NCI Highlights Blinatumomab and Atezolizumab Advances as Childhood Cancer Data Initiative Scales Up
核心洞察
An NCI-supported Children's Oncology Group phase 3 trial found adding blinatumomab to chemotherapy substantially improved disease-free survival in children newly diagnosed with B-cell acute lymphoblastic leukemia (搜索).
An NCI-led trial of about 50 patients supported FDA approval of atezolizumab for adults and children aged 2 and older with advanced alveolar soft part sarcoma (搜索).
NCI's Childhood Cancer Data Initiative provides no-cost tumor characterization at diagnosis, returning clinically relevant results to clinicians within 2-3 weeks.
Adding the immunotherapy blinatumomab to chemotherapy substantially improved disease-free survival in children newly diagnosed with B-cell acute lymphoblastic leukemia (搜索) (B-ALL), largely by reducing relapses, according to an NCI-supported Children's Oncology Group phase 3 trial. The result is already changing initial treatment for many children with the disease, NCI's Dr. Brigitte Widemann said in a Q&A marking Childhood Cancer Awareness Month.
A second advance cited by Widemann centers on atezolizumab for alveolar soft part sarcoma (搜索), an extremely rare cancer for which there had been no approved treatment. An NCI-led trial enrolled about 50 patients, including children treated through NCI's intramural program, and led the FDA to approve the drug for adults and children ages 2 and older with advanced disease. Widemann described the two examples as evidence of the complementary roles of large cooperative-group trials and NIH Clinical Center studies of cancers too rare to investigate easily elsewhere.
A Data Ecosystem for Rare Cancers
NCI launched the Childhood Cancer Data Initiative (CCDI) in 2019 to build a data ecosystem intended to accelerate how childhood cancers are understood and treated. Because these cancers are rare, Widemann said, collaboration and data sharing are necessary "if we hope to learn from every child and ultimately benefit every child." She added that a successful model could provide a blueprint not only for childhood cancer but for cancer research and treatment more broadly.
A major component is the CCDI Molecular Characterization Initiative (MCI), developed with the Children's Oncology Group. MCI provides eligible children, adolescents, and young adults treated at COG-affiliated hospitals with no-cost, state-of-the-art tumor characterization at diagnosis, returning clinically relevant results to participants and their clinicians within 2-3 weeks while making deidentified data available for future research in near real time. According to Widemann, testing across thousands of participants has shown that this characterization can refine, and sometimes change, a diagnosis, inform precision treatment, and help identify eligibility for clinical trials. The initiative is strengthened by complementary efforts supported through the Childhood Cancer Survivorship, Treatment, Access, and Research (STAR) Act and its reauthorization.
The 2026 CCDI Symposium is scheduled for September 18, convening patients, families, advocates, clinicians, researchers, data scientists, industry, and federal partners. Widemann said she expects it to produce practical ideas for using CCDI's data and resources, including AI approaches to important questions in pediatric cancer.
AI, Target Discovery, and Earlier Detection
Widemann identified target identification, drug discovery and development, and survivorship and quality of life as areas where artificial intelligence could make the greatest difference. CCDI is generating the high-quality, connected data that can make AI useful; the Molecular Characterization Initiative, for example, includes digitized pathology slides. By learning from thousands of images derived from childhood cancer patients, AI may eventually help pathologists make more precise diagnoses or predict molecular features from a routinely stained slide — a capability she said could be especially valuable where comprehensive molecular testing is not readily available.
Building on CCDI, the Bio Genesis Mission includes a challenge pillar focused on childhood cancer that will leverage AI to integrate multimodal data, accelerate biomarker and therapeutic target discovery, improve precision medicine and clinical trial design, and speed development of new treatments. AI could also help analyze imaging data to detect tumors earlier, particularly in children with an inherited predisposition to cancer, and match patients with appropriate clinical trials. Widemann noted that NCI brings scientific and clinical expertise, research networks, and pediatric cancer data, while the Department of Energy (搜索) brings advanced AI and computing expertise — capabilities neither organization could deploy alone.
