香港兒童醫院於2023 年11 月，為一名十個月大的 脊髓肌肉萎縮症（SMA）男嬰進行了香港史上首宗 基因療法。 SMA 患者因基因缺陷，無法產生足夠功能的蛋白 質，導致行動、呼吸及吞嚥能力持續衰退。一次 性的基因療法能提高病童存活率，擺脫終身使用藥 物、輪椅和呼吸機，帶來新希望。本院為這重要個 案作了全面準備，超過30 人的跨專業團隊曾接受 培訓，確保所有硬件和流程符合規格。 In November 2023, HKCH administered the first gene therapy in Hong Kong history to a 10-monthold boy with spinal muscular atrophy (SMA). SMA patients are unable to produce enough functioning proteins due to genetic defects, resulting in progressive loss of movement, breathing and swallowing abilities. This one-time gene therapy brings new hope to SMA children by improving their survival rate, and freeing them from lifelong medications, wheelchair and ventilator. HKCH was well prepared for this milestone case. A multidisciplinary team of more than 30 professionals had received training to make sure all hardware and workflows meet the required standards. 父母分享感受 Parents’ sharing 藥劑部人員在無菌環境下調配藥物。基因療法將帶有相關基因的無 害病毒載體注入病人體內，彌補先天不足，從而控制病情。 Aseptic preparation of the drug. The gene therapy involves injecting a harmless viral vector carrying the corresponding genes into the patient’s body to compensate for congenital deficiencies. 醫護人員正為男嬰輸注 能改寫其一生的基因治 療藥物。父母感激醫管 局及主診醫生促成治 療，及醫護團隊的悉心 照料。 The baby boy receiving the life-changing gene therapy infusion. His parents expressed gratitude towards HA and the doctor in charge for introducing the new therapy, and the clinical team for their good care. 本院是醫管局其中一間可提供「嵌合抗原受體T 細 胞」（CAR-T 細胞）治療的中心，配備血液成份分 離機、超低溫儲存設備、隔離病室等配套和專業團 隊，為患有指定的復發性或難治性急性淋巴白血病 或淋巴癌病童帶來治療曙光。由病人自身提取的免 疫T 細胞被送到海外進行基因改造後，再運回本院 輸到病人體內，它們便能辨別癌細胞，將之消滅。 HKCH is one of the centres in HA that provide chimeric antigen receptor T-cell (CAR-T cell) therapy. Designated teams and related apheresis, cryopreservation and isolation facilities are in place to serve patients with specified refractory or relapsed acute lymphoblastic leukaemia or lymphoma. T cells harvested from a patient are sent abroad for genetic modification. They are then transported back to HKCH and re-infused into the patient to identify and eliminate the cancer cells. CAR-T 細胞治療 為血癌病童闢新路 CAR-T Cell Therapy Opens New Door 一名病童在完成CAR-T 細胞 輸注後，與醫護人員合照。 A patient took a photo with his care team after receiving CAR-T cells infusion. 突破性基因療法 將不可能變可能 Gene Therapy Makes the Impossible Possible 51 Hong Kong Children’s Hospital 5th Anniversary Commemorative Album 香港兒童醫院 五周年紀念特刊 追求卓越 Pursuit of Excellence
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