7月10日，美国生物制药公司Athersys宣布，其专有的干细胞疗法MultiStem获FDA授予的孤儿药地位，用于治疗赫尔勒氏综合症（Hurler's Syndrome），即I型粘多糖贮积症（MPS-I）。
赫尔勒氏综合症是种溶酶体贮积病（LSD），在儿童中的发病率大约1/100000。
溶酶体贮积症为源于溶酶体缺陷的一群罕见遗传病，溶酶体是细胞内负责分解复合磷脂类及糖类的小体。溶酶体缺陷时，它只能产生很少量的能分解以上分子的必需酶，从而引起细胞与组织中的有害积累，最终导致神经系统疾病及一系列其他并发症。
赫尔勒氏综合症由分解葡糖胺多糖（GAGs）的专一性酶缺乏所致，GAGs的积累可导致智力低下、呼吸系统及心脏并发症或幼儿期死亡。
此外，Athersys公司近日宣布一项美国专利的发行，其涵盖非胚胎多能干细胞疗法，如MultiStem，用于溶酶体贮积症的治疗。
 

Athersys Gets Orphan Drug Designation For MultiStem In Hurler's Syndrome

Athersys Inc. (ATHX: News ) announced that MultiStem, the company's proprietary cell therapy, has been granted orphan drug designation by the U.S. Food and Drug Administration for the treatment of Hurler's Syndrome, also known as mucopolysaccharidosis type I or MPS-I.

Hurler's Syndrome is a lysosomal storage disorder that affects approximately 1 of every 100,000 children.

Lysosomal storage diseases are a group of rare, inherited diseases that initially result from defects in the lysosome, a component of the cell that is responsible for the breakdown of certain molecules, such as complex lipids and sugars. In such cases, the lysosome produces too little of an enzyme that is necessary for the break-down of these materials, resulting in unwanted accumulations in the cells and tissues, which can lead to neurological disease and a range other complications.

Hurler's Syndrome is caused by a deficiency of a specific enzyme responsible for degrading complex sugars called glycosaminoglycans (GAGs), accumulation of which can lead to mental retardation, respiratory and cardiac complications, and death in early childhood.

 

 

The company also recently announced the issuance of a U.S. patent covering the use of non-embryonic, multipotent stem cells, such as MultiStem, for the treatment of lysosomal storage diseases.