FDA核准第1型糖尿病幹細胞治療孤兒藥
作者：Yael Waknine
出處：WebMD醫學新聞
May 7, 2010 — 美國食品藥物管理局(FDA)以孤兒藥方式核准一種幹細胞治療(商品名Prochymal；Osiris Therapeutics公司)，用於新診斷為第1型糖尿病的患者。
這項產品是靜脈注射型的成人間葉幹細胞(mesenchymal stem cells，MSCs)，從健康年輕捐贈者的骨髓分離，因此避免胚胎和胎兒細胞來源的爭議，細胞培養也使得可以從單一捐贈者大量製造數千次的劑量。
根據藥廠新聞稿，MSCs可藉由控制發炎、促進組織新生、預防疤痕形成而有治療助益。臨床前研究指出，MSCs可以藉由保留β細胞功能而延緩第1型糖尿病惡化。
目前以一個在美國20個醫學中心進行、有62個病患的雙盲安慰劑控制第2期研究評估治療安全性與效果， 適合的病患是年紀12-35歲、納入研究前已經診斷2-20週。
MSC治療在之前被FDA核准為孤兒藥，擴大核准用於移植物之抗宿主疾病，允許用於有生命危險的狀況。
其他研究中的可能適應症包括克隆氏症、心肌梗塞、肺病。
FDA Approves Orphan Drug Status for Type 1 Diabetes Stem Cell Therapy
By Yael Waknine
Medscape Medical News
May 7, 2010 — The US Food and Drug Administration (FDA) has approved orphan drug designation for a stem cell therapy (Prochymal; Osiris Therapeutics, Inc) in patients with newly diagnosed type 1 diabetes mellitus.
The product is an intravenous formulation of adult mesenchymal stem cells (MSCs) that are isolated from the bone marrow of healthy young adult donors, thereby avoiding the controversy associated with embryonic and fetal cell sources. Cell culture allows the large-scale production of thousands of doses from a single donation.
According to a company news release, MSCs are designed to provide therapeutic benefit by controlling inflammation, promoting tissue regeneration, and preventing scar formation. Preclinical studies indicate that MSCs may delay the progression of type 1 diabetes by preserving beta cell function.
Treatment safety and efficacy are currently being evaluated in a 62-patient, double-blind, placebo-controlled phase 2 study conducted at 20 medical centers across the United States. To be eligible, patients must be aged 12 to 35 years and have been diagnosed 2 to 20 weeks before study entry.
The MSC therapy previously was granted FDA orphan drug status and expanded access approval for graft vs host disease, allowing its use in patients whose condition is life-threatening.
Other potential indications under investigation include Crohn's disease, myocardial infarction, and pulmonary disease.
