title: Encapsulated cell therapy for neurodegenerative diseases: from promise to product.
abstract: Delivering therapeutic molecules, including trophic factor proteins, across the blood brain barrier to the brain parenchyma to treat chronic neurodegenerative diseases remains one of the great challenges in biology. To be effective, delivery needs to occur in a long-term and stable manner at sufficient quantities directly to the target region in a manner that is selective but yet covers enough of the target site to be efficacious. One promising approach uses cellular implants that produce and deliver therapeutic molecules directly to the brain region of interest. Implanted cells can be precisely positioned into the desired region and can be protected from host immunological attack by encapsulating them and by surrounding them within an immunoisolatory, semipermeable capsule. In this approach, cells are enclosed within a semiporous capsule with a perm selective membrane barrier that admits oxygen and required nutrients and releases bioactive cell secretions while restricting passage of larger cytotoxic agents from the host immune defense system. Recent advances in human cell line development have increased the levels of secreted therapeutic molecules from encapsulated cells, and membrane extrusion techniques have led to the first ever clinical demonstrations of long-term survival and function of encapsulated cells in the brain parenchyma. As such, cell encapsulation is capable of providing a targeted, continuous, de novo synthesized source of very high levels of therapeutic molecules that can be distributed over significant portions of the brain.
authors.affiliation: NsGene, Inc., Providence, RI, USA. Electronic address: dwaine.emerich@gmail.com.
authors.forename: Dwaine F
authors.initials: DF
authors.lastname: Emerich
authors.affiliation: Laboratory of Pharmacy and Pharmaceutical Technology, Faculty of Pharmacy, University of the Basque Country, Vitoria, Spain; Networking Biomedical Research Center on Bioengineering, Biomaterials and Nanomedicine, CIBER-BBN, SLFPB-EHU, Vitoria-Gasteiz, Spain.
authors.forename: Gorka
authors.initials: G
authors.lastname: Orive
authors.affiliation: Cytosolve, Inc., Povidence, RI, USA.
authors.forename: Christopher
authors.initials: C
authors.lastname: Thanos
authors.affiliation: NsGene, Inc., Providence, RI, USA.
authors.forename: Jens
authors.initials: J
authors.lastname: Tornoe
authors.affiliation: NsGene, Inc., Providence, RI, USA.
authors.forename: Lars U
authors.initials: LU
authors.lastname: Wahlberg
mesh_terms: D000544:Alzheimer Disease / Q000628:therapy; D000818:Animals; D017690:Cell Transplantation / Q000295:instrumentation / Q000379:methods*; D006801:Humans; D006816:Huntington Disease / Q000628:therapy; D020932:Nerve Growth Factor / Q000378:metabolism; D019636:Neurodegenerative Diseases / Q000628:therapy*; D010300:Parkinson Disease / Q000628:therapy; D054457:Tissue Scaffolds
chemical_list: D020932:Nerve Growth Factor
keywords: Cell transplantation; Neurodegeneration; Polymer encapsulation