission!! 🙏
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3moEncouraging news! Keep up the great work National Cancer Institute (NCI)!
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3moThanks for sharing these inspiring updates from Cancer Currents! The approval of tovorafenib (for low-grade glioma) and the use of liquid biopsies for detecting solid cancers are particularly remarkable.