SNUH begins research on self-produced CAR-T treatment for pediatric leukemia

Researchers from Seoul National University Hospital (SNUH) have submitted to the Ministry of Health and Welfare a clinical research plan for CAR-T treatment targeting acute lymphocytic leukemia in children and adolescents.

Seoul National University Hospital has begun research into producing CAR-T treatments to provide new treatment options for pediatric leukemia patients. (SNUH)

If approved, children and adolescents with relapsed and refractory acute lymphocytic leukemia can participate in a clinical trial and receive free CAR-T produced by the SNUH team led by Professor Kim Hyo-soo.

The team also plans to establish the so-called CAR-T one-stop development system by uniting the processes of the preclinical laboratory, good manufacturing practice (GMP) plant and clinical trial center in the hospital through this study.

“I hope this study will give hope to pediatric and adolescent leukemia patients who were unable to receive CAR-T treatment because of the high cost,” said Professor Kim. “We will strive for practical results by leveraging the hospital’s capabilities.”

Treatment outcome for acute lymphocytic leukemia in children and adolescents has steadily improved, but results are still poor in relapsed or refractory patients. To provide such patients with a better treatment option, some global pharmaceutical companies have developed CAR-T (Chimeric antigen receptor-T) treatments.

CAR-T treatment shows a different treatment method than existing medicines. Unlike conventional pharmaceuticals, CAR-T collects T cells in a patient’s white blood cells in a hospital, freezes them, and sends them to a manufacturing facility. After expressing the chimeric antigen receptor (CAR) that recognizes cancer cells in T cells, the facility grows the cells and returns them to the hospital.

The treatment has recently gained attention as a breakthrough by only finding and accurately targeting cancer cells, while minimizing damage to normal cells in the body.

Novartis Korea recently received approval for Kymriah, the CAR-T treatment, from the Ministry of Food and Drug Safety and plans to introduce the drug soon. However, the first CAR-T treatment in Korea will likely become the most expensive drug approved in the country, with a price tag of 500 million won ($ 440,683), making it difficult for patients to access.

Therefore, some doctors have expressed concern about the drug’s high price tag, stressing that the drug would burden the country with significantly higher expenditures than before, given that the number of patients is not small.

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