Seoul National University Hospital (SNUH) said regulatory authorities have approved the hospital’s clinical research plan for CAR-T treatment for acute lymphoblastic leukemia in children and adolescents.
Seoul National University Hospital has begun research to produce CAR-T treatment as a new treatment option for pediatric leukemia patients.
The approval comes eight months after the hospital submitted such clinical trial plans in April. It is also the first time that regulators have given the green light to produce and conduct clinical research on a proprietary CAR-T treatment for a Korean hospital.
The hospital said it plans to initiate a large-scale clinical trial targeting young patients with relapsed and refractory acute lymphoblastic leukemia.
Treatment outcome for acute lymphoblastic leukemia in children and adolescents has steadily improved, but outcomes are still poor in relapsed or refractory patients, SNUH said. Some global pharmaceutical companies have developed Chimeric antigen receptor-T (CAR-T) treatments to provide such patients with a better treatment option.
CAR-T treatment shows a different treatment method than existing drugs. Unlike conventional pharmaceuticals, CAR-T collects T cells in a patient’s white blood cells in a hospital and then 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 sends them back to the hospital.
The treatment has recently attracted attention as a breakthrough in finding only cancer cells and precisely targeting them 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 is likely to 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 since the number of patients is not small.
However, under SNUH’s clinical trial plans, patients get the treatment for free.
The hospital also stressed that it would drastically reduce production time.
While the existing CAR-T treatment took an average of three weeks before the patient’s T cells were sent abroad for proliferation and returned to them for reinjection, this can reduce the production time to 12 days as the hospital manufactures the treatments . House.
The hospital expects children with acute lymphoblastic leukemia, who struggle with time, to be treated quickly.
“I am pleased that regulators have approved the research plan after much deliberation,” said Professor Kang Hyung-jin of the Department of Pediatrics. “Because it was the first study of its kind in Korea, it took a long time to get approval, and I feel sorry for the kids who couldn’t get treatment in the meantime.”
The hospital hopes this study will provide hope for children with childhood and adolescent leukemia in Korea who have not received CAR-T treatment because of the cost, Kang added.
The hospital also plans to establish the so-called CAR-T development one-stop system through this study by integrating the processes of the preclinical laboratory, good manufacturing practice (GMP) manufacturing plant and clinical research center in the hospital. unite, and develop CAR. -T treatment for other diseases as well.