Orphan Drug Status for MultiStem
Drug discovery & development
Orphan Drug Status for MultiStem

Athersys Inc. announced that MultiStem, Athersys' 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. Orphan drug designation, which is intended to facilitate drug development, provides substantial potential benefits to the sponsor, including funding for certain clinical studies, study-design assistance, tax incentives and seven years of market exclusivity for the product upon regulatory approval.

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.

In a recent publication in the scientific journal, Cell Transplantation, investigators at the University of Minnesota, together with scientists from Athersys, described preclinical study results suggesting that MultiStem cells could provide benefit to patients suffering from lysosomal storage disorders, such as Hurler's Syndrome.

The study demonstrated that an injection of MultiStem cells in neonatal MPS-I mice reduces the accumulation of GAGs in the brain thereby resulting in improvements in behavioral and motor function in the diseased mice. 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.

BioRestorative Therapies: A Stem Cell Biotechnology Company