Rare cancers collectively make up 15% of all pediatric cancer diagnoses. These types of cancer can range from lung cancer (common in adults but rare in kids), thyroid cancer (one of the most common rare tumors that just hasn’t been studied), and other tumors that only affect a small number of kids each year. Many of these rare cancers have never had a research study focused specifically on them. 

The lack of research data doesn’t mean progress has stalled. Instead of relying on single institution studies, which often struggle to enroll enough kids,  scientists studying rare pediatric cancers are taking different approaches based on collaboration. By pooling patients and data across institutions, they can run meaningful clinical trials and work toward ensuring children with rare tumors experience the same high cure rates seen in more common childhood cancers.

When a child with a diagnosis of a rare pediatric cancer comes to doctors like Dr. Theodore Laetsch, collaboration is often the first step. He frequently turns to his colleagues for guidance — what he calls a simple but essential rule: call your friends. 

“If you study very rare tumors, make friends,” said Dr. Laetsch, who chairs the Children’s Oncology Group rare tumor committee and leads the ALSF Centers of Excellence Grant at Children’s Hospital of Philadelphia (CHOP).

The Centers of Excellence program (COE) supports pediatric cancer clinical trials, pediatric drug development, and — through its Scholars program — the training of scientists in clinical trial design. There are four Centers of Excellence: CHOP, Texas Children’s Cancer Center at Baylor College of Medicine, the University of California San Francisco, and Dana-Farber Cancer Institute. Together, these centers run collaborative trials across their institutions and at partner sites nationwide — and even globally. With ALSF’s broad support, they can offer clinical trial options to children facing all types of pediatric cancer, including rare cancers that have historically received far less research attention.

In 2024 alone, the COE locations sponsored 24 collaborative and scholar-led trials in 319 locations throughout the United States, Canada and Australia. Funding from foundations like ALSF is especially critical because traditional funding sources can be difficult to secure for rare childhood cancers. And that collaboration combined with funding from ALSF doesn’t just change research outcomes — it changes lives. 

Lakelynn was just 3 years old when she was diagnosed a rare sarcoma tumor in her arm. The tumor was inoperable. Radiation was too dangerous. There was not a standard chemotherapy protocol, but there was a clinical trial open through the COE program for a drug that could treat a genetic mutation in her tumor — the NTRK fusion. Lakelynn joined the trial and it worked. Today Lakelynn is a busy pre-teen. And the drug she received, Larotrectinib, was approved by the FDA for other kids with tumors that have the same mutation. The same drug is now used in the standard treatment for babies with another rare cancer, infantile fibrosarcoma, providing a safer and effective alternative to chemotherapy. 

Prior to the first ALSF grant in 2005, only 18 drugs were approved for pediatric oncology use.  Now, over 50 drugs, including Larotrectinib, have been approved for the treatment of pediatric cancer. Collaboration has driven meaningful progress, increasing survival rates and reducing toxic side effects. But significant gaps remain — especially for children with rare cancers.

“We’ve come a long way. Today, more than 80% of children with cancer are cured. But that progress has not extended as far for patients with rare cancers, where outcomes are often worse,” said Dr. Laetsch.

Support Childhood Cancer Research 
The ALSF Grant Program funds research every step of the way—from early-stage innovative research all the way through to life-saving clinical trials for kids with cancer. ALSF’s initiatives are having an impact on research worldwide.  ALSF is also one of the only childhood cancer research organizations that has been given the NCI peer-reviewed funder designation for rigorous selection of research grants. You can help carry on the mission of helping kids beat cancer through research by joining the One Cup at a Time Club, a community of generous people who give what they can every month to help cure childhood cancer.

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