A 10-year project funded by a 300 billion won donation has expanded diagnosis and treatment infrastructure over five years
Seoul National University Hospital establishes Korea's first standard treatment protocol for pediatric acute lymphoblastic leukemia; more than 20 previously unknown rare diseases identified
Research projects grow from 7 to 190; 1,411 medical professionals at 185 institutions nationwide now participating
Seoul National University Hospital's Support Project for Pediatric Cancer and Rare Diseases announced Monday that the Lee Kun-hee Pediatric Cancer and Rare Disease Overcoming Project — launched with a 300 billion won ($221 million) donation from the late Samsung Chairman Lee Kun-hee's family — had diagnosed 22,646 patients and treated 14,757 others through September this year. The project office also held an international symposium that day, bringing together domestic and foreign experts to share five years of research findings and chart the course for future international collaborative research.
The project launched in April 2021, with Seoul National University Children's Hospital designated as the lead institution and the project office formally established the following month. It is scheduled to run for 10 years through 2030. Over the past five years, the project office has developed diagnostic and treatment technologies for pediatric cancers and rare diseases while expanding the infrastructure needed for patient diagnosis and research.
Five-year highlights of the Lee Kun-hee Pediatric Cancer and Rare Disease Overcoming Project
In the field of pediatric cancer, the project produced Korea's first standard treatment protocol for pediatric acute lymphoblastic leukemia. Patients are classified into five risk groups to guide treatment strategy, and the protocol incorporates genomic analysis and next-generation sequencing-based minimal residual disease monitoring to support precision medicine.
In rare diseases, researchers identified and diagnosed more than 20 previously unknown conditions. A nationwide rare disease diagnostic network was established so that regional hospitals can send specimens and genetic information to a central analysis center, which returns results within four to six weeks. Regional hub institutions are also being supported to access advanced genomic testing, and families of pediatric patients with rare genetic diseases receive genetic counseling and prenatal diagnosis support.
More than 60 percent of Korea's pediatric solid tumor patients are now enrolled in STREAM, a precision medicine platform for pediatric solid cancers. The platform has enabled new diagnoses for patients who had previously gone undiagnosed and allowed existing diagnoses to be reclassified for more effective treatment. Seoul National University Hospital has also built an in-house GMP facility capable of manufacturing CAR-T cells on site, and through September this year the project had produced and supplied CAR-T cells to 21 pediatric patients.
The nationwide research collaboration network has grown substantially. The project office has broadened its research base by working with medical institutions across the country through its cancer, rare disease, and joint research divisions. The number of research projects it supports rose from 7 in 2021 to 190 this year. As of September, 1,411 medical professionals at 185 institutions nationwide are participating, and the pediatric cancer and rare disease patient cohort now holds 57,030 registered data entries.
This collaboration is a critical foundation for giving children with hard-to-diagnose and hard-to-treat cancers and rare diseases better access to care. Rare diseases take an average of 9.2 years to diagnose, and about 50 percent of patients are still never diagnosed. There are only 69 pediatric hematology-oncology specialists nationwide, and more than 70 percent of pediatric cancer patients living outside Seoul receive treatment in a region other than their own. The project office connects the capabilities of medical institutions and researchers across the country to widen the diagnostic base and create an environment in which children can receive appropriate diagnosis and treatment regardless of where they live.
The symposium was held under the theme "Opening the Future of Pediatric Precision Medicine: Advanced Research in Pediatric Cancer and Rare Diseases." Eight domestic and foreign experts — including representatives from the International Agency for Research on Cancer and universities in the United States and United Kingdom — participated in two sessions covering genomics-based precision medicine for pediatric cancer, CAR-T therapy development, international cooperation for rare disease diagnosis, precision gene editing, patient-derived organoids, and digital pathology research. About 200 people attended.
"Pediatric cancers and rare diseases present many challenges that individual research institutions cannot solve on their own, given the small number of patients per condition and the wide variety of characteristics involved," said Project Director Chae Jong-hee. "I hope this symposium will serve as an opportunity to share research findings and clinical experience among domestic and foreign experts, strengthen the foundation for international collaborative research, and chart the direction for the next five years."
"The true value of medical research lies in improving patients' lives," said Hospital President Baek Nam-jong. "This project demonstrates the potential for a virtuous cycle in which a meaningful private donation supports medical research, and the results in turn contribute to the diagnosis and treatment of patients."
"The late Chairman Lee Kun-hee's lifelong philosophy of 'respect for human beings' extended to care and love for all lives, and for children in particular," said Jang Seok-hun, president of Samsung's social contribution operations. "It is deeply meaningful to share the research achievements of these past years with researchers from Korea and abroad at today's symposium. I hope this event will create more hope and more treatment opportunities not only for pediatric cancer and rare disease patients in Korea, but for children around the world."
The project office plans to expand its international cooperation network and advance treatment technologies including CAR-T therapy while strengthening a sustainable research foundation. It will also develop programs for early diagnosis and disease prevention — among them rapid genetic diagnosis for critically ill infants, neonatal genomic screening, prenatal diagnosis, and preimplantation genetic testing. The office also intends to translate research findings into clinical practice and health policy to broaden diagnosis and treatment opportunities for pediatric cancer and rare disease patients.
kty@heraldcorp.com
