アデノ随伴ウイルスベクターを用いた遺伝子治療の臨床実施体制に関する全国調査 ―脊髄性筋萎縮症に対する遺伝子治療の実態把握―

抄録

Onasemnogene abeparvovec-xioi (OA) is the first approved gene therapy product for spinal muscular atrophy (SMA) in Japan. Since the product is a vector derived from adeno-associated virus, however, the clinical use categorized as in vivo gene therapy must be under the type I use of the Cartagena Act to preserve environmental biodiversity. On the other hand, very few health care providers have had experiences of dealing with such as a product and the challenges in clinical sites have still been unknown. To understand the current clinical situation of gene therapy for SMA, we had conducted a nation-wide survey for 41 medical institutions that have used or plan to use the product. Among them, 33 institutes responded to the questionnaire and 21 have provided the gene therapy for the patients. Although each facility conducted the gene therapy by using its own protocols for personal protective equipment, inactivation or disposal of the vector, and patient care, 73％ of the facilities took more than a month to introduce the gene therapy into their hospitals and 82％ found it difficult to establish the implementation system complied with the Cartagena Act. The survey indicated the importance of promptly preparing the standardization of procedure dealing with virus-derived medical products including education materials for health care providers for safe and effective gene therapy nation-wide.