Sustained Investment and the Rationale for Federal Support
Widemann framed sustained federal investment as essential because all childhood cancers are rare, with some diagnoses occurring in only 40 or 50 children in the United States each year. That rarity makes it difficult to build expertise, understand the biology, identify patients, and conduct clinical trials, while commercial incentives to develop treatments are smaller than for common adult cancers. NCI therefore has what she described as a unique role in supporting research to identify promising therapeutic targets and translate discoveries into new treatments for children.
She also cautioned that childhood cancers are often biologically different from most adult cancers, so a drug that works in adults cannot be assumed to work in children. Progress from basic discovery to clinical application can take years, and survivorship questions require decades of follow-up — studies such as the NCI-supported Childhood Cancer Survivor Study are possible only with strong, sustained public support. NCI's research portfolio spans large clinical trials networks including the Children's Oncology Group, the Pediatric Early Phase Clinical Trials Network (PEP-CTN), the Pediatric Immunotherapy Network (PIN), and the My Pediatric and Adult Rare Tumor (myPART) network, which focuses on rare pediatric and young adult tumors. Additional initiatives such as the Fusion Oncoproteins in Childhood Cancers Consortium and the global Cancer Grand Challenges effort bring researchers together across institutions and countries.
Access, Survivorship, and Long-Term Follow-Up
The pediatric cancer community is highly connected, largely through the Children's Oncology Group and its network of more than 200 institutions, which work with primary care providers to connect children and families to pediatric oncologists as close to home as possible or where the most appropriate expertise exists. NCI is also supporting pediatric sites within the NCI Community Oncology Research Network (NCORP) to expand trial reach and awareness. Gaps remain, particularly for adolescents and young adults, for whom insurance transitions and the divide between pediatric and adult care can make specialized care and trial participation harder to access. Telehealth, virtual molecular tumor boards connecting experts across the country, and decentralized clinical trials are among the approaches Widemann cited to reduce practical and geographic burdens of participation.
As treatments improve, more children will live for decades after cancer, shifting attention to physical toxicities as well as the impact of childhood cancer on mental health, fertility, employment, relationships, and access to appropriate follow-up care in adulthood. Widemann said the long-term effects of newer treatments, including immunotherapies, remain poorly understood and may differ substantially from side effects of past therapies. Through the STAR Act and STAR Reauthorization Act, NCI supports projects studying the development of chronic health conditions among childhood and AYA cancer survivors and the development of subsequent cancers. Results from the Childhood Cancer Survivor Study continuously inform COG's Long-Term Follow-Up Guidelines for Survivors of Childhood, Adolescent and Young Adult Cancers.
"Our goal cannot be limited to eliminating the tumor," Widemann said, adding that the objective is for children not only to survive cancer but to live long, healthy, and fulfilling lives through quality of life, psychosocial support, and lifelong care.
Prevention, Liquid Biomarkers, and Community Momentum
Looking forward, Widemann said she is hopeful for earlier diagnosis and treatments that are more precise, less toxic, and better tailored to each child's tumor, as well as the possibility of cancer prevention and earlier detection. A meaningful proportion of children with cancer have an inherited predisposition, she noted; identifying that risk may help monitor patients and families more carefully, reveal new prevention strategies for families at risk, and find tumors when they are smaller and easier to treat. Liquid biomarkers represent another promising area, with a blood sample potentially indicating whether a tumor is present, shrinking, or growing — which could reduce the need for invasive tumor biopsies and allow more precise disease monitoring.
The themes were echoed at a separate event, "Advancing Hope: The Future of Pediatric Cancer Research," convened by The Children's Inn at NIH (搜索) in partnership with NCI, featuring panelists Dr. Anthony Letai, Dr. Bridgitte Widermann, and Dr. Jack Shern with moderator Brian Kelly. In a LinkedIn post, NCI pointed to advances in precision medicine and immunotherapy and the growing potential of data and AI to accelerate discovery, while noting that much work remains to improve outcomes for every child and young person facing cancer.